1 00:00:05,880 --> 00:00:08,880 >> WELCOME TO THE SPRING 2022 2 00:00:08,880 --> 00:00:10,360 MEETING OF THE MUSCULAR 3 00:00:10,360 --> 00:00:13,320 DYSTROPHY COORDINATING 4 00:00:13,320 --> 00:00:13,600 COMMITTEE. 5 00:00:13,600 --> 00:00:17,640 I'M GLEN KNUCKLES A PROGRAM 6 00:00:17,640 --> 00:00:20,080 DIRECTOR AT THE NATIONAL 7 00:00:20,080 --> 00:00:21,800 INSTITUTE OF NEUROLOGICAL 8 00:00:21,800 --> 00:00:23,200 DISORDERS AND STROKE. 9 00:00:23,200 --> 00:00:25,440 LET'S HAVE OUR COMMITTEE MEMBERS 10 00:00:25,440 --> 00:00:26,800 INTRODUCE THEMSELVES AND WE'LL 11 00:00:26,800 --> 00:00:40,440 START WITH TERESA SAN AUGUSTA. 12 00:00:40,440 --> 00:00:44,440 >> GOOD AFTERNOON, EVERYONE. 13 00:00:44,440 --> 00:00:49,320 I'M TERESA SAN AGUSTIN. 14 00:00:49,320 --> 00:00:51,440 I'VE BEEN WITH THIS COMMITTEE 15 00:00:51,440 --> 00:00:54,280 SINCE 2007 AND I'M LOOKING 16 00:00:54,280 --> 00:00:55,880 FORWARD FOR THE AFTERNOON 17 00:00:55,880 --> 00:00:56,280 PRESENTATION. 18 00:00:56,280 --> 00:00:57,640 THANK YOU. 19 00:00:57,640 --> 00:00:58,880 >> THANKS, TERESA. 20 00:00:58,880 --> 00:01:01,760 NOW WE'LL GO BY ALPHABETICAL 21 00:01:01,760 --> 00:01:05,840 ORDER STARTING WITH DR. BIANCHI. 22 00:01:05,840 --> 00:01:08,000 >> GOOD AFTERNOON, EVERYONE. 23 00:01:08,000 --> 00:01:15,040 I'M DR. DIANA BIANCHI. 24 00:01:15,040 --> 00:01:18,880 AND I'VE BEEN IN THIS POSITION 25 00:01:18,880 --> 00:01:19,320 SINCE LATE 2016. 26 00:01:19,320 --> 00:01:20,920 THANK YOU. 27 00:01:20,920 --> 00:01:23,320 >> THANK YOU. 28 00:01:23,320 --> 00:01:28,160 LINDSEY CRISWELL. 29 00:01:28,160 --> 00:01:30,920 >> HELLO EVERYBODY I'M THE 30 00:01:30,920 --> 00:01:35,440 DIRECTOR OF NIAMS. 31 00:01:35,440 --> 00:01:37,400 AND I'VE BEEN IN THIS POSITION 32 00:01:37,400 --> 00:01:39,080 SINCE FEBRUARY OF LAST YEAR. 33 00:01:39,080 --> 00:01:41,080 GLAD TO BE HERE. 34 00:01:41,080 --> 00:01:51,760 >> THANK YOU. 35 00:01:51,760 --> 00:02:03,280 GUSTA VO DZIEWCZAPOLSKI. 36 00:02:03,280 --> 00:02:05,080 >> I'M 9 SIGN AT THIS I CAN 37 00:02:05,080 --> 00:02:07,600 DIRECTOR SINCE MAY OF 2016 AND 38 00:02:07,600 --> 00:02:15,720 PART OF THE -- COMMITTEE -- THEN 39 00:02:15,720 --> 00:02:18,160 OF 2018 AND -- WE HAVE NO 40 00:02:18,160 --> 00:02:21,280 TREATMENT YESTERDAY BUT SOME 41 00:02:21,280 --> 00:02:22,480 INTERESTING LEADS. 42 00:02:22,480 --> 00:02:25,040 WE'RE GETTING SOME INTEREST FROM 43 00:02:25,040 --> 00:02:27,240 THE PRIVATE SECTOR COMING TO ME 44 00:02:27,240 --> 00:02:27,720 HERE. 45 00:02:27,720 --> 00:02:31,880 >> THANK YOU. 46 00:02:31,880 --> 00:02:38,280 EMILY FREILICH 47 00:02:38,280 --> 00:02:39,640 >> GOOD AFTERNOON. 48 00:02:39,640 --> 00:02:41,600 I'M HAPPY TO BE A MEMBER OF THIS 49 00:02:41,600 --> 00:02:44,040 COMMITTEE. 50 00:02:44,040 --> 00:02:48,480 >> THANK YOU. 51 00:02:48,480 --> 00:02:51,200 GENE FREUND 52 00:02:51,200 --> 00:02:52,440 >> HI. 53 00:02:52,440 --> 00:02:57,840 I'M GENE FREUND. 54 00:02:57,840 --> 00:03:01,120 CMS IS RESPONSIBLE FOR MEDICARE, 55 00:03:01,120 --> 00:03:03,040 MEDICAID, THE HEALTH CARE MARKET 56 00:03:03,040 --> 00:03:05,360 PLACE AS WELL AS A LOT OF 57 00:03:05,360 --> 00:03:07,080 HOSPITAL QUALITY AND OTHER 58 00:03:07,080 --> 00:03:09,280 QUALITY ISSUES. 59 00:03:09,280 --> 00:03:10,880 >> THANK YOU. 60 00:03:10,880 --> 00:03:17,800 MICHAIL GOLDSTEIN. 61 00:03:17,800 --> 00:03:24,720 IS MICHAIL ON? 62 00:03:24,720 --> 00:03:27,080 MAYBE NOT YET. 63 00:03:27,080 --> 00:03:36,680 ALISHA KEEN. 64 00:03:36,680 --> 00:03:43,120 IS ALISHA ON? 65 00:03:43,120 --> 00:03:54,560 JIM KYLEE. 66 00:03:54,560 --> 00:04:10,120 JIM, YOU'RE MUTED. 67 00:04:10,120 --> 00:04:12,360 YOU SHOULD BE ABLE TO SPEAK NOW 68 00:04:12,360 --> 00:04:13,720 JIM. 69 00:04:13,720 --> 00:04:14,040 >> HELLO. 70 00:04:14,040 --> 00:04:14,720 SORE E OTHER. 71 00:04:14,720 --> 00:04:17,200 A LITTLE BIT OF A CONNECTION 72 00:04:17,200 --> 00:04:17,760 PROBLEM. 73 00:04:17,760 --> 00:04:20,960 I'M JIM KYLEE. 74 00:04:20,960 --> 00:04:23,120 GREAT TO SEE ALL OF YOU. 75 00:04:23,120 --> 00:04:28,240 >> THANK YOU. 76 00:04:28,240 --> 00:04:34,880 WALTER -- 77 00:04:34,880 --> 00:04:41,040 >> I DO NOT SEE HIM YET. 78 00:04:41,040 --> 00:04:44,320 >> MARY ELANA MAGUIRE. 79 00:04:44,320 --> 00:04:46,120 >> HI, EVERYONE. 80 00:04:46,120 --> 00:04:49,080 I'M THE PROGRAM MANAGER FOR THE 81 00:04:49,080 --> 00:04:50,360 SOMETHING MUSCULAR DYSTROPHY 82 00:04:50,360 --> 00:04:52,880 PROGRAM. 83 00:04:52,880 --> 00:04:54,920 PART OF THE MEDICAL RESEARCH 84 00:04:54,920 --> 00:04:57,160 PROGRAMS FOR THE DoD. 85 00:04:57,160 --> 00:04:59,600 I'VE ACTUALLY BEEN ATTENDING 86 00:04:59,600 --> 00:05:00,080 THESE MEETINGS. 87 00:05:00,080 --> 00:05:05,480 I WANT TO SAY SINCE AROUND 88 00:05:05,480 --> 00:05:07,720 2009-2010 AND THEN BECAME A 89 00:05:07,720 --> 00:05:10,120 PANEL MEMBER BACK IN 2013. 90 00:05:10,120 --> 00:05:13,400 LOOK FORWARD TO TODAY'S MEETING. 91 00:05:13,400 --> 00:05:14,360 >> THANK YOU. 92 00:05:14,360 --> 00:05:23,160 DAN PEREZ. 93 00:05:23,160 --> 00:05:30,080 IS DAN ON? 94 00:05:30,080 --> 00:05:31,520 >> I DON'T THINK HE IS ON YET. 95 00:05:31,520 --> 00:05:32,120 >> OKAY. 96 00:05:32,120 --> 00:05:40,200 NATALIE STREET. 97 00:05:40,200 --> 00:05:43,240 I SEE NATALIE BUT IT LOOKS LIKE 98 00:05:43,240 --> 00:05:47,680 -- SHE IS FROZEN. 99 00:05:47,680 --> 00:05:51,800 LOOKS LIKE CONNECTION PROBLEMS. 100 00:05:51,800 --> 00:06:01,760 >> LET'S GO BACK AND ASK 101 00:06:01,760 --> 00:06:03,720 DR. KOROSHETZ. 102 00:06:03,720 --> 00:06:05,120 >> HAPPY TO BE HERE. 103 00:06:05,120 --> 00:06:06,440 >> ALISHA KEEN. 104 00:06:06,440 --> 00:06:15,440 >> HI, EVERYONE. 105 00:06:15,440 --> 00:06:20,880 >> NATALIE -- ARE YOU ON? 106 00:06:20,880 --> 00:06:22,280 SEEMS LIKE YOUR WINDOW IS 107 00:06:22,280 --> 00:06:22,880 FROZEN. 108 00:06:22,880 --> 00:06:29,720 7 OKAY. 109 00:06:29,720 --> 00:06:31,520 I THINK THAT IS IT FOR CURRENT 110 00:06:31,520 --> 00:06:33,400 MEMBERS AND WE HAVE SOME 111 00:06:33,400 --> 00:06:34,880 INCOMING MEMBERS AND THEY ARE 112 00:06:34,880 --> 00:06:36,920 LISTED ON THE AGENDA AS NEW 113 00:06:36,920 --> 00:06:39,080 MEMBERS AND I'VE LEARNED THEY 114 00:06:39,080 --> 00:06:41,120 ARE STILL PENDING OFFICIAL 115 00:06:41,120 --> 00:06:43,080 APPOINTMENT FROM THE HHS 116 00:06:43,080 --> 00:06:44,880 SECRETARY SO WE STILL EXTEND A 117 00:06:44,880 --> 00:06:47,000 WARM WELCOME AT THIS TIME AND 118 00:06:47,000 --> 00:06:48,880 HOPE YOU'LL GET YOUR OFFICIAL 119 00:06:48,880 --> 00:06:52,080 LETTER SOON SO LET'S INTRODUCE 120 00:06:52,080 --> 00:06:53,720 DENISE MYLER. 121 00:06:53,720 --> 00:06:55,280 >> HI, I'M GLAD TO BE PART OF 122 00:06:55,280 --> 00:06:56,480 THIS. 123 00:06:56,480 --> 00:06:58,600 >> THANK YOU. 124 00:06:58,600 --> 00:07:00,800 ERIC WANG. 125 00:07:00,800 --> 00:07:03,560 >> HI. 126 00:07:03,560 --> 00:07:05,040 REPRESENTING -- FOUNDATION AND 127 00:07:05,040 --> 00:07:07,480 ALSO A FACULTY MEMBER AT THE 128 00:07:07,480 --> 00:07:10,440 UNIVERSITY OF FLORIDA. 129 00:07:10,440 --> 00:07:12,880 >> AND CHRIS ANSELMO. 130 00:07:12,880 --> 00:07:14,040 >> HI, EVERYONE. 131 00:07:14,040 --> 00:07:16,480 THIS IS CHRIS ANSELMO. 132 00:07:16,480 --> 00:07:18,280 REPRESENTING THE MUSCULAR 133 00:07:18,280 --> 00:07:19,680 DYSTROPHY ASSOCIATION AND I'M 134 00:07:19,680 --> 00:07:21,800 REALLY HAPPY TO BE HERE TODAY. 135 00:07:21,800 --> 00:07:25,160 THANKS. 136 00:07:25,160 --> 00:07:32,320 >> DID MICHAIL GOLDSTEIN JOIN? 137 00:07:32,320 --> 00:07:33,400 THERE IS NATALIE. 138 00:07:33,400 --> 00:07:35,120 DO YOU WANT TO INTRODUCE 139 00:07:35,120 --> 00:07:35,560 YOURSELF 140 00:07:35,560 --> 00:07:36,880 >> I APOLOGIZE. 141 00:07:36,880 --> 00:07:38,480 HAVING SOME VERY BAD CONNECTION 142 00:07:38,480 --> 00:07:40,240 ISSUES TODAY. 143 00:07:40,240 --> 00:07:44,040 I'M NATALIE STREET. 144 00:07:44,040 --> 00:07:45,560 I'M THE MUSCULAR DYSTROPHY 145 00:07:45,560 --> 00:07:48,040 SCIENTIFIC LEAD AT THE CDC. 146 00:07:48,040 --> 00:07:50,520 AND I THINK I'VE BEEN ON THE 147 00:07:50,520 --> 00:07:52,160 COMMITTEE SINCE ABOUT 2018. 148 00:07:52,160 --> 00:07:53,840 BUT HAVE BEEN COMING TO 149 00:07:53,840 --> 00:07:57,440 COMMITTEE MEMBERS BEFORE THEN. 150 00:07:57,440 --> 00:08:00,880 >> THANK YOU. 151 00:08:00,880 --> 00:08:02,480 SO I'LL COVER GENERAL STUFF. 152 00:08:02,480 --> 00:08:04,200 SO THIS MEETING IS BEING 153 00:08:04,200 --> 00:08:06,320 CONDUCTED IN COMPLIANCE WITH THE 154 00:08:06,320 --> 00:08:07,800 FEDERAL ADVISORY COMMITTEE ACT 155 00:08:07,800 --> 00:08:09,480 TO ENSURE THAT THE COMMITTEE 156 00:08:09,480 --> 00:08:11,280 PROVIDES EXPERT DEVICE AND 157 00:08:11,280 --> 00:08:13,080 DIVERSE OPINIONS TO INFORM 158 00:08:13,080 --> 00:08:14,960 DECISION MAKING BY OFFICIALS AND 159 00:08:14,960 --> 00:08:16,960 AGENCIES IN THE EXECUTIVE BRANCH 160 00:08:16,960 --> 00:08:20,160 OF THE FEDERAL GOVERNMENT IN A 161 00:08:20,160 --> 00:08:22,080 MANNER THAT IS TRANSPARENT AND 162 00:08:22,080 --> 00:08:23,920 FREE OF CONFLICTS OF INTEREST. 163 00:08:23,920 --> 00:08:26,160 THERE IS NO CLOSED SESSION 164 00:08:26,160 --> 00:08:27,280 PLANNED FOR TODAY. 165 00:08:27,280 --> 00:08:31,000 WE DON'T ANTICIPATE DISCUSSIONS 166 00:08:31,000 --> 00:08:33,680 OR ACTIONS SPECIFIC TO THE 167 00:08:33,680 --> 00:08:35,240 FINANCIAL INTERESTS SO NO NEED 168 00:08:35,240 --> 00:08:37,880 FOR ANY RECUSALS AT THIS TIME. 169 00:08:37,880 --> 00:08:39,920 WE DID POST A NOTICE TO THE 170 00:08:39,920 --> 00:08:41,040 FEDERAL REGISTER ABOUT THIS 171 00:08:41,040 --> 00:08:43,120 MEETING AND DID NOT RECEIVE ANY 172 00:08:43,120 --> 00:08:44,560 REQUESTS TO ADDRESS THE 173 00:08:44,560 --> 00:08:45,840 COMMITTEE SO WE HAVE NOT 174 00:08:45,840 --> 00:08:47,280 SCHEDULED A PUBLIC COMMENT 175 00:08:47,280 --> 00:08:49,080 PERIOD DURING THIS MEETING BUT 176 00:08:49,080 --> 00:08:51,640 THAT IS SOMETHING THAT WE'RE 177 00:08:51,640 --> 00:08:53,880 WELCOME TO DO AT ANY OF OUR 178 00:08:53,880 --> 00:08:54,960 MEETINGS. 179 00:08:54,960 --> 00:08:56,760 THE MEETING IS BEING STREAMED 180 00:08:56,760 --> 00:08:58,440 LIVE FOR PUBLIC VIEWING AND IT'S 181 00:08:58,440 --> 00:09:00,960 ALSO BEING RECORDED AND THE 182 00:09:00,960 --> 00:09:03,080 STREAM AND RECORDING ARE 183 00:09:03,080 --> 00:09:14,600 AVAILABLE AT VIDEO CAST.NIH.GOV. 184 00:09:14,600 --> 00:09:16,480 SO DURING THIS MEETING THE 185 00:09:16,480 --> 00:09:18,880 PANELISTS AND OTHER PARTICIPANTS 186 00:09:18,880 --> 00:09:20,720 CAN POST ANY QUESTIONS THEY HAVE 187 00:09:20,720 --> 00:09:21,880 THROUGH THE CHAT AND WE'LL 188 00:09:21,880 --> 00:09:24,000 MONITOR THE CHAT AND MAKE SURE 189 00:09:24,000 --> 00:09:25,440 THAT WE GET THOSE QUESTIONS 190 00:09:25,440 --> 00:09:27,840 ADDRESSED FOR YOU. 191 00:09:27,840 --> 00:09:29,720 COULD YOU BRING UP THE FIRST 192 00:09:29,720 --> 00:09:35,320 SLIDE THAT I ASKED FOR. 193 00:09:35,320 --> 00:09:38,160 SO I JUST WANTED TO GIVE A BRIEF 194 00:09:38,160 --> 00:09:40,160 UPDATE ON THE TOPIC THAT WE 195 00:09:40,160 --> 00:09:41,400 COVERED FROM OUR MEETING IN 196 00:09:41,400 --> 00:09:42,720 DECEMBER. 197 00:09:42,720 --> 00:09:44,520 AND YOU RECALL IN DECEMBER OUR 198 00:09:44,520 --> 00:09:47,200 MEETING WAS FOCUSED ON 199 00:09:47,200 --> 00:09:49,640 STRATEGIES TO PROMOTE DIVERSITY 200 00:09:49,640 --> 00:09:50,440 IN THE MUSCULAR DYSTROPHY 201 00:09:50,440 --> 00:09:52,200 CLINICAL STUDY PARTICIPATION. 202 00:09:52,200 --> 00:09:53,880 SO WE'VE TAKEN THE LESSONS 203 00:09:53,880 --> 00:09:54,880 LEARNED AND OTHER 204 00:09:54,880 --> 00:09:55,760 RECOMMENDATIONS FROM THAT 205 00:09:55,760 --> 00:09:58,240 MEETING AND SUMMARIZED THEM ON 206 00:09:58,240 --> 00:09:59,360 TO THE WEBSITE. 207 00:09:59,360 --> 00:10:03,440 SO IF YOU GO TO OUR WEBSITE 208 00:10:03,440 --> 00:10:06,080 THERE IS A NEW BUTTON AT THE TOP 209 00:10:06,080 --> 00:10:08,560 THAT SAYS STRATEGIES TO PRE MOAT 210 00:10:08,560 --> 00:10:09,680 DIVERSITY AND IF YOU CLICK THAT 211 00:10:09,680 --> 00:10:11,840 IT TAKES TO YOU THIS PAGE. 212 00:10:11,840 --> 00:10:14,720 WHERE WE PROVIDE GUIDANCE TO 213 00:10:14,720 --> 00:10:16,560 RESEARCHERS WHO ARE PLANNING 214 00:10:16,560 --> 00:10:18,800 RESEARCH STUDIES SO THEY CAN 215 00:10:18,800 --> 00:10:21,760 CONSIDER STEPS THAT ARE KNOWN TO 216 00:10:21,760 --> 00:10:27,280 REDUCE OBSTACLE ELSE TO 217 00:10:27,280 --> 00:10:27,840 PARTICIPATION. 218 00:10:27,840 --> 00:10:33,800 * SO FOR EXAMPLE IF YOU CLICK ON 219 00:10:33,800 --> 00:10:35,080 SOCIOECONOMIC IT EXPANDS TO A 220 00:10:35,080 --> 00:10:38,200 SET OF RECOMMENDATIONS ON THINGS 221 00:10:38,200 --> 00:10:41,400 LIKE TRANSLATING CONSENT FORMS 222 00:10:41,400 --> 00:10:43,360 TO HAVING BILINGUAL STUDY 223 00:10:43,360 --> 00:10:45,760 PERSONNEL AND OTHER EFFECTIVE 224 00:10:45,760 --> 00:10:47,280 STRATEGIES THAT WE TALKED ABOUT 225 00:10:47,280 --> 00:10:49,760 AT OUR PREVIOUS MEETING. 226 00:10:49,760 --> 00:10:52,440 SO THEN ON THE ORIGINAL PAGE THE 227 00:10:52,440 --> 00:10:54,640 SITE ALSO HAS AT THE BOTTOM 228 00:10:54,640 --> 00:10:56,000 LINKS TO ADDITIONAL GUIDANCE 229 00:10:56,000 --> 00:10:59,160 FROM THE FDA AND NIH GUIDANCE ON 230 00:10:59,160 --> 00:11:01,560 THIS TOPIC AND HOPEFULLY THIS 231 00:11:01,560 --> 00:11:02,920 WILL HELP RESEARCHERS TO PLAN 232 00:11:02,920 --> 00:11:05,600 AND CONDUCT STUDIES THAT HAVE 233 00:11:05,600 --> 00:11:07,280 APPROPRIATE ENROLLMENT DIVERSITY 234 00:11:07,280 --> 00:11:09,720 AND WE LOOK FORWARD TO POINTING 235 00:11:09,720 --> 00:11:12,560 TO THIS WEBSITE IN OUR FUTURE 236 00:11:12,560 --> 00:11:13,720 FUNDING OPPORTUNITY 237 00:11:13,720 --> 00:11:15,480 ANNOUNCEMENTS SO THAT THAT 238 00:11:15,480 --> 00:11:16,720 INFORMATION WILL BE AVAILABLE TO 239 00:11:16,720 --> 00:11:17,960 PEOPLE AS THEY ARE PLANNING 240 00:11:17,960 --> 00:11:20,880 THEIR RESEARCH STUDIES. 241 00:11:20,880 --> 00:11:24,120 REALLY WANT TO SAY THANKS TO 242 00:11:24,120 --> 00:11:25,480 MARIE FOR CONSTRUCTING THIS 243 00:11:25,480 --> 00:11:27,160 WEBSITE. 244 00:11:27,160 --> 00:11:29,480 SO, THAT IS ALL FOR MY 245 00:11:29,480 --> 00:11:31,160 INTRODUCTION AND I'LL TURN THE 246 00:11:31,160 --> 00:11:33,240 MEETING BACK OVER TO 247 00:11:33,240 --> 00:11:37,240 DR. BIANCHI. 248 00:11:37,240 --> 00:11:37,960 >> THANK YOU, GLEN. 249 00:11:37,960 --> 00:11:39,880 THIS IS THE FIRST TIME I'M 250 00:11:39,880 --> 00:11:43,280 ACTUALLY DRIVING IN A ZOOM 251 00:11:43,280 --> 00:11:44,520 PRESENTATION FROM MY OFFICE IN 252 00:11:44,520 --> 00:11:49,280 TWO YEARS SO I'M PRETTY -- WITH 253 00:11:49,280 --> 00:11:52,280 MY LAPTOP SO THIS WILL BE 254 00:11:52,280 --> 00:12:05,840 ADVENTURE. 255 00:12:05,840 --> 00:12:06,920 >> ARE YOU SEEING THIS? 256 00:12:06,920 --> 00:12:08,480 OKAY. 257 00:12:08,480 --> 00:12:11,280 YOU'RE SEEING THE PRESENTATION 258 00:12:11,280 --> 00:12:13,240 MODE HOPEFULLY 259 00:12:13,240 --> 00:12:15,360 >> YOU'RE STILL ON SPEAKER VIEW. 260 00:12:15,360 --> 00:12:17,280 JUST SWAP IT AND YOU WILL BE ALL 261 00:12:17,280 --> 00:12:19,920 SET. 262 00:12:19,920 --> 00:12:21,120 PERFECT. 263 00:12:21,120 --> 00:12:21,840 >> ALL RIGHT. 264 00:12:21,840 --> 00:12:23,840 EXCELLENT. 265 00:12:23,840 --> 00:12:25,880 YOU CAN TRAIN OLD DOGS. 266 00:12:25,880 --> 00:12:27,440 IT'S MY GREAT PLEASURE TO BE 267 00:12:27,440 --> 00:12:29,000 WITH YOU TODAY AND I'M LOOKING 268 00:12:29,000 --> 00:12:30,880 FORWARD TO EVERYONE ELSE'S 269 00:12:30,880 --> 00:12:32,240 PRESENTATION SO I WOULD ALSO 270 00:12:32,240 --> 00:12:35,520 LIKE TO ACKNOWLEDGE 271 00:12:35,520 --> 00:12:37,480 DR. ELIZABETH BADEN'S ASSISTANCE 272 00:12:37,480 --> 00:12:38,880 IN PUTTING THIS TOGETHER. 273 00:12:38,880 --> 00:12:40,480 WE'VE WELCOMED EVERYONE AND THE 274 00:12:40,480 --> 00:12:41,760 NEW MEMBERS OF THE COMMITTEE. 275 00:12:41,760 --> 00:12:43,440 I'M GOING TO BE TALKING ABOUT 276 00:12:43,440 --> 00:12:45,880 THESE TOPICS TODAY AND I HAVE 277 00:12:45,880 --> 00:12:49,640 LET A GOOD AMOUNT OF TIME FOR 278 00:12:49,640 --> 00:12:51,320 QUESTIONS SO PUT YOUR QUESTIONS 279 00:12:51,320 --> 00:12:53,280 IN THE CHAT BOX WHICH I CANNOT 280 00:12:53,280 --> 00:12:56,840 SEE NOW BUT WE'LL GET TO IT 281 00:12:56,840 --> 00:12:59,680 AFTERWARDS OR SIMPLY WRITE THEM 282 00:12:59,680 --> 00:13:00,640 DOWN. 283 00:13:00,640 --> 00:13:03,200 WE'RE IN THE MIDDLE OF A BUDGET 284 00:13:03,200 --> 00:13:03,760 SEASON. 285 00:13:03,760 --> 00:13:06,960 WE HAVE THIS WEEK THE HOUSE 286 00:13:06,960 --> 00:13:11,080 BUDGET HEARING FOR FISCAL YEAR 287 00:13:11,080 --> 00:13:15,560 23. AS YOU PROBABLY KNOW WE DID 288 00:13:15,560 --> 00:13:19,720 NOT GET THE FISCAL 22 BUDGET 289 00:13:19,720 --> 00:13:23,200 UNTIL THE MIDDLE OF MARCH. 290 00:13:23,200 --> 00:13:25,520 THAT MEANS EVERYONE WORKS TWICE 291 00:13:25,520 --> 00:13:27,600 AS HARD TO GET THE SAME AMOUNT 292 00:13:27,600 --> 00:13:30,120 OF WORK DONE IN HALF OF THE 293 00:13:30,120 --> 00:13:30,680 TIME. 294 00:13:30,680 --> 00:13:36,280 NIH DID GET AN INCREASE TO THE 295 00:13:36,280 --> 00:13:36,840 BUDGET $45 BILLION AND IN 296 00:13:36,840 --> 00:13:40,200 ADDITION A BILLION DOLLARS IS 297 00:13:40,200 --> 00:13:46,880 GIVEN TO ESTABLISH ARPA-H WHICH 298 00:13:46,880 --> 00:13:49,040 WILL HAVE A DUAL REPORTING 299 00:13:49,040 --> 00:13:50,920 RELATIONSHIP WHERE THEY THEIR 300 00:13:50,920 --> 00:13:54,600 ADMINISTRATIVE ASPECTS WILL BE 301 00:13:54,600 --> 00:13:56,680 AFFILIATED WITH THE NIH BUT THE 302 00:13:56,680 --> 00:13:58,600 DIRECTOR WILL REPORT DIRECTLY TO 303 00:13:58,600 --> 00:14:01,840 HEALTH AND HUMAN SERVICES 304 00:14:01,840 --> 00:14:04,280 SECRETARY -- AGAIN IN PARALLEL 305 00:14:04,280 --> 00:14:08,160 WE'RE RIGHT NOW WORKING ON THE 306 00:14:08,160 --> 00:14:09,160 FISCAL 2023 BUDGET. 307 00:14:09,160 --> 00:14:13,280 WE HAVE PUT FORWARD SOME 308 00:14:13,280 --> 00:14:14,000 RECOMMENDATIONS. 309 00:14:14,000 --> 00:14:18,560 THE PRESIDENT HAS PROPOSED 310 00:14:18,560 --> 00:14:20,680 $62.5 BILLION FOR NIH WHICH 311 00:14:20,680 --> 00:14:23,440 INCLUDES A FLAT BASED BUDGET AND 312 00:14:23,440 --> 00:14:26,040 ANNA ADDITIONAL $5 BILLION FOR 313 00:14:26,040 --> 00:14:28,440 ARPA-H AND $12 BILLION FOR 314 00:14:28,440 --> 00:14:30,560 PANDEMIC PREPAREDNESS. 315 00:14:30,560 --> 00:14:34,680 NOW DON'T WORRY ABOUT THAT 316 00:14:34,680 --> 00:14:37,800 $45 BILLION FLAT REQUEST. 317 00:14:37,800 --> 00:14:41,280 IT WAS BASED ON THE CONTINUING 318 00:14:41,280 --> 00:14:43,880 RESOLUTION FIGURES BECAUSE WHEN 319 00:14:43,880 --> 00:14:46,120 THE PRESIDENT PUT FORWARD HIS 320 00:14:46,120 --> 00:14:49,800 BUC WE DID NOT HAVE A FISCAL 321 00:14:49,800 --> 00:14:50,880 CAREER 23 BUDGET. 322 00:14:50,880 --> 00:14:53,040 THERE WILL BE MORE POTENTIALLY 323 00:14:53,040 --> 00:14:54,040 AVAILABLE BUT THAT IS THE 324 00:14:54,040 --> 00:14:55,920 MISSION THIS WEEK. 325 00:14:55,920 --> 00:14:57,600 I WILL BE TESTIFYING IN THE 326 00:14:57,600 --> 00:15:00,600 HOUSE HEARINGS ALONG WITH FIVE 327 00:15:00,600 --> 00:15:04,680 OTHER NIH DIRECTORS AND THE 328 00:15:04,680 --> 00:15:07,120 INSTITUTE DIRECTORS AND ACTING 329 00:15:07,120 --> 00:15:09,760 DIRECTOR TABAK AND THE FOLLOWING 330 00:15:09,760 --> 00:15:12,080 WEEK FIVE OR SIX DIRECTORS WILL 331 00:15:12,080 --> 00:15:13,840 BE SPEAKING AT THE SENATE BUDGET 332 00:15:13,840 --> 00:15:17,080 HEARINGS. 333 00:15:17,080 --> 00:15:20,080 AGAIN AS YOU PROBABLY KNOW 334 00:15:20,080 --> 00:15:21,320 DR. COLLINS STEPPED DOWN AS THE 335 00:15:21,320 --> 00:15:23,840 NIH DIRECTOR ON DECEMBER 20th 336 00:15:23,840 --> 00:15:24,640 LAST YEAR. 337 00:15:24,640 --> 00:15:29,600 HE THOUGHT HE WAS GOING TO BE 338 00:15:29,600 --> 00:15:32,040 RETIRING TO A LIFE AS A SENIOR 339 00:15:32,040 --> 00:15:33,760 INVESTIGATOR DOING RESEARCH IN 340 00:15:33,760 --> 00:15:37,280 HIS OWN LAB BUT THEN THE 341 00:15:37,280 --> 00:15:39,840 PRESIDENT TAPPED HIM TO BE HIS 342 00:15:39,840 --> 00:15:41,720 SCIENCE ADVISOR IN THE CABINET 343 00:15:41,720 --> 00:15:43,520 AS WELL AS THE PRESIDENT'S 344 00:15:43,520 --> 00:15:46,880 COUNCIL ADVISORY COUNCIL ON STEM 345 00:15:46,880 --> 00:15:48,720 TECHNOLOGY -- SCIENCE AND 346 00:15:48,720 --> 00:15:49,760 TECHNOLOGY. 347 00:15:49,760 --> 00:15:54,120 HE IS THE COCHAIR OF THE P CAST. 348 00:15:54,120 --> 00:15:57,680 HE IS FREQUENTLY AROUND OR IN 349 00:15:57,680 --> 00:15:58,480 THE WHITE HOUSE. 350 00:15:58,480 --> 00:16:01,760 IN THE MEANTIME DR. TABAK IS 351 00:16:01,760 --> 00:16:04,440 DOING A SUPERB JOB AS THE ACTING 352 00:16:04,440 --> 00:16:07,480 NIH DIRECTOR AND THE WHITE HOUSE 353 00:16:07,480 --> 00:16:10,880 IS CONDUCTING THE SEARCH FOR THE 354 00:16:10,880 --> 00:16:13,520 NEXT DIRECTOR. 355 00:16:13,520 --> 00:16:16,320 RUMORS -- WE DON'T KNOW IF THEY 356 00:16:16,320 --> 00:16:19,520 ARE TRUE -- ONCE SOMEONE IS 357 00:16:19,520 --> 00:16:21,200 NAMED THAT PERSON WILL THEN NEED 358 00:16:21,200 --> 00:16:24,400 TO UNDERGO SENATE CONFIRMATION. 359 00:16:24,400 --> 00:16:26,160 AT THE SAME TIME WE'VE GOT TWO 360 00:16:26,160 --> 00:16:30,040 OTHER INSTITUTE DIRECTOR 361 00:16:30,040 --> 00:16:34,000 VACANCIES N KATZ AND THAT SEARCH 362 00:16:34,000 --> 00:16:36,520 HAS MOVED ALONG BUT IT IS 363 00:16:36,520 --> 00:16:42,040 WAITING TO * SEE IF THERE IS A 364 00:16:42,040 --> 00:16:46,280 PERMANENT DIRECTOR NAMED AND NCI 365 00:16:46,280 --> 00:16:48,080 -- THE PRESIDENT WOULD NEED 366 00:16:48,080 --> 00:16:53,040 IDENTIFY AN NCI DIRECTOR WHO 367 00:16:53,040 --> 00:16:55,040 WILL ALSO UPPED GO SENATE 368 00:16:55,040 --> 00:16:55,360 CONFIRMATION. 369 00:16:55,360 --> 00:16:57,880 I'M NOT GOING TO SAY A LOT ABOUT 370 00:16:57,880 --> 00:16:58,320 COVID-19. 371 00:16:58,320 --> 00:17:00,880 I DID WANT TO BRING ONE THING TO 372 00:17:00,880 --> 00:17:02,440 YOUR ATTENTION WHICH I THINK IS 373 00:17:02,440 --> 00:17:06,280 OF INTEREST TO THIS GROUP. 374 00:17:06,280 --> 00:17:07,280 I DON'T KNOW HOW MANY OF YOU 375 00:17:07,280 --> 00:17:10,600 HAVE HAD THE EXPERIENCE OF DOING 376 00:17:10,600 --> 00:17:13,680 AT HOME ANTIGEN TESTING. 377 00:17:13,680 --> 00:17:15,400 I'VE DONE IT FREQUENTLY TO BE 378 00:17:15,400 --> 00:17:17,400 SAFE AT WORK AND ALSO IF I'M 379 00:17:17,400 --> 00:17:19,240 GOING FOR ANY SORT OF EVENT I 380 00:17:19,240 --> 00:17:21,080 WOULD LIKE TO KNOW WHEN I'M 381 00:17:21,080 --> 00:17:24,480 NEGATIVE SO THAT I'M NOT 382 00:17:24,480 --> 00:17:26,040 TRANSMITTING SARS-CoV-2 383 00:17:26,040 --> 00:17:28,280 INFECTION TO COLLEAGUES OR 384 00:17:28,280 --> 00:17:30,480 PEOPLE THAT I MEET BUT I'VE ALSO 385 00:17:30,480 --> 00:17:32,200 WATCHED MY HUSBAND WHO'S NOT 386 00:17:32,200 --> 00:17:34,720 MEDICAL OR SCIENCE BASED I'VE 387 00:17:34,720 --> 00:17:36,480 ALSO WATCHED HIM TRY TO DO IT 388 00:17:36,480 --> 00:17:38,200 AND IT'S NOT SO EASY IF YOU 389 00:17:38,200 --> 00:17:40,640 DON'T HAVE A LABORATORY BASED 390 00:17:40,640 --> 00:17:42,840 BACKGROUND AND IT'S EVEN MORE 391 00:17:42,840 --> 00:17:45,080 DIFFICULT IF YOU HAVE ANY SORT 392 00:17:45,080 --> 00:17:47,960 OF DISABILITY WHETHER IT'S A 393 00:17:47,960 --> 00:17:49,640 PHYSICAL DISABILITY OR BLIND FOR 394 00:17:49,640 --> 00:17:51,880 EXAMPLE. 395 00:17:51,880 --> 00:17:54,320 WE'VE DONE AN A AMAZING JOB IN 396 00:17:54,320 --> 00:17:56,040 MAKING THESE TESTS AVAILABLE. 397 00:17:56,040 --> 00:17:58,600 IN MANY CASES AT NO COST TO THE 398 00:17:58,600 --> 00:17:59,160 GENERAL PUBLIC. 399 00:17:59,160 --> 00:18:02,920 BUT THE PROBLEM IS THEY ARE NOT 400 00:18:02,920 --> 00:18:06,560 ACCESSIBLE FOR PEOPLE WHO HAVE A 401 00:18:06,560 --> 00:18:12,480 VARIETY OF ISSUES SO NIBIB HELD 402 00:18:12,480 --> 00:18:14,920 A MEETING THAT WAS HOSTED BY THE 403 00:18:14,920 --> 00:18:16,600 CONSORTIUM FOR IMPROVING 404 00:18:16,600 --> 00:18:20,120 MEDICINE WITH INNOVATION NAME AS 405 00:18:20,120 --> 00:18:25,440 -- AND CIMIT. 406 00:18:25,440 --> 00:18:28,240 AND THE GOALS WERE TO UNDERSTAND 407 00:18:28,240 --> 00:18:30,080 CHALLENGES TO THE ACCESSIBILITY 408 00:18:30,080 --> 00:18:31,480 OF AT HOME TESTS. 409 00:18:31,480 --> 00:18:33,560 AND TO IDENTIFY BEST PRACTICE 410 00:18:33,560 --> 00:18:35,520 AND GOOD MODELS. 411 00:18:35,520 --> 00:18:36,920 AND THEY ESTABLISHED OPEN LINES 412 00:18:36,920 --> 00:18:38,960 OF COMMUNICATION FOR AN ONGOING 413 00:18:38,960 --> 00:18:40,200 DISCUSSION. 414 00:18:40,200 --> 00:18:41,800 AND THIS IS REALLY IMPORTANT 415 00:18:41,800 --> 00:18:43,800 BECAUSE I THINK WHAT THIS WHOLE 416 00:18:43,800 --> 00:18:46,960 EXPERIENCE HAS SHOWN US IS THAT 417 00:18:46,960 --> 00:18:48,880 IT DOES WORK IF YOU CAN GET 418 00:18:48,880 --> 00:18:50,560 TESTING INTO THE HANDS OF THE 419 00:18:50,560 --> 00:18:51,240 GENERAL PUBLIC. 420 00:18:51,240 --> 00:18:53,800 AND WE HOPE THAT THE PANDEMIC 421 00:18:53,800 --> 00:18:58,840 WILL BE OVER SOON BUT IT MAY NOT 422 00:18:58,840 --> 00:19:00,600 BE AND THERE MAY BE OTHER THINGS 423 00:19:00,600 --> 00:19:03,160 THAT REQUIRE TESTING SO THIS WAS 424 00:19:03,160 --> 00:19:05,480 AN AMAZING ACHIEVEMENT TO GET 425 00:19:05,480 --> 00:19:08,920 THE TESTING OUT THERE BUT WE CAN 426 00:19:08,920 --> 00:19:09,960 DO BETTER. 427 00:19:09,960 --> 00:19:12,640 THERE IS A SPECIAL NOTICE THAT 428 00:19:12,640 --> 00:19:15,720 ADDRESSES ACCESSIBILITY AND 429 00:19:15,720 --> 00:19:17,480 EQUITIES FOR INDIVIDUALS WITH 430 00:19:17,480 --> 00:19:19,280 VISUAL IMPAIRMENT BUT ALSO 431 00:19:19,280 --> 00:19:22,680 LOOKING FOR TESTING 432 00:19:22,680 --> 00:19:23,320 ACCOMMODATIONS FOR PEOPLE WHO 433 00:19:23,320 --> 00:19:30,000 HAVE FINE MOTOR OR OTHER GROSS 434 00:19:30,000 --> 00:19:31,480 MOTOR ISSUES WHERE THEY WOULD 435 00:19:31,480 --> 00:19:35,560 NOT BE ABLE TO PERFORM THE 436 00:19:35,560 --> 00:19:36,880 TESTING. 437 00:19:36,880 --> 00:19:39,480 LAST TIME I MIGHT HAVE MENTIONED 438 00:19:39,480 --> 00:19:44,480 THE SOMATIC CELL GENOME EDITING 439 00:19:44,480 --> 00:19:48,080 PROGRAM BUT THIS IS RELEVANT FOR 440 00:19:48,080 --> 00:19:50,640 THE THERAPIES. 441 00:19:50,640 --> 00:19:52,880 IN ITS FIRST YEAR PHASE IT 442 00:19:52,880 --> 00:19:54,760 FUNDED 45 PROJECTS. 443 00:19:54,760 --> 00:19:56,760 THE SECOND FIVE-YEAR PHASE IS 444 00:19:56,760 --> 00:20:00,160 NOW FOCUSING ON TRANSLATING AND 445 00:20:00,160 --> 00:20:05,040 ACCELERATING SAFE AND EFFECTIVE 446 00:20:05,040 --> 00:20:06,800 GENOME EDITING THERAPEUTICS AND 447 00:20:06,800 --> 00:20:08,000 IT HAS THREE PARTS. 448 00:20:08,000 --> 00:20:10,880 SO THE FIRST PART IS TO DEVELOP 449 00:20:10,880 --> 00:20:11,880 NOVEL TECHNOLOGIES. 450 00:20:11,880 --> 00:20:14,320 THE SECOND PART IS TO BUILD A 451 00:20:14,320 --> 00:20:16,480 FOUNDATION FOR -- APPLICATIONS 452 00:20:16,480 --> 00:20:18,680 AND THE THIRD PART IS TO DELIVER 453 00:20:18,680 --> 00:20:20,880 RESOURCES TO ACCELERATE 454 00:20:20,880 --> 00:20:21,800 TREATMENTS. 455 00:20:21,800 --> 00:20:25,680 AND THIS IS AGAIN NIH WIDE AND 456 00:20:25,680 --> 00:20:28,120 THERE IS A LOT OF DISCUSSION AS 457 00:20:28,120 --> 00:20:30,680 TO WHICH SINGLE GENE DISORDERS 458 00:20:30,680 --> 00:20:32,240 WOULD BENEFIT MOST FROM THIS 459 00:20:32,240 --> 00:20:34,680 NOVEL POINT OF THERAPY. 460 00:20:34,680 --> 00:20:38,000 WE HAVE SEVERAL DIFFERENT 461 00:20:38,000 --> 00:20:38,960 FUNDING OPPORTUNITIES IN THIS 462 00:20:38,960 --> 00:20:40,360 AREA. 463 00:20:40,360 --> 00:20:43,040 SO THE FIRST IS ACTUALLY A 464 00:20:43,040 --> 00:20:44,000 REQUEST FOR INFORMATION. 465 00:20:44,000 --> 00:20:45,960 IT'S NOT A FUNDING OPPORTUNITY 466 00:20:45,960 --> 00:20:50,480 BUT IT IS REALLY A WAY OF 467 00:20:50,480 --> 00:20:52,560 REWARDING PEOPLE FOR THINKING 468 00:20:52,560 --> 00:20:55,240 ABOUT NEW TECHNOLOGIES THAT 469 00:20:55,240 --> 00:21:00,080 COULD DELIVER EITHER NONVIRAL OR 470 00:21:00,080 --> 00:21:01,760 CELL TYPE SPECIFIC THERAPIES. 471 00:21:01,760 --> 00:21:03,280 SO THIS IS YOUR OPPORTUNITY IF 472 00:21:03,280 --> 00:21:07,040 YOU WOULD LIKE TO MAKE 473 00:21:07,040 --> 00:21:08,480 SUGGESTIONS ABOUT WHAT MATERIALS 474 00:21:08,480 --> 00:21:13,280 SHOULD BE IN A CHALLENGE. 475 00:21:13,280 --> 00:21:14,880 WE ALSO HAVE SEVERAL FUNDING 476 00:21:14,880 --> 00:21:16,760 OPPORTUNITY ANNOUNCEMENTS FOR 477 00:21:16,760 --> 00:21:19,480 PLATFORM CLINICAL TRIALS. 478 00:21:19,480 --> 00:21:24,280 AND WE'RE GOING TO BE TALKING 479 00:21:24,280 --> 00:21:25,400 ABOUT THIS. 480 00:21:25,400 --> 00:21:28,280 TODAY AND IN FUTURE MEETINGS. 481 00:21:28,280 --> 00:21:29,720 SO SOME OF THESE TRIALS ARE 482 00:21:29,720 --> 00:21:32,880 INCLUDING AT LEAST TWO DIFFERENT 483 00:21:32,880 --> 00:21:36,160 DISEASES CUES ARE USING THE SAME 484 00:21:36,160 --> 00:21:37,800 GENOMED TOWARD ADMINISTRATION 485 00:21:37,800 --> 00:21:40,680 AND DELIVERY SYSTEM. 486 00:21:40,680 --> 00:21:43,600 AND ANOTHER OPPORTUNITY OUT 487 00:21:43,600 --> 00:21:52,880 THERE THAT IS -- WHERE TEAMS ARE 488 00:21:52,880 --> 00:21:54,480 ESTABLISHED AND DEVELOPED TO 489 00:21:54,480 --> 00:21:57,800 INVESTIGATE RESEARCH TO MAKE THE 490 00:21:57,800 --> 00:21:59,720 TRANSLATION TO THE CLINIC THAT 491 00:21:59,720 --> 00:22:01,800 MUCH FASTER AND THEN LASTLY 492 00:22:01,800 --> 00:22:04,760 THERE IS ANOTHER IND OPPORTUNITY 493 00:22:04,760 --> 00:22:07,480 THAT IS ON TECHNOLOGIES AND 494 00:22:07,480 --> 00:22:12,640 ASSAYS FOR THERAPEUTIC GENOME 495 00:22:12,640 --> 00:22:13,200 EDITING. 496 00:22:13,200 --> 00:22:15,360 THEY ARE SLIGHTLY DIFFERENT. 497 00:22:15,360 --> 00:22:20,880 SO, THAT IS ONE OPPORTUNITY TO 498 00:22:20,880 --> 00:22:23,560 HELP GET THERAPIES TO THE CLINIC 499 00:22:23,560 --> 00:22:24,000 FASTER. 500 00:22:24,000 --> 00:22:27,080 AND I SHOULD SAY TODAY'S 501 00:22:27,080 --> 00:22:29,680 PRESENTATION IS LIKE A LITTLE 502 00:22:29,680 --> 00:22:32,040 POTPOURRI OF THINGS THAT WE 503 00:22:32,040 --> 00:22:33,560 WANTED TO GET TO YOUR ATTENTION 504 00:22:33,560 --> 00:22:35,800 BUT DON'T NECESSARILY NEED TO 505 00:22:35,800 --> 00:22:38,080 TAKE UP AN ENTIRE PRESENTATION. 506 00:22:38,080 --> 00:22:39,640 BUT THERE ARE THINGS THAT ARE 507 00:22:39,640 --> 00:22:39,880 IMPORTANT. 508 00:22:39,880 --> 00:22:43,960 SO ONE IS OUR PROGRESS IN EARLY 509 00:22:43,960 --> 00:22:45,760 STAGE INVESTIGATORS. 510 00:22:45,760 --> 00:22:50,160 SO WE REALLY NEED THE NEXT 511 00:22:50,160 --> 00:22:54,040 GENERATION TO BE FUNDED AND TO 512 00:22:54,040 --> 00:22:55,920 HAVE THE SECURITY OF KNOWING 513 00:22:55,920 --> 00:22:58,360 THAT THEY HAVE A CARRERA HEAD OF 514 00:22:58,360 --> 00:23:00,480 THEM AND I JUST THOUGHT THIS WAS 515 00:23:00,480 --> 00:23:01,640 QUITE INTERESTING. 516 00:23:01,640 --> 00:23:05,600 IF YOU IGNORE THE DIP WHICH WAS 517 00:23:05,600 --> 00:23:09,600 AROUND THE TYPE OF FLAT FUNDING 518 00:23:09,600 --> 00:23:13,280 OF NIH AND LOOKING AT 2017 519 00:23:13,280 --> 00:23:15,360 ONWARDS YOU CAN SEE THE INCREASE 520 00:23:15,360 --> 00:23:17,240 IN THE NUMBER OF EARLY STAGE 521 00:23:17,240 --> 00:23:19,760 INVESTIGATORS AND THIS IS THE 522 00:23:19,760 --> 00:23:23,040 RESULT OF BOTH NIH AND 523 00:23:23,040 --> 00:23:25,240 CONGRESSIONAL INTEREST IN 524 00:23:25,240 --> 00:23:27,560 PREFERENTIAL FUNDING OF EARLY 525 00:23:27,560 --> 00:23:28,120 STAGE INVESTIGATORS. 526 00:23:28,120 --> 00:23:31,120 I KNOW THAT AT NIH THEY HAVE A 527 00:23:31,120 --> 00:23:32,960 MUCH MORE GENEROUS PAY LINE. 528 00:23:32,960 --> 00:23:36,480 SO WE'RE ABLE TO PAY MORE OF 529 00:23:36,480 --> 00:23:38,240 THEM WHO WOULD HAVE HAVE 530 00:23:38,240 --> 00:23:41,160 RECEIVED GRAND FUNDING OTHERWISE 531 00:23:41,160 --> 00:23:42,680 AND WE HAVE HAD A GOAL FOR A 532 00:23:42,680 --> 00:23:46,120 NUMBER OF YEARS OF FUNDING AT 533 00:23:46,120 --> 00:23:47,680 LEAST 1100 AND YOU CAN SEE THAT 534 00:23:47,680 --> 00:23:49,560 WE'VE EXCEEDED THAT SO, THAT IS 535 00:23:49,560 --> 00:23:53,040 VERY GOOD NEWS. 536 00:23:53,040 --> 00:23:54,680 ANOTHER THINGS THAT RELATESES TO 537 00:23:54,680 --> 00:23:57,760 OUR PREVIOUS MEETING IS 538 00:23:57,760 --> 00:23:59,880 PROMOTING SCIENTIFIC RESEARCH 539 00:23:59,880 --> 00:24:01,040 TOWARDS DIVERSITY. 540 00:24:01,040 --> 00:24:02,880 LAST TIME WE TALKED ABOUT THE 541 00:24:02,880 --> 00:24:07,680 WORKFORCE AS WELL AS INCLUDING A 542 00:24:07,680 --> 00:24:09,080 DIVERSE WORKFORCE POPULATION. 543 00:24:09,080 --> 00:24:12,480 BUT WE HAVE A NUMBER OF 544 00:24:12,480 --> 00:24:14,640 INITIATIVES FROM DR. BERNARD'S 545 00:24:14,640 --> 00:24:17,280 OFFICE THE NIH CHIEF OFFICER FOR 546 00:24:17,280 --> 00:24:19,840 DIVERSITY AND A NUMBER OF 547 00:24:19,840 --> 00:24:23,160 INSTITUTES ARE CONTRIBUTING TO A 548 00:24:23,160 --> 00:24:26,480 POTENTIAL PRIZE FOR 549 00:24:26,480 --> 00:24:30,800 INSTITUTIONAL EXCELLENCE IN DEIA 550 00:24:30,800 --> 00:24:33,960 ISSUES AND NICHD IS CONTRIBUTING 551 00:24:33,960 --> 00:24:34,720 TO THIS. 552 00:24:34,720 --> 00:24:36,600 MY FELLOW DIRECTORS MIGHT WANT 553 00:24:36,600 --> 00:24:39,040 TO COMMENT AS TO WHETHER THEY 554 00:24:39,040 --> 00:24:40,120 ARE ALSO CONTRIBUTING. 555 00:24:40,120 --> 00:24:43,800 THIS IS FOCUSING ON THE 556 00:24:43,800 --> 00:24:46,080 EXTRAMURAL COURSE IN THE 557 00:24:46,080 --> 00:24:47,800 UNIVERSITY ARE UNIVERSITY AND 558 00:24:47,800 --> 00:24:49,120 CHANGING INSTITUTIONAL CULTURE 559 00:24:49,120 --> 00:24:51,800 IN HIGHER EDUCATION AND REALLY 560 00:24:51,800 --> 00:24:53,800 LOOKING FOR BEST PRACTICES. 561 00:24:53,800 --> 00:24:55,200 HIGHLIGHTING SYSTEMS. 562 00:24:55,200 --> 00:24:57,640 PROJECTS AND PROCESSES AND 563 00:24:57,640 --> 00:24:59,000 CREATE RESEARCH AND ENVIRONMENTS 564 00:24:59,000 --> 00:25:02,080 THAT PROMOTE DIVERSITY, EQUITY 565 00:25:02,080 --> 00:25:05,280 AND INCLUSION AND ACCESSIBILITY. 566 00:25:05,280 --> 00:25:08,400 THIS IS AN OPPORTUNITY FOR WHICH 567 00:25:08,400 --> 00:25:11,680 RESPONSES ARE DUE AT THE END OF 568 00:25:11,680 --> 00:25:13,720 JULY SO THERE IS THREE MONTHS OR 569 00:25:13,720 --> 00:25:19,680 SO THAT YOU COULD BE ABLE TO 570 00:25:19,680 --> 00:25:22,120 PARTICIPATE AND THERE IS A NOTE 571 00:25:22,120 --> 00:25:26,920 NOTICE OF SPECIAL INTEREST TO 572 00:25:26,920 --> 00:25:28,880 RECOGNIZE THIS AND THIS IS A 573 00:25:28,880 --> 00:25:30,680 WONDERFUL WAY OF RECOGNIZING 574 00:25:30,680 --> 00:25:32,080 FACULTY MEMBERS WHO HAVE 575 00:25:32,080 --> 00:25:33,720 DEMONSTRATED OUTSTANDING 576 00:25:33,720 --> 00:25:35,480 MENTORSHIP TRAINING ESPECIALLY 577 00:25:35,480 --> 00:25:37,040 TO INDIVIDUALS FROM GROUPS 578 00:25:37,040 --> 00:25:38,640 IDENTIFIED AS UNDER REPRESENTED 579 00:25:38,640 --> 00:25:42,160 IN THE BIOMEDICAL SCIENCES. 580 00:25:42,160 --> 00:25:44,000 UNFORTUNATELY THAT ONE HAS 581 00:25:44,000 --> 00:25:48,200 CLOSED BUT I WANTED YOU TO BE 582 00:25:48,200 --> 00:25:48,880 AWARE OF IT. 583 00:25:48,880 --> 00:25:50,800 THE LAST PART IS HIGHLIGHTING 584 00:25:50,800 --> 00:25:53,440 SOME ADVANCES IN MUSCULAR 585 00:25:53,440 --> 00:25:54,240 DYSTROPHY RESEARCH. 586 00:25:54,240 --> 00:25:57,320 AND SO I HAVE A NUMBER OF SLIDES 587 00:25:57,320 --> 00:26:01,480 FROM PUBLICATION THAT'S WE'VE 588 00:26:01,480 --> 00:26:03,040 SELECTED AND IF YOU THINK THERE 589 00:26:03,040 --> 00:26:07,120 IS SOMETHING MAJOR THAT WE 590 00:26:07,120 --> 00:26:09,840 SOMEHOW NEGLECTED TO MENTION 591 00:26:09,840 --> 00:26:11,320 FEEL FREE TO SPEAK UP AT THE 592 00:26:11,320 --> 00:26:12,320 END. 593 00:26:12,320 --> 00:26:14,400 THE FIRST ONE IS AND A NUMBER 594 00:26:14,400 --> 00:26:18,840 ARE FOCUSED ON BIOMARKERS AND IN 595 00:26:18,840 --> 00:26:22,120 PARTICULAR SEEKING NONINVASIVE 596 00:26:22,120 --> 00:26:25,520 BIOMARKERS OF HSHD AND 597 00:26:25,520 --> 00:26:29,280 CORRELATING THEM * WITH DISEASE 598 00:26:29,280 --> 00:26:30,680 PROGRESSION THAT COULD BE USED 599 00:26:30,680 --> 00:26:36,040 IN CLINICAL TRIALS AND THERAPY. 600 00:26:36,040 --> 00:26:47,120 FSHD -- -- ALSO LOOKING AT 601 00:26:47,120 --> 00:26:48,680 BIOMARKERS AND CLINICAL 602 00:26:48,680 --> 00:26:50,760 MEASURES. 603 00:26:50,760 --> 00:26:52,760 AND UNFORTUNATELY THERE WAS NOT 604 00:26:52,760 --> 00:26:54,880 ONE BIOMARKER THAT SHOWED A 605 00:26:54,880 --> 00:26:58,840 STRONG CORRELATION WITH OVER-ALL 606 00:26:58,840 --> 00:27:01,720 DISEASE PROJECTION. 607 00:27:01,720 --> 00:27:03,480 -- PROGRESSION. 608 00:27:03,480 --> 00:27:14,760 *. 609 00:27:14,760 --> 00:27:17,280 MRI IS A LITTLE BIT MORE 610 00:27:17,280 --> 00:27:23,360 NONINVASIVE AND A RELATIVELY NEW 611 00:27:23,360 --> 00:27:29,080 TECHNIQUE -- -- TIRM. 612 00:27:29,080 --> 00:27:31,520 AND PARTICULARLY LOOKING AT AN 613 00:27:31,520 --> 00:27:34,880 INCREASE IN FAT FRACTION AS 614 00:27:34,880 --> 00:27:36,600 ASSOCIATED WITH LATER STAGE 615 00:27:36,600 --> 00:27:37,640 DISEASE. 616 00:27:37,640 --> 00:27:39,960 AND WHILE THERE WAS NOT ONE 617 00:27:39,960 --> 00:27:43,480 IDEAL BIOMARKER, DATA IS HELPING 618 00:27:43,480 --> 00:27:48,800 TO GUIDE BIOMARKER SELECTION FOR 619 00:27:48,800 --> 00:27:49,360 FUTURE TRIALS. 620 00:27:49,360 --> 00:27:52,080 THERE IS A POTENTIAL NEW 621 00:27:52,080 --> 00:27:55,840 TREATMENT APPROACH FOR FSHD. 622 00:27:55,840 --> 00:27:57,440 MUSCLE TISSUE IS PARTICULARLY 623 00:27:57,440 --> 00:28:01,600 DIFFICULT TO TARGET. 624 00:28:01,600 --> 00:28:04,040 AND THIS STUDY IDENTIFIED 625 00:28:04,040 --> 00:28:05,920 SEVERAL NATURALLY OCCURRING 626 00:28:05,920 --> 00:28:11,720 MICRO RNAS THAT IS COULD INHIBIT 627 00:28:11,720 --> 00:28:13,960 DUX4 TRANSCRIPTION. 628 00:28:13,960 --> 00:28:24,760 MICRO RNA675 SUPPRESSES. 629 00:28:24,760 --> 00:28:29,160 AND DELIVERING -- BY GENE 630 00:28:29,160 --> 00:28:37,680 THERAPY IN A DUX4 MODEL LED TO A 631 00:28:37,680 --> 00:28:39,680 SIGNIFICANT REDUCTION. 632 00:28:39,680 --> 00:28:47,400 SMALL MOLECULES INCREASED THE 633 00:28:47,400 --> 00:28:53,320 LEVELS OF MIR675 AND ASSOCIATED 634 00:28:53,320 --> 00:28:56,960 DISEASE OUTCOMES IN 635 00:28:56,960 --> 00:28:58,280 PATIENTLY-DERIVED MUSCLE CELLS. 636 00:28:58,280 --> 00:29:02,880 THIS STUDY HAS BEEN SUPPORTED BY 637 00:29:02,880 --> 00:29:09,080 BOTH NIAMS AND THE PREVIOUS 638 00:29:09,080 --> 00:29:12,800 STUDY WAS SUPPORTED BY NIADS. 639 00:29:12,800 --> 00:29:16,120 THE NEXT STUDY IS ADDRESSING 640 00:29:16,120 --> 00:29:18,160 COGNITIVE FUNCTION IN DM1 641 00:29:18,160 --> 00:29:19,080 CLINICAL TRIALS. 642 00:29:19,080 --> 00:29:31,600 THIS WAS SUPPORTED BY N NINDS, AND 643 00:29:31,600 --> 00:29:32,280 THE MUSCULAR DYSTROPHY 644 00:29:32,280 --> 00:29:33,640 ASSOCIATION. 645 00:29:33,640 --> 00:29:36,920 THE BEST METHODS TO ADDRESS OR 646 00:29:36,920 --> 00:29:39,880 ASSESS -- ACROSS MULTIPLE SITES 647 00:29:39,880 --> 00:29:41,760 STILL REMAINS UNDETERMINED. 648 00:29:41,760 --> 00:29:45,080 SO THIS WAS A PERSPECTIVE 649 00:29:45,080 --> 00:29:47,640 LONGITUDINAL STUDY OF 113 650 00:29:47,640 --> 00:29:50,280 ADULTS. 651 00:29:50,280 --> 00:29:53,800 TO VALIDATE USE OF COMPUTERIZED 652 00:29:53,800 --> 00:29:55,360 TESTS AND THE RESULT SHOWED THAT 653 00:29:55,360 --> 00:29:59,120 IT WAS FEASIBLE TO USE 654 00:29:59,120 --> 00:30:01,160 COMPUTERIZED COGNITIVE TESTS IN 655 00:30:01,160 --> 00:30:02,600 MULTI-SITE STUDIES. 656 00:30:02,600 --> 00:30:08,320 ALSO THE LOWER PERFORMANCE IN 657 00:30:08,320 --> 00:30:09,640 WORKING MEMORY. 658 00:30:09,640 --> 00:30:13,160 AND FUTURE TESTS THAT INCLUDE 659 00:30:13,160 --> 00:30:18,320 MEASURES OF VISUAL SPATIAL 660 00:30:18,320 --> 00:30:28,680 PERFEC--PERCEPTION COULD BE VALID. 661 00:30:28,680 --> 00:30:34,280 -- A CLINICAL TRIAL TESTING 662 00:30:34,280 --> 00:30:43,960 COURT CENTER STRIDE -- COURT 663 00:30:43,960 --> 00:30:58,080 CO-STEROID DOSING REGIMEN. 664 00:30:58,080 --> 00:31:03,800 -- IT WAS THE DAILY REGIMEN THAT 665 00:31:03,800 --> 00:31:08,080 RESULTED IN SIGNIFICANTLY BETTER 666 00:31:08,080 --> 00:31:09,160 OUTCOMES. 667 00:31:09,160 --> 00:31:11,400 THERE WERE NO SIGNIFICANT 668 00:31:11,400 --> 00:31:12,240 DIFFERENCES BETWEEN THE TWO 669 00:31:12,240 --> 00:31:17,160 TYPES OF DAILY CORTICO STEROIDS. 670 00:31:17,160 --> 00:31:20,600 THE FUTURE * TRIALS WILL TEST 671 00:31:20,600 --> 00:31:25,720 NOVEL TREATMENT FOR DIFFERENT 672 00:31:25,720 --> 00:31:26,120 DOSAGE. 673 00:31:26,120 --> 00:31:28,000 FOR EXAMPLE A HIGHER DOSE. 674 00:31:28,000 --> 00:31:30,480 THIS STUDY DID SHOW A 675 00:31:30,480 --> 00:31:32,040 THERAPEUTIC ADVANTAGE BUT ALSO 676 00:31:32,040 --> 00:31:35,000 PROVIDES VALID BASELINE DATA AND 677 00:31:35,000 --> 00:31:37,640 THIS AS WELL IS PUBLISHED IN 678 00:31:37,640 --> 00:31:43,120 JAMA AND SUPPORTED BY NAINDS. 679 00:31:43,120 --> 00:31:46,680 AND THEN THERE IS UNDERSTANDING 680 00:31:46,680 --> 00:31:50,200 RESPIRATORY MUSCLE DEGENERATION. 681 00:31:50,200 --> 00:31:53,400 THERE IS NOT MUCH KNOWN ABOUT 682 00:31:53,400 --> 00:31:58,360 ABDOMINAL MUSCLE PATHOLOGY AND 683 00:31:58,360 --> 00:31:59,120 ITS RELATIONSHIP. 684 00:31:59,120 --> 00:32:01,800 HOWEVER THE LEVEL OF MUSCLE 685 00:32:01,800 --> 00:32:03,600 REPLACEMENT BY FAT CAN BE 686 00:32:03,600 --> 00:32:06,360 ESTIMATED USING M-R AND 687 00:32:06,360 --> 00:32:08,880 EXPRESSED AS A FAT FRACTION. 688 00:32:08,880 --> 00:32:11,360 INDIVIDUALS IN THIS STUDY WHO 689 00:32:11,360 --> 00:32:17,640 HAD DMD WHO UNDERWENT ABNORMAL 690 00:32:17,640 --> 00:32:20,640 MRI TO DETERMINE THE FAT 691 00:32:20,640 --> 00:32:21,600 FRACTION. 692 00:32:21,600 --> 00:32:23,720 AND THE DATA WERE USED TO 693 00:32:23,720 --> 00:32:27,600 ESTIMATE A MODEL EXPERFECT TORI 694 00:32:27,600 --> 00:32:31,280 MODEL * DEGENERATION. 695 00:32:31,280 --> 00:32:33,600 HERE AGAIN CORTICO STEROIDS WERE 696 00:32:33,600 --> 00:32:37,280 USED TO TREAT AND A MILD 697 00:32:37,280 --> 00:32:39,680 PHENOTYPE WERE ASSOCIATED WITH 698 00:32:39,680 --> 00:32:42,720 SLOWING OF EXPERFECT TORI MUSCLE 699 00:32:42,720 --> 00:32:44,120 FATTY IN FILTRATION. 700 00:32:44,120 --> 00:32:58,040 * AND THIS STUDY WAS SUPPORTED 701 00:32:58,040 --> 00:33:05,120 BY NIAMS, NHLBI AND NICHD. 702 00:33:05,120 --> 00:33:07,040 7 SO THE LAST STUDY THAT I WANT 703 00:33:07,040 --> 00:33:10,880 TO HIGHLIGHT IS A GENED ITING 704 00:33:10,880 --> 00:33:16,720 STUDY WITH CRISPR CAS9 IN A DOG 705 00:33:16,720 --> 00:33:17,280 MODEL. 706 00:33:17,280 --> 00:33:20,800 MOST OF WHAT WE'VE KNOWN TO DATE 707 00:33:20,800 --> 00:33:23,280 HAS BEEN DONE IN MICE. 708 00:33:23,280 --> 00:33:24,840 THESE ARE TWO STUDIES ONE OF 709 00:33:24,840 --> 00:33:31,760 WHICH WAS PUBLISHED IN MOLECULAR 710 00:33:31,760 --> 00:33:37,280 THERAPY -. 711 00:33:37,280 --> 00:33:41,160 THESE STUDIES COMPARED MUSCLE 712 00:33:41,160 --> 00:33:43,920 SPECIFIC TO GENE EDITING WITH 713 00:33:43,920 --> 00:33:48,880 CRISPR CASS 9 IN DOGS TO MICRO 714 00:33:48,880 --> 00:34:11,760 -- GENE PLACEMENT. 715 00:34:11,760 --> 00:34:15,840 -- DOG MODELS OF DMD AND 716 00:34:15,840 --> 00:34:18,480 IMPORTANTLY UNLIKE THE SITUATION 717 00:34:18,480 --> 00:34:22,680 IN MICE DOGS EXHIBITS A ROBUST 718 00:34:22,680 --> 00:34:23,680 NEW RESPONSE TO THE 719 00:34:23,680 --> 00:34:28,080 CAS9 L0 PROTEIN. 720 00:34:28,080 --> 00:34:32,080 THIS COULD BE MINIMIZED BY NOT 721 00:34:32,080 --> 00:34:33,840 ENTIRELY AVOIDED. 722 00:34:33,840 --> 00:34:38,640 IT DOES UNFORTUNATELY * A 723 00:34:38,640 --> 00:34:41,200 POTENTIAL ROADBLOCK TO THE 724 00:34:41,200 --> 00:34:43,320 THERAPEUTIC USES OF CRISPR 725 00:34:43,320 --> 00:34:44,480 TECHNOLOGY. 726 00:34:44,480 --> 00:34:47,680 A GOOD NEWS BAD NEWS STORY. 727 00:34:47,680 --> 00:34:50,200 SO THOSE ARE THE STUDIES THAT I 728 00:34:50,200 --> 00:34:52,400 WANTED TO HIGHLIGHT AT THIS 729 00:34:52,400 --> 00:34:54,880 POINT. 730 00:34:54,880 --> 00:34:55,800 WE'RE WELCOMING QUESTIONS. 731 00:34:55,800 --> 00:34:57,840 THERE ARE SOME IN THE CHAT BOX 732 00:34:57,840 --> 00:35:00,480 SO LET ME STOP SHARING. 733 00:35:00,480 --> 00:35:02,440 AND I JUST WANT TO MAKE NOTE OF 734 00:35:02,440 --> 00:35:06,920 THE FACT THAT THE NICHD 735 00:35:06,920 --> 00:35:09,480 BACKGROUND ACKNOWLEDGES THAT 736 00:35:09,480 --> 00:35:12,280 WE'RE IN 2022 CELEBRATING OUR 737 00:35:12,280 --> 00:35:15,320 60th ANNIVERSARY. 738 00:35:15,320 --> 00:35:22,680 SO NOW WE MOVE TO THE CHAT BOX. 739 00:35:22,680 --> 00:35:25,400 AND GLEN, DO YOU WANT TO COMMENT 740 00:35:25,400 --> 00:35:27,120 ON THE AWARDS. 741 00:35:27,120 --> 00:35:28,600 >> YOU HAD BROUGHT UP COMMITMENT 742 00:35:28,600 --> 00:35:31,280 TO SUPPORTING EARLY STAGE 743 00:35:31,280 --> 00:35:34,600 INVESTIGATORS FOR RO1s. 744 00:35:34,600 --> 00:35:36,800 NIH HAS ESTABLISHED A NEW 745 00:35:36,800 --> 00:35:39,080 PROGRAM SPECIFICALLY FOR EARLY 746 00:35:39,080 --> 00:35:41,760 STAGE INVESTIGATOR RO1s AND 747 00:35:41,760 --> 00:35:44,120 SPECIFICALLY FOR RESEARCHERS WHO 748 00:35:44,120 --> 00:35:45,360 ARE CHANGING THEIR RESEARCH 749 00:35:45,360 --> 00:35:47,120 DIRECTION AND THIS PROGRAM HAS 750 00:35:47,120 --> 00:35:50,440 BEEN NAMED AFTER STEVEN CATS. 751 00:35:50,440 --> 00:35:54,320 THE FORMER CHAIR AND FORMER 752 00:35:54,320 --> 00:35:57,920 DIRECTOR OF NAIMS. 753 00:35:57,920 --> 00:35:59,160 THAT PROGRAM HAS BEEN GOING ON 754 00:35:59,160 --> 00:36:00,960 FOR ABOUT A YEAR NOW AND 755 00:36:00,960 --> 00:36:03,640 HOPEFULLY IT'S HAVING A POSITIVE 756 00:36:03,640 --> 00:36:05,880 IMPACT IN ALLOWING JUNIOR 757 00:36:05,880 --> 00:36:07,760 INVESTIGATORS TO START EXCITING 758 00:36:07,760 --> 00:36:11,280 NEW PROJECTS AND NEW DIRECTIONS. 759 00:36:11,280 --> 00:36:12,520 >> GLEN, THANK YOU SO MUCH FOR 760 00:36:12,520 --> 00:36:13,560 BRINGING THAT UP. 761 00:36:13,560 --> 00:36:17,120 IT'S IMPORTANT FOR THE AUDIENCE 762 00:36:17,120 --> 00:36:19,360 TO KNOW THAT WHAT MAKES THE CATS 763 00:36:19,360 --> 00:36:21,680 AWARDS DIFFERENT IS THAT YOU 764 00:36:21,680 --> 00:36:23,880 DON'T NEED PRELIMINARY DATA. 765 00:36:23,880 --> 00:36:28,000 SO THE TYPICAL RO1 WOULD BE 766 00:36:28,000 --> 00:36:31,320 EXPECT TO HAVE DATA AND THE ESI 767 00:36:31,320 --> 00:36:32,680 THE EARLY STAGE INVESTIGATORS 768 00:36:32,680 --> 00:36:34,880 CAN BE FUNDED WITH A DIFFERENT 769 00:36:34,880 --> 00:36:36,160 PAY LINE. 770 00:36:36,160 --> 00:36:38,920 BUT THE CATS AWARDS REALLY 771 00:36:38,920 --> 00:36:40,400 HIGHLIGHT THE FACT THAT THIS IS 772 00:36:40,400 --> 00:36:44,880 A YOUNG INVESTIGATOR AND THE 773 00:36:44,880 --> 00:36:46,360 EVALUATIONS OR REVIEW OF THE 774 00:36:46,360 --> 00:36:49,960 GRANTS ARE MADE FOCUSING ON 775 00:36:49,960 --> 00:36:53,000 POTENTIAL AND NOT HAVING THE 776 00:36:53,000 --> 00:36:53,720 EXPECTATIONS OF PRELIMINARY 777 00:36:53,720 --> 00:36:54,760 DATA. 778 00:36:54,760 --> 00:37:05,360 SO IT'S A GREAT OPPORTUNITY. 779 00:37:05,360 --> 00:37:07,120 OTHER QUESTIONS? 780 00:37:07,120 --> 00:37:09,000 DAN, I SEE THAT YOU'VE SIGNED 781 00:37:09,000 --> 00:37:11,080 ON. 782 00:37:11,080 --> 00:37:14,680 I PUT IT FIRST 783 00:37:14,680 --> 00:37:17,760 >> THANK YOU, KINDLY. 784 00:37:17,760 --> 00:37:20,960 GOOD TO BE HERE. 785 00:37:20,960 --> 00:37:26,880 DANIEL PEREZ AND CO-FOUNDER AND 786 00:37:26,880 --> 00:37:30,640 DIRECTOR -- THANK YOU FOR HAVING 787 00:37:30,640 --> 00:37:31,200 ME HERE. 788 00:37:31,200 --> 00:37:36,840 I JUST HAD A QUESTION -- WILL WE 789 00:37:36,840 --> 00:37:40,000 BE REASSESSING THE FUNDING 790 00:37:40,000 --> 00:37:47,840 LEVELS -- THE COMPOSITION -- OUT 791 00:37:47,840 --> 00:37:59,720 OF $100 MILLION -- -- -- BECAUSE 792 00:37:59,720 --> 00:38:01,560 AS I LOOK AT THE FUNDING LEVEL 793 00:38:01,560 --> 00:38:06,600 FOR THE DISEASE -- IT IS 794 00:38:06,600 --> 00:38:09,520 DIFFICULT TO KNOW WHETHER OR NOT 795 00:38:09,520 --> 00:38:14,480 WE'RE ACTUALLY -- -- FOR THE 796 00:38:14,480 --> 00:38:15,240 DISCOVERY. 797 00:38:15,240 --> 00:38:18,240 AND I JUST DIDN'T KNOW IF THAT 798 00:38:18,240 --> 00:38:23,120 WAS UNDERLINED OR WHAT YOUR 799 00:38:23,120 --> 00:38:29,880 THINKING IS -- ---IN TERMS OF 800 00:38:29,880 --> 00:38:33,360 EXAMINING THAT -- --- 801 00:38:33,360 --> 00:38:36,520 >> THANK YOU FOR THAT QUESTION. 802 00:38:36,520 --> 00:38:41,400 GLEN AND WALTER, WOULD YOU WANT 803 00:38:41,400 --> 00:38:43,280 TO ADD YOUR CONTINUES AND WHAT 804 00:38:43,280 --> 00:38:45,280 YOU'RE SUGGESTING SOUNDS TO ME 805 00:38:45,280 --> 00:38:47,280 LIKE STRATEGIC PLANNING A. AND 806 00:38:47,280 --> 00:38:51,840 REALLY TAKING THE LONGVIEW OF * 807 00:38:51,840 --> 00:38:53,920 OF WHAT ARE WE DOING NOW. 808 00:38:53,920 --> 00:38:55,600 WHERE ARE THE GAPS AND WHERE DO 809 00:38:55,600 --> 00:38:58,480 WE NEED TO MOVE MORE QUICKLY. 810 00:38:58,480 --> 00:39:00,720 BUT MAYBE GLEN AND WALTER WANT 811 00:39:00,720 --> 00:39:03,040 TO ADD THEIR COMMENTS. 812 00:39:03,040 --> 00:39:04,600 >> WELL I'M GOING TO COMMENT 813 00:39:04,600 --> 00:39:06,920 THAT WE DON'T HAVE PLANS FOR 814 00:39:06,920 --> 00:39:08,200 TODAY'S MEETING TO GO BACK AND 815 00:39:08,200 --> 00:39:10,960 LOOK AT HISTORIC LEVELS AND 816 00:39:10,960 --> 00:39:12,200 CURRENT LEVELS OF FUNDING FOR 817 00:39:12,200 --> 00:39:13,680 THE DIFFERENT FORMS OF MUSCULAR 818 00:39:13,680 --> 00:39:14,080 DYSTROPHY. 819 00:39:14,080 --> 00:39:15,520 THAT INFORMATION IS AVAILABLE AT 820 00:39:15,520 --> 00:39:17,880 ANY TIME ON THE NIH WEBSITE ON 821 00:39:17,880 --> 00:39:20,080 THE NIH REPORTER. 822 00:39:20,080 --> 00:39:22,400 IT'S SOMETHING THAT WE CAN 823 00:39:22,400 --> 00:39:23,760 CONSIDER FOR PERHAPS THE FALL 824 00:39:23,760 --> 00:39:25,480 MEETING THAT WE COULD REVIEW 825 00:39:25,480 --> 00:39:27,360 WHERE WE ARE IN TERMS OF FUNDING 826 00:39:27,360 --> 00:39:29,760 LEVELS FOR DIFFERENT MUSCULAR 827 00:39:29,760 --> 00:39:35,080 DYSTROPHYs. 828 00:39:35,080 --> 00:39:41,760 >> -- -- WHEN I LOOK AT NIH RCDC 829 00:39:41,760 --> 00:39:46,080 -- RC -- 9:00 OR $10 MILLION -- 830 00:39:46,080 --> 00:39:49,560 BUT WHEN I LOOK AT NIH REPORTER 831 00:39:49,560 --> 00:39:55,880 -- I SEE ABOUT $18 MILLION. 832 00:39:55,880 --> 00:39:57,920 $17 MILLION TO $18 MILLION. 833 00:39:57,920 --> 00:40:00,560 THERE IS QUITE A DISCREPANCY 834 00:40:00,560 --> 00:40:04,440 BETWEEN THE TWO. 835 00:40:04,440 --> 00:40:08,080 I WONDERED WHETHER OR NOT -- WE 836 00:40:08,080 --> 00:40:09,840 MIGHT WANT TO TAKE A LOOK AT 837 00:40:09,840 --> 00:40:11,480 THAT. 838 00:40:11,480 --> 00:40:14,760 PROJECT THAT'S I SEE UNDER RCDC 839 00:40:14,760 --> 00:40:21,880 -- ARE TAGGED * -- APPROPRIATELY 840 00:40:21,880 --> 00:40:28,320 BUT ---THEY ARE NOT DEAD CENTER 841 00:40:28,320 --> 00:40:35,960 ON -- -- APPLICATION -- SO AGAIN 842 00:40:35,960 --> 00:40:41,720 THERE IS A $10 MILLION VERSUS 843 00:40:41,720 --> 00:40:43,440 $18 MILLION. 844 00:40:43,440 --> 00:40:49,880 CDC FUNDING. 845 00:40:49,880 --> 00:40:52,840 IT'S TOUGH TO KNOW -- THE 846 00:40:52,840 --> 00:40:55,280 $10 MILLION. 847 00:40:55,280 --> 00:41:00,320 WE'VE GONE DOWN -- SINCE FIVE 848 00:41:00,320 --> 00:41:04,080 YEARS AGO. 849 00:41:04,080 --> 00:41:12,200 -- AND -- -- -- WE'RE NOT REALLY 850 00:41:12,200 --> 00:41:15,720 IT'S NOT INCREASING. 851 00:41:15,720 --> 00:41:19,520 SO I'M LITTLE CONFUSED. 852 00:41:19,520 --> 00:41:24,080 IT'S UNCLEAR TO ME WHAT THE 853 00:41:24,080 --> 00:41:24,920 TRAJECTORY IS. 854 00:41:24,920 --> 00:41:26,840 >> I CAN MAKE A COMMENT BUT I'M 855 00:41:26,840 --> 00:41:28,880 GOING TO ACKNOWLEDGE LINDSEY AND 856 00:41:28,880 --> 00:41:31,160 THEN WALTER TURNED ON HIS VIDEO 857 00:41:31,160 --> 00:41:33,080 TOO. 858 00:41:33,080 --> 00:41:35,680 RCDC EACH CATEGORY IS NOT 859 00:41:35,680 --> 00:41:36,680 INDEPENDENT OF THE OTHER. 860 00:41:36,680 --> 00:41:40,560 THERE IS DUPLICATION IN RCDC AND 861 00:41:40,560 --> 00:41:42,440 THE REPORTER SO, ITS POSSIBLE 862 00:41:42,440 --> 00:41:44,040 THAT REPORTER HAS A LARGER 863 00:41:44,040 --> 00:41:47,080 AMOUNT OF DOLLARS BECAUSE THERE 864 00:41:47,080 --> 00:41:50,640 ARE OTHER GRANTS THAT ARE NOT 865 00:41:50,640 --> 00:41:54,840 FOCUSED EXCLUSIVELY ON FSHD BUT 866 00:41:54,840 --> 00:41:56,280 MENTIONED IN THE ABSTRACT OR 867 00:41:56,280 --> 00:41:58,160 SOMEWHERE IN THE APPLICATION SO 868 00:41:58,160 --> 00:42:01,040 IT GETS ADDED TO THAT CATEGORY 869 00:42:01,040 --> 00:42:04,480 BUT I'M GOING TO HAND IT OVER TO 870 00:42:04,480 --> 00:42:04,760 DR. GRISWALD 871 00:42:04,760 --> 00:42:06,160 >> THANK YOU FOR THAT QUESTION 872 00:42:06,160 --> 00:42:10,080 AND I JUST WAS GOING TO COMMENT 873 00:42:10,080 --> 00:42:12,720 THAT PERHAPS THE CRISWELL. 874 00:42:12,720 --> 00:42:14,480 -- *. 875 00:42:14,480 --> 00:42:18,280 THERE IS AN ENTIRE OFFICE 876 00:42:18,280 --> 00:42:20,040 DEVOTED TO PORTFOLIO ANALYSIS 877 00:42:20,040 --> 00:42:21,800 AND THEY ARE DEVELOPING AND 878 00:42:21,800 --> 00:42:23,360 APPLYING TOOLS THAT CAN HELP 879 00:42:23,360 --> 00:42:25,920 UNDERSTAND IT AT A DEEPER LEVEL 880 00:42:25,920 --> 00:42:28,640 THAT GOES BEYOND THE CODES. 881 00:42:28,640 --> 00:42:30,080 THE NATURE OF PORTFOLIO AND HOW 882 00:42:30,080 --> 00:42:32,520 THEY ARE EVOLVING OVER TIME. 883 00:42:32,520 --> 00:42:34,480 SO IF THERE ARE CHALLENGES WE 884 00:42:34,480 --> 00:42:37,280 MIGHT WANT TO CONSIDER REACHING 885 00:42:37,280 --> 00:42:41,080 OUT TO THEM. 886 00:42:41,080 --> 00:42:44,640 AND HE HAS BEEN RESPONSIVE TO 887 00:42:44,640 --> 00:42:49,080 SOME OUTREACH THAT WE'VE DONE AT 888 00:42:49,080 --> 00:42:50,480 NIAMS. 889 00:42:50,480 --> 00:42:50,880 >> WALTER 890 00:42:50,880 --> 00:42:55,280 >> DAN, THE RCDC WAS BUILT TO BE 891 00:42:55,280 --> 00:42:58,280 CONSISTENT YEAR TO YEAR A. AND 892 00:42:58,280 --> 00:43:00,840 IT CODES * FOR DISEASE AREAS. 893 00:43:00,840 --> 00:43:02,360 REPORTER DOES NOT DO THAT. 894 00:43:02,360 --> 00:43:03,920 REPORTER IS JUST ANYTHING THAT 895 00:43:03,920 --> 00:43:05,640 HAS ANYTHING TO DO WITH ANYTHING 896 00:43:05,640 --> 00:43:07,160 THAT IS EVEN CLOSE. 897 00:43:07,160 --> 00:43:09,280 SO YOU CANNOT RELY ON REPORTER 898 00:43:09,280 --> 00:43:12,760 FOR -- YOU CANNOT USE REPORTER 899 00:43:12,760 --> 00:43:14,000 TO TALK LATE THE DOLLARS. 900 00:43:14,000 --> 00:43:18,680 YOU HAVE TO STICK WITH RCDC. 901 00:43:18,680 --> 00:43:20,880 >> BUT EVEN RCDC IS NOT 902 00:43:20,880 --> 00:43:21,960 ACCURATE. 903 00:43:21,960 --> 00:43:24,520 THERE ARE OVERLAP SO WE'RE 904 00:43:24,520 --> 00:43:26,200 LOOKING AT CERTAIN TOPICS MORE 905 00:43:26,200 --> 00:43:27,000 THAN ONCE. 906 00:43:27,000 --> 00:43:30,320 SO IT DOESN'T GIVE YOU AN 907 00:43:30,320 --> 00:43:31,280 ABSOLUTE AMOUNT. 908 00:43:31,280 --> 00:43:32,880 THAT WOULD REQUIRE A LOT MORE 909 00:43:32,880 --> 00:43:38,840 WORK. 910 00:43:38,840 --> 00:43:41,600 >> THAT -- THE GRANTS THAT ARE 911 00:43:41,600 --> 00:43:44,800 IDENTIFIED IN THE RECORDER ARE 912 00:43:44,800 --> 00:43:49,960 ALL -- THEY ARE ALL ON TARGET. 913 00:43:49,960 --> 00:43:55,760 ---THE RCDC NUMBER IS THAT LOW. 914 00:43:55,760 --> 00:44:01,280 I UNDERSTAND WHAT YOU'RE SAYING. 915 00:44:01,280 --> 00:44:03,240 I DON'T THINK THEY ARE THAT FAR 916 00:44:03,240 --> 00:44:07,960 OFF THE MARK. 917 00:44:07,960 --> 00:44:11,480 BUT -- -- ANY WAY -- THANK YOU. 918 00:44:11,480 --> 00:44:13,440 THANK YOU. 919 00:44:13,440 --> 00:44:14,560 FOR YOUR QUESTION. 920 00:44:14,560 --> 00:44:26,440 OTHER QUESTIONS? 921 00:44:26,440 --> 00:44:27,920 I DON'T SEE ANY. 922 00:44:27,920 --> 00:44:29,960 PLEASE RAISE YOUR HAND OR PUT 923 00:44:29,960 --> 00:44:34,080 SOMETHING IN THE CHAT BOX. 924 00:44:34,080 --> 00:44:35,280 WE CAN GO ON. 925 00:44:35,280 --> 00:44:37,520 WE'RE ACTUALLY ON TIME. 926 00:44:37,520 --> 00:44:40,480 SO WE CAN MOVE ON TO THE NEXT 927 00:44:40,480 --> 00:44:41,840 SPEAKER. 928 00:44:41,840 --> 00:44:45,080 SO AS YOU KNOW WE WELCOME OTHER 929 00:44:45,080 --> 00:44:45,680 ORGANIZATIONS MEMBER 930 00:44:45,680 --> 00:44:47,680 ORGANIZATIONS TO HIGHLIGHT 931 00:44:47,680 --> 00:44:48,480 STUDIES THAT THEY HAVE 932 00:44:48,480 --> 00:44:50,360 SUPPORTED. 933 00:44:50,360 --> 00:44:52,200 TO SO OUR NEXT PRESENTATION WILL 934 00:44:52,200 --> 00:44:54,560 BE FROM DR. NATALIE STREET WHO'S 935 00:44:54,560 --> 00:44:56,680 A HEALTH SCIENTISTS FOR THE 936 00:44:56,680 --> 00:44:58,480 DIVISION OF HUMAN DEVELOPMENT 937 00:44:58,480 --> 00:45:00,040 AND DISABILITY AT THE CENTERS 938 00:45:00,040 --> 00:45:01,840 FOR DISEASE CONTROL AND 939 00:45:01,840 --> 00:45:02,960 PREVENTION. 940 00:45:02,960 --> 00:45:05,080 THERE HAVE BEEN SEVERAL EXCITING 941 00:45:05,080 --> 00:45:06,880 PAPERS REPORTING RESULTS FROM 942 00:45:06,880 --> 00:45:08,720 CDC FUNDED PROJECTS THAT HAVE 943 00:45:08,720 --> 00:45:10,880 BEEN PUBLISHED SINCE OUR LAST 944 00:45:10,880 --> 00:45:12,680 MEETING SO WE'RE VERY FORTUNATE 945 00:45:12,680 --> 00:45:14,480 TO HAVE NATALIE HERE TODAY TO 946 00:45:14,480 --> 00:45:16,080 TELL US A LITTLE BIT MORE ABOUT 947 00:45:16,080 --> 00:45:16,480 THEM. 948 00:45:16,480 --> 00:45:18,160 OVER TO YOU, NATALIE. 949 00:45:18,160 --> 00:45:20,520 THANK YOU FOR BEING HERE. 950 00:45:20,520 --> 00:45:22,480 >> THANK YOU FOR INVITING ME TO 951 00:45:22,480 --> 00:45:24,160 TALK ABOUT SOME OF THE RECENT 952 00:45:24,160 --> 00:45:25,680 FINDINGS THAT HAVE COME OUT. 953 00:45:25,680 --> 00:45:32,960 I'M GOING TO SHARE MY SCREEN AND 954 00:45:32,960 --> 00:45:36,480 SO -- LET ME KNOW -- WHEN YOU 955 00:45:36,480 --> 00:45:42,880 CAN SEE IT. 956 00:45:42,880 --> 00:45:45,800 DOES EVERYBODY SEE MY SCREEN? 957 00:45:45,800 --> 00:45:58,160 >> NOT YET. 958 00:45:58,160 --> 00:45:59,520 >> IT LOOKS LIKE IT SHOULD BE 959 00:45:59,520 --> 00:46:32,120 SHARING SO LET ME KNOW. 960 00:46:32,120 --> 00:46:32,680 >> ALL RIGHT. 961 00:46:32,680 --> 00:46:33,480 THANK YOU VERY MUCH. 962 00:46:33,480 --> 00:46:35,360 NOW YOU CAN SEE THE SCREEN. 963 00:46:35,360 --> 00:46:40,520 SO I THINK I CAN GO TO THE NEXT 964 00:46:40,520 --> 00:46:44,800 SLIDE. 965 00:46:44,800 --> 00:46:49,800 SO AS -- WE SAID EARLIER TODAY 966 00:46:49,800 --> 00:46:53,800 -- I'M GOING TO BE PRESENTING ON 967 00:46:53,800 --> 00:46:55,200 SOME RECENTLY PUBLISHED FINDINGS 968 00:46:55,200 --> 00:46:57,720 BUT I DO WANT TO GIVE A VERY 969 00:46:57,720 --> 00:47:02,800 BRIEF OVERVIEW OF -- FIRST SO 970 00:47:02,800 --> 00:47:08,160 THAT WE'RE ALL ON THE SAME PAGE. 971 00:47:08,160 --> 00:47:13,080 I'VE SUMMARIZED THE RECENTLY 972 00:47:13,080 --> 00:47:14,880 PUBLISHED FINDINGS AND THEN 973 00:47:14,880 --> 00:47:18,280 FUTURE CONTRIBUTIONS. 974 00:47:18,280 --> 00:47:19,360 MD STAR. 975 00:47:19,360 --> 00:47:22,480 *. 976 00:47:22,480 --> 00:47:26,480 SO AS YOU ARE AWARE *. 977 00:47:26,480 --> 00:47:30,760 BACK IN 2001 THE MD CARE ACT WAS 978 00:47:30,760 --> 00:47:33,520 PASSED AND AT THAT TIME ONE 979 00:47:33,520 --> 00:47:37,040 THING THAT IS FOR CDC TO CREATE 980 00:47:37,040 --> 00:47:38,720 A NATIONAL SURVEILLANCE PROGRAM 981 00:47:38,720 --> 00:47:40,000 FOR MUSCULAR DYSTROPHY. 982 00:47:40,000 --> 00:47:46,480 SO IN 2002 STARTED WITH FUNDING 983 00:47:46,480 --> 00:48:02,160 OF FOUR SITES. 984 00:48:02,160 --> 00:48:09,880 ON MD STAR NET IS A MULTI-SITE 985 00:48:09,880 --> 00:48:10,960 SURVEILLANCE SYSTEM FOR MUSCULAR 986 00:48:10,960 --> 00:48:13,600 DYSTROPHY AND FUNDED BY CDC. 987 00:48:13,600 --> 00:48:17,800 THROUGH ACTIVE SURVEILLANCE WE 988 00:48:17,800 --> 00:48:19,640 IDENTIFY ALL PEOPLE WITH 989 00:48:19,640 --> 00:48:21,280 ELIGIBLE MUSCULAR DYSTROPHIES 990 00:48:21,280 --> 00:48:22,880 WHO SEEK CLINICAL CARE AND 991 00:48:22,880 --> 00:48:24,360 FOLLOW THEM OVER TIME. 992 00:48:24,360 --> 00:48:27,480 THE SOURCES THAT WE USE FOR 993 00:48:27,480 --> 00:48:30,920 IDENTIFYING CASES INCLUDE MU ROW 994 00:48:30,920 --> 00:48:33,160 MUSCULAR CLINICS. 995 00:48:33,160 --> 00:48:36,240 VITAL RECORDS AND HOSPITALS AND 996 00:48:36,240 --> 00:48:38,880 OTHER OUTPATIENT CLINICS. 997 00:48:38,880 --> 00:48:44,960 EACH RECEIVES APPROVAL AND MANY 998 00:48:44,960 --> 00:48:47,840 HAVE PUBLIC HEALTH AUTHORITY TO 999 00:48:47,840 --> 00:48:48,840 CONDUCT SURVEILLANCE FOR 1000 00:48:48,840 --> 00:48:56,280 MUSCULAR DYSTROPHY. 1001 00:48:56,280 --> 00:48:58,000 THE FUNDED SITES HAVE CHANGED 1002 00:48:58,000 --> 00:49:00,560 OVER THE YEARS SO WE STARTED OFF 1003 00:49:00,560 --> 00:49:01,320 WITH FOUR. 1004 00:49:01,320 --> 00:49:05,440 THE DATA COLLECTION THEN STARTED 1005 00:49:05,440 --> 00:49:09,000 WITH -- BECKER AND WE'VE STARTED 1006 00:49:09,000 --> 00:49:15,080 DATA COLLECTION FOR OTHER MDs. 1007 00:49:15,080 --> 00:49:17,600 SO AS YOU CAN SEE FROM THE MAP 1008 00:49:17,600 --> 00:49:18,680 CURRENTLY WE HAVE SEVEN SITES 1009 00:49:18,680 --> 00:49:19,680 THAT ARE FUNDED. 1010 00:49:19,680 --> 00:49:23,440 SO THERE IS A PART OF FLORIDA 1011 00:49:23,440 --> 00:49:26,000 THAT IS FUNDED, OHIO, NORTH 1012 00:49:26,000 --> 00:49:27,360 CAROLINA, WESTERN NORTH 1013 00:49:27,360 --> 00:49:28,840 CAROLINA, SOUTH CAROLINA. 1014 00:49:28,840 --> 00:49:33,000 UTAH AND VIRGINIA. 1015 00:49:33,000 --> 00:49:37,720 AND WE USE DATA TO LOOK AT 1016 00:49:37,720 --> 00:49:39,880 DISPARITIES AS WELL AS 1017 00:49:39,880 --> 00:49:41,040 DIAGNOSIS, HEALTH CARE SERVICE 1018 00:49:41,040 --> 00:49:43,160 USE, COMPARISON OF ACTUAL CARE 1019 00:49:43,160 --> 00:49:45,480 AND RECOMMENDED CARE. 1020 00:49:45,480 --> 00:49:48,240 DISEASE PROGRESSION AND THE 1021 00:49:48,240 --> 00:49:50,240 ASSOCIATION OF TREATMENTSES ON 1022 00:49:50,240 --> 00:49:50,720 OUTCOMES. 1023 00:49:50,720 --> 00:49:52,640 PERFECT CALLLY WE CONDUCT 1024 00:49:52,640 --> 00:49:55,480 SURVEYS ON TOPIC THAT'S CANNOT 1025 00:49:55,480 --> 00:49:58,400 BE EASILY RESEARCHED USING THE 1026 00:49:58,400 --> 00:49:58,880 SURVEILLANCE DATA. 1027 00:49:58,880 --> 00:50:03,120 WE WILL BE FIELDING A SURVEY ON 1028 00:50:03,120 --> 00:50:06,360 ADULTS'S EXPERIENCES WITH COVID 1029 00:50:06,360 --> 00:50:08,400 AS WELL AS VACCINATION. 1030 00:50:08,400 --> 00:50:10,920 PAIN AND FATIGUE AND FREEING 1031 00:50:10,920 --> 00:50:18,520 NANCY AND INFERTILITY. 1032 00:50:18,520 --> 00:50:20,560 AND SOME OF THE RESULTS ARE WHAT 1033 00:50:20,560 --> 00:50:30,960 I WILL BE DISCUSSING NEXT. 1034 00:50:30,960 --> 00:50:33,120 SO NOW I'M GOING TO SWITCH OVER 1035 00:50:33,120 --> 00:50:35,640 TO SOME OF THE ARTICLES THAT 1036 00:50:35,640 --> 00:50:37,000 HAVE BEEN PUBLISHED WITHIN THE 1037 00:50:37,000 --> 00:50:49,720 PAST SIX MONTHS. 1038 00:50:49,720 --> 00:50:51,520 THIS ARTICLE RECENTLY PUBLISHED 1039 00:50:51,520 --> 00:50:55,880 IN MARCH AND THIS UPDATED THE 1040 00:50:55,880 --> 00:50:58,720 2009 PAPER THAT DESCRIBED THE 1041 00:50:58,720 --> 00:51:00,160 DIAGNOSTIC MILESTONES AND 1042 00:51:00,160 --> 00:51:03,200 AVERAGE AGE OF DIAGNOSIS IN 1043 00:51:03,200 --> 00:51:05,480 PATIENTS WITH DMD. 1044 00:51:05,480 --> 00:51:07,880 AND THIS STUDY WAS DONE IN A 1045 00:51:07,880 --> 00:51:13,680 COHORT OR OF MALES WHO WERE BORN 1046 00:51:13,680 --> 00:51:14,880 BETWEEN 2000 AND 2015. 1047 00:51:14,880 --> 00:51:16,960 AND LIKE THE PREVIOUS STUDY IT 1048 00:51:16,960 --> 00:51:18,920 LOOKED AT THE MILESTONES AND THE 1049 00:51:18,920 --> 00:51:23,240 MEAN AGES AT REACHING THOSE 1050 00:51:23,240 --> 00:51:24,480 MILESTONES. 1051 00:51:24,480 --> 00:51:29,280 FIRST SIGNS AND SYMPTOMS 2.7 1052 00:51:29,280 --> 00:51:29,480 YEARS. 1053 00:51:29,480 --> 00:51:35,760 FIRST CREATINE WAS 4.6. 1054 00:51:35,760 --> 00:51:38,040 DIAGNOSTIC WAS 4.9 YEARS. 1055 00:51:38,040 --> 00:51:44,280 THERE WAS AN AVERAGE TIME OF 2.2 1056 00:51:44,280 --> 00:51:47,960 YEARS AND THE MOST NOTABLE THING 1057 00:51:47,960 --> 00:51:51,160 IS THERE HAS NOT BEEN A CHANGE. 1058 00:51:51,160 --> 00:51:53,600 NO SIGNIFICANT DIFFERENCES OVER 1059 00:51:53,600 --> 00:51:55,000 TIMES IN WHICH THESE MILESTONES 1060 00:51:55,000 --> 00:51:56,080 HAVE BEEN REACHED SO 1061 00:51:56,080 --> 00:51:58,000 HIGHLIGHTING THAT WE NEED TO DO 1062 00:51:58,000 --> 00:52:02,200 MORE WORK ON EARLIER DIAGNOSIS. 1063 00:52:02,200 --> 00:52:09,160 NEXT SLIDE PLEASE. 1064 00:52:09,160 --> 00:52:10,880 SO THIS IS ANOTHER ARTICLE THAT 1065 00:52:10,880 --> 00:52:14,920 JUST CAME OUT. 1066 00:52:14,920 --> 00:52:17,680 INVESTIGATORS IDENTIFIED THE SUB 1067 00:52:17,680 --> 00:52:18,160 PHENOTYPES. 1068 00:52:18,160 --> 00:52:24,520 THEY ANALYZED A COHORT OF 268 1069 00:52:24,520 --> 00:52:27,880 MALES -- 1070 00:52:27,880 --> 00:52:30,440 [ AUDIO DIFFICULTIES ] 1071 00:52:30,440 --> 00:52:32,280 USING THE VARIABLES THAT ARE 1072 00:52:32,280 --> 00:52:33,400 LISTED HERE. 1073 00:52:33,400 --> 00:52:36,320 THE VARIABLES THAT THEY USED 1074 00:52:36,320 --> 00:52:42,840 WERE THE AGE AT LAST AMBULATION. 1075 00:52:42,840 --> 00:52:47,280 SUM OF -- SYMPTOM ONSET. 1076 00:52:47,280 --> 00:52:49,600 AND THEN THEY ELSE DETERMINED 1077 00:52:49,600 --> 00:52:54,320 THE SEVERITY LOOKING AT CARDIO 1078 00:52:54,320 --> 00:52:56,320 MYOPATHY ONSET. 1079 00:52:56,320 --> 00:52:59,680 AND ALSO INCORPORATING CORTICO 1080 00:52:59,680 --> 00:53:10,000 STEROID TREATMENTS. 1081 00:53:10,000 --> 00:53:15,280 FROM THESE VARIABLES FOUR SUB 1082 00:53:15,280 --> 00:53:16,520 PHENOTYPIC CLUSTERS WERE 1083 00:53:16,520 --> 00:53:18,480 IDENTIFIED. 1084 00:53:18,480 --> 00:53:22,720 WITH A MEAN AGE OF LOSS OF 1085 00:53:22,720 --> 00:53:25,960 AMBULATION BETWEEN 11 AND 9.4% 1086 00:53:25,960 --> 00:53:31,120 OF THOSE WERE STILL AM BUY 1087 00:53:31,120 --> 00:53:33,800 LATING. 1088 00:53:33,800 --> 00:53:37,080 -- ABOUT 90% WERE STILL * LIVING 1089 00:53:37,080 --> 00:53:44,240 AND THE MEAN AGE WAS 13.3 YEARS. 1090 00:53:44,240 --> 00:53:48,520 IN THE BLUE BOX IS THE CLASSICAL 1091 00:53:48,520 --> 00:53:50,760 DMD AND THEY ALSO HAD A MEAN AGE 1092 00:53:50,760 --> 00:53:53,960 OF LOSS OF AMBULATION OF LESS 1093 00:53:53,960 --> 00:53:55,520 THAN 11. 1094 00:53:55,520 --> 00:53:58,840 1% WAS STILL AM BUY LATING AS OF 1095 00:53:58,840 --> 00:54:03,840 THE LAST CLINIC VISIT. 1096 00:54:03,840 --> 00:54:16,560 66% OF THOSE WERE STILL LIVING. 1097 00:54:16,560 --> 00:54:20,480 THEY HAD LATER AGES AT DEATH AND 1098 00:54:20,480 --> 00:54:24,240 ON SET OF CARDIO MYOPATHY AND 1099 00:54:24,240 --> 00:54:25,480 INITIATION OF VENTILATION THAN 1100 00:54:25,480 --> 00:54:28,560 THE SEVERE GROUP. 1101 00:54:28,560 --> 00:54:31,480 THEN GOING DOWN TO THE GREEN 1102 00:54:31,480 --> 00:54:35,320 THIS WAS THE LATE AMBULATORY 1103 00:54:35,320 --> 00:54:37,800 GROUP AND THESE PATIENTS HAD 1104 00:54:37,800 --> 00:54:40,160 LATER AGES WHEN THEY STOPPED 1105 00:54:40,160 --> 00:54:41,360 AMBULATING. 1106 00:54:41,360 --> 00:54:44,200 30% WERE STILL AMBULATING. 1107 00:54:44,200 --> 00:54:49,280 THEY HAD EARLIER SYMPTOM ON SET. 1108 00:54:49,280 --> 00:54:55,880 HOWEVER 82.3% WERE STILL LIVING. 1109 00:54:55,880 --> 00:54:58,560 AND IS YOU MIGHT IMAGINE WAS THE 1110 00:54:58,560 --> 00:55:01,520 GROUP THAT HAD THE HIGHEST 1111 00:55:01,520 --> 00:55:04,680 PERCENTAGE OF CORTICO STEROID 1112 00:55:04,680 --> 00:55:05,800 USERS. 1113 00:55:05,800 --> 00:55:09,200 LASTLY THE CLINICAL BMD. 1114 00:55:09,200 --> 00:55:13,560 THEY HAD LATER AGES AT LOSS OF 1115 00:55:13,560 --> 00:55:20,600 AMBULATION AND 92% WAS STILL 1116 00:55:20,600 --> 00:55:22,040 AMBULATING AND ALL OF THE 1117 00:55:22,040 --> 00:55:23,800 INDIVIDUALS ARE STILL LIVING 1118 00:55:23,800 --> 00:55:29,680 WITH THE MEAN AGE OF 22 YEARS. 1119 00:55:29,680 --> 00:55:34,680 NEXT SLIDE PLEASE. 1120 00:55:34,680 --> 00:55:36,440 SO THIS IS ONE OF THE ARTICLES 1121 00:55:36,440 --> 00:55:38,760 THAT HAS COME OUT OF THE SURVEY 1122 00:55:38,760 --> 00:55:39,480 DATA. 1123 00:55:39,480 --> 00:55:41,880 AND SO THIS WAS A SURVEY THAT 1124 00:55:41,880 --> 00:55:52,880 WAS DONE I THINK BACK I 2011-12. 1125 00:55:52,880 --> 00:55:54,280 WE WERE REALLY TRYING TO 1126 00:55:54,280 --> 00:55:58,320 DETERMINE THE MEDICAL KNOWLEDGE, 1127 00:55:58,320 --> 00:56:01,680 HOW INDEPENDENT THIS GROUP WAS. 1128 00:56:01,680 --> 00:56:04,480 HOW THE PERCENTAGE WHO WERE 1129 00:56:04,480 --> 00:56:06,480 EMPLOYED AND LOOKED AT THEIR 1130 00:56:06,480 --> 00:56:08,400 RELATIONSHIPS. 1131 00:56:08,400 --> 00:56:09,600 OF 60 MALES THAT WERE IN THE 1132 00:56:09,600 --> 00:56:15,200 STUDY YOU CAN SEE THE VARYING 1133 00:56:15,200 --> 00:56:16,560 PERCENTAGE HERE OF THOSE THAT 1134 00:56:16,560 --> 00:56:17,160 WERE INDEPENDENT. 1135 00:56:17,160 --> 00:56:21,600 SO AS YOU CAN SEE THE GROUP THAT 1136 00:56:21,600 --> 00:56:24,240 IS HAD BECKERS WAS MORE 1137 00:56:24,240 --> 00:56:26,000 INDEPENDENT THAN THOSE WERE WITH 1138 00:56:26,000 --> 00:56:32,400 -- THOSE WITH BECKERS HAD A 1139 00:56:32,400 --> 00:56:34,760 HIGHER PERCENTAGE -- 1140 00:56:34,760 --> 00:56:36,240 [ AUDIO DIFFICULTIES ] 1141 00:56:36,240 --> 00:56:38,240 WITHOUT SUPERVISION AS WELL AS 1142 00:56:38,240 --> 00:56:40,200 PERFORMING TASKS OF DAILY 1143 00:56:40,200 --> 00:56:43,280 LIVING. 1144 00:56:43,280 --> 00:56:46,640 AND THEY ALWAYS WERE -- 1145 00:56:46,640 --> 00:56:48,480 MEDICATIONS IN 67% AND THERE WAS 1146 00:56:48,480 --> 00:56:52,600 A SIMILAR PERCENTAGE WHO HAD 1147 00:56:52,600 --> 00:56:55,160 EXPERIENCED -- ROMANTIC 1148 00:56:55,160 --> 00:56:56,160 RELATIONSHIPS. 1149 00:56:56,160 --> 00:56:57,960 WHAT THIS STUDY HIGHLIGHTED WAS 1150 00:56:57,960 --> 00:56:59,520 THE NEED FOR RESEARCH INTO THE 1151 00:56:59,520 --> 00:57:01,800 BARRIERS AND FACILITATORS OF 1152 00:57:01,800 --> 00:57:03,360 INDEPENDENCE IN MEN WITH 1153 00:57:03,360 --> 00:57:05,720 MUSCULAR DYSTROPHY. 1154 00:57:05,720 --> 00:57:15,320 NEXT SLIDE, PLEASE. 1155 00:57:15,320 --> 00:57:17,520 THIS NEXT ARTICLE ESTIMATED THE 1156 00:57:17,520 --> 00:57:19,080 PRODUCTIVITY COST OF A PARENT 1157 00:57:19,080 --> 00:57:22,120 CARING FOR A MALE WITH MUSCULAR 1158 00:57:22,120 --> 00:57:24,440 DYSTROPHY -- AND THIS WAS 1159 00:57:24,440 --> 00:57:26,320 PUBLISHED LAST FALL. 1160 00:57:26,320 --> 00:57:31,960 IT COMPARED DATA FROM THE SURVEY 1161 00:57:31,960 --> 00:57:34,080 -- FROM THE CURRENT POPULATION 1162 00:57:34,080 --> 00:57:35,520 SURVEY AND MATCHED ON THE STATE, 1163 00:57:35,520 --> 00:57:38,120 THE YEAR OF THE SURVEY, 1164 00:57:38,120 --> 00:57:40,120 RESPONDENT'S AGE, THE CHILD'S 1165 00:57:40,120 --> 00:57:41,960 AGE AND NUMBER OF MINOR CHILDREN 1166 00:57:41,960 --> 00:57:44,480 AND WHAT THE STUDY FOUND WAS 1167 00:57:44,480 --> 00:57:51,200 THAT CAREGIVERS OF BOYS WITH 1168 00:57:51,200 --> 00:57:52,920 DMMD WERE APPROXIMATELY 300 1169 00:57:52,920 --> 00:57:56,000 FEWER HOURS A YEAR CORRESPONDING 1170 00:57:56,000 --> 00:57:59,640 TO ABOUT A $9,000 LOSS IN 1171 00:57:59,640 --> 00:58:01,400 PREDICTED EARNINGS. 1172 00:58:01,400 --> 00:58:03,920 CAREGIVERS -- OF MORE THAN FOUR 1173 00:58:03,920 --> 00:58:07,040 YEARS OF AMBULATION LOSS WERE 1174 00:58:07,040 --> 00:58:15,560 LESS LIKELY TO WORK. 1175 00:58:15,560 --> 00:58:18,040 CAREGIVERS WHO LOST AMBULATION 1176 00:58:18,040 --> 00:58:19,800 WERE 48% LESS LIKELY TO WORK 1177 00:58:19,800 --> 00:58:22,760 THAN CAREGIVERS OF THOSE STILL 1178 00:58:22,760 --> 00:58:25,560 AMBULATING. 1179 00:58:25,560 --> 00:58:29,200 THE ANNUAL LOSS -- WAS ABOUT 1180 00:58:29,200 --> 00:58:37,600 $24,000 AND IT WAS $13,828 FOR 1181 00:58:37,600 --> 00:58:38,840 CAREGIVERS OF BOYS WITH LESS 1182 00:58:38,840 --> 00:58:40,640 THAN FOUR YEARS OF AMBULATION 1183 00:58:40,640 --> 00:58:41,960 LOSS. 1184 00:58:41,960 --> 00:58:44,520 THIS HIGHLIGHTS -- ONE OF THE 1185 00:58:44,520 --> 00:58:47,160 BENEFITS -- IS TO REALLY TRY TO 1186 00:58:47,160 --> 00:58:51,720 PROLONG AMBULATION AND ALSO 1187 00:58:51,720 --> 00:58:53,000 PROVIDE SUPPORTERS FOR 1188 00:58:53,000 --> 00:59:04,920 CAREGIVERS. 1189 00:59:04,920 --> 00:59:07,480 THE NEXT ARTICLE EXAMINED 1190 00:59:07,480 --> 00:59:10,480 ASSOCIATIONS BETWEEN CLINICAL 1191 00:59:10,480 --> 00:59:28,560 PREDICTORS, THEY ANALYZED THE 1192 00:59:28,560 --> 00:59:31,080 CURVATURE . 1193 00:59:31,080 --> 00:59:34,680 THE AFTERNOON MAXIMUM CURVE WAS 1194 00:59:34,680 --> 00:59:37,600 25 DEGREES AND 42.8 DEGREES 1195 00:59:37,600 --> 00:59:38,960 AMONG THOSE RECOMMENDED FOR 1196 00:59:38,960 --> 00:59:48,120 SURGERY. 1197 00:59:48,120 --> 00:59:51,160 OTHERS FOUND A HIGHER ADJUSTED 1198 00:59:51,160 --> 00:59:52,920 HAZARD RATIO FOR INDIVIDUALS WHO 1199 00:59:52,920 --> 00:59:55,680 HAD LOST AMBULATION WHO DID NOT 1200 00:59:55,680 --> 01:00:01,200 USE CORTICO STEROIDS AND THEN 1201 01:00:01,200 --> 01:00:03,840 AFTER LOSING AMBULATION. 1202 01:00:03,840 --> 01:00:05,680 THEY ARE MORE THAN TWICE AS 1203 01:00:05,680 --> 01:00:09,480 LIKELY TO UNDERGO SPINAL SURGERY 1204 01:00:09,480 --> 01:00:15,400 FOR SCOLIOSIS. 1205 01:00:15,400 --> 01:00:18,400 CORTICO STEROIDS MAY DELAY 1206 01:00:18,400 --> 01:00:30,800 SPINAL CURVATURE PROGRESSION. 1207 01:00:30,800 --> 01:00:32,880 SO THE OBJECTIVE OF THIS ARTICLE 1208 01:00:32,880 --> 01:00:38,080 WAS TO EVALUATE WHETHER CORTICO 1209 01:00:38,080 --> 01:00:40,480 STEROID TREATMENT IS BENEFIT FOR 1210 01:00:40,480 --> 01:00:43,480 CARDIAC OR PULMONARY FUNCTIONS 1211 01:00:43,480 --> 01:00:49,240 A. INVESTIGATORS ANALYZED THE 1212 01:00:49,240 --> 01:00:50,640 ASSOCIATION -- 1213 01:00:50,640 --> 01:01:00,120 [ AUDIO DIFFICULTIES ] 1214 01:01:00,120 --> 01:01:04,120 YOU CAN SEE THE NUMBER OF 1215 01:01:04,120 --> 01:01:06,960 INDIVIDUALS IN EACH CART 1216 01:01:06,960 --> 01:01:25,080 CO-STEROID GROUP. 1217 01:01:25,080 --> 01:01:27,560 THIS STUDY FOUND NO DIFFERENCE 1218 01:01:27,560 --> 01:01:32,600 IN THE AGE -- BY CORTICO STEROID 1219 01:01:32,600 --> 01:01:33,680 USE GROUPS. 1220 01:01:33,680 --> 01:01:35,640 HOWEVER THERE WAS A DIFFERENCE 1221 01:01:35,640 --> 01:01:40,760 AT FIRST ABNORMAL PPFEC IN MALES 1222 01:01:40,760 --> 01:01:42,560 TREATED MORE THAN A YEAR AFTER 1223 01:01:42,560 --> 01:01:43,920 LOSS OF AMBULATION. 1224 01:01:43,920 --> 01:01:47,760 AND ON THE CARDIAC FINDING THE 1225 01:01:47,760 --> 01:01:51,320 AUTHORS HAD PROPOSED THAT THE 1226 01:01:51,320 --> 01:01:53,960 SHORTER TIME FROM LOSS TO 1227 01:01:53,960 --> 01:01:56,920 AMBULATION TOGETHER ON SET OF 1228 01:01:56,920 --> 01:01:59,920 ABNORMAL LEFT VENTRICULAR 1229 01:01:59,920 --> 01:02:03,480 FUNCTION FOR BOYS WHOM STEROID 1230 01:02:03,480 --> 01:02:05,360 TREATMENT CONTINUED BEYOND -- 1231 01:02:05,360 --> 01:02:08,880 LIKELY REFLECTS THE -- 1232 01:02:08,880 --> 01:02:10,360 [ INDISCERNIBLE ] 1233 01:02:10,360 --> 01:02:14,480 LOSS OF AMBULATION AND ON SET OF 1234 01:02:14,480 --> 01:02:21,080 ABNORMAL LEFT CONVENIENT TRICK R 1235 01:02:21,080 --> 01:02:39,200 -- VENTRICULAR FUNCTION. 1236 01:02:39,200 --> 01:02:45,040 -- THE ASSOCIATION BETWEEN 1237 01:02:45,040 --> 01:02:46,160 GLUCOCORTICOIDS AND ALL CAUSE 1238 01:02:46,160 --> 01:02:50,200 MORTALITY. 1239 01:02:50,200 --> 01:02:55,880 MEDIAN SURVIVAL TIME WAS 23.7 1240 01:02:55,880 --> 01:02:58,200 YEARS. 1241 01:02:58,200 --> 01:03:01,200 AFTER LOSS OF AMBULATION -- 1242 01:03:01,200 --> 01:03:04,080 USERS HAD LOWER RISK OF DEATH 1243 01:03:04,080 --> 01:03:05,760 THAN NONUSERS. 1244 01:03:05,760 --> 01:03:07,400 WE DO HAVE PLANS TO SHARE MORE 1245 01:03:07,400 --> 01:03:09,200 ABOUT THE FINDINGS THROUGH 1246 01:03:09,200 --> 01:03:11,400 VARIOUS CHANNELS OVER THE NEXT 1247 01:03:11,400 --> 01:03:14,200 FEW WEEKS. 1248 01:03:14,200 --> 01:03:22,400 NEXT SLIDE PLEASE. 1249 01:03:22,400 --> 01:03:25,160 AS YOU MAY HAVE NOTICED THESE 1250 01:03:25,160 --> 01:03:28,560 RECENT PUBLICATIONS ARE 1251 01:03:28,560 --> 01:03:29,840 PRIMARILY ABOUT MUSCULAR 1252 01:03:29,840 --> 01:03:30,320 DYSTROPHY. 1253 01:03:30,320 --> 01:03:31,840 HOWEVER SEVERAL PROJECTS USING 1254 01:03:31,840 --> 01:03:33,280 DATA ON OTHER TYPES OF MUSCULAR 1255 01:03:33,280 --> 01:03:35,520 DYSTROPHY ARE IN VARIOUS PHASES 1256 01:03:35,520 --> 01:03:36,600 OF DEVELOPMENT. 1257 01:03:36,600 --> 01:03:37,760 AND SOME OF THE KEY PROJECTS 1258 01:03:37,760 --> 01:03:45,280 INCLUDE -- DYSTROPHY -- THAT 1259 01:03:45,280 --> 01:03:47,120 LOOKS AT VARIOUS CLUSTERS AND 1260 01:03:47,120 --> 01:03:48,320 THE TYPES OF SYMPTOMS THAT WE 1261 01:03:48,320 --> 01:03:50,280 HAVE IN EACH ONE. 1262 01:03:50,280 --> 01:03:55,680 THERE IS -- EPIDEMIOLOGY AND 1263 01:03:55,680 --> 01:03:59,120 DIAGNOSIS IN -- DISTAL MUSCULAR 1264 01:03:59,120 --> 01:04:00,360 DYSTROPHY. 1265 01:04:00,360 --> 01:04:03,720 LOOKING AT SOME OF THE -- 1266 01:04:03,720 --> 01:04:05,880 VARIANTS ALSO WITHIN THE 1267 01:04:05,880 --> 01:04:08,120 MUTATIONS OR WITHIN THE GENES. 1268 01:04:08,120 --> 01:04:11,880 THERE IS ALSO A RESPIRATORY 1269 01:04:11,880 --> 01:04:14,320 FUNCTION, EVALUATION AND 1270 01:04:14,320 --> 01:04:18,480 OUTCOMES PAPERS BEING DEVELOPED 1271 01:04:18,480 --> 01:04:25,720 FOR -- DM AND FSHD. 1272 01:04:25,720 --> 01:04:27,920 ANOTHER ONE THAT IS UNDERWAY AS 1273 01:04:27,920 --> 01:04:28,360 WELL. 1274 01:04:28,360 --> 01:04:30,160 AND THEN WHAT WE'RE GOING TO BE 1275 01:04:30,160 --> 01:04:33,000 LOOKING AT SHORTLY WITH DATA 1276 01:04:33,000 --> 01:04:36,240 THAT IS GOING TO BE COMING OUT 1277 01:04:36,240 --> 01:04:39,520 THIS YEAR IS PREVALENCE IN 1278 01:04:39,520 --> 01:04:41,560 SURVIVAL BY MUSCULAR DYSTROPHY 1279 01:04:41,560 --> 01:04:47,280 TYPE SO THAT WOULD BE MY TONIC 1280 01:04:47,280 --> 01:04:49,480 DYSTROPHY, FSHD. 1281 01:04:49,480 --> 01:04:53,840 -- -- AND WE'LL ALSO UPDATE * 1282 01:04:53,840 --> 01:04:57,360 UPDATE NUMBERS IN A COUPLE OF 1283 01:04:57,360 --> 01:04:57,920 YEARS. 1284 01:04:57,920 --> 01:05:00,280 AND A NOTE ABOUT THAT PREVALENCE 1285 01:05:00,280 --> 01:05:02,280 IS THAT WE DO RECOGNIZE THAT 1286 01:05:02,280 --> 01:05:05,000 THIS IS GOING TO BE A CLINICAL 1287 01:05:05,000 --> 01:05:09,400 PREVALENCE SO IT WILL BE A 1288 01:05:09,400 --> 01:05:10,120 CONSERVATIVE ESTIMATE AND THAT 1289 01:05:10,120 --> 01:05:12,800 WILL BE ADDRESSED WITHIN THE 1290 01:05:12,800 --> 01:05:14,240 PAPERS WHEN THEY COME OUT. 1291 01:05:14,240 --> 01:05:16,680 SO I THINK THAT IS ALL I HAVE. 1292 01:05:16,680 --> 01:05:17,920 SO NEXT SLIDE, PLEASE. 1293 01:05:17,920 --> 01:05:40,920 ANY QUESTIONS? 1294 01:05:40,920 --> 01:05:42,600 >> VERY NICE, NATALIE. 1295 01:05:42,600 --> 01:05:43,120 THANK YOU SO MUCH. 1296 01:05:43,120 --> 01:05:44,280 7 1297 01:05:44,280 --> 01:05:49,600 >> YOU'RE WELCOMED. 1298 01:05:49,600 --> 01:05:51,400 >> I DON'T SEE QUESTIONS IN THE 1299 01:05:51,400 --> 01:05:53,160 CHAT BOX. 1300 01:05:53,160 --> 01:05:55,320 IT WAS A GREAT PRESENTATION. 1301 01:05:55,320 --> 01:05:58,000 I THINK WE'RE HEARING FROM 1302 01:05:58,000 --> 01:06:22,520 MULTIPLE DIFFERENT ROUNDS. 1303 01:06:22,520 --> 01:06:25,280 >> I WANT TO THANK YOU FOR THE 1304 01:06:25,280 --> 01:06:25,880 UPDATE. 1305 01:06:25,880 --> 01:06:27,880 NEXT WE'RE GOING TO INTRODUCE 1306 01:06:27,880 --> 01:06:31,680 AND WELCOME THREE MEMBERS OUR 1307 01:06:31,680 --> 01:06:33,400 NEW MEMBERS AND WE'RE GOING TO 1308 01:06:33,400 --> 01:06:36,280 INVITE THEM TO BRIEFLY TELL 1309 01:06:36,280 --> 01:06:37,240 ABOUT THEMSELVES AND THE 1310 01:06:37,240 --> 01:06:38,040 ORGANIZATIONS THAT THEY 1311 01:06:38,040 --> 01:06:38,600 REPRESENT. 1312 01:06:38,600 --> 01:06:41,680 SO FIRST UP IS DENISE MYLER 1313 01:06:41,680 --> 01:06:44,240 WHO'S REPRESENTING THE IDAHO 1314 01:06:44,240 --> 01:06:46,680 CAREGIVERS ALLIANCE AND THE 1315 01:06:46,680 --> 01:06:49,800 IDAHO STATE INDEPENDENT LIVING 1316 01:06:49,800 --> 01:06:50,120 COUNCIL. 1317 01:06:50,120 --> 01:06:52,040 WELCOME, DENISE AND I DON'T SEE 1318 01:06:52,040 --> 01:06:53,680 YOUR VIDEO ON. 1319 01:06:53,680 --> 01:06:56,880 SO -- THERE YOU ARE. 1320 01:06:56,880 --> 01:06:57,360 GREAT. 1321 01:06:57,360 --> 01:06:58,120 WELCOME. 1322 01:06:58,120 --> 01:07:01,040 >> THANK YOU, DIANA. 1323 01:07:01,040 --> 01:07:04,240 IT'S -- GOING TO BE FUN TO BE 1324 01:07:04,240 --> 01:07:05,480 PART OF THIS GROUP. 1325 01:07:05,480 --> 01:07:08,200 I'VE LEARNED A LOT ALREADY. 1326 01:07:08,200 --> 01:07:11,280 I LIVE IN EASTERN IDAHO. 1327 01:07:11,280 --> 01:07:14,000 TO GIVE YOU A LITTLE GEOGRAPHY 1328 01:07:14,000 --> 01:07:16,000 I'M ABOUT 90 MINUTES TO 1329 01:07:16,000 --> 01:07:19,080 YELLOWSTONE AND I'M A FOUR HOUR 1330 01:07:19,080 --> 01:07:22,000 DRIVE SOUTH TO SALT LAKE CITY. 1331 01:07:22,000 --> 01:07:25,640 I WAS BORN WITH CLUB FEET. 1332 01:07:25,640 --> 01:07:28,800 AND A NEUROMUSCULAR PROBLEM. 1333 01:07:28,800 --> 01:07:32,680 THE CLUB FEET CAUSED THE DOCTORS 1334 01:07:32,680 --> 01:07:35,560 TO SLOWLY PULL MY FEET OUT WITH 1335 01:07:35,560 --> 01:07:37,200 CASTS FOR THE FIRST YEAR OF MY 1336 01:07:37,200 --> 01:07:37,760 LIFE. 1337 01:07:37,760 --> 01:07:40,280 AS I STARTED TO STAND AND WALK I 1338 01:07:40,280 --> 01:07:43,920 STARTED TO SEVERAL A LOWER CURVE 1339 01:07:43,920 --> 01:07:46,200 AND I HAD A TWO INCH DIFFERENCE 1340 01:07:46,200 --> 01:07:48,440 BETWEEN MY LEFT LEG AND MY RIGHT 1341 01:07:48,440 --> 01:07:49,360 LEG. 1342 01:07:49,360 --> 01:07:54,080 THE DOCTORS TRIED TO DO A CATH 1343 01:07:54,080 --> 01:07:57,040 BIOPSY TO DETERMINE EXACTLY MY 1344 01:07:57,040 --> 01:07:58,560 NEUROLOGICAL DISABILITY. 1345 01:07:58,560 --> 01:08:02,320 BUT WERE UNABLE TO GET ANY CLEAR 1346 01:08:02,320 --> 01:08:02,880 EVIDENCE 1347 01:08:02,880 --> 01:08:04,680 THEN THEY TRIED TO OPERATE ON MY 1348 01:08:04,680 --> 01:08:06,920 RIGHT KNEE TO TRY TO EQUAL OUT 1349 01:08:06,920 --> 01:08:09,760 THE LENGTH OF MY LEGS AND MISSED 1350 01:08:09,760 --> 01:08:12,080 IT BY JUST A LITTLE BIT. 1351 01:08:12,080 --> 01:08:14,840 IT WASN'T UNTIL I WAS 60 YEARS 1352 01:08:14,840 --> 01:08:17,960 OLD I'M NOW 64 SO ETC. ADJUST 1353 01:08:17,960 --> 01:08:19,520 BEEN WITHIN THE LAST FOUR YEARS 1354 01:08:19,520 --> 01:08:23,480 THAT I'VE GOTTEN MY DIAGNOSIS OF 1355 01:08:23,480 --> 01:08:25,720 HAVING LIMB GIRDLE MUSCULAR 1356 01:08:25,720 --> 01:08:26,120 DYSTROPHY. 1357 01:08:26,120 --> 01:08:28,840 AND I USE EITHER A POWER 1358 01:08:28,840 --> 01:08:31,920 WHEELCHAIR OR A WALKER TO HELP 1359 01:08:31,920 --> 01:08:34,080 MY MOBILITY BECAUSE WHEN I WALK 1360 01:08:34,080 --> 01:08:41,280 I HAVE A SWAYING GAIT AND I AM 1361 01:08:41,280 --> 01:08:42,400 GLAD TO KNOW THE TYPE OF 1362 01:08:42,400 --> 01:08:44,480 MUSCULAR DYSTROPHY I'VE GOT SO I 1363 01:08:44,480 --> 01:08:46,680 CAN MAKE PLANS FOR MY FUTURE. 1364 01:08:46,680 --> 01:08:51,560 I'VE BEEN A DISABLED ADVOCATE 1365 01:08:51,560 --> 01:08:53,240 SINCE 1990 AND I'VE BEEN 1366 01:08:53,240 --> 01:08:57,120 INVOLVED WITH A WIDE VARIETY OF 1367 01:08:57,120 --> 01:08:59,120 ORGANIZATIONS SOME WITH 1368 01:08:59,120 --> 01:09:00,680 TRANSPORTATION STATEWIDE. 1369 01:09:00,680 --> 01:09:02,200 MY LOCAL CENTER FOR INDEPENDENT 1370 01:09:02,200 --> 01:09:02,440 LIVING. 1371 01:09:02,440 --> 01:09:07,880 AND I'M JUST WRAPPING UP MY TERM 1372 01:09:07,880 --> 01:09:09,520 ON IDAHO STATE INDEPENDENT 1373 01:09:09,520 --> 01:09:11,320 LIVING COUNCIL AND I'VE GOTTEN 1374 01:09:11,320 --> 01:09:12,560 INVOLVED WITH THE IDAHO 1375 01:09:12,560 --> 01:09:15,760 CAREGIVERS ALLIANCE AND THE 1376 01:09:15,760 --> 01:09:17,440 AGING AND DISABLED RESOURCE 1377 01:09:17,440 --> 01:09:18,200 CENTER. 1378 01:09:18,200 --> 01:09:20,080 THE ALZHEIMER'S DISEASE AND 1379 01:09:20,080 --> 01:09:23,880 RELATED DEMENTIA AND THE 1380 01:09:23,880 --> 01:09:25,960 CONSORTIUM FOR IDAHOANS WITH 1381 01:09:25,960 --> 01:09:26,440 DISABILITIES. 1382 01:09:26,440 --> 01:09:28,880 LET ME TELL YOU MORE ABOUT THE 1383 01:09:28,880 --> 01:09:30,840 ADULT CAREGIVERS ALLIANCE. 1384 01:09:30,840 --> 01:09:32,880 A COALITION OF INDIVIDUALS AND 1385 01:09:32,880 --> 01:09:35,080 ORGANIZATIONS FOCUSED ON 1386 01:09:35,080 --> 01:09:37,040 EXPANDING THE OPPORTUNITIES OF 1387 01:09:37,040 --> 01:09:39,240 RESPID ACROSS THE LIFE-SPAN AND 1388 01:09:39,240 --> 01:09:44,760 TO A ADVANCE THE WELL-BEING OF 1389 01:09:44,760 --> 01:09:46,760 CAREGIVERS BY PROMOTING 1390 01:09:46,760 --> 01:09:48,400 COLLABORATION THAT IT IMPROVES 1391 01:09:48,400 --> 01:09:50,720 ACCESS TO QUALITY SUPPORTS AND 1392 01:09:50,720 --> 01:09:51,520 SERVICES. 1393 01:09:51,520 --> 01:09:52,040 ONE 1394 01:09:52,040 --> 01:09:55,480 MAJOR PROJECTS OF THE IDAHO CARE 1395 01:09:55,480 --> 01:09:57,800 GIVER ALLIANCE IS WHAT WE CALL A 1396 01:09:57,800 --> 01:10:04,200 FAMILY NAVIGATOR, FAMILY CARE 1397 01:10:04,200 --> 01:10:05,680 GIVER NAVIGATOR AND WHAT THAT 1398 01:10:05,680 --> 01:10:10,640 DOES IS WE DO AN ASSESS MEANT OF 1399 01:10:10,640 --> 01:10:12,320 A CARE GIVER THAT IS HAVING 1400 01:10:12,320 --> 01:10:14,680 PROBLEMS AND THEN WE BUILD A 1401 01:10:14,680 --> 01:10:16,320 CARE PLAN THAT HELPS THAT CARE 1402 01:10:16,320 --> 01:10:21,120 GIVER HAVE OTHER RESOURCES OTHER 1403 01:10:21,120 --> 01:10:22,960 SERVICES THEY CAN CONTACT AND WE 1404 01:10:22,960 --> 01:10:24,520 HELP MAKE THOSE CONTACTS WITH 1405 01:10:24,520 --> 01:10:26,080 THEM. 1406 01:10:26,080 --> 01:10:28,440 AND THE PROGRAM STARTED IN BOISE 1407 01:10:28,440 --> 01:10:30,760 AND IS NOW EXPANDING INTO 1408 01:10:30,760 --> 01:10:33,960 EASTERN IDEA. 1409 01:10:33,960 --> 01:10:35,640 AND THEN THE IDAHO STATE 1410 01:10:35,640 --> 01:10:37,520 INDEPENDENT LIVING COUNCIL IS 1411 01:10:37,520 --> 01:10:39,680 ACROSS DISABILITY ORGANIZATIONS. 1412 01:10:39,680 --> 01:10:42,360 IT'S RAN BY PEOPLE WITH 1413 01:10:42,360 --> 01:10:43,600 DISABILITIES. 1414 01:10:43,600 --> 01:10:46,680 AND WE WORK ON DISABLED ISSUES 1415 01:10:46,680 --> 01:10:54,520 SUCH AS EQUAL ACCESS -- TO JOBS 1416 01:10:54,520 --> 01:11:00,800 -- HOUSING -- HEALTH CARE -- WE 1417 01:11:00,800 --> 01:11:04,720 WORK ON SYSTEMATIC CHANGES IN 1418 01:11:04,720 --> 01:11:09,000 PROGRAMS FROM HUD TO HHS TO 1419 01:11:09,000 --> 01:11:10,880 LABOR, EDUCATION. 1420 01:11:10,880 --> 01:11:15,080 AND PROBABLY ONE OF THE BIG 1421 01:11:15,080 --> 01:11:17,440 THINGS THAT THE SILK HAS BEEN 1422 01:11:17,440 --> 01:11:19,160 ABLE TO ACCOMPLISH IS WE GOT A 1423 01:11:19,160 --> 01:11:22,600 TABLE A SEAT AT THE EMERGENCY 1424 01:11:22,600 --> 01:11:25,040 MANAGEMENT TABLE AND WHEN COVID 1425 01:11:25,040 --> 01:11:26,840 HIT BEING AT THAT TABLE PROVED 1426 01:11:26,840 --> 01:11:30,040 TO BE A BIG BLESSING BECAUSE WE 1427 01:11:30,040 --> 01:11:35,200 WERE ABLE TO TELL THE EMERGENCY 1428 01:11:35,200 --> 01:11:38,760 MANAGEMENT AND OTHER RESULTED 1429 01:11:38,760 --> 01:11:40,360 GROUPS THIS IS WHAT HAPPENS IN 1430 01:11:40,360 --> 01:11:43,960 THE DISABILITY COMMUNITY. 1431 01:11:43,960 --> 01:11:49,040 AND ONE OF THE FOCUSES FOR THE 1432 01:11:49,040 --> 01:11:51,040 FUTURE OF SILK IS IN IDAHO WE 1433 01:11:51,040 --> 01:11:55,120 HAVE A HORRID HOUSING CRISIS AND 1434 01:11:55,120 --> 01:11:58,240 A DIRECTION CARE WORKFORCE 1435 01:11:58,240 --> 01:12:00,240 CRISIS AND THEY ARE SO 1436 01:12:00,240 --> 01:12:08,520 INTERUPTED THAINTEREST-- INTERUPTED -- IT' S TIME TO 1437 01:12:08,520 --> 01:12:10,440 UNWIND THEM AND GET SOME HELP. 1438 01:12:10,440 --> 01:12:11,440 THANK YOU, DIANE 1439 01:12:11,440 --> 01:12:13,760 >> THANK YOU, DENISE FOR YOUR 1440 01:12:13,760 --> 01:12:15,080 PERSONAL AND LIVED EXPERIENCE. 1441 01:12:15,080 --> 01:12:17,280 I'M JUST CURIOUS. 1442 01:12:17,280 --> 01:12:19,080 HOW MUCH DO YOU THINK THE DELAY 1443 01:12:19,080 --> 01:12:20,960 IN YOUR DIAGNOSIS WAS RELATED TO 1444 01:12:20,960 --> 01:12:24,640 YOU LIVING IN A RELATIVELY RURAL 1445 01:12:24,640 --> 01:12:26,360 OR UNDERSERVED AREA? 1446 01:12:26,360 --> 01:12:29,560 IF YOU LIVED IN A MORE OF A 1447 01:12:29,560 --> 01:12:32,080 METROPOLITAN AREA YOU WOULD HAVE 1448 01:12:32,080 --> 01:12:33,760 HAVE BEEN DIAGNOSED EARLIER? 1449 01:12:33,760 --> 01:12:34,240 >> NO. 1450 01:12:34,240 --> 01:12:36,760 BECAUSE I SPENT A WEEK WITHIN 1451 01:12:36,760 --> 01:12:40,440 WHEN I WAS 10 YEARS OLD AT THE 1452 01:12:40,440 --> 01:12:43,520 UNIVERSITY OF UTAH WHICH IS THE 1453 01:12:43,520 --> 01:12:47,040 MDA CLINIC IS THAT I GO TO AND 1454 01:12:47,040 --> 01:12:52,000 THAT WAS IN ABOUT '68. 1455 01:12:52,000 --> 01:12:54,840 AND THEY COULD NOT EVEN SPENDING 1456 01:12:54,840 --> 01:12:56,400 A WEEK THERE AND POKING AND 1457 01:12:56,400 --> 01:12:58,600 PRODDING AND WHATEVER THEY STILL 1458 01:12:58,600 --> 01:13:00,480 HAD TROUBLES COMING UP WITH IT 1459 01:13:00,480 --> 01:13:03,120 SO I THINK PART OF THE PROBLEM 1460 01:13:03,120 --> 01:13:07,360 OF WHY IT TOOK SO LONG TO GET MY 1461 01:13:07,360 --> 01:13:09,920 DIAGNOSIS WAS THE MEDICINE AT 1462 01:13:09,920 --> 01:13:13,560 THE TIME IN THE '60s WAS NOT 1463 01:13:13,560 --> 01:13:16,520 SOPHISTICATED ENOUGH TO 1464 01:13:16,520 --> 01:13:19,000 DISTINGUISH THE EXACT AND KIND 1465 01:13:19,000 --> 01:13:21,040 OF NEUROMUSCULAR PROBLEMS THAT I 1466 01:13:21,040 --> 01:13:27,040 HAD. 1467 01:13:27,040 --> 01:13:27,640 >> INTERESTING 1468 01:13:27,640 --> 01:13:28,320 OTHER QUESTIONS? 1469 01:13:28,320 --> 01:13:31,200 >> A FOLLOW-UP QUESTION TO 1470 01:13:31,200 --> 01:13:31,960 YOURS. 1471 01:13:31,960 --> 01:13:36,240 IN THE LAST 10 YEARS WERE YOU 1472 01:13:36,240 --> 01:13:37,800 SEEKING FOR YOUR DIAGNOSIS AND 1473 01:13:37,800 --> 01:13:43,640 HOW IT WAS THAT WHEN WE HAVE 1474 01:13:43,640 --> 01:13:49,480 MUCH MORE TECHNICAL 1475 01:13:49,480 --> 01:13:52,600 OPPORTUNITIES FOR DIAGNOSING OR 1476 01:13:52,600 --> 01:13:55,080 WERE YOU NOT SEEKING IN THE LAST 1477 01:13:55,080 --> 01:13:56,240 10 YEARS? 1478 01:13:56,240 --> 01:13:58,560 >> I'VE BEEN SEEKING FOR A 1479 01:13:58,560 --> 01:14:00,120 DIAGNOSIS MY WHOLE LIFE SO WHEN 1480 01:14:00,120 --> 01:14:03,200 I WAS ABLE TO GET ONE OF MY 1481 01:14:03,200 --> 01:14:04,880 DOCTORS HERE IN IDAHO FALLS DID 1482 01:14:04,880 --> 01:14:07,520 A TEST ON MY LEGS AND HE WENT -- 1483 01:14:07,520 --> 01:14:09,160 SOMETHING IS NOT KOSHER. 1484 01:14:09,160 --> 01:14:15,680 SO HE SENT ME TO THE MDA CLINIC. 1485 01:14:15,680 --> 01:14:19,880 AND I HAVE BEEN TALKING WITH 1486 01:14:19,880 --> 01:14:24,280 DR. MARK BRAMBERG AND HE WAS THE 1487 01:14:24,280 --> 01:14:25,960 ONE THAT ABOUT THE SECOND TIME 1488 01:14:25,960 --> 01:14:31,120 WE MET SAID THAT I HAD THE LIMB 1489 01:14:31,120 --> 01:14:33,680 GIRDLE AFTER HE DID SOME 1490 01:14:33,680 --> 01:14:34,680 ADDITIONAL TESTING. 1491 01:14:34,680 --> 01:14:37,560 SO IT WAS REALLY -- IT'S BEEN 1492 01:14:37,560 --> 01:14:38,560 REALLY GOOD TO HAVE THAT 1493 01:14:38,560 --> 01:14:42,640 INFORMATION. 1494 01:14:42,640 --> 01:14:44,200 BECAUSE I USE THAT INFORMATION 1495 01:14:44,200 --> 01:14:46,080 TO THEN SAY OKAY. 1496 01:14:46,080 --> 01:14:48,920 HERE IS MY BASE. 1497 01:14:48,920 --> 01:14:50,880 I'VE GOT LIMB GIRDLE MUSCULAR 1498 01:14:50,880 --> 01:14:52,360 DYSTROPHY AND THEN START 1499 01:14:52,360 --> 01:14:54,160 STACKING EVERYTHING ON TOP OF 1500 01:14:54,160 --> 01:14:58,560 THAT AND SO BEES ABLE TO SAY 1501 01:14:58,560 --> 01:15:00,320 WELL IT'S NOT YOUR MUSCULAR 1502 01:15:00,320 --> 01:15:02,280 DYSTROPHY SO WE'LL RULE THAT OUT 1503 01:15:02,280 --> 01:15:04,840 HAS MADE IT EASIER FOR ME TO 1504 01:15:04,840 --> 01:15:07,280 WORK WITH MY DOCTORS IN THE TYPE 1505 01:15:07,280 --> 01:15:10,360 OF HEALTH CARE AND OTHER THINGS 1506 01:15:10,360 --> 01:15:11,560 I NEED. 1507 01:15:11,560 --> 01:15:16,200 >> WOULD YOU -- MEDICALLY 1508 01:15:16,200 --> 01:15:17,800 TESTED? 1509 01:15:17,800 --> 01:15:20,440 >> IT WAS A LIMITED GENETIC 1510 01:15:20,440 --> 01:15:22,520 TEST. 1511 01:15:22,520 --> 01:15:27,520 THE MORE ACCURATE OR SPECIFIC 1512 01:15:27,520 --> 01:15:32,040 GENETIC TESTING IS NOT 1513 01:15:32,040 --> 01:15:34,160 AFFORDABLE BECAUSE MY INSURANCE 1514 01:15:34,160 --> 01:15:37,120 I'M ON MEDICARE AND MEDICAID AND 1515 01:15:37,120 --> 01:15:38,600 THEY WON'T PAY FOR IT 1516 01:15:38,600 --> 01:15:46,880 >> I THINK JANE -- -- FOUNDATION 1517 01:15:46,880 --> 01:15:47,600 -- 1518 01:15:47,600 --> 01:15:50,040 >> THEY ARE NO LONGER DOING 1519 01:15:50,040 --> 01:15:52,320 GENETIC TESTING. 1520 01:15:52,320 --> 01:15:54,280 IN MUSCULAR DYSTROPHY. 1521 01:15:54,280 --> 01:15:56,000 BECAUSE THAT WAS ONE OF THE 1522 01:15:56,000 --> 01:16:00,200 OTHER TESTS THAT WE HAD WAND TO 1523 01:16:00,200 --> 01:16:04,400 DO AND -- 1524 01:16:04,400 --> 01:16:11,440 >> I TALKED -- LIMB GIRDLE 1525 01:16:11,440 --> 01:16:13,360 DISEASES -- DOES ANYONE ON THE 1526 01:16:13,360 --> 01:16:14,680 PANEL KNOW WHAT I'M TALKING 1527 01:16:14,680 --> 01:16:21,680 ABOUT? 1528 01:16:21,680 --> 01:16:22,560 OKAY. 1529 01:16:22,560 --> 01:16:23,600 I'LL LOOK INTO IT. 1530 01:16:23,600 --> 01:16:26,720 THANK YOU. 1531 01:16:26,720 --> 01:16:30,920 >> DONOVAN HAS SUBJECTED INVITA. 1532 01:16:30,920 --> 01:16:32,280 THAT THEY ARE DOING TESTING FOR 1533 01:16:32,280 --> 01:16:32,880 FREE. 1534 01:16:32,880 --> 01:16:34,120 >> THANK YOU, DONOVAN. 1535 01:16:34,120 --> 01:16:39,720 I WILL MENTION THAT TO 1536 01:16:39,720 --> 01:16:41,240 DR. BRAMBERG WHEN WE MEET NEXT 1537 01:16:41,240 --> 01:16:42,200 MONTH 1538 01:16:42,200 --> 01:16:44,520 >> AND ANDREA CLIVE HAS ALSO 1539 01:16:44,520 --> 01:16:50,480 CONFIRMED THAT. 1540 01:16:50,480 --> 01:16:52,120 THAT WOULD BE SOMETHING TO 1541 01:16:52,120 --> 01:16:52,720 PURSUE. 1542 01:16:52,720 --> 01:16:54,400 THE OTHER THING I WAS THINKING 1543 01:16:54,400 --> 01:16:57,480 ABOUT IS ALL OF US BECAUSE WE 1544 01:16:57,480 --> 01:16:59,640 WOULD GET FREE SEQUENCING WITH 1545 01:16:59,640 --> 01:17:02,240 ALL OF US BUT THEY ARE MUCH MORE 1546 01:17:02,240 --> 01:17:03,520 INTERESTED BECAUSE OF 1547 01:17:03,520 --> 01:17:05,240 THERAPEUTIC IMPLICATIONS. 1548 01:17:05,240 --> 01:17:08,240 I WOULD DEFINITELY START WITH 1549 01:17:08,240 --> 01:17:10,840 THEM. 1550 01:17:10,840 --> 01:17:13,760 >> -- A POINT OF JUST TO ADD A 1551 01:17:13,760 --> 01:17:15,360 COMMENT -- I THINK THERE IS A 1552 01:17:15,360 --> 01:17:21,560 LOT OF MISDIAGNOSIS US NOT A LOT 1553 01:17:21,560 --> 01:17:25,040 -- BUT NOT UNCOMMON THAT 1554 01:17:25,040 --> 01:17:27,680 SOMEBODY WITH LIMB GIRDLE IS 1555 01:17:27,680 --> 01:17:34,280 ALSO -- -- -- WHAT IS AN ISSUE 1556 01:17:34,280 --> 01:17:37,040 IS WHEN THE -- MATERIALS GET 1557 01:17:37,040 --> 01:17:40,960 INTO THE REGISTRIES AND THEY ARE 1558 01:17:40,960 --> 01:17:46,480 MISS CHARACTERIZED -- -- ---AND 1559 01:17:46,480 --> 01:17:58,120 THE CELL LINES -- -- AND SO TO 1560 01:17:58,120 --> 01:18:00,800 IMAGINE THAT -- PEOPLE ARE 1561 01:18:00,800 --> 01:18:04,360 ACCESSING MATERIAL FROM THE 1562 01:18:04,360 --> 01:18:09,080 REPOSITORIES THAT ARE -- -- -- 1563 01:18:09,080 --> 01:18:11,720 -- THEY ARE LOOKING AT THE 1564 01:18:11,720 --> 01:18:15,040 GENETICS OF THEIR CELL LINES. 1565 01:18:15,040 --> 01:18:22,800 THEY WERE NOT LOOKING AT -- --- 1566 01:18:22,800 --> 01:18:25,680 ---SO OVER THE YEARS I THINK 1567 01:18:25,680 --> 01:18:27,240 IT'S ALWAYS IMPORTANT TO 1568 01:18:27,240 --> 01:18:30,320 UNDERSTAND HOW IS IT THAT WE 1569 01:18:30,320 --> 01:18:33,920 CAME TO THE DETERMINATION THAT 1570 01:18:33,920 --> 01:18:41,600 THIS IS -- IS IT GENETICALLY. 1571 01:18:41,600 --> 01:18:43,280 METABOLICALLY. 1572 01:18:43,280 --> 01:18:44,000 CLINICALLY? 1573 01:18:44,000 --> 01:18:49,480 JUST A COMMENT. 1574 01:18:49,480 --> 01:18:51,320 BECAUSE I THINK WE CAN REALLY 1575 01:18:51,320 --> 01:18:52,320 SLOW DOWN RESEARCH. 1576 01:18:52,320 --> 01:18:54,720 THANK YOU. 1577 01:18:54,720 --> 01:18:57,400 >> THANK YOU, DAN. 1578 01:18:57,400 --> 01:18:59,960 ANDREA KLEIN HAS SAID AND PLEASE 1579 01:18:59,960 --> 01:19:01,760 ANDREA FEEL FREE TO COME OFF 1580 01:19:01,760 --> 01:19:03,560 MUTE IF YOU WOULD LIKE TO. 1581 01:19:03,560 --> 01:19:06,880 YOUR SUBTYPE OF LIMB GIRDLE WILL 1582 01:19:06,880 --> 01:19:09,040 HELP GUIDE YOU TO KNOW ABOUT 1583 01:19:09,040 --> 01:19:15,440 CARDIAC AND RESPIRATORY MUSCLE 1584 01:19:15,440 --> 01:19:16,520 INVOLVEMENT. 1585 01:19:16,520 --> 01:19:18,800 AND DONOVAN DECKER HAS ALSO 1586 01:19:18,800 --> 01:19:21,200 PROVIDED HIS E-MAIL ADDRESS TO 1587 01:19:21,200 --> 01:19:23,880 HELP YOU FACILITATE THE 1588 01:19:23,880 --> 01:19:25,560 CONNECTION AND GLEN IS GOING TO 1589 01:19:25,560 --> 01:19:28,400 GET THAT TO YOU, DENISE. 1590 01:19:28,400 --> 01:19:28,880 >> TERRIFIC. 1591 01:19:28,880 --> 01:19:30,800 THANK YOU. 1592 01:19:30,800 --> 01:19:34,680 >> AND THEN YOU CAN GIVE US 1593 01:19:34,680 --> 01:19:38,240 FOLLOW-UP IF YOU DO GET 1594 01:19:38,240 --> 01:19:40,240 SEQUENCING AND THEN HOPEFULLY 1595 01:19:40,240 --> 01:19:42,880 YOU'LL FIND OUT A CONFIRMATION 1596 01:19:42,880 --> 01:19:44,560 FROM A GENETIC PERSPECTIVE 1597 01:19:44,560 --> 01:19:46,960 >> I WILL DO THAT, DIANA. 1598 01:19:46,960 --> 01:19:48,520 BECAUSE THAT WOULD BE VERY NICE 1599 01:19:48,520 --> 01:19:50,080 TO KNOW. 1600 01:19:50,080 --> 01:19:51,560 I'M ALWAYS A SPONGE TRYING TO 1601 01:19:51,560 --> 01:19:53,640 GET AS MUCH INFORMATION AS I 1602 01:19:53,640 --> 01:19:54,560 CAN. 1603 01:19:54,560 --> 01:19:55,880 >> WELL IT SOUNDS LIKE YOU 1604 01:19:55,880 --> 01:19:57,240 DIDN'T GET TO THE POINT AND DAN 1605 01:19:57,240 --> 01:19:59,920 WAS MAKING THE POINT THAT THERE 1606 01:19:59,920 --> 01:20:02,600 WAS CONFUSION -- BUT IT SOUNDS 1607 01:20:02,600 --> 01:20:05,520 LIKE YOU DIDN'T GET TO THAT 1608 01:20:05,520 --> 01:20:05,720 POINT. 1609 01:20:05,720 --> 01:20:07,240 THEY KNEW YOU HAD SOMETHING BUT 1610 01:20:07,240 --> 01:20:09,360 NEVER SPECIFIED. 1611 01:20:09,360 --> 01:20:11,520 >> CORRECT. 1612 01:20:11,520 --> 01:20:12,680 >> OKAY. 1613 01:20:12,680 --> 01:20:14,440 WELL AGAIN THANK YOU SO MUCH FOR 1614 01:20:14,440 --> 01:20:16,200 SHARING YOUR PERSONAL STORY AND 1615 01:20:16,200 --> 01:20:19,080 OUR NEXT NEW MEMBER IS DR. ERIC 1616 01:20:19,080 --> 01:20:23,840 WANG WHO'S REPRESENTING THE 1617 01:20:23,840 --> 01:20:26,120 MYOTONIC DYSTROPHY FOUNDATION 1618 01:20:26,120 --> 01:20:32,400 AND ALSO AN ASSOCIATE PROFESSOR 1619 01:20:32,400 --> 01:20:34,880 FOR NEURO GENETICS FROM THE 1620 01:20:34,880 --> 01:20:36,200 UNIVERSITY OF FLORIDA. 1621 01:20:36,200 --> 01:20:36,800 WELCOME. 1622 01:20:36,800 --> 01:20:38,680 >> THANK YOU. 1623 01:20:38,680 --> 01:20:41,000 THANK YOU SO MUCH. 1624 01:20:41,000 --> 01:20:49,440 SO * I AM HERE TO REPRESENT THE 1625 01:20:49,440 --> 01:20:50,880 MYOTONIC DYSTROPHY FOUNDATION 1626 01:20:50,880 --> 01:20:53,760 AND IN MY CAPACITY AT THE 1627 01:20:53,760 --> 01:20:55,560 UNIVERSITY OF FLORIDA I'M ALSO 1628 01:20:55,560 --> 01:20:59,840 DEPARTMENT OF MICROBIOLOGY. 1629 01:20:59,840 --> 01:21:02,720 SO I WOULD START OFF WITH SOME 1630 01:21:02,720 --> 01:21:06,080 PERSON BACKGROUND ON WHY I STUDY 1631 01:21:06,080 --> 01:21:07,720 THIS AND WHY I GOT INVOLVED AND 1632 01:21:07,720 --> 01:21:10,640 THAT STARTS WITH -- THIS IS A 1633 01:21:10,640 --> 01:21:12,680 PICTURE OF ME AND MY DAD WHEN I 1634 01:21:12,680 --> 01:21:16,360 THINK WE HAD RETURNED FROM A 1635 01:21:16,360 --> 01:21:20,480 TRIP BACK TO TAIWAN WHERE MY 1636 01:21:20,480 --> 01:21:22,480 GRANDPARENTS LIVE AND IT LETS ME 1637 01:21:22,480 --> 01:21:26,680 -- THE KID -- AND MY DAD WAS 1638 01:21:26,680 --> 01:21:30,320 VERY HEALTHY AND A VERY STRONG 1639 01:21:30,320 --> 01:21:30,960 GUY. 1640 01:21:30,960 --> 01:21:33,200 VERY SHARP GUY AND WHEN I WAS 1641 01:21:33,200 --> 01:21:38,320 AROUND 8 HE ENDED UP GETTING A 1642 01:21:38,320 --> 01:21:40,360 DIAGNOSED WITH MYOTONIC 1643 01:21:40,360 --> 01:21:41,080 DYSTROPHY. 1644 01:21:41,080 --> 01:21:42,880 EXPERIENCING PROBLEMS WITH HIS 1645 01:21:42,880 --> 01:21:45,080 HANDS WHERE HE WOULD USE A 1646 01:21:45,080 --> 01:21:46,760 HAMMER OR GRAB ON TO SOMETHING 1647 01:21:46,760 --> 01:21:49,960 TIGHTLY AND COULD NOT LET GO 1648 01:21:49,960 --> 01:21:54,200 EASILY AND THAT IS THE SYMPTOM. 1649 01:21:54,200 --> 01:21:56,680 AND THAT CAUSED HIM TO GET A 1650 01:21:56,680 --> 01:21:58,760 DIAGNOSIS AND IN 89 WHICH WAS 1651 01:21:58,760 --> 01:22:02,480 BEFORE THE GENE WAS FORMALLY 1652 01:22:02,480 --> 01:22:04,160 DISCOVERED SO HIS DIAGNOSES WAS 1653 01:22:04,160 --> 01:22:09,720 DONE BY A MUSCLE BIOPSY AND 1654 01:22:09,720 --> 01:22:10,760 CLINICAL EXAM. 1655 01:22:10,760 --> 01:22:12,560 SO I'VE WATCHED THE DISEASE 1656 01:22:12,560 --> 01:22:15,520 PROGRESS OVER MANY YEARS. 1657 01:22:15,520 --> 01:22:18,760 IT'S A HIGHLY MULTI-SYSTEMATIC 1658 01:22:18,760 --> 01:22:19,680 DISEASE. 1659 01:22:19,680 --> 01:22:22,480 AFFECTING NOT JUST THE SKELETAL 1660 01:22:22,480 --> 01:22:24,040 MUSCLE BUT ALSO THE CARDIAC 1661 01:22:24,040 --> 01:22:26,360 MUSCLE AND THE CNS AND A NUMBER 1662 01:22:26,360 --> 01:22:27,800 OF OTHER TISSUES. 1663 01:22:27,800 --> 01:22:30,240 THIS IS ME AND MY WIFE AND 1664 01:22:30,240 --> 01:22:32,800 PARENTS -- A FEW YEARS AGO NOW 1665 01:22:32,800 --> 01:22:34,000 HERE IN FLORIDA. 1666 01:22:34,000 --> 01:22:37,040 AND YOU CAN APPRECIATE MY DAD 1667 01:22:37,040 --> 01:22:39,120 HAS GOT A LOT WEAKER. 1668 01:22:39,120 --> 01:22:44,760 HE HAS A CARDIO DEFIBRILLATOR TO 1669 01:22:44,760 --> 01:22:47,200 DEAL WITH THE ARRHYTHMIAS THAT 1670 01:22:47,200 --> 01:22:48,200 CAN HAPPEN. 1671 01:22:48,200 --> 01:22:53,680 AND I WOULD SAY THAT AMONG MANY 1672 01:22:53,680 --> 01:22:58,400 OTHER -- PATIENTS -- HIS CNS 1673 01:22:58,400 --> 01:23:00,600 ASPECTS -- HE IS EXTREMELY 1674 01:23:00,600 --> 01:23:01,360 SHARP. 1675 01:23:01,360 --> 01:23:03,920 SO THEY MAY NOT AFFECTED HIM AS 1676 01:23:03,920 --> 01:23:05,640 MUCH THROUGH THE A LOT OF HIS 1677 01:23:05,640 --> 01:23:06,960 ADULTHOOD BUT HE DEFINITELY 1678 01:23:06,960 --> 01:23:09,280 DEALS WITH A LOT OF THE FATIGUE 1679 01:23:09,280 --> 01:23:11,720 AND THE HYPER SOMNIA. 1680 01:23:11,720 --> 01:23:14,200 AND EVEN BRAIN FOG THAT COMES 1681 01:23:14,200 --> 01:23:15,880 ALONG WITH THIS DISEASE. 1682 01:23:15,880 --> 01:23:18,720 SO THIS IS RECENTLY BUT IN 1683 01:23:18,720 --> 01:23:20,880 BETWEEN THEM I HAD GONE TO 1684 01:23:20,880 --> 01:23:23,000 COLLEGE AT HARVARD AND THEN 1685 01:23:23,000 --> 01:23:24,880 DECIDED TO WORK IN A RESEARCH 1686 01:23:24,880 --> 01:23:27,560 LAB AFTERWARDS. 1687 01:23:27,560 --> 01:23:30,440 I WORKED ON SOME SCIENTIFIC 1688 01:23:30,440 --> 01:23:32,160 TOPICS UNREPRESENTED. 1689 01:23:32,160 --> 01:23:34,000 I WANTED TO GET INVOLVED IN 1690 01:23:34,000 --> 01:23:36,920 RESEARCH AFTER COLLEGE. 1691 01:23:36,920 --> 01:23:39,080 I APPLIED TO MEDICAL SCHOOL AND 1692 01:23:39,080 --> 01:23:41,680 ALMOST WENT TO MEDICAL SCHOOL 1693 01:23:41,680 --> 01:23:43,400 BUT GOT SUCKED INTO BASIC 1694 01:23:43,400 --> 01:23:45,760 SCIENCE RESEARCH AND APPLIED TO 1695 01:23:45,760 --> 01:23:49,120 THE HARVARD PROGRAM AND MY TWO 1696 01:23:49,120 --> 01:23:52,600 MENTORS WERE DAVID HOUSE MAN AND 1697 01:23:52,600 --> 01:23:53,680 CHRIS BERGE. 1698 01:23:53,680 --> 01:23:56,760 AND DAVID HAD DISCOVERED THE 1699 01:23:56,760 --> 01:24:05,240 GEN--GENE AND I FIRST MET DAN PEREZ 1700 01:24:05,240 --> 01:24:09,880 THROUGH DAVID'S HST160 CLASS. 1701 01:24:09,880 --> 01:24:12,080 I WAS IN THE AUDIENCE. 1702 01:24:12,080 --> 01:24:16,280 AND THIS WAS BACK IN 1703 01:24:16,280 --> 01:24:16,800 MID-2000s. 1704 01:24:16,800 --> 01:24:20,440 AND THEN CHRIS BURGE WAS AN 1705 01:24:20,440 --> 01:24:23,200 EXPERT IN COMPUTATIONAL BIOLOGY 1706 01:24:23,200 --> 01:24:24,400 WHO HELPED REALLY TEACH ME ABOUT 1707 01:24:24,400 --> 01:24:27,880 THE VALUE OF BASIC SCIENCE AND 1708 01:24:27,880 --> 01:24:30,000 HOW IT CAN TEACH YOU ABOUT HOW 1709 01:24:30,000 --> 01:24:31,800 THE SYSTEM WORKS SO THAT YOU CAN 1710 01:24:31,800 --> 01:24:33,720 LEVERAGE THAT TO MORE 1711 01:24:33,720 --> 01:24:34,920 EFFECTIVELY DEVELOP 1712 01:24:34,920 --> 01:24:35,880 THERAPEUTICS. 1713 01:24:35,880 --> 01:24:42,480 I WAS FORTUNATE TO GET AN NIH 1714 01:24:42,480 --> 01:24:43,760 DIRECTOR'S EARLY INDEPENDENCE 1715 01:24:43,760 --> 01:24:43,960 AWARD. 1716 01:24:43,960 --> 01:24:47,160 THAT WAS FUNDED BY NINDS. 1717 01:24:47,160 --> 01:24:49,480 JOHN PORTER WAS MY PROGRAM 1718 01:24:49,480 --> 01:24:50,880 OFFICER BACK THEN AND THAT 1719 01:24:50,880 --> 01:24:55,080 ALLOWED ME TO START MY LAB AS A 1720 01:24:55,080 --> 01:24:56,760 RESEARCH FELLOW FOR CANCER 1721 01:24:56,760 --> 01:24:59,080 RESEARCH. 1722 01:24:59,080 --> 01:25:02,240 IN 2015 I ENDED UP MOVING MY LAB 1723 01:25:02,240 --> 01:25:03,560 TO THE UNIVERSITY OF FLORIDA 1724 01:25:03,560 --> 01:25:05,560 BECAUSE I WANTED TO WORK MORE 1725 01:25:05,560 --> 01:25:07,040 CLOSELY WITH OTHER SCIENTISTS 1726 01:25:07,040 --> 01:25:13,720 AND OFFERS THAT WERE STUDYING MY 1727 01:25:13,720 --> 01:25:17,760 TONIC DYSTROPHY. 1728 01:25:17,760 --> 01:25:29,600 *. 1729 01:25:29,600 --> 01:25:32,240 -- AND ALSO HAVE THE ABILITY TO 1730 01:25:32,240 --> 01:25:35,480 BE CLOSE TO WHERE CLINICAL 1731 01:25:35,480 --> 01:25:37,080 TRIALS WERE BEING DONE. 1732 01:25:37,080 --> 01:25:41,720 AND ALSO CLOSE TO A VERY ROBUST 1733 01:25:41,720 --> 01:25:44,040 PATIENT COMMUNITY LOCALLY. 1734 01:25:44,040 --> 01:25:47,040 AND SO, ITS BEEN ABOUT SEVEN 1735 01:25:47,040 --> 01:25:49,800 YEARS NOW SINCE I'VE BEEN HERE. 1736 01:25:49,800 --> 01:25:53,440 AND I'VE GROWN MY LAB. 1737 01:25:53,440 --> 01:25:55,720 VERY EXCITED EVERY DAY TO COME 1738 01:25:55,720 --> 01:25:58,480 INTO THE LAB AND MAKE -- AND DO 1739 01:25:58,480 --> 01:26:00,160 BOTH BASIC AND TRANSLATIONAL 1740 01:26:00,160 --> 01:26:02,840 RESEARCH. 1741 01:26:02,840 --> 01:26:08,120 IN THE INTERIM I'VE WORKED 1742 01:26:08,120 --> 01:26:10,800 CLOSELY -- AND EARLIER THIS YEAR 1743 01:26:10,800 --> 01:26:13,320 I BECAME A SIGN AT THISIC 1744 01:26:13,320 --> 01:26:15,480 ADVISORY BOARD MEMBER FOR THE 1745 01:26:15,480 --> 01:26:16,720 FOUNDATION AND HAVE BEEN ABLE TO 1746 01:26:16,720 --> 01:26:19,040 WORK TOGETHER WITH DIFFERENT 1747 01:26:19,040 --> 01:26:20,960 DRUG COMPANIES HOPING TO DEVELOP 1748 01:26:20,960 --> 01:26:25,640 TREATMENTS FOR DM AND OTHER 1749 01:26:25,640 --> 01:26:26,920 DISEASES AND ALSO HAVE BEEN ABLE 1750 01:26:26,920 --> 01:26:29,680 TO INVENT APPROACH THAT'S ARE 1751 01:26:29,680 --> 01:26:31,840 BEING ACTIVELY FURTHER DEVELOPED 1752 01:26:31,840 --> 01:26:33,480 BY COMPANIES. 1753 01:26:33,480 --> 01:26:35,680 AND I'M JUST A BRIEF PLUG. 1754 01:26:35,680 --> 01:26:38,720 I KNOW CHRIS IS GOING TO PRESENT 1755 01:26:38,720 --> 01:26:40,240 NEXT BUT TOGETHER WITH CHRIS AND 1756 01:26:40,240 --> 01:26:43,240 OTHER COLLEAGUES AT MIT AND 1757 01:26:43,240 --> 01:26:44,920 HARVARD WE PUT TOGETHER 1758 01:26:44,920 --> 01:26:46,520 FUND-RAISING AND AWARENESS 1759 01:26:46,520 --> 01:26:49,520 EVENT. 1760 01:26:49,520 --> 01:26:49,880 S. 1761 01:26:49,880 --> 01:26:52,520 WE DID A FEW OF THESE IN BOSTON 1762 01:26:52,520 --> 01:26:54,240 AND THERE IS TALK ABOUT REVIVING 1763 01:26:54,240 --> 01:26:56,520 THIS AS WELL GIVEN CHRIS'S ROLE 1764 01:26:56,520 --> 01:26:58,760 AT MDA AND WE WERE ABLE TO FUND 1765 01:26:58,760 --> 01:27:01,600 A COUPLE OF FELLOWSHIPS FOCUSED 1766 01:27:01,600 --> 01:27:04,600 ON MUSCLE DISEASE RESEARCH. 1767 01:27:04,600 --> 01:27:09,320 AND SO I FIGURED I WOULD DO A 1768 01:27:09,320 --> 01:27:11,080 BRIEF OVERVIEW OF SOME OF THE 1769 01:27:11,080 --> 01:27:12,480 TOPIC THAT'S WE STUDY IN THE 1770 01:27:12,480 --> 01:27:13,040 LAB. 1771 01:27:13,040 --> 01:27:16,360 THERE ARE THREE AREAS THAT WE 1772 01:27:16,360 --> 01:27:22,960 FOCUS ON. 1773 01:27:22,960 --> 01:27:24,200 USING INFORMATION FROM THESE 1774 01:27:24,200 --> 01:27:26,680 AREAS FOR THERAPEUTIC 1775 01:27:26,680 --> 01:27:27,000 DEVELOPMENT. 1776 01:27:27,000 --> 01:27:29,480 AND I WON'T GO INTO DETAIL 1777 01:27:29,480 --> 01:27:31,360 BECAUSE THIS IS NOT A TALK 1778 01:27:31,360 --> 01:27:32,800 FOCUSED ON SCIENCE. 1779 01:27:32,800 --> 01:27:36,680 WE'VE TRIED TO UNDERSTAND HOW 1780 01:27:36,680 --> 01:27:40,240 MOLECULAR EVENTS IN MYOTONIC 1781 01:27:40,240 --> 01:27:41,800 DYSTROPHY LEAD TO DIFFERENT 1782 01:27:41,800 --> 01:27:42,560 PHENOTYPES. 1783 01:27:42,560 --> 01:27:45,120 ONE IS BETTER UNDERSTANDING THE 1784 01:27:45,120 --> 01:27:45,720 PROCESS. 1785 01:27:45,720 --> 01:27:49,760 ANOTHER AREA IS UNDERSTANDING 1786 01:27:49,760 --> 01:27:52,680 HOW REPEATS SPEND AND HOW THEY 1787 01:27:52,680 --> 01:27:55,440 CONNECT TO SPECIFIC SYMPTOMSES 1788 01:27:55,440 --> 01:27:58,400 AND PHENOTYPES AND TISSUES. 1789 01:27:58,400 --> 01:28:00,320 UNDERSTANDING HOW RNA WHICH IS 1790 01:28:00,320 --> 01:28:03,760 THE KEY MESSAGE IN CELLS IS 1791 01:28:03,760 --> 01:28:05,240 MOVED AROUND AND PROCESSED AND 1792 01:28:05,240 --> 01:28:07,240 MOVED AROUND SO WE USE A VARIETY 1793 01:28:07,240 --> 01:28:10,600 OF APPROACHES TO LOOK AT RNAs 1794 01:28:10,600 --> 01:28:13,040 IN MUSCLE TO UNDERSTAND HOW 1795 01:28:13,040 --> 01:28:17,360 RNAs AND PARTICLES MOVE AROUND 1796 01:28:17,360 --> 01:28:18,920 AND THEN WE HAVE A NUMBER OF 1797 01:28:18,920 --> 01:28:22,000 APPROACHES THAT ARE FOCUSED ON 1798 01:28:22,000 --> 01:28:24,320 USING INSIGHTS TO DEVELOP 1799 01:28:24,320 --> 01:28:26,960 THERAPEUTIC MODALITIES INCLUDING 1800 01:28:26,960 --> 01:28:31,400 COLLABORATIONS WITH OTHER 1801 01:28:31,400 --> 01:28:40,880 INDIVIDUALS -- GENE THERAPY AND 1802 01:28:40,880 --> 01:28:41,240 COLLABORATIONS. 1803 01:28:41,240 --> 01:28:43,000 AND THEN DEFINITELY AN EYE 1804 01:28:43,000 --> 01:28:46,080 TOWARDS THE FUTURE OF GETTING TO 1805 01:28:46,080 --> 01:28:48,000 HOW DO YOU MEASURE THERAPEUTIC 1806 01:28:48,000 --> 01:28:50,320 OUTCOMES AND HAVING THOSE 1807 01:28:50,320 --> 01:28:52,240 BIOMARKERS IN PARTICULAR FOR THE 1808 01:28:52,240 --> 01:28:53,680 CNS ARE VERY IMPORTANT. 1809 01:28:53,680 --> 01:28:58,080 SO I'LL CLOSE WITH A BRIEF 1810 01:28:58,080 --> 01:29:00,600 DESCRIPTION OF THE MYOTONIC 1811 01:29:00,600 --> 01:29:02,200 DYSTROPHY FOUNDATION. 1812 01:29:02,200 --> 01:29:05,760 THE FOUNDATION IS FOCUSED ON 1813 01:29:05,760 --> 01:29:07,360 REALIZING THIS VISION WHERE YOU 1814 01:29:07,360 --> 01:29:11,000 WILL HAVE TREATMENTS AND A CURE. 1815 01:29:11,000 --> 01:29:12,960 AND THE MISSION IS THREEFOLD. 1816 01:29:12,960 --> 01:29:15,000 IT'S REALLY FOCUSED ON THREE 1817 01:29:15,000 --> 01:29:15,600 AREAS. 1818 01:29:15,600 --> 01:29:17,040 THE FIRST IS COMMUNITY TO 1819 01:29:17,040 --> 01:29:19,480 SUPPORT AND CONNECT THE GREATER 1820 01:29:19,480 --> 01:29:20,600 COMMUNITY WHICH INVOLVES MANY 1821 01:29:20,600 --> 01:29:22,720 DIFFERENT STAKEHOLDERS INCLUDING 1822 01:29:22,720 --> 01:29:24,840 PATIENTS, CAREGIVERS AND 1823 01:29:24,840 --> 01:29:25,880 RESEARCHERS. 1824 01:29:25,880 --> 01:29:29,480 AND DRUG DEVELOPERS. 1825 01:29:29,480 --> 01:29:30,480 PROVIDING AND DEVELOPING 1826 01:29:30,480 --> 01:29:31,200 RESOURCES FOR CARE. 1827 01:29:31,200 --> 01:29:34,240 SO DEALING WITH THE IMMEDIATE 1828 01:29:34,240 --> 01:29:34,960 NEEDS OF THE COMMUNITY RIGHT 1829 01:29:34,960 --> 01:29:35,920 NOW. 1830 01:29:35,920 --> 01:29:39,120 AND THEN THE THIRD FOCUSED ON 1831 01:29:39,120 --> 01:29:40,800 TREATMENTS AND CURES. 1832 01:29:40,800 --> 01:29:42,560 THE IDEA OF BUILDING AND 1833 01:29:42,560 --> 01:29:43,840 SUPPORTING THE INFRASTRUCTURE 1834 01:29:43,840 --> 01:29:45,240 THAT IS GOING TO BE REQUIRED TO 1835 01:29:45,240 --> 01:29:47,480 GET TO ACTUAL TREATMENTS THAT 1836 01:29:47,480 --> 01:29:50,040 WILL MAKE A DIFFERENCE IN 1837 01:29:50,040 --> 01:29:50,960 PATIENT'S LIVES. 1838 01:29:50,960 --> 01:29:52,720 AND IT'S BEEN EXCITING TO WATCH 1839 01:29:52,720 --> 01:29:54,080 THE FOUNDATION FORM AND GET 1840 01:29:54,080 --> 01:29:54,680 STARTED. 1841 01:29:54,680 --> 01:29:57,080 IT STARTED WHEN I WAS A GRADUATE 1842 01:29:57,080 --> 01:29:57,480 STUDENT. 1843 01:29:57,480 --> 01:29:59,600 I REMEMBER GOING TO A MEETING IN 1844 01:29:59,600 --> 01:30:02,400 ITALY AND INTERACT WITH SOME OF 1845 01:30:02,400 --> 01:30:04,000 THE KEY FOUNDERS OF THIS 1846 01:30:04,000 --> 01:30:05,840 FOUNDATION AND IT WAS INITIATED 1847 01:30:05,840 --> 01:30:08,400 OUT OF A NEED TO GET HELP AND 1848 01:30:08,400 --> 01:30:11,280 PROVIDE HELP TO PEOPLE WHO ARE 1849 01:30:11,280 --> 01:30:12,840 LIVING WITH THIS DISEASE. 1850 01:30:12,840 --> 01:30:14,600 THE FOUNDATION HAS GONE THROUGH 1851 01:30:14,600 --> 01:30:16,400 A NUMBER OF ITERATIONS AND 1852 01:30:16,400 --> 01:30:18,840 GROWTH AND NAVIGATED A LOT OF 1853 01:30:18,840 --> 01:30:21,080 DIFFERENT ISSUES AND CONSIDERS 1854 01:30:21,080 --> 01:30:22,240 THAT HAPPEN WITH ANY 1855 01:30:22,240 --> 01:30:23,520 ORGANIZATION AS IT GROWS AND 1856 01:30:23,520 --> 01:30:25,320 IT'S BEEN EXCITING TO WATCH THAT 1857 01:30:25,320 --> 01:30:26,880 PROCESS AND THEN I'VE BEEN 1858 01:30:26,880 --> 01:30:28,880 INVOLVED IN A NUMBER OF 1859 01:30:28,880 --> 01:30:32,640 CAPACITIES AS A REVIEWER, 1860 01:30:32,640 --> 01:30:36,080 FUND-RAISER -- SAB MEMBER AND 1861 01:30:36,080 --> 01:30:36,920 COMMUNITY MEMBER. 1862 01:30:36,920 --> 01:30:41,800 BUT THROUGHOUT THESE ENDEAVORS 1863 01:30:41,800 --> 01:30:43,600 AND RELATIONSHIPS THERE ARE 1864 01:30:43,600 --> 01:30:45,480 QUESTIONS THAT I GRAPPLE WITH A 1865 01:30:45,480 --> 01:30:47,120 LOT AND ARE SHARED ISSUES THAT 1866 01:30:47,120 --> 01:30:48,920 PEOPLE ON THIS CALL THINK ABOUT 1867 01:30:48,920 --> 01:30:49,880 OFTEN. 1868 01:30:49,880 --> 01:30:53,520 AND THEY ARE ISSUE THAT'S DEAL 1869 01:30:53,520 --> 01:30:55,680 WITH THE TRANSIENT NATURE WE 1870 01:30:55,680 --> 01:30:57,480 HAVE ON THIS EARTH WHICH IS HOW 1871 01:30:57,480 --> 01:31:01,760 DO YOU ALLOCATE YOUR RESOURCES. 1872 01:31:01,760 --> 01:31:04,960 DO YOU FOCUS ON BASIC SCIENCE OR 1873 01:31:04,960 --> 01:31:06,720 TRANSLATIONAL RESEARCH OR BOTH? 1874 01:31:06,720 --> 01:31:08,160 I'VE DEALT WITH THIS IN 1875 01:31:08,160 --> 01:31:09,160 DIFFERENT WAYS. 1876 01:31:09,160 --> 01:31:11,240 I WAS INVITED TO WRITE AN 1877 01:31:11,240 --> 01:31:12,440 ARTICLE. 1878 01:31:12,440 --> 01:31:15,720 I DESCRIBE IT AS BOTH ARE PIECES 1879 01:31:15,720 --> 01:31:17,480 OF A ROPE THAT ARE BEING PULLED 1880 01:31:17,480 --> 01:31:20,160 AGAINST EACH OTHER. 1881 01:31:20,160 --> 01:31:23,240 ONE ATTITUDE I'VE COME TO 1882 01:31:23,240 --> 01:31:26,600 APPRECIATE IS THAT THEY MIGHT BE 1883 01:31:26,600 --> 01:31:31,120 TWO ENDS OF A ROPE BUT HAVE A 1884 01:31:31,120 --> 01:31:32,680 PULLEY IN THE MIDDLE. 1885 01:31:32,680 --> 01:31:35,560 HOW DO YOU FACILITATE THE 1886 01:31:35,560 --> 01:31:37,960 RESEARCH? 1887 01:31:37,960 --> 01:31:44,160 YOU HAVE MUSCLE SKELETAL MUSCLE 1888 01:31:44,160 --> 01:31:46,520 BUT THERE MIGHT BE OTHER TISSUES 1889 01:31:46,520 --> 01:31:48,000 AFFECTED AND HOW DO YOU GAIN 1890 01:31:48,000 --> 01:31:49,680 SUPPORT AND BRING NEW PEOPLE IN 1891 01:31:49,680 --> 01:31:51,800 WITH THE EXPERTISE NEEDED TO 1892 01:31:51,800 --> 01:31:53,480 DEAL WITH AND MAKE PROGRESS IN 1893 01:31:53,480 --> 01:31:54,320 THOSE AREAS. 1894 01:31:54,320 --> 01:31:56,360 AND THEN FINALLY THIS ECOSYSTEM 1895 01:31:56,360 --> 01:31:58,160 THAT IS REQUIRED TO GET TO 1896 01:31:58,160 --> 01:32:00,760 TREATMENTS IS QUITE COMPLEX AND 1897 01:32:00,760 --> 01:32:01,040 EXTENSIVE. 1898 01:32:01,040 --> 01:32:04,960 SO HOW DO YOU BEST SUPPORT THAT 1899 01:32:04,960 --> 01:32:05,800 ECOSYSTEM. 1900 01:32:05,800 --> 01:32:07,960 WHERE TO PLACE YOUR EFFORTS AND 1901 01:32:07,960 --> 01:32:09,840 IT'S NOT AN EASY TASK. 1902 01:32:09,840 --> 01:32:12,240 I THINK MANY US ARE THINKING 1903 01:32:12,240 --> 01:32:13,720 ABOUT THIS AND DEALING WITH THIS 1904 01:32:13,720 --> 01:32:15,200 AND IT'S FANTASTIC TO GO TO 1905 01:32:15,200 --> 01:32:17,360 SPEAK TO OTHERS AND ENGAGE WITH 1906 01:32:17,360 --> 01:32:18,080 OTHERS THAT ARE ALSO DEALING 1907 01:32:18,080 --> 01:32:20,160 WITH THESE ISSUE. 1908 01:32:20,160 --> 01:32:21,720 I'LL LEAVE IT AT THAT. 1909 01:32:21,720 --> 01:32:22,680 THANK YOU 1910 01:32:22,680 --> 01:32:25,240 >> THANK YOU SO MUCH ERIC FOR 1911 01:32:25,240 --> 01:32:26,480 DESCRIBING BOTH YOUR PERSONAL 1912 01:32:26,480 --> 01:32:28,840 CONNECTION AS WELL AS YOUR 1913 01:32:28,840 --> 01:32:30,160 PROFESSIONAL CONNECTION. 1914 01:32:30,160 --> 01:32:33,360 AND WE'RE DELIGHTED TO HAVE YOU 1915 01:32:33,360 --> 01:32:35,040 AS A MEMBER OF THE COMMITTEE. 1916 01:32:35,040 --> 01:32:43,040 THER-- ARE THERE QUESTIONS FOR ERIC? 1917 01:32:43,040 --> 01:32:46,240 >> JUST A COMMENT -- EXCELLENT 1918 01:32:46,240 --> 01:32:48,880 PRESENTATION. 1919 01:32:48,880 --> 01:32:52,360 THANK YOU FOR YOUR BACKGROUND 1920 01:32:52,360 --> 01:33:08,800 AND YOUR ENDEAVOUR. 1921 01:33:08,800 --> 01:33:10,920 -- RIGHT ON THE MARK IN PURSUING 1922 01:33:10,920 --> 01:33:12,320 THE DISEASE AND TRYING TO UNDER 1923 01:33:12,320 --> 01:33:14,720 WHAT IS GOING ON WITH THE 1924 01:33:14,720 --> 01:33:17,720 DISEASE AND STAYING CLOSE TO 1925 01:33:17,720 --> 01:33:21,240 GENETICS AND SITTING AT THE 1926 01:33:21,240 --> 01:33:23,360 INTERSECTION OF THE PHENOTYPE 1927 01:33:23,360 --> 01:33:28,920 AND TRYING TO RATIONALIZE 1928 01:33:28,920 --> 01:33:31,640 RESOURCES -- -- IT WAS A GREAT 1929 01:33:31,640 --> 01:33:32,440 PRESENTATION. 1930 01:33:32,440 --> 01:33:32,680 THANK YOU. 1931 01:33:32,680 --> 01:33:36,120 >> I HAVE TO SAY -- I DON'T KNOW 1932 01:33:36,120 --> 01:33:38,480 IF I'VE DIRECTLY SPOKEN TO YOU 1933 01:33:38,480 --> 01:33:39,760 EVER BUT I DEFINITELY THINK 1934 01:33:39,760 --> 01:33:41,600 ABOUT THE WORK YOU'VE DONE AND 1935 01:33:41,600 --> 01:33:44,200 IT'S BEEN VERY INSPIRATIONAL AND 1936 01:33:44,200 --> 01:33:45,480 POWERFUL SO THANK YOU FOR THAT. 1937 01:33:45,480 --> 01:33:48,560 I THINK ABOUT FHSD OFTEN. 1938 01:33:48,560 --> 01:33:52,200 THERE IS A LOT OF PARALLELS 1939 01:33:52,200 --> 01:33:53,160 SCIENTIFICALLY SO I HAVE 1940 01:33:53,160 --> 01:33:54,080 DEFINITELY BENEFITED FROM 1941 01:33:54,080 --> 01:33:57,040 THINKING ABOUT MULTIPLE DISEASES 1942 01:33:57,040 --> 01:34:01,480 IN PARALLEL. 1943 01:34:01,480 --> 01:34:03,400 >> YOU KNOW IT'S NOT JUST A 1944 01:34:03,400 --> 01:34:05,000 MATTER OF THE HEAD OR KNOWLEDGE 1945 01:34:05,000 --> 01:34:09,080 OF GENETICS. 1946 01:34:09,080 --> 01:34:11,520 YOU -- YOU TALK ABOUT THE HEART 1947 01:34:11,520 --> 01:34:16,080 AS WELL AND YOUR FATHER IS 1948 01:34:16,080 --> 01:34:17,760 INSPIRATIONAL AND CONTINUES TO 1949 01:34:17,760 --> 01:34:19,240 BE THE SOURCE OF PASSION FOR THE 1950 01:34:19,240 --> 01:34:21,080 WORK AND I THINK THAT IS WHAT 1951 01:34:21,080 --> 01:34:22,240 MAKES THE DIFFERENCE. 1952 01:34:22,240 --> 01:34:23,880 AND WE APPRECIATE YOUR 1953 01:34:23,880 --> 01:34:25,120 WILLINGNESS TO SHARE THAT WITH 1954 01:34:25,120 --> 01:34:25,680 US. 1955 01:34:25,680 --> 01:34:27,480 THERE IS SOMETHING IN THE CHAT 1956 01:34:27,480 --> 01:34:28,360 BOX. 1957 01:34:28,360 --> 01:34:29,160 >> THANK YOU, ANGELA. 1958 01:34:29,160 --> 01:34:31,240 I THINK THIS IS PROBABLY A 1959 01:34:31,240 --> 01:34:32,520 COMMON THEME ALSO WITH MANY 1960 01:34:32,520 --> 01:34:34,400 PEOPLE WORKING ON DISEASES THAT 1961 01:34:34,400 --> 01:34:37,720 ARE CLOSE TO THEM AND CERTAINLY 1962 01:34:37,720 --> 01:34:40,240 ANGELA GETS THAT AND MANY PEOPLE 1963 01:34:40,240 --> 01:34:44,000 GET THAT SO I THINK AN 1964 01:34:44,000 --> 01:34:46,040 INTERESTING TOPIC AND IT 1965 01:34:46,040 --> 01:34:48,040 PROBABLY COMES UP BUT HOW DO WE 1966 01:34:48,040 --> 01:34:49,600 FURTHER LEVERAGE THAT TO HELP 1967 01:34:49,600 --> 01:34:51,680 THIS COMMUNITY AND FURTHER 1968 01:34:51,680 --> 01:34:53,280 CAPITALIZE ON THAT 1969 01:34:53,280 --> 01:34:54,760 >> HOLD THAT THOUGHT AND WE'LL 1970 01:34:54,760 --> 01:34:56,760 GET BACK TO THAT AND BRING THAT 1971 01:34:56,760 --> 01:34:58,280 UP. 1972 01:34:58,280 --> 01:35:02,400 SO NOW OUR LAST AND THIRD MEMBER 1973 01:35:02,400 --> 01:35:04,920 IS CHRIS ANSELMO WHO IS FROM THE 1974 01:35:04,920 --> 01:35:06,520 MUSCULAR DYSTROPHY ASSOCIATION. 1975 01:35:06,520 --> 01:35:09,440 CHRIS IS THE MARKET INTELLIGENCE 1976 01:35:09,440 --> 01:35:11,280 MANAGER AT THE NATIONAL HEALTH 1977 01:35:11,280 --> 01:35:12,280 CARE PARTNERSHIP TEAM. 1978 01:35:12,280 --> 01:35:14,720 I DON'T KNOW IF YOU'RE A DOCTOR 1979 01:35:14,720 --> 01:35:15,520 OR NOT. 1980 01:35:15,520 --> 01:35:17,440 I DON'T HAVE THAT INFORMATION. 1981 01:35:17,440 --> 01:35:25,680 I MADE THE A ESSENTIALS 1982 01:35:25,680 --> 01:35:27,240 >> WELCOME, CHRIS. 1983 01:35:27,240 --> 01:35:28,880 AND ARE YOU ON? 1984 01:35:28,880 --> 01:35:29,920 >> YEP. 1985 01:35:29,920 --> 01:35:31,800 >> ARE YOU A DOCTOR I WANT TO BE 1986 01:35:31,800 --> 01:35:32,360 CORRECTED 1987 01:35:32,360 --> 01:35:33,520 >> I WOULD LIKE TO BE BUT NO. 1988 01:35:33,520 --> 01:35:34,680 I'M NOT. 1989 01:35:34,680 --> 01:35:40,240 MAYBE FOR TODAY. 1990 01:35:40,240 --> 01:35:41,840 I'M CHRIS ANSELMO. 1991 01:35:41,840 --> 01:35:45,320 THE DIRECTOR OF MARKET 1992 01:35:45,320 --> 01:35:46,480 INTELLIGENCE AT THE MUSCULAR 1993 01:35:46,480 --> 01:35:47,840 DYSTROPHY ASSOCIATION AND ALSO A 1994 01:35:47,840 --> 01:35:50,720 MEMBER OF THE NEUROMUSCULAR 1995 01:35:50,720 --> 01:35:51,680 DISEASE COMMUNITY. 1996 01:35:51,680 --> 01:35:53,760 I LIVED WITH -- MUSCULAR 1997 01:35:53,760 --> 01:35:55,840 DYSTROPHY TYPE 2B. 1998 01:35:55,840 --> 01:35:59,480 JUST A QUICK WORD OF AUCTION. 1999 01:35:59,480 --> 01:36:01,800 I'M DOING THIS FROM MY PHONE. 2000 01:36:01,800 --> 01:36:04,480 THERE IS A SLIGHT CHANCE THAT I 2001 01:36:04,480 --> 01:36:06,640 MIGHT CUT OUT AT SOME POINT 2002 01:36:06,640 --> 01:36:10,320 >> WE HEAR YOU CLEARLY. 2003 01:36:10,320 --> 01:36:12,040 >> A LITTLE BIT OF BACKGROUND 2004 01:36:12,040 --> 01:36:14,040 ABOUT MY JOURNEY. 2005 01:36:14,040 --> 01:36:16,480 IT'S KIND OF INTERESTING BECAUSE 2006 01:36:16,480 --> 01:36:17,960 I'M 35 NOW. 2007 01:36:17,960 --> 01:36:20,440 I LIVE IN CONNECTICUT. 2008 01:36:20,440 --> 01:36:23,000 FOR THE FIRST 18 YEARS OF MY 2009 01:36:23,000 --> 01:36:25,680 LIFE I DIDN'T EVEN KNOW THAT I 2010 01:36:25,680 --> 01:36:27,000 HAD ANY SORT OF MUSCLE 2011 01:36:27,000 --> 01:36:27,600 CONDITION. 2012 01:36:27,600 --> 01:36:29,240 IT WAS A FLUKE THING. 2013 01:36:29,240 --> 01:36:31,720 I WAS IN I A CAR ACCIDENT MY 2014 01:36:31,720 --> 01:36:33,480 SENIOR YEAR OF HIGH SCHOOL AND 2015 01:36:33,480 --> 01:36:37,680 AT THE HOSPITAL THEY WERE JUST 2016 01:36:37,680 --> 01:36:39,840 ABOUT TO DISCHARGE ME AND TAKING 2017 01:36:39,840 --> 01:36:42,240 ROUTINE BLOOD TEST AND THE 2018 01:36:42,240 --> 01:36:44,680 DOCTOR CAME IN SAYING THAT WE 2019 01:36:44,680 --> 01:36:49,200 NEED TO KEEP YOU BECAUSE YOUR 2020 01:36:49,200 --> 01:36:51,520 CREATINE LEVELS ARE -- IN THE 2021 01:36:51,520 --> 01:36:52,320 THOUSANDS. 2022 01:36:52,320 --> 01:36:54,800 SO THEY THOUGHT I SUFFERED SOME 2023 01:36:54,800 --> 01:36:57,080 SORT OF INTERNAL INJURY. 2024 01:36:57,080 --> 01:36:59,680 THEY DETERMINED THAT I WAS FINE 2025 01:36:59,680 --> 01:37:00,800 OUTWARDLY BUT THEY KNEW 2026 01:37:00,800 --> 01:37:02,320 SOMETHING WAS OFF. 2027 01:37:02,320 --> 01:37:05,680 THAT STARTED ABOUT A YEAR LONG 2028 01:37:05,680 --> 01:37:08,080 DIAGNOSTIC JOURNEY WHERE AFTER 2029 01:37:08,080 --> 01:37:11,720 SEEING VARIOUS SPECIALISTS AND 2030 01:37:11,720 --> 01:37:14,400 HAVING A THIGH BIOPSY THEY WERE 2031 01:37:14,400 --> 01:37:15,840 ABLE TO DETERMINE THAT I HAD 2032 01:37:15,840 --> 01:37:18,480 SOMETHING WHAT THEY CALLED -- 2033 01:37:18,480 --> 01:37:20,880 THAT I WAS MISSING THE -- 2034 01:37:20,880 --> 01:37:22,920 PROTEIN. 2035 01:37:22,920 --> 01:37:27,360 THIS WAS 2004 SO THEY KNEW A 2036 01:37:27,360 --> 01:37:28,920 LITTLE BIT ABOUT IT BUT NOT TOO 2037 01:37:28,920 --> 01:37:30,680 MUCH. 2038 01:37:30,680 --> 01:37:32,680 SO OFF TO COLLEGE I WENT. 2039 01:37:32,680 --> 01:37:33,640 I FELT FINE. 2040 01:37:33,640 --> 01:37:36,120 AND SHORTLY AFTER I GRADUATED 2041 01:37:36,120 --> 01:37:39,040 FROM NORTHEASTERN UNIVERSITY IN 2042 01:37:39,040 --> 01:37:40,800 2008 I WAS MOVING TO A NEW 2043 01:37:40,800 --> 01:37:44,680 APARTMENT AND CARRYING MY DESK 2044 01:37:44,680 --> 01:37:47,520 CHAIR AND THINGS WERE MORE 2045 01:37:47,520 --> 01:37:49,240 DIFFICULT. 2046 01:37:49,240 --> 01:37:52,080 IT WAS THE FIRST SORT OF SYMPTOM 2047 01:37:52,080 --> 01:37:53,720 THAT I COULD REMEMBER THAT 2048 01:37:53,720 --> 01:37:55,040 ULTIMATELY COULD BE TIED BACK TO 2049 01:37:55,040 --> 01:37:56,240 THE DISEASE. 2050 01:37:56,240 --> 01:37:58,440 AND ONCE THAT STARTED THAT 2051 01:37:58,440 --> 01:38:00,400 REALLY KIND OF ACCELERATED SO I 2052 01:38:00,400 --> 01:38:03,360 WENT FROM BEING FAIRLY ATHLETIC 2053 01:38:03,360 --> 01:38:05,480 BEING ABLE TO RUN AND JUMP AND 2054 01:38:05,480 --> 01:38:08,000 PLAY SPORTS TO WITHIN THREE 2055 01:38:08,000 --> 01:38:10,760 YEARS FALLING TO WHERE I AM TO 2056 01:38:10,760 --> 01:38:13,800 YOU RIGHT NOW I USE A WHEELCHAIR 2057 01:38:13,800 --> 01:38:15,960 FULL-TIME AND THIS HAS IMPACTED 2058 01:38:15,960 --> 01:38:17,760 MY CAREER TRAJECTORY. 2059 01:38:17,760 --> 01:38:20,640 SO WHEN I GRADUATED I WORKED AT 2060 01:38:20,640 --> 01:38:23,640 A FEW DIFFERENT TECH COMPANIES 2061 01:38:23,640 --> 01:38:26,400 IN THE BOSTON AREA. 2062 01:38:26,400 --> 01:38:29,760 I ENJOYED THAT BUT AS MY DISEASE 2063 01:38:29,760 --> 01:38:32,160 PROGRESSED AND THE WEAKNESS 2064 01:38:32,160 --> 01:38:33,840 ACCELERATED ALL I COULD THINK 2065 01:38:33,840 --> 01:38:37,000 ABOUT WAS MY DISEASE SO I 2066 01:38:37,000 --> 01:38:38,600 REALIZED AT SOME POINT THAT IS 2067 01:38:38,600 --> 01:38:41,120 WHAT I WANTED TO DO WAS DO 2068 01:38:41,120 --> 01:38:43,040 SOMETHING RELATED TO HEALTH CARE 2069 01:38:43,040 --> 01:38:45,240 OR MY PARTICULAR CONDITION. 2070 01:38:45,240 --> 01:38:47,680 I MIGHT AS WELL MAKE IT INTO A 2071 01:38:47,680 --> 01:38:49,240 CAREER BECAUSE OTHERWISE I COULD 2072 01:38:49,240 --> 01:38:52,880 NOT FOCUS ON OTHER THINGS. 2073 01:38:52,880 --> 01:38:53,880 I QUIT MY JOB. 2074 01:38:53,880 --> 01:38:56,200 I WENT TO BUSINESS SCHOOL AND 2075 01:38:56,200 --> 01:38:59,440 GRADUATED FROM BOSTON COLLEGE IN 2076 01:38:59,440 --> 01:39:00,880 2016. 2077 01:39:00,880 --> 01:39:03,600 THAT IS WHEN I MET ERIC. 2078 01:39:03,600 --> 01:39:06,680 JUST BY COINCIDENCE I WAS 2079 01:39:06,680 --> 01:39:11,920 SCROLLING FACEBOOK AND SAW AN 2080 01:39:11,920 --> 01:39:18,760 INVITATION AND I SENT AN E-MAIL 2081 01:39:18,760 --> 01:39:20,320 SAYING I WOULD LIKE TO GET 2082 01:39:20,320 --> 01:39:22,560 INVOLVED AND HE HAS BEEN A GREAT 2083 01:39:22,560 --> 01:39:25,480 FRIEND AND SOMEONE I COULD ASK A 2084 01:39:25,480 --> 01:39:26,360 LOT OF QUESTIONS. 2085 01:39:26,360 --> 01:39:28,480 HE HAS A RESEARCH BACKGROUND. 2086 01:39:28,480 --> 01:39:30,360 I FOUND THAT CONNECT WITH 2087 01:39:30,360 --> 01:39:32,200 RESEARCHERS AND CONNECTING WITH 2088 01:39:32,200 --> 01:39:33,400 OTHER PATIENTS AND CONNECTING 2089 01:39:33,400 --> 01:39:35,880 WITH PEOPLE THAT JUST LIVED IN 2090 01:39:35,880 --> 01:39:38,880 THIS WORLD WAS REALLY BENEFICIAL 2091 01:39:38,880 --> 01:39:44,400 FOR ME TO BETTER ACCEPT WHAT WAS 2092 01:39:44,400 --> 01:39:45,160 GOING ON. 2093 01:39:45,160 --> 01:39:47,080 YOU THINK LIFE IS GOING TO GO 2094 01:39:47,080 --> 01:39:49,400 ONE WAY BUT IT GOES IN A 2095 01:39:49,400 --> 01:39:50,240 DIFFERENT DIRECTION. 2096 01:39:50,240 --> 01:39:55,000 AND THAT ULTIMATELY LED ME TO 2097 01:39:55,000 --> 01:39:57,680 NBA I JOINED FOUR YEARS AGO BUT 2098 01:39:57,680 --> 01:39:59,880 HAD BEEN INVOLVED FOR SEVERAL 2099 01:39:59,880 --> 01:40:05,680 YEARS. WAS A FREELANCE WRITER. 2100 01:40:05,680 --> 01:40:09,200 NOW I'M PART OF THE NATIONAL 2101 01:40:09,200 --> 01:40:10,000 MARKETING TEAM. 2102 01:40:10,000 --> 01:40:13,400 AND WITHIN THE LAST COUPLE OF 2103 01:40:13,400 --> 01:40:15,280 YEARS I BECAME MORE INTERESTED 2104 01:40:15,280 --> 01:40:18,280 IN POLICY -- OBVIOUSLY POLICY 2105 01:40:18,280 --> 01:40:19,960 PLAYS AN IMPORTANT ROLE IN ALL 2106 01:40:19,960 --> 01:40:22,080 OF OUR LIVES BUT I REALLY 2107 01:40:22,080 --> 01:40:23,200 STARTED TO LEARN ABOUT THE 2108 01:40:23,200 --> 01:40:28,600 DIFFERENT -- INITIATIVES THAT 2109 01:40:28,600 --> 01:40:31,680 IMPACTED PEOPLE WITH MUSCULAR 2110 01:40:31,680 --> 01:40:32,280 DYSTROPHY. 2111 01:40:32,280 --> 01:40:33,120 AND STARTED TO UNDERSTAND SOME 2112 01:40:33,120 --> 01:40:35,080 OF THE DIFFERENT LAWS AND 2113 01:40:35,080 --> 01:40:36,880 DIFFERENT TYPES OF THINGS LIKE 2114 01:40:36,880 --> 01:40:39,480 THAT THAT DIRECTED OUR LIVES IN 2115 01:40:39,480 --> 01:40:42,120 TERMS OF WHETHER IT COULD BE -- 2116 01:40:42,120 --> 01:40:43,680 SOMETHING THAT COULD BE A 2117 01:40:43,680 --> 01:40:47,240 BENEFIT TO US AND JUST BECAME 2118 01:40:47,240 --> 01:40:48,920 INTERESTED IN THAT JUST HOW ALL 2119 01:40:48,920 --> 01:40:50,680 OF THE DIFFERENT POLICIES AND 2120 01:40:50,680 --> 01:40:54,040 LAWS AND ADVOCATING FOR THAT. 2121 01:40:54,040 --> 01:40:56,360 EVEN ON A PERSONAL LEVEL. 2122 01:40:56,360 --> 01:40:59,960 FINDING ANOTHER PATIENT WITH MY 2123 01:40:59,960 --> 01:41:02,000 PARTICULAR DISEASE SO THEY COULD 2124 01:41:02,000 --> 01:41:05,000 BE TESTED AND BE A PART OF THE 2125 01:41:05,000 --> 01:41:05,960 COMMUNITY. 2126 01:41:05,960 --> 01:41:08,120 BECAUSE IT'S INCREDIBLY RARE SO 2127 01:41:08,120 --> 01:41:09,600 ALL OF THE PATIENTS THAT WE CAN 2128 01:41:09,600 --> 01:41:11,000 FIND THE BETTER. 2129 01:41:11,000 --> 01:41:12,920 SO I'M HERE REPRESENTING THE 2130 01:41:12,920 --> 01:41:14,680 MUSCULAR DYSTROPHY ASSOCIATION. 2131 01:41:14,680 --> 01:41:17,080 I SEE MOST OF YOU PROBABLY KNOW 2132 01:41:17,080 --> 01:41:20,080 WHAT MDA IS. 2133 01:41:20,080 --> 01:41:21,720 WE'RE THE NATIONAL UMBRELLA 2134 01:41:21,720 --> 01:41:24,080 ORGANIZATION COVERING I THINK 2135 01:41:24,080 --> 01:41:26,160 NOW IT'S OVER 40 DIFFERENT 2136 01:41:26,160 --> 01:41:28,000 MUSCULAR DYSTROPHY DISEASES AND 2137 01:41:28,000 --> 01:41:31,080 THERE ARE DOZENS AND DOZENS OF 2138 01:41:31,080 --> 01:41:33,080 SUBTYPES AND WE PROVIDE SERVICES 2139 01:41:33,080 --> 01:41:33,880 TO OUR COMMUNITY. 2140 01:41:33,880 --> 01:41:38,680 WE ADVANCE RESEARCH AND CARE. 2141 01:41:38,680 --> 01:41:42,080 WE FUND A LOT OF RESEARCH THAT'S 2142 01:41:42,080 --> 01:41:43,520 BUILDING BLOCKS FOR THE 2143 01:41:43,520 --> 01:41:45,480 THERAPIES THAT WE RECEIVE TODAY. 2144 01:41:45,480 --> 01:41:47,240 WE PROVIDE SERVICES TO OUR 2145 01:41:47,240 --> 01:41:49,840 COMMUNITY SUCH AS MDA SUMMER 2146 01:41:49,840 --> 01:41:52,480 CAMP. 2147 01:41:52,480 --> 01:41:53,960 IT'S A VALUE TO THE CHILDREN AND 2148 01:41:53,960 --> 01:41:54,960 THE FAMILIES AND THE 2149 01:41:54,960 --> 01:41:56,240 ORGANIZATION. 2150 01:41:56,240 --> 01:41:59,080 AND ALSO WE HAVE A VERY STRONG 2151 01:41:59,080 --> 01:42:01,720 POLICY AND ADVOCACY TEAM AS 2152 01:42:01,720 --> 01:42:02,120 WELL. 2153 01:42:02,120 --> 01:42:06,560 AND IN RECENT -- -- I'M THE ONE 2154 01:42:06,560 --> 01:42:09,000 KEEPING EVERYONE FROM BREAK TIME 2155 01:42:09,000 --> 01:42:15,800 -- WE SE -- PUT ON THE CONFERENCE 2156 01:42:15,800 --> 01:42:17,600 IN NASHVILLE IN MARCH. 2157 01:42:17,600 --> 01:42:20,600 WE HAD A VIRTUAL CONFERENCE AND 2158 01:42:20,600 --> 01:42:23,880 IT IS NICE TO BE ABLE TO CONVENE 2159 01:42:23,880 --> 01:42:27,400 AND BRING DIFFERENT STAKEHOLDERS 2160 01:42:27,400 --> 01:42:28,560 TOGETHER TO FIGURE OUT WHAT IS 2161 01:42:28,560 --> 01:42:29,880 GOING ON IN THE SPACE AND 2162 01:42:29,880 --> 01:42:32,960 HITTING CLOSER TO HOME FOR ME 2163 01:42:32,960 --> 01:42:37,280 MDA AND OUR PARTNER -- 2164 01:42:37,280 --> 01:42:39,880 ORGANIZATIONS HELPED TO GET 2165 01:42:39,880 --> 01:42:40,800 ICD10 CODES FOR MUSCULAR 2166 01:42:40,800 --> 01:42:43,880 DYSTROPHY -- I DON'T KNOW IF 2167 01:42:43,880 --> 01:42:46,480 THEY ARE 100% APPROVED YET BUT 2168 01:42:46,480 --> 01:42:47,880 THEY ARE CLOSE TO THE FINISH 2169 01:42:47,880 --> 01:42:49,360 LINE IN TERMS OF GETTING 2170 01:42:49,360 --> 01:42:52,320 APPROVED AND HOPEFULLY 2171 01:42:52,320 --> 01:42:53,560 IMPLEMENTED SOON. 2172 01:42:53,560 --> 01:42:56,480 BECAUSE SO MAN OTHER US WHEN WE 2173 01:42:56,480 --> 01:42:59,040 WERE DIAGNOSED WE WERE BUCKET 2174 01:42:59,040 --> 01:43:01,560 INTO THAT OTHER NEUROMUSCULAR 2175 01:43:01,560 --> 01:43:04,240 DISEASE CATEGORY AND TO HAVE A 2176 01:43:04,240 --> 01:43:06,880 DIAGNOSTIC CODE WOULD HELP TO 2177 01:43:06,880 --> 01:43:08,040 IDENTIFY PATIENTS WITH LIMB 2178 01:43:08,040 --> 01:43:11,040 GIRDLE -- DIFFERENT SUBTYPES AND 2179 01:43:11,040 --> 01:43:13,480 THAT WILL HELP TO ACCELERATE 2180 01:43:13,480 --> 01:43:15,680 ADVANCES AND CARE AND TREATMENTS 2181 01:43:15,680 --> 01:43:17,400 AS WELL. 2182 01:43:17,400 --> 01:43:21,760 SO I'M VERY THANKFUL TO BE PART 2183 01:43:21,760 --> 01:43:25,480 OF THE COMMITTEE AND I'M HERE TO 2184 01:43:25,480 --> 01:43:26,080 ANSWER QUESTIONS 2185 01:43:26,080 --> 01:43:27,680 >> THANK YOU SO MUCH CHRIS FOR 2186 01:43:27,680 --> 01:43:29,680 YOUR PERSONAL STORY AND REALLY 2187 01:43:29,680 --> 01:43:33,560 HOW IT INFLUENCES THE EVOLUTION 2188 01:43:33,560 --> 01:43:36,360 IN YOUR PROFESSIONAL CAREER AS 2189 01:43:36,360 --> 01:43:36,560 WELL. 2190 01:43:36,560 --> 01:43:38,240 AND REALIZING A LOT OF PEOPLE 2191 01:43:38,240 --> 01:43:42,000 HAVE A BOSTON CONNECTION HERE. 2192 01:43:42,000 --> 01:43:43,520 I'M IN BETHESDA. 2193 01:43:43,520 --> 01:43:45,720 BUT I'M A FULL-TIME 2194 01:43:45,720 --> 01:43:47,640 MASSACHUSETTS RESIDENT WHEN I'M 2195 01:43:47,640 --> 01:43:49,480 NOT HERE AT THE NIH. 2196 01:43:49,480 --> 01:43:52,160 AND IF ERIC WAS A LITTLE OLDER I 2197 01:43:52,160 --> 01:43:53,760 MIGHT HAVE TAUGHT HIM IN HIS 2198 01:43:53,760 --> 01:43:57,440 FIRST YEAR GENETICS COURSE BUT I 2199 01:43:57,440 --> 01:43:59,080 THINK HE IS A LITTLE TOO YOUNG 2200 01:43:59,080 --> 01:44:04,360 FOR THAT BECAUSE I LEFT HARVARD 2201 01:44:04,360 --> 01:44:05,360 IN 1993. 2202 01:44:05,360 --> 01:44:07,880 >> I JUST WANTED TO TELL OUR 2203 01:44:07,880 --> 01:44:09,760 THREE NEW MEMBERS HOW MUCH I 2204 01:44:09,760 --> 01:44:11,240 THOROUGHLY ENJOYED YOUR 2205 01:44:11,240 --> 01:44:11,840 PRESENTATIONS. 2206 01:44:11,840 --> 01:44:12,680 THE PERSONAL STORIES. 2207 01:44:12,680 --> 01:44:19,520 THEY ARE REALLY COMPELLING AND 2208 01:44:19,520 --> 01:44:19,680 -- 2209 01:44:19,680 --> 01:44:20,480 [ AUDIO DIFFICULTIES ] 2210 01:44:20,480 --> 01:44:21,880 THANK YOU FOR JOINING AND I 2211 01:44:21,880 --> 01:44:22,680 SHARING. 2212 01:44:22,680 --> 01:44:28,400 I ENJOYED IT VERY MUCH. 2213 01:44:28,400 --> 01:44:29,960 >> THANK YOU, LINDSEY FOR THAT 2214 01:44:29,960 --> 01:44:30,880 COMMENT. 2215 01:44:30,880 --> 01:44:33,600 ALSO A COMMENT -- THANKING CHRIS 2216 01:44:33,600 --> 01:44:38,240 FOR REPRESENTING MDA SO WELL. 2217 01:44:38,240 --> 01:44:47,760 DO WE HAVE QUESTIONS FOR CHRIS? 2218 01:44:47,760 --> 01:44:50,080 >> I DON'T SEE ANY BUT MAYBE A 2219 01:44:50,080 --> 01:44:52,560 LITTLE BREAK WILL INSPIRE PEOPLE 2220 01:44:52,560 --> 01:44:55,360 TO ADD QUESTIONS TO THE CHAT BOX 2221 01:44:55,360 --> 01:44:56,920 IF THEY SO DESIRE. 2222 01:44:56,920 --> 01:44:59,920 I WANT TO THANK OUR THREE NEW 2223 01:44:59,920 --> 01:45:04,160 MEMBERS AS WELL FOR REALLY 2224 01:45:04,160 --> 01:45:09,080 PUTTING THE PERSONAL IN THE 2225 01:45:09,080 --> 01:45:14,280 STORY THIS IS WHY WE HAVE 2226 01:45:14,280 --> 01:45:15,480 SOMETHING CALLED THE VOICE OF 2227 01:45:15,480 --> 01:45:18,960 THE PARTICIPANT BECAUSE YOU CAN 2228 01:45:18,960 --> 01:45:21,440 INTELLECTUAL LIES BUT THERE IS * 2229 01:45:21,440 --> 01:45:24,080 NOTHING ABOUT HEARING ABOUT 2230 01:45:24,080 --> 01:45:25,200 SOMEONE'S INDIVIDUAL LIVED 2231 01:45:25,200 --> 01:45:26,880 EXPERIENCE WHETHER IT'S THE TIME 2232 01:45:26,880 --> 01:45:29,000 IT TOOK TO GET THE DIAGNOSIS OR 2233 01:45:29,000 --> 01:45:32,160 THE FACT THAT SOMEBODY HAS 2234 01:45:32,160 --> 01:45:33,800 PARTICIPATED IN THERAPY AND IT 2235 01:45:33,800 --> 01:45:34,560 HAS HELPED THEM. 2236 01:45:34,560 --> 01:45:37,640 IT MAKES A HUGE DIFFERENCE. 2237 01:45:37,640 --> 01:45:39,440 ALL THREE OF YOU DO HAVE A 2238 01:45:39,440 --> 01:45:41,440 PERSONAL CONNECTION AND WE 2239 01:45:41,440 --> 01:45:43,000 APPRECIATE YOU SHARING YOUR 2240 01:45:43,000 --> 01:45:43,760 STORIES. 2241 01:45:43,760 --> 01:45:45,120 SO WE LOOK FORWARD TO WORKING 2242 01:45:45,120 --> 01:45:46,680 WITH YOU OVER THE NEXT FEW 2243 01:45:46,680 --> 01:45:48,000 MONTHS AND YEARS. 2244 01:45:48,000 --> 01:45:49,480 SO WITH THAT BEING RIGHT ON TIME 2245 01:45:49,480 --> 01:45:51,760 WE'RE GOING TO TAKE A SHORT 2246 01:45:51,760 --> 01:45:52,840 BREAK AND WE'LL COME BACK AT 2247 01:45:52,840 --> 01:45:55,000 3:00. 2248 01:45:55,000 --> 01:45:56,440 >> I'D LIKE TO WELCOME EVERYONE 2249 01:45:56,440 --> 01:45:58,880 BACK AND THE REST OF THE MANAGES 2250 01:45:58,880 --> 01:46:00,720 WILL BE DEVOTED TO PRESENTATIONS 2251 01:46:00,720 --> 01:46:06,360 AND DISCUSSIONS ON THE -- 2252 01:46:06,360 --> 01:46:14,320 SUBJECT OF DRUG REPURPOSING. 2253 01:46:14,320 --> 01:46:16,280 THERE ARE MANY EXAMPLES IN 2254 01:46:16,280 --> 01:46:18,440 MEDICINE IN WHICH A DRUG 2255 01:46:18,440 --> 01:46:19,560 ORIGINALLY DEVELOPED AND 2256 01:46:19,560 --> 01:46:21,880 APPROVED FOR ONE CONDITION IS 2257 01:46:21,880 --> 01:46:24,320 FOUND TO BE EFFICACIOUS FOR 2258 01:46:24,320 --> 01:46:25,040 ANOTHER CONDITION. 2259 01:46:25,040 --> 01:46:27,480 BY SEEKING OUT OPPORTUNITIES WE 2260 01:46:27,480 --> 01:46:29,480 MAY BE ABLE TO ACCELERATE THE 2261 01:46:29,480 --> 01:46:32,040 IDENTIFICATION OF DRUGS FOR 2262 01:46:32,040 --> 01:46:34,040 TREATING MUSCULAR DYSTROPHYs. 2263 01:46:34,040 --> 01:46:43,400 SO I'M GOIN I'LL -- I'M NOW GOING TO TURN 2264 01:46:43,400 --> 01:46:45,120 THE MEETING OVER TO GLEN 2265 01:46:45,120 --> 01:46:46,680 NUCKOLLS. 2266 01:46:46,680 --> 01:46:48,080 >> THANK YOU. 2267 01:46:48,080 --> 01:46:50,160 CAN YOU SEE MY SLIDES OKAY? 2268 01:46:50,160 --> 01:46:50,960 IS THAT A YES. 2269 01:46:50,960 --> 01:46:53,840 I WANT TO GIVE A BREACH 2270 01:46:53,840 --> 01:46:54,960 INTRODUCTION TO THIS SECTION 2271 01:46:54,960 --> 01:46:57,160 WHICH IS GOING TO BE 2272 01:46:57,160 --> 01:46:58,200 INTERESTING. 2273 01:46:58,200 --> 01:47:01,280 DRUG REPURPOSING AS DR. BIANCHI 2274 01:47:01,280 --> 01:47:02,720 MENTIONED IS TAKING A DRUG THAT 2275 01:47:02,720 --> 01:47:05,240 IS APPROVED FOR ONE CONDITION 2276 01:47:05,240 --> 01:47:07,840 AND TESTING IT FOR SAFETY AND 2277 01:47:07,840 --> 01:47:09,520 EFFICACY IN OTHER CONDITIONS AND 2278 01:47:09,520 --> 01:47:12,960 REASON THIS WORKS IS THAT THERE 2279 01:47:12,960 --> 01:47:15,080 SOMETIMES IS A COMMON SET OF 2280 01:47:15,080 --> 01:47:17,120 PATHOLOGICAL PROCESSES THAT LIE 2281 01:47:17,120 --> 01:47:19,680 DOWNSTREAM OF MANY DIFFERENT 2282 01:47:19,680 --> 01:47:22,560 GENE MUTATIONS AND SO THESE 2283 01:47:22,560 --> 01:47:24,880 COMMON PATHOLOGIES WHICH MIGHT 2284 01:47:24,880 --> 01:47:28,000 INCLUDE ABNORMAL IMMUNE 2285 01:47:28,000 --> 01:47:30,320 RESPONSES, FIBROSIS, 2286 01:47:30,320 --> 01:47:32,720 MITOCHONDRIAL FALL FUNCTION. 2287 01:47:32,720 --> 01:47:35,240 COMPROMISED CELLULAR REPAIR -- 2288 01:47:35,240 --> 01:47:36,720 CAN CAUSE DIFFERENT CONDITIONS 2289 01:47:36,720 --> 01:47:38,320 DEPENDING ON THE TISSUES AND 2290 01:47:38,320 --> 01:47:40,920 CELLS THAT ARE AFFECTED. 2291 01:47:40,920 --> 01:47:46,160 AND FROM A DRUG DEVELOPMENT 2292 01:47:46,160 --> 01:47:49,480 STANDPOINT REPURPOSING HAS SOME 2293 01:47:49,480 --> 01:47:49,920 ADVANTAGES. 2294 01:47:49,920 --> 01:47:52,680 THIS SLIDE SHOWS THE CLASSIC 2295 01:47:52,680 --> 01:47:56,680 PIPELINE OF DRUG DISCOVERY FROM 2296 01:47:56,680 --> 01:47:58,680 DISEASE MECHANISMS THROUGH 2297 01:47:58,680 --> 01:48:02,640 TARGETS AND PRECLINICAL 2298 01:48:02,640 --> 01:48:04,080 DEVELOPMENT THROUGH CLINICAL 2299 01:48:04,080 --> 01:48:14,360 TRIALS AND THEN EVEN THEN TO FDA APPROVAL AND 2300 01:48:14,360 --> 01:48:16,360 THEN TO PATIENTS. 2301 01:48:16,360 --> 01:48:19,080 THIS IS A VALLEY OF DEATH. 2302 01:48:19,080 --> 01:48:21,040 THAT IS BECAUSE A LOT OF 2303 01:48:21,040 --> 01:48:24,280 DISCOVERIES COME INTO THIS STEPS 2304 01:48:24,280 --> 01:48:26,920 AND VERY FEW SAFE AND EFFECTIVE 2305 01:48:26,920 --> 01:48:28,160 DRUGS RESULT FROM THE END OF 2306 01:48:28,160 --> 01:48:30,720 THIS PIPELINE. 2307 01:48:30,720 --> 01:48:33,880 AND SO DRUG DEVELOPMENT -- I 2308 01:48:33,880 --> 01:48:35,880 MEAN DRUG REPURPOSING CAN HAVE 2309 01:48:35,880 --> 01:48:40,080 AN OPPORTUNITY TO LINK DISEASE 2310 01:48:40,080 --> 01:48:42,280 MECHANISMS DIRECTLY TO APPROVED 2311 01:48:42,280 --> 01:48:42,840 DRUGS. 2312 01:48:42,840 --> 01:48:44,920 IT'S PROBABLY NOT LIKELY THAT 2313 01:48:44,920 --> 01:48:47,760 YOU CAN SKIP ALL OF THE STEPS IN 2314 01:48:47,760 --> 01:48:48,240 BETWEEN. 2315 01:48:48,240 --> 01:48:50,800 THERE MAY BE THE NEED FOR SOME 2316 01:48:50,800 --> 01:48:52,280 CHEMICAL MODIFICATION OR 2317 01:48:52,280 --> 01:48:53,800 FORMULATION OR ADDITIONAL 2318 01:48:53,800 --> 01:48:56,080 PRECLINICAL TESTING BUT DRUG 2319 01:48:56,080 --> 01:48:58,080 REPURPOSING CAN KIND OF 2320 01:48:58,080 --> 01:49:00,280 STREAMLINE MANY OF THESE STEPS 2321 01:49:00,280 --> 01:49:03,120 AND RESULT IN MUCH HIGHER 2322 01:49:03,120 --> 01:49:07,200 SUCCESS RATE THAN WHAT IS SEEN 2323 01:49:07,200 --> 01:49:09,000 IN REGULAR NEW DRUG DEVELOPMENT. 2324 01:49:09,000 --> 01:49:14,240 SO HERE IS A LIST OF CURRENTLY 2325 01:49:14,240 --> 01:49:15,760 ACTIVE CLINICAL TRIALS THAT ARE 2326 01:49:15,760 --> 01:49:18,160 REPURPOSING DRUGS FOR MUSCULAR 2327 01:49:18,160 --> 01:49:21,880 DYSTROPHYs INCLUDING STUDIES 2328 01:49:21,880 --> 01:49:26,520 IN -- FHSN AND THERE ARE MORE 2329 01:49:26,520 --> 01:49:30,080 STUDIES GOING ON FOR PRECLINICAL 2330 01:49:30,080 --> 01:49:33,160 TESTING OF DRUG REPURPOSING AND 2331 01:49:33,160 --> 01:49:36,800 ALSO DEPRIORITIZED DRUGS SO 2332 01:49:36,800 --> 01:49:39,320 DRUGS THAT ARE BEING DEVELOPED 2333 01:49:39,320 --> 01:49:42,240 FOR ONE CONDITION AND FOR SOME 2334 01:49:42,240 --> 01:49:44,880 REASON EITHER FAILED IN CLINICAL 2335 01:49:44,880 --> 01:49:46,920 TRIALS OR WHATEVER REASON DIDN'T 2336 01:49:46,920 --> 01:49:49,440 BECOME APPROVED BUT STILL HAVE A 2337 01:49:49,440 --> 01:49:50,680 LOT OF IMPORTANT INFORMATION 2338 01:49:50,680 --> 01:49:53,040 WITH THEM AND MAY BE QUITE 2339 01:49:53,040 --> 01:49:54,680 EFFECTIVE IN HITTING THEIR 2340 01:49:54,680 --> 01:49:58,200 TARGETS AND SAFE IN HUMANS. 2341 01:49:58,200 --> 01:50:00,720 SO I WANT TO BRING TO YOUR 2342 01:50:00,720 --> 01:50:02,200 ATTENTION A TWO-DAY WORKSHOP 2343 01:50:02,200 --> 01:50:06,480 THAT TOOK PLACE IN 2019 AND IT 2344 01:50:06,480 --> 01:50:08,480 WAS ORGANIZED BY HEATHER STONE 2345 01:50:08,480 --> 01:50:10,960 FROM THE FDA AND SHE IS WITH OUR 2346 01:50:10,960 --> 01:50:15,360 MEETING TODAY AND BOBBY FROM 2347 01:50:15,360 --> 01:50:18,640 NCATS AND THIS IS FOCUSING ON 2348 01:50:18,640 --> 01:50:22,160 REPURPOSE EGG OF OFF PATENT 2349 01:50:22,160 --> 01:50:23,520 DRUGS. 2350 01:50:23,520 --> 01:50:26,080 THIS SEEMS TO BE THE MOST 2351 01:50:26,080 --> 01:50:28,080 DIFFICULT CASE WHEN THERE IS NO 2352 01:50:28,080 --> 01:50:29,960 PATENT PROTECTIONS. 2353 01:50:29,960 --> 01:50:31,840 THEY TALKED ABOUT REGULATORY 2354 01:50:31,840 --> 01:50:33,840 PATHWAYS AND INCENTIVES. 2355 01:50:33,840 --> 01:50:35,400 IF YOU'RE IMPLEMENTED IN A 2356 01:50:35,400 --> 01:50:37,600 DEEPER DIVE IN THIS TOPIC I 2357 01:50:37,600 --> 01:50:40,040 RECOMMEND THAT YOU CHECK OUT THE 2358 01:50:40,040 --> 01:50:42,040 VIDEO WHICH IS AVAILABLE AT THE 2359 01:50:42,040 --> 01:50:45,040 NIH VIDEO CAST AND THEY HAVE ON 2360 01:50:45,040 --> 01:50:47,880 THE NCATS WEBSITE A NICE SUMMARY 2361 01:50:47,880 --> 01:50:50,320 OF THE CHALLENGES AND POSSIBLE 2362 01:50:50,320 --> 01:50:52,000 SOLUTIONS FOR SOME OF THE 2363 01:50:52,000 --> 01:50:53,760 CHALLENGES AND HEATHER IS GOING 2364 01:50:53,760 --> 01:50:55,640 TO BE TELLING US ABOUT SOME OF 2365 01:50:55,640 --> 01:50:56,680 THE PROGRESS FROM THE 2366 01:50:56,680 --> 01:50:57,480 RECOMMENDATIONS OF THAT 2367 01:50:57,480 --> 01:50:59,760 WORKSHOP. 2368 01:50:59,760 --> 01:51:02,000 SO FOR TODAY'S SESSION WE ONLY 2369 01:51:02,000 --> 01:51:03,360 HAVE ABOUT TWO HOURS TO TALK 2370 01:51:03,360 --> 01:51:04,120 ABOUT THIS. 2371 01:51:04,120 --> 01:51:07,560 BUT WE HAVE SOME REALLY GREAT 2372 01:51:07,560 --> 01:51:10,200 PRESENTATIONS FROMLY SWEENEY 2373 01:51:10,200 --> 01:51:14,440 FROM THE UNIVERSITY OF FLORIDA. 2374 01:51:14,440 --> 01:51:16,280 LIZOTTE UNGER FROM NCATS. 2375 01:51:16,280 --> 01:51:19,200 BARBARA GOODMAN AND HEATHER 2376 01:51:19,200 --> 01:51:21,240 STONE FROM FDA. 2377 01:51:21,240 --> 01:51:25,960 SO WE WILL BE TALKING SOME ABOUT 2378 01:51:25,960 --> 01:51:28,080 RESOURCE THAT'S ARE AVAILABLE TO 2379 01:51:28,080 --> 01:51:31,800 SUPPORT DRUG REPURPOSING 2380 01:51:31,800 --> 01:51:33,040 STUDIES. 2381 01:51:33,040 --> 01:51:36,600 MANY OF THE MUSCULAR DYSTROPHY 2382 01:51:36,600 --> 01:51:38,600 NIH INSTITUTES WE HAVE A VARIETY 2383 01:51:38,600 --> 01:51:40,960 OF EXISTING FUNDING OPPORTUNITY 2384 01:51:40,960 --> 01:51:42,600 ANNOUNCEMENTS THAT CAN ALSO BE 2385 01:51:42,600 --> 01:51:44,840 USED FOR DRUG REPURPOSING 2386 01:51:44,840 --> 01:51:45,480 STUDIES. 2387 01:51:45,480 --> 01:51:48,040 THESE INCLUDE STUDIES OF 2388 01:51:48,040 --> 01:51:49,960 PRECLINICAL THERAPY DEVELOPMENT 2389 01:51:49,960 --> 01:51:52,600 OR OUR CLINICAL TRIAL FUNDING 2390 01:51:52,600 --> 01:51:53,840 OPPORTUNITIES. 2391 01:51:53,840 --> 01:51:55,880 SO IF ANYONE IS INTERESTED IN 2392 01:51:55,880 --> 01:51:57,040 PURSUING THESE FUNDING 2393 01:51:57,040 --> 01:51:58,520 OPPORTUNITIES CONTACT ME. 2394 01:51:58,520 --> 01:52:02,600 AND I CAN PUT YOU IN CONTACT 2395 01:52:02,600 --> 01:52:04,080 WITH THE PROGRAM DIRECTORS FROM 2396 01:52:04,080 --> 01:52:05,160 THE OTHER INSTITUTES. 2397 01:52:05,160 --> 01:52:07,480 THE GOAL FOR TODAY'S SESSION ARE 2398 01:52:07,480 --> 01:52:09,280 TO INCREASE AWARENESS OF THE 2399 01:52:09,280 --> 01:52:11,440 PROCESS OF DRUG PREPURPOSING AND 2400 01:52:11,440 --> 01:52:12,760 THEN THE OPPORTUNITIES AND THE 2401 01:52:12,760 --> 01:52:14,680 CHALLENGES THAT ARE PROPOSED BY 2402 01:52:14,680 --> 01:52:15,880 THIS TYPE OF RESEARCH. 2403 01:52:15,880 --> 01:52:19,440 AND THEN TO HIGHLIGHT RESOURCES 2404 01:52:19,440 --> 01:52:20,960 BOTH FINANCIAL AND SCIENTIFIC 2405 01:52:20,960 --> 01:52:22,880 THAT ARE AVAILABLE FOR 2406 01:52:22,880 --> 01:52:25,400 PRECLINICAL STUDIES AND REALLY 2407 01:52:25,400 --> 01:52:29,480 TO TRY TO ENCOURAGE 2408 01:52:29,480 --> 01:52:30,680 PUBLIC/PRIVATE PARTNERSHIPS AND 2409 01:52:30,680 --> 01:52:32,400 TO PROMOTE PARTNERSHIPS IN THIS 2410 01:52:32,400 --> 01:52:34,880 TYPE REPURPOSING STUDIES. 2411 01:52:34,880 --> 01:52:36,760 SO WITH THAT I'LL TURN THE 2412 01:52:36,760 --> 01:52:39,920 MEETING BACK OVER TO 2413 01:52:39,920 --> 01:52:40,440 DR. BIANCHI. 2414 01:52:40,440 --> 01:52:42,040 >> THANK YOU FOR SETTING THE 2415 01:52:42,040 --> 01:52:45,320 STAGE FOR THIS SESSION. 2416 01:52:45,320 --> 01:52:48,320 OUR FIRST PRESENTINGER IS LEE 2417 01:52:48,320 --> 01:52:50,440 SWEENEY FROM THE UNIVERSITY OF 2418 01:52:50,440 --> 01:52:56,680 FLORIDA. 2419 01:52:56,680 --> 01:53:00,520 AND TITLE IS REPURPOSING DRUGS 2420 01:53:00,520 --> 01:53:08,840 FOR THE TREATMENT OF DUCHENNE 2421 01:53:08,840 --> 01:53:10,120 MUSCULAR DYSTROPHY 2422 01:53:10,120 --> 01:53:14,640 >> THANK YOU. 2423 01:53:14,640 --> 01:53:16,640 THANK YOU FOR THE INVITATION. 2424 01:53:16,640 --> 01:53:19,560 I WANT TO TALK ABOUT REPURPOSING 2425 01:53:19,560 --> 01:53:21,640 FROM THE ACADEMIC STANDPOINT 2426 01:53:21,640 --> 01:53:23,280 BECAUSE IT IS SOMETHING THAT MY 2427 01:53:23,280 --> 01:53:25,400 LAB IS INVOLVED IN AND HAS BEEN 2428 01:53:25,400 --> 01:53:26,480 INVOLVED IN FOR A NUMBER OF 2429 01:53:26,480 --> 01:53:28,680 YEARS. 2430 01:53:28,680 --> 01:53:30,800 FIRST OF ALL I'LL MAKE A 2431 01:53:30,800 --> 01:53:31,680 DISCLOSURE. 2432 01:53:31,680 --> 01:53:34,240 I DO CONSULT WITH A NUMBER OF 2433 01:53:34,240 --> 01:53:36,480 BIOTECH AND PHARMA COMPANIES BUT 2434 01:53:36,480 --> 01:53:38,480 IT SHOULD NOT HAVE ANY IMPACT ON 2435 01:53:38,480 --> 01:53:40,680 ANYTHING I'M GOING TO SAY TODAY. 2436 01:53:40,680 --> 01:53:43,280 I'M GOING TO TALK ABOUT 2437 01:53:43,280 --> 01:53:47,120 SPECIFICALLY DRUGS FOR DUCHENNE 2438 01:53:47,120 --> 01:53:48,280 MUSCULAR DYSTROPHY WHICH NEEDS 2439 01:53:48,280 --> 01:53:52,000 NO INTRODUCTION TO THIS GROUP. 2440 01:53:52,000 --> 01:53:53,240 THESE ARE THE POINTS THAT I WANT 2441 01:53:53,240 --> 01:53:54,440 TO MAKE. 2442 01:53:54,440 --> 01:53:56,400 THE STEPS INVOLVED AT LEAST IN 2443 01:53:56,400 --> 01:53:59,080 OUR EFFORTS IS TO FIRST A FEW 2444 01:53:59,080 --> 01:54:02,720 DRUGS THAT ENGAGE A PATHWAY THAT 2445 01:54:02,720 --> 01:54:06,680 WE THINK DRIVES COUCHE-TARD 2446 01:54:06,680 --> 01:54:10,920 DISEASE IN ANIMAL MODELS AND IN 2447 01:54:10,920 --> 01:54:22,200 PATIENTS. 2448 01:54:22,200 --> 01:54:27,120 -- 2449 01:54:27,120 --> 01:54:29,880 >> WE WOULD LIKE TO IDENTIFY THE 2450 01:54:29,880 --> 01:54:31,480 APPROPRIATE DISEASE STAGE WHERE 2451 01:54:31,480 --> 01:54:34,280 WE WOULD INTERVENE IN PATIENTS 2452 01:54:34,280 --> 01:54:35,800 AND POSSIBLE END POINTS TO QUINN 2453 01:54:35,800 --> 01:54:38,000 THE DESIGN OF THE DUCHESS OF 2454 01:54:38,000 --> 01:54:40,480 CAMBRIDGE TRIAL AND THEN 2455 01:54:40,480 --> 01:54:41,920 DEPENDING ON THE DRUG STATISTICS 2456 01:54:41,920 --> 01:54:56,480 WE TAKE ONE OF TWO ROUTES. 2457 01:54:56,480 --> 01:54:58,080 AS YOU KNOW IF THE DRUG IS 2458 01:54:58,080 --> 01:55:00,560 APPROVED FOR A COMMON INDICATION 2459 01:55:00,560 --> 01:55:04,280 AS MANY THESE DRUGS ARE THEN THE 2460 01:55:04,280 --> 01:55:06,080 POSSIBILITY OF GETTING AN 2461 01:55:06,080 --> 01:55:09,000 APPROVAL FOR AN ORPHAN DISEASE 2462 01:55:09,000 --> 01:55:10,680 INDICATION IS -- QUITE 2463 01:55:10,680 --> 01:55:12,120 ECONOMICALLY APPEALING TO THE 2464 01:55:12,120 --> 01:55:14,280 SPONSOR BECAUSE IT EXTENDS THEIR 2465 01:55:14,280 --> 01:55:15,320 PATENT PROTECTION. 2466 01:55:15,320 --> 01:55:18,520 IF THE DRUG IS OFF PATENT IT'S 2467 01:55:18,520 --> 01:55:19,840 MORE DIFFICULT SO WE HAVE TO 2468 01:55:19,840 --> 01:55:22,560 RAISE FUNDS TO DO AN ACADEMIC 2469 01:55:22,560 --> 01:55:25,280 PROOF OF CONCEPT STUDY AT OUR 2470 01:55:25,280 --> 01:55:26,440 INSTITUTION AND THEN IF 2471 01:55:26,440 --> 01:55:29,000 NECESSARY AND WANTED FROM THE 2472 01:55:29,000 --> 01:55:30,880 DATA WE WOULD THEN TRY TO 2473 01:55:30,880 --> 01:55:32,880 FOLLOW-UP WITH THE LARGER STUDY 2474 01:55:32,880 --> 01:55:36,280 AND THE GOAL IS TO CONVINCE THE 2475 01:55:36,280 --> 01:55:40,000 PRESCRIBERS TO ADOPT THE DRUG AS 2476 01:55:40,000 --> 01:55:41,720 PART OF THE STANDARD OF CARE NOT 2477 01:55:41,720 --> 01:55:43,400 NECESSARILY TO GET A LABEL 2478 01:55:43,400 --> 01:55:45,680 CHANGE FROM THE FDA BECAUSE OF 2479 01:55:45,680 --> 01:55:47,880 THE TIME AND EXPENSE INVOLVED IN 2480 01:55:47,880 --> 01:55:51,400 THAT BUT AS YOU KNOW PREDNISONE 2481 01:55:51,400 --> 01:55:53,720 WAS NEVER APPROVED FOR DUCHENNE 2482 01:55:53,720 --> 01:55:55,200 MUSCULAR DYSTROPHY YET IT IS THE 2483 01:55:55,200 --> 01:55:56,440 STANDARD OF CARE. 2484 01:55:56,440 --> 01:55:58,960 ALTHOUGH -- WAS LATER APPROVED 2485 01:55:58,960 --> 01:56:01,120 FOR DUCHENNE MUSCULAR DYSTROPHY. 2486 01:56:01,120 --> 01:56:03,040 OUR GOAL IS TO STAY OFF OF THIS 2487 01:56:03,040 --> 01:56:05,120 LIST AND THIS IS THE LIST THAT 2488 01:56:05,120 --> 01:56:09,640 WAS RECENTLY PUBLISHED FROM I 2489 01:56:09,640 --> 01:56:11,320 FORGOT WHAT THE PUBLICATION WAS. 2490 01:56:11,320 --> 01:56:12,680 IT'S UP THERE. 2491 01:56:12,680 --> 01:56:16,040 BUT AT ANY RATE IT'S THE GROWING 2492 01:56:16,040 --> 01:56:17,760 LIST OF DRUGS THAT HAVE FAILED 2493 01:56:17,760 --> 01:56:20,320 IN TRIALS FOR DUCHENNE AND THEY 2494 01:56:20,320 --> 01:56:22,760 FAIL FOR A VARIETY OF REASONS. 2495 01:56:22,760 --> 01:56:25,320 BUT I WOULD LIKE TO POINT OUT 2496 01:56:25,320 --> 01:56:26,880 THAT THERE WERE THREE ATTEMPTS 2497 01:56:26,880 --> 01:56:29,440 OF DRUG REPURPOSING ON THIS 2498 01:56:29,440 --> 01:56:30,040 LIST. 2499 01:56:30,040 --> 01:56:33,680 ONE OF WHICH MY LAB WAS HEAVILY 2500 01:56:33,680 --> 01:56:35,000 INVOLVED IN AND I'M GOING TO 2501 01:56:35,000 --> 01:56:36,560 TALK ABOUT THAT AS AN EXAMPLE. 2502 01:56:36,560 --> 01:56:38,280 BUT I WOULD LIKE TO POINT OUT 2503 01:56:38,280 --> 01:56:41,440 THAT THE AUTHORS OF THE PAPER 2504 01:56:41,440 --> 01:56:45,000 POINT OUT THAT THESE DRUGS 2505 01:56:45,000 --> 01:56:49,280 USUALLY FAIL FOR TWO MAJOR 2506 01:56:49,280 --> 01:56:58,960 REASONS' ONE IS IN ADD QUIE -- ADEQUACY 2507 01:56:58,960 --> 01:57:06,160 OF THE MOST COMMON PRECLINICAL 2508 01:57:06,160 --> 01:57:09,680 MODELS NAMELY THE MOUSE ON THE 2509 01:57:09,680 --> 01:57:10,000 BACKGROUND. 2510 01:57:10,000 --> 01:57:12,240 BUT ALSO MANY PROBLEMS WITH 2511 01:57:12,240 --> 01:57:13,280 TRIAL DESIGN. 2512 01:57:13,280 --> 01:57:16,040 AND THAT STEMS FROM HAVING TOO 2513 01:57:16,040 --> 01:57:18,320 FEW PATIENT ETC. IN THE TRIAL 2514 01:57:18,320 --> 01:57:19,880 WHICH CREATES A PROBLEM BECAUSE 2515 01:57:19,880 --> 01:57:22,520 OF THE HIGH DISEASE VARIABILITY, 2516 01:57:22,520 --> 01:57:24,480 PATIENT TO PATIENT AND ALSO 2517 01:57:24,480 --> 01:57:26,160 INAPPROPRIATE END POINTS IN MANY 2518 01:57:26,160 --> 01:57:29,480 CASES GIVEN THE MECHANISM OF THE 2519 01:57:29,480 --> 01:57:30,720 THERAPEUTIC THAT WAS BEING 2520 01:57:30,720 --> 01:57:32,880 LOOKED AT AND SOMETIMES THIS IS 2521 01:57:32,880 --> 01:57:35,080 DUE TO INADEQUATE UNDERSTANDING 2522 01:57:35,080 --> 01:57:36,280 OF THE DISEASE. 2523 01:57:36,280 --> 01:57:39,480 AND SO I WANT TO ADDRESS THE 2524 01:57:39,480 --> 01:57:41,600 FACT THAT REPURPOSING REALLY 2525 01:57:41,600 --> 01:57:45,120 STARTS WITH A DETAILED 2526 01:57:45,120 --> 01:57:47,400 UNDERSTANDING OF THE DISEASE 2527 01:57:47,400 --> 01:57:48,960 PATHOGENESIS IN THE HUMAN 2528 01:57:48,960 --> 01:57:51,480 PATIENTS AS WELL AS THE DISEASE 2529 01:57:51,480 --> 01:57:53,080 PATHOGENESIS IN OUR ANIMAL 2530 01:57:53,080 --> 01:57:54,840 MODELS THAT WE'RE GOING TO TRY 2531 01:57:54,840 --> 01:57:59,880 TO USE TO VALIDATE THE DESIRE TO 2532 01:57:59,880 --> 01:58:01,800 MOVE THE DRUG FORWARD. 2533 01:58:01,800 --> 01:58:06,280 TO ADDRESS THE HUMAN SIDE MY 2534 01:58:06,280 --> 01:58:09,080 COLLEGES AND I REALIZED THAT WE 2535 01:58:09,080 --> 01:58:10,880 NEEDED TO HAVE MORE DETAILED 2536 01:58:10,880 --> 01:58:14,080 NATURAL HISTORY SO WE STARTED A 2537 01:58:14,080 --> 01:58:16,280 NATURAL HISTORY STUDY THAT WAS 2538 01:58:16,280 --> 01:58:23,880 FUNDED BY INITIALLY BY NINDS AND 2539 01:58:23,880 --> 01:58:27,560 CONTINUES TO BE FUNDED -- WHICH 2540 01:58:27,560 --> 01:58:31,320 WE CALLED THE IMAGING PROJECT TO 2541 01:58:31,320 --> 01:58:33,520 REFLECT THE FACT THAT WE LOOK AT 2542 01:58:33,520 --> 01:58:35,520 MORE THAN JUST DUCHENNE BUT THE 2543 01:58:35,520 --> 01:58:38,800 IDEA WAS NOT ONLY TO DEVELOP A 2544 01:58:38,800 --> 01:58:44,800 DATABASE BUT ALSO TO DEVELOP 2545 01:58:44,800 --> 01:58:48,360 MRI/MRS AS A BIOMARKER TO GIVE 2546 01:58:48,360 --> 01:58:50,600 QUANTITATIVE MEASURES AND TOOLS 2547 01:58:50,600 --> 01:58:51,880 FOR CLINICAL TRIAL DEVELOPMENT. 2548 01:58:51,880 --> 01:58:53,600 ESPECIALLY TOOLS THAT WOULD GIVE 2549 01:58:53,600 --> 01:58:57,240 YOU EARLIER READ OUTS ON DRUG 2550 01:58:57,240 --> 01:58:58,120 EFFICACY THAN YOU CAN GET FROM 2551 01:58:58,120 --> 01:59:00,160 THE FUNCTIONAL MEASURES ALONE 2552 01:59:00,160 --> 01:59:01,680 AND THIS IS TO SHOW YOU THAT 2553 01:59:01,680 --> 01:59:05,440 WHAT THE M-R AND THE THIGH 2554 01:59:05,440 --> 01:59:05,680 MUSCLES. 2555 01:59:05,680 --> 01:59:07,880 DUCHENNE PATIENTS APPEARS AND 2556 01:59:07,880 --> 01:59:10,000 THE MOST STRIKING FEATURE IS 2557 01:59:10,000 --> 01:59:11,840 THIS PROGRESSIVE REPLACEMENT OF 2558 01:59:11,840 --> 01:59:14,480 THE MUSCLE WHICH IS IN DARK GRAY 2559 01:59:14,480 --> 01:59:17,560 BY THIS HYPER INTENSE AREA WHICH 2560 01:59:17,560 --> 01:59:22,680 IS FAT AS THE MUSCLES BASICALLY 2561 01:59:22,680 --> 01:59:23,600 REGENERATION FAILS AS THE 2562 01:59:23,600 --> 01:59:27,680 PATIENTS GET OLDER AND THERE IS 2563 01:59:27,680 --> 01:59:30,760 TOTAL MUSCLE REPLACEMENT AS THE 2564 01:59:30,760 --> 01:59:35,800 DISEASE PROGRESSES AND THIS IS A 2565 01:59:35,800 --> 01:59:38,000 FATTY MATRIX BUT THE FIBROSIS 2566 01:59:38,000 --> 01:59:41,120 WOULD BE VERY DARK IN THESE 2567 01:59:41,120 --> 01:59:42,400 IMAGES AND YOU CAN SEE THE 2568 01:59:42,400 --> 01:59:45,080 PROBLEM IS THAT HERE NOW HALLING 2569 01:59:45,080 --> 01:59:47,400 THE FATTY IN FILTRATION AND WHAT 2570 01:59:47,400 --> 01:59:51,880 WE USE AS A SENTINEL MUSCLE -- 2571 01:59:51,880 --> 01:59:54,440 BECAUSE IT PROGRESSES RAPIDLY 2572 01:59:54,440 --> 02:00:00,680 OVER THE AGE RANGE AND THERE FOR 2573 02:00:00,680 --> 02:00:02,920 IS A CANDIDATE FOR A SURROGATE 2574 02:00:02,920 --> 02:00:03,800 IN CLINICAL TRIALS. 2575 02:00:03,800 --> 02:00:07,680 THERE IS A LOT OF HETEROGENEITY 2576 02:00:07,680 --> 02:00:08,520 PATIENT TO PATIENT. 2577 02:00:08,520 --> 02:00:10,880 YOU HAVE TO USE LARGE NUMBERS OR 2578 02:00:10,880 --> 02:00:13,680 USE THESE SORTS OF TOOLS TO 2579 02:00:13,680 --> 02:00:16,440 SELECT A SUBSET OF THE PATIENTS 2580 02:00:16,440 --> 02:00:18,720 THAT ARE PROGRESSING AT A 2581 02:00:18,720 --> 02:00:19,960 SIMILAR RATE IN ORDER TO PERFORM 2582 02:00:19,960 --> 02:00:22,880 NEW TRIALS. 2583 02:00:22,880 --> 02:00:24,760 THE REASON THE MUSCLE IS BEING 2584 02:00:24,760 --> 02:00:27,440 REPLACED IS THE NORMAL 2585 02:00:27,440 --> 02:00:29,760 REGENERATION THAT FOLLOWS -- 2586 02:00:29,760 --> 02:00:32,080 DEGENERATION BY MUSCLE ENERGY 2587 02:00:32,080 --> 02:00:34,520 GOES AWRY IN THIS DISEASE AS IT 2588 02:00:34,520 --> 02:00:35,760 DOES IN A NUMBER OF MUSCULAR 2589 02:00:35,760 --> 02:00:38,640 DYSTROPHY AND THE INFLAMMATORY 2590 02:00:38,640 --> 02:00:40,680 PHASE IS NEVER RESOLVED SO YOU 2591 02:00:40,680 --> 02:00:43,680 NEVER PROGRESS TO NORMAL MUSCLE 2592 02:00:43,680 --> 02:00:44,800 REPAIR. 2593 02:00:44,800 --> 02:00:48,280 IT INVOLVES REPLACEMENT OF THE 2594 02:00:48,280 --> 02:00:50,680 CELLS WITH CELLS THAT COME FROM 2595 02:00:50,680 --> 02:00:58,360 OTHER STEM CELLS -- THAT CAN 2596 02:00:58,360 --> 02:01:03,680 RESULT IN DEPOSITION FIBROSIS 2597 02:01:03,680 --> 02:01:03,880 AND FAT. 2598 02:01:03,880 --> 02:01:05,680 THERE ARE A NUMBER OF MECHANISMS 2599 02:01:05,680 --> 02:01:07,760 THAT ARE AT PLAY HERE. 2600 02:01:07,760 --> 02:01:11,840 THAT REALLY PROVIDE THE TARGETS 2601 02:01:11,840 --> 02:01:13,640 OTHER THAN REPLACEMENT OF THE 2602 02:01:13,640 --> 02:01:19,320 MISSING PROTEIN WHICH IS DESTROY 2603 02:01:19,320 --> 02:01:22,320 EN. 2604 02:01:22,320 --> 02:01:31,200 * COURT CORD STEROIDS -- THEY 2605 02:01:31,200 --> 02:01:36,360 SLOW THE INFLAMMATION AND ALLOW 2606 02:01:36,360 --> 02:01:37,400 GENERATION. 2607 02:01:37,400 --> 02:01:40,080 SLOWING THE DISEASE. 2608 02:01:40,080 --> 02:01:43,040 THE MAJOR PRECLINICAL CHALLENGE 2609 02:01:43,040 --> 02:01:45,920 IS TO FIND A MODEL THAT MIMICS 2610 02:01:45,920 --> 02:01:50,080 THE HUMAN DISEASE SO WE CAN LOOK 2611 02:01:50,080 --> 02:01:57,800 AT DIFFERENT TARGETED PATHWAYSES 2612 02:01:57,800 --> 02:01:58,080 PATHWAYSES. 2613 02:01:58,080 --> 02:02:00,920 THE PROBLEM ALL OF THESE FACE IS 2614 02:02:00,920 --> 02:02:03,840 NONE OF THEM SHOW THE DEGREE OF 2615 02:02:03,840 --> 02:02:07,480 FATTY REPLACEMENT THAT WE SEE IN 2616 02:02:07,480 --> 02:02:18,480 HUMANS. 2617 02:02:18,480 --> 02:02:22,200 -- SO I THINK THE ONLY MODEL 2618 02:02:22,200 --> 02:02:25,080 HERE THAT IS REALLY NOT THAT 2619 02:02:25,080 --> 02:02:26,840 USEFUL IS THE ONE THAT HAS BEEN 2620 02:02:26,840 --> 02:02:29,200 USED THE MOST WHICH IS THE 2621 02:02:29,200 --> 02:02:30,680 STANDARD MDX MOUSE. 2622 02:02:30,680 --> 02:02:33,920 MANY HAVE A LOT OF THE HALLMARKS 2623 02:02:33,920 --> 02:02:38,640 OF THE HUMAN DISEASE THAT 2624 02:02:38,640 --> 02:02:44,560 RESULTS IN BASICALLY A 2625 02:02:44,560 --> 02:02:45,800 CHESSATION ARE MUSCLE 2626 02:02:45,800 --> 02:02:46,280 GENERATION. 2627 02:02:46,280 --> 02:02:54,480 THIS IS COMPARING * AND LOOKING 2628 02:02:54,480 --> 02:02:56,400 AT PRECLINICAL STUDIES AT THIS 2629 02:02:56,400 --> 02:02:57,880 POINT AND I WANT TO RETURN TO 2630 02:02:57,880 --> 02:03:01,880 WHAT I SAID ABOUT THE FAILED 2631 02:03:01,880 --> 02:03:02,480 STUDY. 2632 02:03:02,480 --> 02:03:05,000 THE REASON THAT WE EMBARKED 2633 02:03:05,000 --> 02:03:08,320 ABOUT THAT WAS BECAUSE OF THE 2634 02:03:08,320 --> 02:03:12,680 FACT THAT IT CAN -- INCREASE 2635 02:03:12,680 --> 02:03:16,000 BLOOD FLOW AND BLOOD FLOW 2636 02:03:16,000 --> 02:03:17,120 REGULATION IS INAPPROPRIATE IN 2637 02:03:17,120 --> 02:03:22,280 THE DUCHENNE PATIENTS BECAUSE 2638 02:03:22,280 --> 02:03:25,120 THEY CANNOT INCREASE BLOOD FLOW 2639 02:03:25,120 --> 02:03:26,840 DURING EXERCISE. 2640 02:03:26,840 --> 02:03:31,080 IT COULD LEAD TO MUSCLE DAMAGE 2641 02:03:31,080 --> 02:03:32,360 BUT ALSO THERE ARE EFFECTS 2642 02:03:32,360 --> 02:03:36,400 INSIDE THE MUSCLE CELLS 2643 02:03:36,400 --> 02:03:44,360 THEMSELVES OF THE -- INHIBITION 2644 02:03:44,360 --> 02:03:49,200 WHICH CAN LEAD TO POTENTIALLY 2645 02:03:49,200 --> 02:03:50,240 MITOCHONDRIAL FUNCTION. 2646 02:03:50,240 --> 02:03:53,080 BUT WHAT THESE IN HIPPORS DO IS 2647 02:03:53,080 --> 02:03:57,000 RAISE LEVELS WHICH CAN LEAD TO 2648 02:03:57,000 --> 02:03:59,280 PKG ACTIVATIONS AND IT HAS A 2649 02:03:59,280 --> 02:04:01,880 NUMBER OF IMPORTANT TARGETS 2650 02:04:01,880 --> 02:04:04,200 INCLUDING TRIP C CHANNELS WHICH 2651 02:04:04,200 --> 02:04:07,320 ARE NOT REGULATED BOTH IN THE 2652 02:04:07,320 --> 02:04:10,760 HEART AND IN THE SKELETAL MUSCLE 2653 02:04:10,760 --> 02:04:13,080 AND SILENCING THOSE COULD LEAD 2654 02:04:13,080 --> 02:04:14,880 TO A NUMBER OF BENEFITS AND 2655 02:04:14,880 --> 02:04:17,320 WITHIN THE VASCULAR SMOOTH 2656 02:04:17,320 --> 02:04:22,480 MUSCLE THE INCREASING -- LEVELS 2657 02:04:22,480 --> 02:04:24,160 LEADS TO BASAL DILATION AND 2658 02:04:24,160 --> 02:04:27,080 BETTER BLOOD FLOW. 2659 02:04:27,080 --> 02:04:29,960 NOW THE PRECLINICAL DATA AS WELL 2660 02:04:29,960 --> 02:04:37,040 AS HUMAN DATA -- WAS NUMBER ONE 2661 02:04:37,040 --> 02:04:41,720 SHOWING THAT THE MICE GIVEN 2662 02:04:41,720 --> 02:04:43,400 COULD EXERCISE BETTER. 2663 02:04:43,400 --> 02:04:45,960 THEY COULD HAVE REPEAT BOUTS OF 2664 02:04:45,960 --> 02:04:49,280 EXCITED WHERE THEY NORMALLY DO 2665 02:04:49,280 --> 02:04:50,880 NOT. 2666 02:04:50,880 --> 02:04:54,400 IT ALSO PREVENTED FUNCTIONAL US 2667 02:04:54,400 --> 02:05:00,960 THEME YA -- * ISCHEMIA -- AND IT 2668 02:05:00,960 --> 02:05:02,960 HAD A MUCH EVEN GREATER IMPACT 2669 02:05:02,960 --> 02:05:05,520 ON THE HEARTS OF THOSE DOGS 2670 02:05:05,520 --> 02:05:07,760 BECAUSE IT IMPROVED BOTH 2671 02:05:07,760 --> 02:05:09,760 DIASTOLIC FUNCTION AND DELAYED 2672 02:05:09,760 --> 02:05:17,360 THE ON SET OF DISSTOLLIC 2673 02:05:17,360 --> 02:05:18,400 FUNCTION. 2674 02:05:18,400 --> 02:05:19,760 THE SIX MINUTE WALK TEST. 2675 02:05:19,760 --> 02:05:26,200 THE TRIAL HAD TWO DOSES AS WELL 2676 02:05:26,200 --> 02:05:29,000 AS A PLACEBO GROUP. 2677 02:05:29,000 --> 02:05:30,840 WE HAD IN MIND THAT WE WOULD DO 2678 02:05:30,840 --> 02:05:33,680 THE STUDY BUT SINCE THEY HAD 2679 02:05:33,680 --> 02:05:35,720 ENOUGH TIME LEFT ON THEIR PATENT 2680 02:05:35,720 --> 02:05:37,120 THAT THEY COULD ORGANIZE AND 2681 02:05:37,120 --> 02:05:41,000 CONDUCT A STUDY WHICH WOULD GIVE 2682 02:05:41,000 --> 02:05:43,120 THEM AN ORPHAN DISEASE 2683 02:05:43,120 --> 02:05:44,680 APPLICATION THEY DECIDED THEY 2684 02:05:44,680 --> 02:05:45,880 WOULD DO THE STUDY. 2685 02:05:45,880 --> 02:05:47,880 AT THE TIME WE WERE DOING THIS 2686 02:05:47,880 --> 02:05:50,000 THE SIX MINUTE WALK TEST WAS THE 2687 02:05:50,000 --> 02:05:51,680 ONLY OUTCOME MEASURE THAT HAD 2688 02:05:51,680 --> 02:05:54,240 BEEN USED SO WE THOUGHT IT 2689 02:05:54,240 --> 02:05:56,800 REASONABLE GIVEN THE IMPACT ON 2690 02:05:56,800 --> 02:05:58,280 EXERCISE ABILITY BUT THAT SHOWED 2691 02:05:58,280 --> 02:06:00,360 A LACK OF UNDERSTANDING OF HOW 2692 02:06:00,360 --> 02:06:01,840 THE PATIENTS REALLY CON CONDUCT 2693 02:06:01,840 --> 02:06:04,640 THE SIX MINUTE WALK AND IT'S NOT 2694 02:06:04,640 --> 02:06:06,880 REALLY A FATIGUE TEST AT ALL. 2695 02:06:06,880 --> 02:06:09,080 THEY TAKE A VERY MEASURED PACE 2696 02:06:09,080 --> 02:06:12,560 AND REALLY PROBABLY ARE NOT 2697 02:06:12,560 --> 02:06:16,680 PUTTING THEIR MUSCLES IN ANY 2698 02:06:16,680 --> 02:06:18,600 FUNCTION,S SCHEME WHAT. 2699 02:06:18,600 --> 02:06:21,280 * IT COULD BE A FAILURE OF OUR 2700 02:06:21,280 --> 02:06:23,840 CHOSEN END POINT OR IT COULD BE 2701 02:06:23,840 --> 02:06:26,600 THAT BOYS WERE NOT ACTIVE ENOUGH 2702 02:06:26,600 --> 02:06:29,000 WHEN THEY WERE NOT BEING TESTED 2703 02:06:29,000 --> 02:06:32,080 TO ACTUALLY HAVE ANY FUNCTIONAL 2704 02:06:32,080 --> 02:06:34,520 ISCHEMIA IN THEIR MUSCLES OR IT 2705 02:06:34,520 --> 02:06:36,240 COULD BE THAT THE LEVELS WAS TOO 2706 02:06:36,240 --> 02:06:39,680 LOW IN THE HUMAN PARENTS VERSUS 2707 02:06:39,680 --> 02:06:41,280 THE MICE SO WE'VE GONE BACK TO 2708 02:06:41,280 --> 02:06:43,760 LOOK AT THIS BECAUSE THE SUB 2709 02:06:43,760 --> 02:06:45,520 STUDIES DID SHOW SOME TRENDS 2710 02:06:45,520 --> 02:06:49,200 TOWARD IMPROVED ARM FUNCTION AND 2711 02:06:49,200 --> 02:06:49,880 CARDIAC FUNCTION. 2712 02:06:49,880 --> 02:06:53,480 7 AND SO WE NOW HAVE HOPE THAT 2713 02:06:53,480 --> 02:06:55,920 IN FACT THEY DO HAVE A ADEQUATE 2714 02:06:55,920 --> 02:07:00,240 NUMBERS OF PDE5 IN THEIR LEG 2715 02:07:00,240 --> 02:07:03,200 MUSCLES SO THAT THE DRUG DOES 2716 02:07:03,200 --> 02:07:05,560 OVERCOME FUNCTIONAL ISCHEMIA IN 2717 02:07:05,560 --> 02:07:06,520 THE LEGS. 2718 02:07:06,520 --> 02:07:08,840 THEY PROBABLY NEVER EXERCISED 2719 02:07:08,840 --> 02:07:10,600 ENOUGH TO EXPOSE THE PROBLEM AND 2720 02:07:10,600 --> 02:07:13,360 IF WE WANTED AN OUTCOME MEASURE 2721 02:07:13,360 --> 02:07:15,440 WE SHOULD HAVE CHOSEN SOMETHING 2722 02:07:15,440 --> 02:07:16,960 THAT SHOWED THE DRUG IMPROVED 2723 02:07:16,960 --> 02:07:19,680 THEIR ABILITY TO EXERCISE EVEN 2724 02:07:19,680 --> 02:07:21,640 THOUGH IT MAY NOT HAVE AFFECTED 2725 02:07:21,640 --> 02:07:23,080 THEIR LONG-TERM DISEASE 2726 02:07:23,080 --> 02:07:25,600 PROGRESSION. 2727 02:07:25,600 --> 02:07:30,760 A CARDIAC MEASURE WOULD BE BEST 2728 02:07:30,760 --> 02:07:34,200 BUT DUE TO LIMITATIONS WE DID 2729 02:07:34,200 --> 02:07:35,720 NOT GO THAT ROUTE. 2730 02:07:35,720 --> 02:07:37,840 I'LL GIVE YOU ONE LAST EXAMPLE. 2731 02:07:37,840 --> 02:07:39,840 TRYING TO GO AFTER THE SAME 2732 02:07:39,840 --> 02:07:46,440 PATHWAY THAT WE THINK THESE 2733 02:07:46,440 --> 02:07:51,920 CORTICO STEROIDS -- MAY EFFECT. 2734 02:07:51,920 --> 02:07:53,680 IT FAILS BECAUSE OF THE CROSS 2735 02:07:53,680 --> 02:07:56,280 TALK BETWEEN THE VARIOUS 2736 02:07:56,280 --> 02:07:59,080 STEM-CELL POPULATIONS AND THE 2737 02:07:59,080 --> 02:08:01,080 INFLAMMATORY CELLS THAT DRIVE 2738 02:08:01,080 --> 02:08:03,640 REGENERATION BUT ALSO DRIVE -- 2739 02:08:03,640 --> 02:08:05,960 TO REGENERATION DURING THE 2740 02:08:05,960 --> 02:08:10,680 DISEASE AND THE MICROFAUNALS ARE 2741 02:08:10,680 --> 02:08:14,080 A BIG PLAYER. 2742 02:08:14,080 --> 02:08:16,440 AND THEY CHANGE THE SIGNALS THAT 2743 02:08:16,440 --> 02:08:18,520 THEY SECRET WHICH ARE REALLY 2744 02:08:18,520 --> 02:08:22,240 IMPORTANT IN TRIGGERING THE FAT 2745 02:08:22,240 --> 02:08:23,360 DIFFERENTIATION INTO CELLS THAT 2746 02:08:23,360 --> 02:08:26,600 DO NOT ALLOW THE RECOVERY FROM 2747 02:08:26,600 --> 02:08:29,320 THE INJURY AS WELL AS THE MAYO 2748 02:08:29,320 --> 02:08:32,520 BLAST SIGNALS TO RECOVER FROM 2749 02:08:32,520 --> 02:08:35,200 THE INJURY SO YOU GET STUCK IN 2750 02:08:35,200 --> 02:08:37,520 THIS INFLAMMATORY PHASE. 2751 02:08:37,520 --> 02:08:40,080 THE IDEA IS TO DEVELOP A WAY TO 2752 02:08:40,080 --> 02:08:43,200 PUSH THE MACROPHAGES OUT AND 2753 02:08:43,200 --> 02:08:44,440 PROMOTE REGENERATION. 2754 02:08:44,440 --> 02:08:46,440 WHAT WE'VE CURRENTLY IDENTIFIED 2755 02:08:46,440 --> 02:08:49,840 IS A POSSIBLE CANDIDATE FOR DRUG 2756 02:08:49,840 --> 02:08:59,040 REPURPOSING OR THE PPAR GAMMA -- 2757 02:08:59,040 --> 02:09:02,040 RECEPTORS WHICH ARE -- FOR THE 2758 02:09:02,040 --> 02:09:03,040 RECEPTOR. 2759 02:09:03,040 --> 02:09:06,880 TURNS OUT THAT PPAR DRIVES THE 2760 02:09:06,880 --> 02:09:10,240 PROGRESSION AS WELL AS THE 2761 02:09:10,240 --> 02:09:11,600 SECRETION OF THE GDF3. 2762 02:09:11,600 --> 02:09:17,720 7 THESE DRUGS ARE OFF PATENT SO 2763 02:09:17,720 --> 02:09:19,680 WE'RE IN THE PROCESS OF TRYING 2764 02:09:19,680 --> 02:09:22,040 TO GENERATE ENOUGH PRECLINICAL 2765 02:09:22,040 --> 02:09:27,000 DATA TO GET FUNDS TO DO A SMALL 2766 02:09:27,000 --> 02:09:29,400 -- -- PATIENTS TO SHOW THAT IT 2767 02:09:29,400 --> 02:09:32,600 WILL BOTH REDUCE FIBROSIS, 2768 02:09:32,600 --> 02:09:34,680 DECREASE INFLAMMATION AND 2769 02:09:34,680 --> 02:09:37,840 IMPROVE REGENERATION AND 2770 02:09:37,840 --> 02:09:39,600 THEREFORE SLOW DISEASE 2771 02:09:39,600 --> 02:09:40,920 REGRESSION IN THOSE PATIENTS. 2772 02:09:40,920 --> 02:09:43,680 THIS IS THE PATHWAY THAT WE'RE 2773 02:09:43,680 --> 02:09:44,480 TAKING. 2774 02:09:44,480 --> 02:09:46,280 IT ALL BEGINS WITH HAVING AN 2775 02:09:46,280 --> 02:09:47,400 APPROPRIATE UNDERSTANDING OF THE 2776 02:09:47,400 --> 02:09:49,400 HUMAN DISEASE AS WELL AS THE 2777 02:09:49,400 --> 02:09:51,680 ANIMAL MODELS THAT WILL MODEL 2778 02:09:51,680 --> 02:09:54,080 THE KEY ASPECTS OF THE DISEASE 2779 02:09:54,080 --> 02:09:56,160 AND THEN THE DRUG STATUS WILL 2780 02:09:56,160 --> 02:09:57,600 DETERMINE THE ROUTE THAT WE 2781 02:09:57,600 --> 02:09:59,720 ULTIMATELY HAVE TO TAKE WHETHER 2782 02:09:59,720 --> 02:10:03,600 WE GO BACK TO THE SPONSOR OR THE 2783 02:10:03,600 --> 02:10:05,920 HEAVY LIFTING IS LEFT TO US AND 2784 02:10:05,920 --> 02:10:06,680 THE COMMUNITY. 2785 02:10:06,680 --> 02:10:09,800 I WANT TO KNOWLEDGE THAT THE 2786 02:10:09,800 --> 02:10:11,680 PROJECT GIVES US A LOT OF 2787 02:10:11,680 --> 02:10:14,160 SUPPORT TO ALLOW US TO GO AFTER 2788 02:10:14,160 --> 02:10:15,880 THESE PRECLINICAL TESTING OF 2789 02:10:15,880 --> 02:10:17,520 DRUGS THAT WE DON'T ACTUALLY 2790 02:10:17,520 --> 02:10:19,840 HAVE FORMAL GRANT SUPPORT FOR. 2791 02:10:19,840 --> 02:10:22,280 AS WELL AS HELPING US RECRUIT 2792 02:10:22,280 --> 02:10:25,040 PATIENTS FOR PROOF OF CONCEPT 2793 02:10:25,040 --> 02:10:27,560 STUDIES AND OF COURSE OUR 2794 02:10:27,560 --> 02:10:30,640 SUPPORT FOR OUR CENTER -- WHICH 2795 02:10:30,640 --> 02:10:33,520 ALLOWS US TO DO MUCH OF THE WORK 2796 02:10:33,520 --> 02:10:35,480 AND CAN SPIN-OFF INTO OTHER 2797 02:10:35,480 --> 02:10:35,960 PROJECTS. 2798 02:10:35,960 --> 02:10:38,600 SO I'M SORRY IF I WENT A LITTLE 2799 02:10:38,600 --> 02:10:42,240 TOO LONG BUT I'M HAPPY TO TAKE 2800 02:10:42,240 --> 02:10:42,560 ANY QUESTIONS. 2801 02:10:42,560 --> 02:10:44,560 >> THANK YOU, LEE. 2802 02:10:44,560 --> 02:10:47,800 SO WE ARE JUST AT TIME FOR 2803 02:10:47,800 --> 02:10:49,160 ELIZABETH'S TALK BUT WE CAN TAKE 2804 02:10:49,160 --> 02:10:49,720 ONE QUESTION. 2805 02:10:49,720 --> 02:10:53,080 AND YOU DID GIVE A VERY HELPFUL 2806 02:10:53,080 --> 02:10:55,720 OVERVIEW. 2807 02:10:55,720 --> 02:11:02,320 THANK YOU FOR THAT. 2808 02:11:02,320 --> 02:11:05,280 I'M NOT SEEING QUESTIONS. 2809 02:11:05,280 --> 02:11:07,240 ANYBODY WANT TO RAISE THEIR 2810 02:11:07,240 --> 02:11:07,440 HAND. 2811 02:11:07,440 --> 02:11:12,760 SCROLLING THROUGH THE 2812 02:11:12,760 --> 02:11:17,840 PARTICIPANTS. 2813 02:11:17,840 --> 02:11:18,040 OKAY. 2814 02:11:18,040 --> 02:11:19,720 IN THE INTEREST OF KEEPING 2815 02:11:19,720 --> 02:11:21,560 PEOPLE ON TIME I'M GOING TO SAY 2816 02:11:21,560 --> 02:11:24,200 THANK YOU AND FEEL FREE TO ASK 2817 02:11:24,200 --> 02:11:25,880 QUESTIONS IN THE CHAT BOX AND 2818 02:11:25,880 --> 02:11:28,880 THEN WE CAN PROBABLY ANSWER THEM 2819 02:11:28,880 --> 02:11:31,520 IN WRITING. 2820 02:11:31,520 --> 02:11:34,640 SO NEXT WE WOULD LIKE TO WELCOME 2821 02:11:34,640 --> 02:11:41,560 DR. ELIZABETH -- OTTINGER. 2822 02:11:41,560 --> 02:12:08,680 WHO SPEAKING FROM NCATS. 2823 02:12:08,680 --> 02:12:09,760 >> HI, THANK YOU. 2824 02:12:09,760 --> 02:12:10,600 WELCOME EVERYBODY. 2825 02:12:10,600 --> 02:12:12,480 CAN YOU HEAR ME AND SEE MY 2826 02:12:12,480 --> 02:12:13,360 SCREEN? 2827 02:12:13,360 --> 02:12:14,360 >> YES. 2828 02:12:14,360 --> 02:12:15,680 >> OKAY. 2829 02:12:15,680 --> 02:12:16,160 YES. 2830 02:12:16,160 --> 02:12:18,480 SO I'M REALLY -- THANK YOU FOR 2831 02:12:18,480 --> 02:12:20,160 THIS OPPORTUNITY TO TELL YOU 2832 02:12:20,160 --> 02:12:21,880 ABOUT NCATS AND THE RESOURCE 2833 02:12:21,880 --> 02:12:22,520 THAT'S WE HAVE. 2834 02:12:22,520 --> 02:12:25,640 I'M GOING TO BE FOCUSING ON THE 2835 02:12:25,640 --> 02:12:27,680 PRECLINICAL SPACE AND GIVING YOU 2836 02:12:27,680 --> 02:12:28,680 AN EXAMPLE. 2837 02:12:28,680 --> 02:12:30,280 WE'VE DONE SOME REPURPOSING 2838 02:12:30,280 --> 02:12:32,480 PROJECTS AND TRY TO GIVE YOU OUR 2839 02:12:32,480 --> 02:12:36,240 PERSPECTIVE OF WHAT WE'VE 2840 02:12:36,240 --> 02:12:36,720 ENCOUNTERED. 2841 02:12:36,720 --> 02:12:38,360 I CAN TELL YOU A LITTLE BIT 2842 02:12:38,360 --> 02:12:39,280 ABOUT MYSELF. 2843 02:12:39,280 --> 02:12:42,920 I'VE BEEN AT NCATS ABOUT 12 2844 02:12:42,920 --> 02:12:43,520 YEARS. 2845 02:12:43,520 --> 02:12:46,400 EVEN BEFORE IT BECAME NCATS. 2846 02:12:46,400 --> 02:12:49,400 WHICH STARTED IN 2011 AND I WAS 2847 02:12:49,400 --> 02:12:50,760 PREVIOUSLY IN INDUSTRY. 2848 02:12:50,760 --> 02:12:52,200 AND JUST BECAUSE WE WERE TALKING 2849 02:12:52,200 --> 02:12:55,080 ABOUT BOSTON I DID MY POSTDOC IN 2850 02:12:55,080 --> 02:12:56,800 BOSTON. 2851 02:12:56,800 --> 02:13:00,560 SO AGAIN THIS IS JUST THE 2852 02:13:00,560 --> 02:13:02,000 MISSION OF NCATS 2853 02:13:02,000 --> 02:13:05,120 AS YOU HAVE HEARD TODAY DRUG 2854 02:13:05,120 --> 02:13:06,840 DEVELOPMENT IS DIFFICULT. 2855 02:13:06,840 --> 02:13:09,200 IT'S OFTEN 10-15 YEARS THAT WE 2856 02:13:09,200 --> 02:13:11,760 SAY TO GET A DRUG FROM FINDING 2857 02:13:11,760 --> 02:13:14,560 IT ALL THE WAY THROUGH ACTUAL 2858 02:13:14,560 --> 02:13:16,040 APPROVAL INTO PATIENTS. 2859 02:13:16,040 --> 02:13:18,680 AND AGAIN THERE IS USUALLY ABOUT 2860 02:13:18,680 --> 02:13:20,880 A 10% SUCCESS RATE AS YOU GO 2861 02:13:20,880 --> 02:13:21,480 THROUGH THE PROCESS. 2862 02:13:21,480 --> 02:13:24,560 AND I THINK OUR PREVIOUS SPEAKER 2863 02:13:24,560 --> 02:13:26,400 FOCUSED ON THE EFFICACY AND THE 2864 02:13:26,400 --> 02:13:30,520 CLINICAL HURDLES BUT THERE IS 2865 02:13:30,520 --> 02:13:32,680 ALSO EFFICACY AND TOXICITY 2866 02:13:32,680 --> 02:13:34,360 HURDLES ESPECIALLY IN THE 2867 02:13:34,360 --> 02:13:35,160 PRECLINICAL SPACE. 2868 02:13:35,160 --> 02:13:37,840 SO OUR MISSION IS ALSO TO 2869 02:13:37,840 --> 02:13:39,280 PROVIDE HEALTH SOLUTIONS AND TO 2870 02:13:39,280 --> 02:13:40,680 DO THAT THROUGH THE 2871 02:13:40,680 --> 02:13:43,960 TRANSLATIONAL SCIENCE AND TO 2872 02:13:43,960 --> 02:13:45,560 SUPPORT A MORE TREATMENTS TO 2873 02:13:45,560 --> 02:13:47,880 PATIENTS MORE QUICKLY. 2874 02:13:47,880 --> 02:13:50,040 NCATS APPROACH IS THAT WE DO A 2875 02:13:50,040 --> 02:13:52,880 LOT -- BASED ON COLLABORATIONS 2876 02:13:52,880 --> 02:13:56,640 ON SETTING UP DIFFERENT 2877 02:13:56,640 --> 02:13:57,600 PUBLIC/PRIVATE PARTNERSHIPS. 2878 02:13:57,600 --> 02:13:59,920 WE WORK WITH BOTH ACADEMICS. 2879 02:13:59,920 --> 02:14:02,480 WE WORK WITH OTHER ICs. 2880 02:14:02,480 --> 02:14:04,560 OTHER AGENCIES. 2881 02:14:04,560 --> 02:14:06,920 ALSO NONPROFITS AND WE WORK WITH 2882 02:14:06,920 --> 02:14:08,840 BIOTECH AND ALSO PHARMA. 2883 02:14:08,840 --> 02:14:11,840 AND SO WE DO THIS BY TRYING TO 2884 02:14:11,840 --> 02:14:13,520 BUILD PLATFORMS. 2885 02:14:13,520 --> 02:14:14,880 WE HAVE MANY DIFFERENT 2886 02:14:14,880 --> 02:14:15,120 PLATFORMS. 2887 02:14:15,120 --> 02:14:18,280 WE BUILD IN ORDER TO SUPPORT THE 2888 02:14:18,280 --> 02:14:20,960 TYPE OF PROJECTS IN 2889 02:14:20,960 --> 02:14:22,960 COLLABORATIONS THAT WE DO AND 2890 02:14:22,960 --> 02:14:27,280 OUR GOAL IS TO DECREASE THE TIME 2891 02:14:27,280 --> 02:14:28,880 TO DEVELOP AND GET MORE 2892 02:14:28,880 --> 02:14:31,240 THERAPIES FOR MORE DISEASES 2893 02:14:31,240 --> 02:14:34,680 FASTER WHICH IS IS A LOT OF US 2894 02:14:34,680 --> 02:14:37,040 IS OUR GOAL IN TRANSLATIONAL 2895 02:14:37,040 --> 02:14:38,840 SPACE TO REALLY HELP PATIENTS. 2896 02:14:38,840 --> 02:14:40,080 THIS IS WHERE I SIT. 2897 02:14:40,080 --> 02:14:44,480 IT'S THE DIVISION OF PRECLINICAL 2898 02:14:44,480 --> 02:14:45,160 INNOVATION. 2899 02:14:45,160 --> 02:14:47,680 SO AGAIN WE'RE NOT A GRANT 2900 02:14:47,680 --> 02:14:48,520 FUNDING. 2901 02:14:48,520 --> 02:14:50,480 WE DON'T PROVIDE GRANT FUNDING 2902 02:14:50,480 --> 02:14:52,800 FOR OUR PROJECTS' WE ACTUALLY 2903 02:14:52,800 --> 02:14:55,360 WORK WITH OUR COLLABORATORS AND 2904 02:14:55,360 --> 02:14:57,040 SET UP PROJECT TEAMS WHERE WE 2905 02:14:57,040 --> 02:14:59,600 WORK TOGETHER TO ACTUALLY 2906 02:14:59,600 --> 02:15:00,960 PROGRESS PROJECTS THROUGH THEIR 2907 02:15:00,960 --> 02:15:01,960 GOALS. 2908 02:15:01,960 --> 02:15:05,320 AND SO AGAIN OUR MISSION IN THE 2909 02:15:05,320 --> 02:15:07,440 BRANCH COMES DOWN FROM NCATS AND 2910 02:15:07,440 --> 02:15:09,440 WE'RE TRYING TO ADVANCE BOTH 2911 02:15:09,440 --> 02:15:11,200 LIKE THE ART OF COLLABORATION 2912 02:15:11,200 --> 02:15:13,960 SETTING UP THE SCIENTIFIC AND 2913 02:15:13,960 --> 02:15:16,000 OPERATIONAL WAYS TO ACTUALLY 2914 02:15:16,000 --> 02:15:18,080 CARRY OUT PROJECTS AND TO 2915 02:15:18,080 --> 02:15:19,960 DELIVER EFFECTIVE THERAPIES FOR 2916 02:15:19,960 --> 02:15:22,240 HUMAN DISEASES. 2917 02:15:22,240 --> 02:15:23,680 THESE ARE THE DIFFERENT BRANCHES 2918 02:15:23,680 --> 02:15:25,280 AND I WANT TO FOCUS HERE. 2919 02:15:25,280 --> 02:15:27,280 THIS IS A THERAPEUTIC 2920 02:15:27,280 --> 02:15:31,080 DEVELOPMENT BRANCH BUT WE ALSO 2921 02:15:31,080 --> 02:15:36,360 HAVE THE EARLY TRANSLATIONAL 2922 02:15:36,360 --> 02:15:39,600 BRANCH. 2923 02:15:39,600 --> 02:15:43,680 SO IN THE THERAPEUTIC PART OF 2924 02:15:43,680 --> 02:15:46,600 DPI THE WAY THAT WE'RE SET UP -- 2925 02:15:46,600 --> 02:15:48,280 AGAIN THESE ARE TO GET US AS 2926 02:15:48,280 --> 02:15:50,680 WE'VE SEEN WE KNOW THAT THIS IS 2927 02:15:50,680 --> 02:15:53,040 THE CHEVRON OF THE DRUG 2928 02:15:53,040 --> 02:15:53,840 DEVELOPMENT PROCESS. 2929 02:15:53,840 --> 02:15:57,360 AND THE THERAPEUTIC DEVELOPMENT 2930 02:15:57,360 --> 02:15:59,880 BRANCH SITS HERE WHERE AGAIN 2931 02:15:59,880 --> 02:16:02,160 THROUGH THE VALLEY OF DEATH HAVE 2932 02:16:02,160 --> 02:16:03,760 PROJECTS THROUGH PRECLINICAL 2933 02:16:03,760 --> 02:16:05,560 DEVELOPMENT INTO THE CLINICAL 2934 02:16:05,560 --> 02:16:06,080 PHASE. 2935 02:16:06,080 --> 02:16:08,000 AND WHAT WE DO IS WE THEN 2936 02:16:08,000 --> 02:16:09,760 SUPPORT THIS BY SETTING UP TEAMS 2937 02:16:09,760 --> 02:16:11,240 WHERE WE HAVE EXPERTISE IN ALL 2938 02:16:11,240 --> 02:16:14,080 OF THE DIFFERENT AREAS OF THE 2939 02:16:14,080 --> 02:16:16,360 ENABLING STUDIES THAT YOU NEED 2940 02:16:16,360 --> 02:16:19,720 IN ORDER TO PROGRESS INTO YOUR 2941 02:16:19,720 --> 02:16:23,520 CLINICAL PHASE OF YOUR PROJECT. 2942 02:16:23,520 --> 02:16:25,600 AND IF YOU LOOKING ATS BIT MORE 2943 02:16:25,600 --> 02:16:28,800 I WANT TO SHINE A SPOTLIGHT. 2944 02:16:28,800 --> 02:16:31,640 MORE IN DETAIL AT THIS PHASE 2945 02:16:31,640 --> 02:16:34,000 THERE IS A LOT OF AREAS THAT GO 2946 02:16:34,000 --> 02:16:34,920 INTO IT. 2947 02:16:34,920 --> 02:16:37,040 NORMALLY YOU HAVE WHICH OTHERS 2948 02:16:37,040 --> 02:16:38,840 HAVE TALKED ABOUT IS YOU HAVE 2949 02:16:38,840 --> 02:16:40,720 THAT PROOF OF CONCEPT IN YOUR 2950 02:16:40,720 --> 02:16:42,520 ANIMAL MODELS WHERE YOU HAVE 2951 02:16:42,520 --> 02:16:44,160 THAT CANDIDATE SELECTION AND 2952 02:16:44,160 --> 02:16:46,200 ONCE YOU CHOOSE THAT THEN ONCE 2953 02:16:46,200 --> 02:16:48,480 YOU HAVE YOUR CANDIDATE SELECTED 2954 02:16:48,480 --> 02:16:50,120 THEN YOU GO INTO THE DISEASE 2955 02:16:50,120 --> 02:16:53,120 ANIMAL MODELS TO SHOW YOUR 2956 02:16:53,120 --> 02:16:54,480 PHARMACOLOGY AND THERE ARE 2957 02:16:54,480 --> 02:16:59,280 STUDIES NEEDED FOR YOUR INVESTAL 2958 02:16:59,280 --> 02:17:01,680 NEW DRUG APPLICATION AND THESE 2959 02:17:01,680 --> 02:17:03,280 ARE THE AREAS THAT WE NORM ALLEY 2960 02:17:03,280 --> 02:17:04,080 WORK WITH. 2961 02:17:04,080 --> 02:17:06,280 WE DON'T NORMALLY SUPPORT 2962 02:17:06,280 --> 02:17:08,080 CLINICAL TRIALS BUT WE DO WORK 2963 02:17:08,080 --> 02:17:09,640 WITH OTHER ICs. 2964 02:17:09,640 --> 02:17:11,280 SOMETIMES THE CLINICAL TRIALS 2965 02:17:11,280 --> 02:17:12,760 ESPECIALLY IN THE AREA DISEASE 2966 02:17:12,760 --> 02:17:15,520 SPACE ARE RUN AT THE CLINICAL 2967 02:17:15,520 --> 02:17:17,560 CENTER OR IF A PARTICULAR TRIAL 2968 02:17:17,560 --> 02:17:19,440 NEEDS FOR EXAMPLE PHASE II 2969 02:17:19,440 --> 02:17:20,560 CLINICAL MATERIAL WE SUPPORT 2970 02:17:20,560 --> 02:17:22,760 THAT OR IF THEY NEED REGULATORY 2971 02:17:22,760 --> 02:17:24,840 SUPPORT. 2972 02:17:24,840 --> 02:17:26,360 AND SO AGAIN EVERYTHING IN THE 2973 02:17:26,360 --> 02:17:28,360 MODEL OF WHAT THE OPERATIONAL 2974 02:17:28,360 --> 02:17:33,080 AND THE WAY THIS WE'VE TRIED TO 2975 02:17:33,080 --> 02:17:35,440 OPERATIONAL LIES THE PROCESS WE 2976 02:17:35,440 --> 02:17:37,080 HAVE THIS TEAM ALL OF OUR 2977 02:17:37,080 --> 02:17:38,600 PROJECTS ARE BASED ON TEAM 2978 02:17:38,600 --> 02:17:40,920 SCIENCE AND THEY HAVE THIS * 2979 02:17:40,920 --> 02:17:42,280 INFRASTRUCTURE. 2980 02:17:42,280 --> 02:17:43,440 PRECLINICAL DEVELOPMENT PLATFORM 2981 02:17:43,440 --> 02:17:45,000 AND ONE THING THAT I WANT TO 2982 02:17:45,000 --> 02:17:48,480 POINT OUT IS THAT NCATS 2983 02:17:48,480 --> 02:17:49,880 FUNCTIONS WITH PROJECT 2984 02:17:49,880 --> 02:17:51,760 MANAGEMENT AND THAT WE TRY TO 2985 02:17:51,760 --> 02:17:54,520 PROVIDE THAT SUPPORT TO EACH 2986 02:17:54,520 --> 02:17:56,880 PROJECT WHERE THEY HAVE THAT 2987 02:17:56,880 --> 02:17:58,680 CHAMPION THAT ONE PERSON THAT 2988 02:17:58,680 --> 02:18:00,240 MAKES SURE THE PROJECT AND 2989 02:18:00,240 --> 02:18:01,880 EVERYTHING IS COORDINATED AND 2990 02:18:01,880 --> 02:18:02,360 ALL. 2991 02:18:02,360 --> 02:18:04,280 THE ACTIVITIES ARE COMING 2992 02:18:04,280 --> 02:18:04,600 TOGETHER. 2993 02:18:04,600 --> 02:18:06,080 BECAUSE THERE IS A LOT THAT GOES 2994 02:18:06,080 --> 02:18:07,040 ON IN'S PROJECT. 2995 02:18:07,040 --> 02:18:08,440 YOU HAVE TO COORDINATE. 2996 02:18:08,440 --> 02:18:09,760 IT TAKES A VILLAGE. 2997 02:18:09,760 --> 02:18:11,680 A LOT OF DIFFERENT AREAS IN 2998 02:18:11,680 --> 02:18:13,400 ORDER TO MOVE A PROJECT FORWARD. 2999 02:18:13,400 --> 02:18:15,520 SO WE WANT TO MAKE SURE THAT 3000 02:18:15,520 --> 02:18:17,800 CLINICAL IS TALKING TO 3001 02:18:17,800 --> 02:18:20,080 PRECLINICAL AND THAT EVERYBODY 3002 02:18:20,080 --> 02:18:22,000 IS COMMUNICATING AND THAT IS 3003 02:18:22,000 --> 02:18:23,320 TRANSPARENT AND THE SCIENCE IS 3004 02:18:23,320 --> 02:18:25,000 MOVING ALONG. 3005 02:18:25,000 --> 02:18:29,440 SO IF WE GET INTO DRUG 3006 02:18:29,440 --> 02:18:31,600 REPURPOSING -- THIS WAS ADAPTED 3007 02:18:31,600 --> 02:18:34,080 FROM A DRUG DISCOVERY TO SAY 3008 02:18:34,080 --> 02:18:36,280 THAT AGAIN THERE ARE DIFFERENT 3009 02:18:36,280 --> 02:18:38,880 WAYS THAT YOU CAN TALK ABOUT 3010 02:18:38,880 --> 02:18:40,760 REPURPOSING OF THE WAY THAT 3011 02:18:40,760 --> 02:18:42,800 PEOPLE DESCRIBE IT, DIFFERENT 3012 02:18:42,800 --> 02:18:45,480 WORDING AND THERE ARE DIFFERENT 3013 02:18:45,480 --> 02:18:47,080 THINGS THAT YOU CAN BE TALKING 3014 02:18:47,080 --> 02:18:49,400 ABOUT WHETHER IT'S CURRENT 3015 02:18:49,400 --> 02:18:50,960 CANDIDATES, APPROVED DRUGS. 3016 02:18:50,960 --> 02:18:52,600 FAILED CANDIDATES. 3017 02:18:52,600 --> 02:18:55,680 WHETHER FROM A COMMON INDICATION 3018 02:18:55,680 --> 02:18:57,280 TO AN ORPHAN INDICATION. 3019 02:18:57,280 --> 02:18:59,840 FOR RARE DISEASES AND DIFFERENT 3020 02:18:59,840 --> 02:19:01,880 WAYS TO REPURPOSE A MOLECULE SO 3021 02:19:01,880 --> 02:19:04,960 YOU MIGHT HAVE TO CHANGE THE 3022 02:19:04,960 --> 02:19:07,840 FORMULATION THE DOSAGE AND MAKE 3023 02:19:07,840 --> 02:19:09,160 IT A COMBINATION WITH ANOTHER 3024 02:19:09,160 --> 02:19:12,080 DRUG OR THE WAY THAT YOU DELIVER 3025 02:19:12,080 --> 02:19:16,480 IT AND ONE THING IS THE MORE 3026 02:19:16,480 --> 02:19:18,720 CHANGES YOU MAKE THE MORE TIME 3027 02:19:18,720 --> 02:19:20,960 IT TAKES AND THE MORE MONEY IT 3028 02:19:20,960 --> 02:19:22,040 ACTUALLY COSTS. 3029 02:19:22,040 --> 02:19:23,680 SO THERE IS A LOT MORE INVOLVED 3030 02:19:23,680 --> 02:19:25,920 WHEN YOU TAKE SOMETHING FROM ITS 3031 02:19:25,920 --> 02:19:28,280 ORIGINAL PURPOSE AND YOU TRY TO 3032 02:19:28,280 --> 02:19:30,040 REPOSITION OR REPURPOSE IT. 3033 02:19:30,040 --> 02:19:33,360 AND SO THE POWER OF REPURPOSING 3034 02:19:33,360 --> 02:19:34,920 IS THAT I THINK ALL OF US FEEL 3035 02:19:34,920 --> 02:19:37,760 IS THERE THE POTENTIAL IS THAT 3036 02:19:37,760 --> 02:19:42,920 IF YOU HAVE NORMALLY THAT 15 3037 02:19:42,920 --> 02:19:45,440 YEAR SPAN IS THERE A WAY TO CUT 3038 02:19:45,440 --> 02:19:47,040 THAT DOWN IF YOU HAVE AN 3039 02:19:47,040 --> 02:19:48,280 APPROVED THERAPY ALREADY. 3040 02:19:48,280 --> 02:19:49,680 CAN YOU UTILIZE THAT. 3041 02:19:49,680 --> 02:19:52,360 SO WE HAVE THE NCATS 3042 02:19:52,360 --> 02:19:53,920 PHARMACEUTICAL COLLECTION AND 3043 02:19:53,920 --> 02:19:56,320 THIS IS ONE WAY THAT 3044 02:19:56,320 --> 02:19:57,280 COLLABORATORS INTERACT AND IF 3045 02:19:57,280 --> 02:20:00,200 YOU HAVE A SCREEN -- AN ASSAY 3046 02:20:00,200 --> 02:20:02,080 SET UP AND YOU WOULD LIKE TO 3047 02:20:02,080 --> 02:20:03,760 SCREEN YOU CAN REACH OUT AND TRY 3048 02:20:03,760 --> 02:20:05,800 TO COLLABORATE AND HAVE THAT 3049 02:20:05,800 --> 02:20:07,280 SCREENED AGAINST -- THERE ARE 3050 02:20:07,280 --> 02:20:10,200 ABOUT 3,000 APPROVED DRUGS IN 3051 02:20:10,200 --> 02:20:11,840 THE COLLECTION AND WHAT YOU HOPE 3052 02:20:11,840 --> 02:20:14,440 IS THAT IF YOU FIND A DRUG THAT 3053 02:20:14,440 --> 02:20:16,280 YOU -- IF YOU HAVE A HIT THAT 3054 02:20:16,280 --> 02:20:17,680 POSSIBLY YOU COULD SHAVE OFF A 3055 02:20:17,680 --> 02:20:24,120 LOT OF THAT TIME RATHER -- THAN 3056 02:20:24,120 --> 02:20:26,080 GOING BACK TO THE BEGINNING. 3057 02:20:26,080 --> 02:20:29,200 AND CAN BE EASIER TO GET TO THE 3058 02:20:29,200 --> 02:20:30,520 FDA APPROVAL. 3059 02:20:30,520 --> 02:20:32,960 THE IDEA FOR THE FASTEST ROUTE 3060 02:20:32,960 --> 02:20:34,880 TO PATIENTS WOULD BE IF YOU HAVE 3061 02:20:34,880 --> 02:20:36,680 PRIOR APPROVAL IN THE U.S. 3062 02:20:36,680 --> 02:20:41,240 IF YOU'RE PURSUING A U.S. FDA 3063 02:20:41,240 --> 02:20:43,160 IND AND IF YOU HAVE THE ACCESS 3064 02:20:43,160 --> 02:20:45,560 AND ABILITY TO USE EXISTING 3065 02:20:45,560 --> 02:20:47,040 DATA. 3066 02:20:47,040 --> 02:20:48,920 SO SOMETIMES IF YOU CAN HAVE 3067 02:20:48,920 --> 02:20:52,240 ACCESS TO THE DRUG OR THE DRUG 3068 02:20:52,240 --> 02:20:53,680 MASTER FILE SO THAT YOU WOULD 3069 02:20:53,680 --> 02:20:56,480 KNOW HOW TO ACTUALLY -- OR TO 3070 02:20:56,480 --> 02:20:58,160 UTILIZE THE MANUFACTURING OF 3071 02:20:58,160 --> 02:21:01,360 THAT SUBSTANCE -- IF YOU DON'T 3072 02:21:01,360 --> 02:21:03,280 CHANGE THE CMC PROCESS. 3073 02:21:03,280 --> 02:21:06,880 IF YOU HAVE THE SAME ROUTE OF 3074 02:21:06,880 --> 02:21:07,240 ADMINISTRATION. 3075 02:21:07,240 --> 02:21:09,280 YOU REALLY USE THE SAME DOSAGE 3076 02:21:09,280 --> 02:21:11,640 AND FORMULATION AND IF THERE WAS 3077 02:21:11,640 --> 02:21:13,520 ANY PRECEDENT IF YOU'RE TRYING 3078 02:21:13,520 --> 02:21:16,000 TO MOVE INTO A PEDIATRIC 3079 02:21:16,000 --> 02:21:17,960 POPULATION IF THAT WAS NEEDED 3080 02:21:17,960 --> 02:21:19,720 AND OF COURSE THIS IS NOT ALWAYS 3081 02:21:19,720 --> 02:21:21,840 THE CASE AND MOST TIMES IT'S NOT 3082 02:21:21,840 --> 02:21:22,600 THE CASE. 3083 02:21:22,600 --> 02:21:23,760 YOU HAVE TO CHANGE SOMETHING. 3084 02:21:23,760 --> 02:21:26,720 SO THE MORE YOU CHANGE THE MORE 3085 02:21:26,720 --> 02:21:28,200 THE PROCESS GETS MORE 3086 02:21:28,200 --> 02:21:31,520 COMPLICATED AS TO HOW YOU'LL 3087 02:21:31,520 --> 02:21:34,520 ACTUALLY GET TO IND FILING AND 3088 02:21:34,520 --> 02:21:36,680 TO TEST IN PATIENTS' WE WORK IN 3089 02:21:36,680 --> 02:21:39,000 THE RARE DISEASE SPACE SO 3090 02:21:39,000 --> 02:21:40,880 REPURPOSING FOR RARE DISEASES 3091 02:21:40,880 --> 02:21:42,680 THE PROBLEM IS THAT THERE IS 3092 02:21:42,680 --> 02:21:47,080 LIKE MORE THAN 7,000 DISEASES. 3093 02:21:47,080 --> 02:21:48,280 ABOUT 30 MILLION PEOPLE IN THE 3094 02:21:48,280 --> 02:21:50,520 U.S. AND LESS THAN 10% APPROVED 3095 02:21:50,520 --> 02:21:52,760 THERAPIES FOR THE NUMBER OF RARE 3096 02:21:52,760 --> 02:21:55,760 DISEASES AND ABOUT 50% THAT ARE 3097 02:21:55,760 --> 02:21:56,560 AFFECTED ARE CHILDREN. 3098 02:21:56,560 --> 02:21:58,560 SO I THINK EVERYONE HERE HAS 3099 02:21:58,560 --> 02:22:01,360 DISCUSSED AND IS AWARE THERE ARE 3100 02:22:01,360 --> 02:22:06,360 MANY CHALLENGES ESPECIALLY FOR 3101 02:22:06,360 --> 02:22:08,000 REPURPOSED MOLECULES -- AND 3102 02:22:08,000 --> 02:22:10,080 THERE IS OFTEN CHALLENGES AND 3103 02:22:10,080 --> 02:22:12,000 PEOPLE HAVING THE FUNDING AND 3104 02:22:12,000 --> 02:22:15,000 RESOURCES THE ACADEMIC 3105 02:22:15,000 --> 02:22:16,400 EXPERIENCE, DRUG DEVELOPMENT 3106 02:22:16,400 --> 02:22:18,840 EXPERTISE AND CLINICAL TRIALS 3107 02:22:18,840 --> 02:22:20,120 ARE DIFFICULT AND ESPECIALLY 3108 02:22:20,120 --> 02:22:22,080 COMMERCIAL DEVELOPMENT PATHS AND 3109 02:22:22,080 --> 02:22:24,680 INTELLECTUAL PROPERTY IS OFTEN 3110 02:22:24,680 --> 02:22:26,880 DIFFICULT WITH PATENTS. 3111 02:22:26,880 --> 02:22:29,800 AND WHERE WE SEE THE OPPORTUNITY 3112 02:22:29,800 --> 02:22:33,040 WHERE WE'VE TRIED TO HELP PEOPLE 3113 02:22:33,040 --> 02:22:34,520 AND ESPECIALLY IN REPURPOSING 3114 02:22:34,520 --> 02:22:37,240 CAN YOU REPOSITION AND LEVERAGE 3115 02:22:37,240 --> 02:22:38,680 EXISTING DRUGS AND CAN WE 3116 02:22:38,680 --> 02:22:40,680 PROVIDE THOSE PRECLINICAL 3117 02:22:40,680 --> 02:22:45,960 EXPERTISE AND REGULATORY 3118 02:22:45,960 --> 02:22:49,920 RESOURCES TO INCREASE 3119 02:22:49,920 --> 02:22:51,400 ACCESSIBILITY AND THERAPIES. 3120 02:22:51,400 --> 02:22:53,640 I WAS GOING TO GIVE ONE EXAMPLE. 3121 02:22:53,640 --> 02:22:54,760 IT'S NOT IN THE MUSCULAR 3122 02:22:54,760 --> 02:22:56,240 DYSTROPHY SPACE BUT IT BRINGS UP 3123 02:22:56,240 --> 02:22:57,800 SOME OF THE ISSUE THAT'S WE CAME 3124 02:22:57,800 --> 02:23:00,160 ACROSS AS WE WERE TRYING TO DO 3125 02:23:00,160 --> 02:23:03,360 REPURPOSING AND IT WAS 3126 02:23:03,360 --> 02:23:07,040 REPURPOSING A EUROPEAN UNION 3127 02:23:07,040 --> 02:23:07,480 THERAPEUTIC. 3128 02:23:07,480 --> 02:23:11,840 AND WE HAD A COLLABORATOR -- AND 3129 02:23:11,840 --> 02:23:14,800 THIS WAS A SMALL BIOTECH AND SHE 3130 02:23:14,800 --> 02:23:16,120 WAS ASSOCIATED WITH BOSTON 3131 02:23:16,120 --> 02:23:17,320 UNIVERSITY OR IS ASSOCIATED AND 3132 02:23:17,320 --> 02:23:20,440 SO THESE ARE A GROUP OF THE 3133 02:23:20,440 --> 02:23:22,440 INHERITED BLOOD DISORDERS AND 3134 02:23:22,440 --> 02:23:25,880 DISEASES THAT AFFECT RED BLOOD 3135 02:23:25,880 --> 02:23:26,080 CELLS. 3136 02:23:26,080 --> 02:23:33,080 YOU KNOW CYCLICAL CELL AND -- *. 3137 02:23:33,080 --> 02:23:35,080 THERE IS ONLY ABOUT 2,000 3138 02:23:35,080 --> 02:23:38,800 PATIENTS IN THE U.S. AND 3139 02:23:38,800 --> 02:23:40,720 SICKLE-CELL HAS ABOUT 1,000. 3140 02:23:40,720 --> 02:23:42,280 THERE ARE CERTAIN POPULATIONS 3141 02:23:42,280 --> 02:23:44,880 THAT ARE MORE PROPORTIONATELY 3142 02:23:44,880 --> 02:23:47,320 AFFECTED ESPECIALLY BLACK AND 3143 02:23:47,320 --> 02:23:48,760 AFRICAN-AMERICAN AND HISPANICS 3144 02:23:48,760 --> 02:23:51,160 AND ONE IN 13 AFRICAN-AMERICAN 3145 02:23:51,160 --> 02:23:54,120 BABIES ARE BORN WITH THE 3146 02:23:54,120 --> 02:23:55,640 SICKLE-CELL TRAIT WHICH MAKES 3147 02:23:55,640 --> 02:23:57,480 THEM MORE SUSCEPTIBLE TO PASS 3148 02:23:57,480 --> 02:23:58,160 THAT ON. 3149 02:23:58,160 --> 02:23:59,960 IT'S A SIGNIFICANT HEALTH 3150 02:23:59,960 --> 02:24:02,720 PROBLEM. 3151 02:24:02,720 --> 02:24:04,520 AND SO THE CHALLENGE WAS THERE 3152 02:24:04,520 --> 02:24:07,960 WAS A POTENTIAL DRUG THAT WAS 3153 02:24:07,960 --> 02:24:09,520 APPROVED GREATER THAN 40 YEARS 3154 02:24:09,520 --> 02:24:12,200 AGO AND IT WAS ONLY AS PART OF A 3155 02:24:12,200 --> 02:24:13,840 COMBINATION PRODUCT FOR 3156 02:24:13,840 --> 02:24:16,880 PARKINSON'S DISEASES BY THE EMA. 3157 02:24:16,880 --> 02:24:20,040 THE EUROPEAN MEDICINES AND THE 3158 02:24:20,040 --> 02:24:20,840 HEALTH CANADA. 3159 02:24:20,840 --> 02:24:22,760 SO THE QUESTION WAS WHAT NEW 3160 02:24:22,760 --> 02:24:26,520 DATA WOULD BE NEEDED TO APPROVE 3161 02:24:26,520 --> 02:24:30,160 IT. 3162 02:24:30,160 --> 02:24:32,600 AGAIN THEY ALL HAVE MUTATIONS IN 3163 02:24:32,600 --> 02:24:37,720 THE BETA GLOBE IN GENE. 3164 02:24:37,720 --> 02:24:39,160 THIS IS THE HEMOGLOBIN AND IT'S 3165 02:24:39,160 --> 02:24:41,040 IN THE BETA CHAIN WHERE YOU GET 3166 02:24:41,040 --> 02:24:42,400 THE MUTATIONS. 3167 02:24:42,400 --> 02:24:45,960 SO BOTH HAVE A COMMON TARGET. 3168 02:24:45,960 --> 02:24:48,440 A COMMON MECHANISM OF ACTION. 3169 02:24:48,440 --> 02:24:53,280 AFTER YOU'RE BORN YOUR FETAL 3170 02:24:53,280 --> 02:24:56,680 HUMAN GLOBE IN STARTS TO 3171 02:24:56,680 --> 02:24:59,360 INCREASE AND YOU HAVE THE BETA 3172 02:24:59,360 --> 02:25:02,160 FORM THAT STARTS INCREASING WHEN 3173 02:25:02,160 --> 02:25:04,680 YOU HAVE THE MUTATIONS THE 3174 02:25:04,680 --> 02:25:06,360 DISEASE ONSET OCCURS. 3175 02:25:06,360 --> 02:25:08,600 SO ONE OF THE COMMON MECHANISMS 3176 02:25:08,600 --> 02:25:11,720 IS CAN YOU INCREASE THIS BETA 3177 02:25:11,720 --> 02:25:15,080 GAME A HEMOGLOBIN AGAIN TO START 3178 02:25:15,080 --> 02:25:16,320 EXPRESSING AND TO PROTECT 3179 02:25:16,320 --> 02:25:16,880 PEOPLE. 3180 02:25:16,880 --> 02:25:22,120 SO THIS MOLECULE CAME OUT OF A 3181 02:25:22,120 --> 02:25:23,800 SCREEN AND YOU SEE OVER HERE ON 3182 02:25:23,800 --> 02:25:25,920 THE RIGHT WHEN IT WAS GIVEN 3183 02:25:25,920 --> 02:25:27,960 AGAINST VEHICLE THEY SAW THE 3184 02:25:27,960 --> 02:25:31,960 INCREASES IN THE FETAL 3185 02:25:31,960 --> 02:25:32,280 HEMOGLOBIN. 3186 02:25:32,280 --> 02:25:34,080 SO WHAT DID WITH HAVE TO DO? 3187 02:25:34,080 --> 02:25:36,640 AGAIN THE DATA WAS VERY OLD. 3188 02:25:36,640 --> 02:25:38,680 THERE WAS NOT MUCH THAT WE COULD 3189 02:25:38,680 --> 02:25:40,360 LEVERAGE IN WILL TERMS AND WE 3190 02:25:40,360 --> 02:25:42,600 HAD TO SHOW EFFICACY AGAIN IN 3191 02:25:42,600 --> 02:25:44,120 THIS NEW INDICATION AND THESE 3192 02:25:44,120 --> 02:25:46,560 NEW DISEASES SO WE HAD TO DO 3193 02:25:46,560 --> 02:25:47,840 EFFICACY STUDIES. 3194 02:25:47,840 --> 02:25:49,840 WE HAD TO DO DISTRIBUTION 3195 02:25:49,840 --> 02:25:51,480 BECAUSE IT WAS GOING TO 3196 02:25:51,480 --> 02:25:53,600 CONCENTRATE -- WHAT WAS THE 3197 02:25:53,600 --> 02:25:54,640 DOSAGE GOING TO BE. 3198 02:25:54,640 --> 02:25:58,800 WE HAD TO WE WERE ABLE TO 3199 02:25:58,800 --> 02:26:01,200 ACQUIRE SOME OF THE EXISTING 3200 02:26:01,200 --> 02:26:02,880 GMPAPI BUT IT HAD TO BE 3201 02:26:02,880 --> 02:26:05,000 FORMULATED AND OF THE DRUG 3202 02:26:05,000 --> 02:26:07,320 PRODUCT AND WE HAD TO AGAIN DO 3203 02:26:07,320 --> 02:26:08,640 SAFETY AND EVALUATION. 3204 02:26:08,640 --> 02:26:10,480 BUT WHAT I DO WANT TO POINT OUT 3205 02:26:10,480 --> 02:26:13,240 IS THAT THE TIME LINE FOR US WE 3206 02:26:13,240 --> 02:26:14,640 FELT IT IS ACCELERATED. 3207 02:26:14,640 --> 02:26:17,640 WE STARTED WORKING ON THIS IN 3208 02:26:17,640 --> 02:26:17,840 2014. 3209 02:26:17,840 --> 02:26:20,440 GOT THROUGH THE EFFICACY AND THE 3210 02:26:20,440 --> 02:26:22,160 TOXICOLOGY STUDIES AND THIS WAS 3211 02:26:22,160 --> 02:26:23,920 FILED BOTH IN THE U.S. AND 3212 02:26:23,920 --> 02:26:27,480 HEALTH CANADA AND BY 2019 AGAIN 3213 02:26:27,480 --> 02:26:28,720 COLLABORATING IN THE CLINICAL 3214 02:26:28,720 --> 02:26:31,200 SPACE THE SITES WERE ESTABLISHED 3215 02:26:31,200 --> 02:26:33,520 AND THIS IS NOW ONGOING AND 3216 02:26:33,520 --> 02:26:35,080 THERE IS AS YOU CAN SEE HERE THE 3217 02:26:35,080 --> 02:26:37,760 SITES ARE BOTH IN THE U.S. AND 3218 02:26:37,760 --> 02:26:40,280 IN CANADA. 3219 02:26:40,280 --> 02:26:42,800 7 AND IT'S -- THEY PLAN TO 3220 02:26:42,800 --> 02:26:46,480 TRANSITION TO THE SICKLE-CELL. 3221 02:26:46,480 --> 02:26:48,280 SO I JUST WANTED TO STOP THERE. 3222 02:26:48,280 --> 02:26:50,240 I HOPE THAT WAS A GOOD EXAMPLE 3223 02:26:50,240 --> 02:26:53,080 TO SHOW YOU AGAIN JUST SOME 3224 02:26:53,080 --> 02:26:55,320 INFORMATION ABOUT THE CHALLENGES 3225 02:26:55,320 --> 02:26:57,040 THAT COME AND THAT YOU HAVE TO 3226 02:26:57,040 --> 02:26:59,120 THINK ABOUT IN THE PRECLINICAL 3227 02:26:59,120 --> 02:27:01,360 SPACE WHEN YOU ACTUALLY TROY TO 3228 02:27:01,360 --> 02:27:02,560 REPURPOSE A MOLECULE. 3229 02:27:02,560 --> 02:27:05,480 SO I WANT TO THANK, WE WORK IN 3230 02:27:05,480 --> 02:27:06,840 COLLABORATION AND I COULD NOT 3231 02:27:06,840 --> 02:27:07,480 LIST EVERYBODY. 3232 02:27:07,480 --> 02:27:10,960 THERE IS ABOUT 60 OF US THAT 3233 02:27:10,960 --> 02:27:12,640 WORK ON THESE THERAPEUTIC 3234 02:27:12,640 --> 02:27:13,400 DEVELOPMENT PROJECTS. 3235 02:27:13,400 --> 02:27:15,440 I WANTED TO MENTION THAT WE DID 3236 02:27:15,440 --> 02:27:18,440 HAVE A COLLABORATION WHERE WE 3237 02:27:18,440 --> 02:27:21,880 WORKED ON THE -- WHICH WHEN IT 3238 02:27:21,880 --> 02:27:27,080 CAME TO NCATS IT WAS THE V BP15 3239 02:27:27,080 --> 02:27:30,000 SO WE WORKED ON A LOT OF THAT IN 3240 02:27:30,000 --> 02:27:31,880 HELPING WITH THE PRECLINICAL 3241 02:27:31,880 --> 02:27:34,080 STUDIES IN DEVELOPING A NEW 3242 02:27:34,080 --> 02:27:35,480 SYNTHETIC ROUTE AND THE 3243 02:27:35,480 --> 02:27:36,840 MANUFACTURING AND SOME OF THE 3244 02:27:36,840 --> 02:27:38,600 PRECLINICAL AND I KNOW THAT HAS 3245 02:27:38,600 --> 02:27:39,640 MOVED THROUGH DEVELOPMENT. 3246 02:27:39,640 --> 02:27:42,160 SO AGAIN WE TRY TO PHYSICAL THAT 3247 02:27:42,160 --> 02:27:44,280 GAP THAT VALLEY OF DEATH TO HELP 3248 02:27:44,280 --> 02:27:45,120 THE PROJECTS. 3249 02:27:45,120 --> 02:27:46,480 SO THANK YOU. 3250 02:27:46,480 --> 02:27:48,040 >> THANK YOU VERY MUCH, 3251 02:27:48,040 --> 02:27:49,680 ELIZABETH AND WE'RE NOW OPEN FOR 3252 02:27:49,680 --> 02:27:50,360 QUESTIONS. 3253 02:27:50,360 --> 02:27:52,880 I THINK THAT WAS REALLY HELPFUL 3254 02:27:52,880 --> 02:27:54,000 SHOWING RESOURCE THAT'S ARE 3255 02:27:54,000 --> 02:27:56,840 AVAILABLE. 3256 02:27:56,840 --> 02:27:59,960 SO THOSE PEOPLE WHO ARE 3257 02:27:59,960 --> 02:28:02,280 ATTENDING LIVE AND PEOPLE 3258 02:28:02,280 --> 02:28:06,080 WATCHING THE WEBCAST THEY WILL 3259 02:28:06,080 --> 02:28:07,720 HAVE THE CONTACT. 3260 02:28:07,720 --> 02:28:11,600 QUESTIONS. 3261 02:28:11,600 --> 02:28:14,080 >> I PUT UP HERE IF YOU WANT TO 3262 02:28:14,080 --> 02:28:16,280 SEE AGAIN YOU CAN ALWAYS LOOK AT 3263 02:28:16,280 --> 02:28:18,840 THE NCATS WEBSITE AND MOSTLY 3264 02:28:18,840 --> 02:28:22,000 PEOPLE JUST CONTACT US IF THEY 3265 02:28:22,000 --> 02:28:23,520 ARE INTERESTED IN 3266 02:28:23,520 --> 02:28:24,000 COLLABORATIONS. 3267 02:28:24,000 --> 02:28:26,200 WE HAVE SOME APPLICATION THAT'S 3268 02:28:26,200 --> 02:28:27,880 PEOPLE DO PUT IN BUT IT'S MORE 3269 02:28:27,880 --> 02:28:29,600 ON A ROLLING BASES. 3270 02:28:29,600 --> 02:28:31,920 WE TRY TO TALK TO PEOPLE AND 3271 02:28:31,920 --> 02:28:33,400 UNDERSTAND WHAT THEIR DRUG 3272 02:28:33,400 --> 02:28:35,040 DEVELOPMENT PROJECTS ARE AND 3273 02:28:35,040 --> 02:28:38,480 WHAT THEY REALLY NEED AND TO SEE 3274 02:28:38,480 --> 02:28:40,520 IF IT FITS AND IF WE CAN SUPPORT 3275 02:28:40,520 --> 02:28:42,800 IT. 3276 02:28:42,800 --> 02:28:43,440 >> THANK YOU. 3277 02:28:43,440 --> 02:28:45,800 I'M NOT SEEING ANYTHING IN THE 3278 02:28:45,800 --> 02:28:46,440 CHAT BOX. 3279 02:28:46,440 --> 02:28:49,080 IT'S EASIER FOR ME TO SEE THE 3280 02:28:49,080 --> 02:28:50,880 CHAT BOX THAN TO SEE EVERYBODY'S 3281 02:28:50,880 --> 02:28:51,840 FACES. 3282 02:28:51,840 --> 02:28:54,600 >> I HAVE A HAND RAISED. 3283 02:28:54,600 --> 02:28:56,840 IT'S GLEN. 3284 02:28:56,840 --> 02:28:58,080 GREAT GREAT TALK. 3285 02:28:58,080 --> 02:29:00,080 LIZ. 3286 02:29:00,080 --> 02:29:02,360 I WONDER IF YOU CAN COMMENT ON 3287 02:29:02,360 --> 02:29:04,440 HOW YOUR BRANCH SELECTS PROJECTS 3288 02:29:04,440 --> 02:29:04,920 TO WORK ON. 3289 02:29:04,920 --> 02:29:09,080 HOW WOULD A RESEARCHER THAT HAS 3290 02:29:09,080 --> 02:29:12,280 A PROJECT THAT NEEDS YOUR KIND 3291 02:29:12,280 --> 02:29:13,800 OF EXPERTISE HOW WOULD THEY BE 3292 02:29:13,800 --> 02:29:15,480 ABLE TO COLLABORATE WITH YOU? 3293 02:29:15,480 --> 02:29:16,320 >> YES. 3294 02:29:16,320 --> 02:29:19,160 SO WHAT THEY NORMALLY DO IS THEY 3295 02:29:19,160 --> 02:29:20,040 CONTACT. 3296 02:29:20,040 --> 02:29:22,960 THEY CAN LOOK FOR THE CONTACT 3297 02:29:22,960 --> 02:29:26,360 HERE ON THE WEBSITE. 3298 02:29:26,360 --> 02:29:28,560 I SHOULD HAVE PUT DIRECT 3299 02:29:28,560 --> 02:29:29,160 CONTACTS. 3300 02:29:29,160 --> 02:29:31,920 WE HAVE ANDRE -- I CAN MAYBE 3301 02:29:31,920 --> 02:29:33,480 SEND THAT TO YOU IF PEOPLE ARE 3302 02:29:33,480 --> 02:29:34,160 INTERESTED. 3303 02:29:34,160 --> 02:29:36,960 THAT PEOPLE CAN CONTACT AND JUST 3304 02:29:36,960 --> 02:29:39,960 SET UP A TIME MOSTLY FIRST WE 3305 02:29:39,960 --> 02:29:41,960 WOULD TRY TO TALK TO PEOPLE AND 3306 02:29:41,960 --> 02:29:43,360 SEE WHAT THE PROJECT IS. 3307 02:29:43,360 --> 02:29:46,040 AND TO SEE IF IT FITS SORT OF IN 3308 02:29:46,040 --> 02:29:46,960 THE SCOPE. 3309 02:29:46,960 --> 02:29:49,920 A LOT DEPENDS ON SCOPE AND 3310 02:29:49,920 --> 02:29:51,640 FUNDING TOO. 3311 02:29:51,640 --> 02:29:53,920 WE'RE A SMALL PROGRAM SO WE 3312 02:29:53,920 --> 02:29:56,640 DON'T HAVE -- ENDLESS AMOUNTS OF 3313 02:29:56,640 --> 02:29:58,720 FUNDING SO WE'VE BEEN MOVING 3314 02:29:58,720 --> 02:30:01,200 MORE TOWARDS WHERE IF IT'S 3315 02:30:01,200 --> 02:30:04,080 SOMETHING THAT HAS A PARTICULAR 3316 02:30:04,080 --> 02:30:05,760 TRANSLATIONAL PROBLEM AND WE 3317 02:30:05,760 --> 02:30:07,600 FEEL IT COULD BE HELPFUL FOR THE 3318 02:30:07,600 --> 02:30:09,080 COMMUNITY IF IT'S SOMETHING IN 3319 02:30:09,080 --> 02:30:11,760 REPURPOSING OR WE'VE BEEN DOING 3320 02:30:11,760 --> 02:30:13,000 GENE THERAPY PLATFORM WHERE 3321 02:30:13,000 --> 02:30:15,240 WE'RE TRYING TO DISSEMINATE. 3322 02:30:15,240 --> 02:30:17,680 WE'RE TRYING TO I THINK WE WERE 3323 02:30:17,680 --> 02:30:20,280 MOVING FROM -- SETTING UP 3324 02:30:20,280 --> 02:30:21,520 OPERATIONAL AND SCIENTIFIC 3325 02:30:21,520 --> 02:30:23,240 PLATFORM AND NOW HOW DO WE MOVE 3326 02:30:23,240 --> 02:30:25,320 TO WHERE WE ACTUALLY KIND OF 3327 02:30:25,320 --> 02:30:28,440 PROVIDE THE INFORMATION OF WHAT 3328 02:30:28,440 --> 02:30:31,280 WE'VE LEARNED OR HOW TO GO ABOUT 3329 02:30:31,280 --> 02:30:32,640 THINGS SO SOMETIMES WHEN 3330 02:30:32,640 --> 02:30:35,600 PROJECTS HAVE A GOOD 3331 02:30:35,600 --> 02:30:36,160 TRANSLATIONAL LEARNING THAT 3332 02:30:36,160 --> 02:30:39,360 COULD BE HELPFUL LIKE AS YOU'RE 3333 02:30:39,360 --> 02:30:41,840 IN THE REPURPOSING SPACE, HOW 3334 02:30:41,840 --> 02:30:43,360 DID YOU GO ABOUT IT AND HOW 3335 02:30:43,360 --> 02:30:44,680 COULD THAT BENEFIT THE REST OF 3336 02:30:44,680 --> 02:30:45,680 THE COMMUNITY? 3337 02:30:45,680 --> 02:30:46,760 THOSE ARE THE PROJECT THAT'S I 3338 02:30:46,760 --> 02:30:49,760 THINK ARE GOING MORE TOWARDS 3339 02:30:49,760 --> 02:30:51,280 WHERE WE FEEL WE COULD HAVE A 3340 02:30:51,280 --> 02:30:53,280 LARGER IMPACT BECAUSE IT COULD 3341 02:30:53,280 --> 02:30:54,600 AFFECT MORE THAN ONE DISEASE IF 3342 02:30:54,600 --> 02:30:56,040 WE CAN PROVIDE THAT INFORMATION 3343 02:30:56,040 --> 02:30:57,800 TO PEOPLE THROUGH OUR DRUG 3344 02:30:57,800 --> 02:30:58,480 DEVELOPMENT PROCESS. 3345 02:30:58,480 --> 02:31:00,840 SO WE'RE TRYING TO BECOME VERY 3346 02:31:00,840 --> 02:31:02,440 OPEN IN SHARING INFORMATION. 3347 02:31:02,440 --> 02:31:05,400 AND GETTING THOSE STORIES OUT 3348 02:31:05,400 --> 02:31:07,960 THERE LIKE WHAT DID IT REALLY 3349 02:31:07,960 --> 02:31:09,440 TAKE IN THE PARTICULAR PROJECTS 3350 02:31:09,440 --> 02:31:11,320 FOR ABILITY TO GET TO THE CLINIC 3351 02:31:11,320 --> 02:31:21,680 AND THE PRECLINICAL SPACE. 3352 02:31:21,680 --> 02:31:23,520 THIS IS DANIEL PEREZ. 3353 02:31:23,520 --> 02:31:27,480 I HAVE A QUESTION OR COMMENT. 3354 02:31:27,480 --> 02:31:38,720 JUST IN TERMS OF -- THE NEGATIVE 3355 02:31:38,720 --> 02:31:45,800 SIDE OF TRYING TO -- THE DRUGS. 3356 02:31:45,800 --> 02:31:56,320 I OFTEN WONDER ABOUT THE 3357 02:31:56,320 --> 02:31:59,160 RATIONAL -- THAT IS CLOSE TO 3358 02:31:59,160 --> 02:32:01,520 TARGET BUT I ALSO WONDER WHAT 3359 02:32:01,520 --> 02:32:08,800 THE ISSUES ARE IN TERMS OF -- 3360 02:32:08,800 --> 02:32:12,000 THE ONE THOUGHT IS ARE WE GOING 3361 02:32:12,000 --> 02:32:13,080 THIS ROUTE BECAUSE WE HAVE NOT 3362 02:32:13,080 --> 02:32:19,680 DONE ENOUGH TO LEARN -- -- FOR 3363 02:32:19,680 --> 02:32:21,080 THE PRELIMINARY WORK. 3364 02:32:21,080 --> 02:32:23,400 WE'VE GOTTEN FRUSTRATED WITH 3365 02:32:23,400 --> 02:32:24,880 UNDERSTANDING THE MECHANISM OF 3366 02:32:24,880 --> 02:32:27,280 DISEASE SO WE'RE TRYING TO MOVE 3367 02:32:27,280 --> 02:32:28,440 BEYOND TO TRY ANYTHING THAT WE 3368 02:32:28,440 --> 02:32:32,800 CAN. 3369 02:32:32,800 --> 02:32:36,320 AND THAT IS NOT NECESSARILY THE 3370 02:32:36,320 --> 02:32:41,960 ONE -- LIKE TO BUILD MUSCLES 3371 02:32:41,960 --> 02:32:47,160 FASTER -- WE'RE NOT CONSIDERING 3372 02:32:47,160 --> 02:32:51,240 THE WHOLE CHARACTER OF THE 3373 02:32:51,240 --> 02:32:52,920 DISEASE AND THE SECOND QUESTION 3374 02:32:52,920 --> 02:32:56,880 IS -- IS WE DO HAVE A LOT OF 3375 02:32:56,880 --> 02:33:15,400 CLINICAL TRIALS -- EXCUSE ME. 3376 02:33:15,400 --> 02:33:20,680 WE DO A CLINICAL TRIAL THAT -- 3377 02:33:20,680 --> 02:33:27,080 -- ARE WE EXHAUSTING THE PATIENT 3378 02:33:27,080 --> 02:33:29,320 POPULATION THAT WITH CAN RUN THE 3379 02:33:29,320 --> 02:33:31,080 CLINICAL TRIALS ON. 3380 02:33:31,080 --> 02:33:37,240 IF I'M IN A TRIAL COULD -- -- 3381 02:33:37,240 --> 02:33:38,840 AND THERE IS A COMPETING TRIAL 3382 02:33:38,840 --> 02:33:47,800 -- -- ---CRITERIA IS NOT TO BE 3383 02:33:47,800 --> 02:33:50,680 IN ANOTHER TRIAL THEN ARE WE 3384 02:33:50,680 --> 02:33:55,680 TRYING TO GET -- THE LOW HANGING 3385 02:33:55,680 --> 02:34:12,080 FRUIT -- -- -- YOUR QUESTIONS OR 3386 02:34:12,080 --> 02:34:13,040 THOUGHTS? 3387 02:34:13,040 --> 02:34:15,400 >> I THINK AUTHORIZES REALLY 3388 02:34:15,400 --> 02:34:18,240 DIFFICULT QUESTIONS THAT WE DEAL 3389 02:34:18,240 --> 02:34:19,400 WITH ALL OF THE TIME ESPECIALLY 3390 02:34:19,400 --> 02:34:22,480 IN THE RARE DISEASE SPACE. 3391 02:34:22,480 --> 02:34:25,120 ONLY SO MANY PATIENTS AND TO TRY 3392 02:34:25,120 --> 02:34:31,840 TO TEST THE THERAPEUTICS. 3393 02:34:31,840 --> 02:34:34,640 IT'S HARD BECAUSE A LOT OF THEM 3394 02:34:34,640 --> 02:34:35,720 DON'T HAVE ANY APPROVED 3395 02:34:35,720 --> 02:34:37,080 THERAPIES. 3396 02:34:37,080 --> 02:34:39,320 SO WE REALLY HAVE BEEN WORKING 3397 02:34:39,320 --> 02:34:40,840 TOWARDS ANYTHING THAT WOULD HELP 3398 02:34:40,840 --> 02:34:42,360 IN THE DISEASE SPACE. 3399 02:34:42,360 --> 02:34:46,120 THAT WOULD ACTUALLY HELP IN THE 3400 02:34:46,120 --> 02:34:48,360 MANAGING SYMPTOMS AND I THINK 3401 02:34:48,360 --> 02:34:50,840 WE'VE BEEN MOVING MORE TOWARDS A 3402 02:34:50,840 --> 02:34:53,920 LOT OF GENE THERAPIES OR TRYING 3403 02:34:53,920 --> 02:34:56,280 TO TARGET MECHANISMS WHERE WE 3404 02:34:56,280 --> 02:35:00,080 COULD HOPEFULLY HAVE MORE OF A 3405 02:35:00,080 --> 02:35:01,560 CURATIVE EFFECT THAT WOULD HAVE 3406 02:35:01,560 --> 02:35:03,120 MORE ON THE OVER-ALL DISEASE BUT 3407 02:35:03,120 --> 02:35:06,760 I DON'T KNOW IF OTHERS WOULD 3408 02:35:06,760 --> 02:35:09,120 LIKE TO CHIME IN THAT HAVE 3409 02:35:09,120 --> 02:35:10,160 EXPERIENCE IN THE DRUG 3410 02:35:10,160 --> 02:35:59,000 DEVELOPMENT SPACE. 3411 02:35:59,000 --> 02:36:04,480 -- YOU KNOW LIMITED NUMBERS OF 3412 02:36:04,480 --> 02:36:07,320 POTENTIAL PARTICIPANTS FOR THE 3413 02:36:07,320 --> 02:36:08,920 TRIALS -- AND SO TRIALS MAY 3414 02:36:08,920 --> 02:36:11,360 START COMPETING BUT I DO AGREE 3415 02:36:11,360 --> 02:36:12,280 WITH DAN. 3416 02:36:12,280 --> 02:36:15,040 THERE NEEDS TO BE VERY GOOD 3417 02:36:15,040 --> 02:36:16,400 COMMUNICATION TO THE PATIENT 3418 02:36:16,400 --> 02:36:18,480 COMMUNITY SO THAT THEY ARE 3419 02:36:18,480 --> 02:36:20,280 MAKING INFORMED DECISIONS ABOUT 3420 02:36:20,280 --> 02:36:26,120 WHAT TRIALS TO PARTICIPATE IN. 3421 02:36:26,120 --> 02:36:27,160 >> ONE THING IN ADDITION TO THE 3422 02:36:27,160 --> 02:36:28,640 PATIENT COMMUNITY THAT I'VE 3423 02:36:28,640 --> 02:36:30,880 HEARD SECONDHAND IS -- NOT IN 3424 02:36:30,880 --> 02:36:33,120 ADDITION TO THE PATIENT 3425 02:36:33,120 --> 02:36:34,800 COMMUNITY OR PATIENT LIMITATION 3426 02:36:34,800 --> 02:36:36,920 THERE IS ALSO INFRASTRUCTURE AND 3427 02:36:36,920 --> 02:36:38,840 STAFF LIMITATIONS ON THE PEOPLE 3428 02:36:38,840 --> 02:36:40,520 RUNNING THE TRIALS AND PEOPLE 3429 02:36:40,520 --> 02:36:43,160 INVOLVED AT VARIOUS CENTERS. 3430 02:36:43,160 --> 02:36:44,720 THERE IS DEFINITELY A 3431 02:36:44,720 --> 02:36:46,200 REQUIREMENT TO BE ABLE TO SCALE 3432 02:36:46,200 --> 02:36:50,320 UP AND DOWN TO MEET THOSE NEEDS. 3433 02:36:50,320 --> 02:36:57,840 >> THIS IS CHRISTINE COLVIS. 3434 02:36:57,840 --> 02:37:01,280 I'LL JUST SAY THAT IN TERMS OF 3435 02:37:01,280 --> 02:37:02,520 REPURPOSING PARTICULARLY TALKING 3436 02:37:02,520 --> 02:37:03,760 ABOUT A DRUG THAT IS ALREADY ON 3437 02:37:03,760 --> 02:37:05,760 THE MARKET I DON'T KNOW THAT I 3438 02:37:05,760 --> 02:37:10,240 WOULD NECESSARILY GIVE THAT SORT 3439 02:37:10,240 --> 02:37:13,560 OF A LOWER RANKING THAN I WOULD 3440 02:37:13,560 --> 02:37:15,880 FOR A NEW -- FOR SOMETHING NEW 3441 02:37:15,880 --> 02:37:20,360 JUST BECAUSE IT'S PERHAPS A MORE 3442 02:37:20,360 --> 02:37:22,040 VALIDATED DRUG. 3443 02:37:22,040 --> 02:37:24,920 IF IT'S ALREADY MARKETED FOR AN 3444 02:37:24,920 --> 02:37:26,600 INDICATION. 3445 02:37:26,600 --> 02:37:29,120 PRESUMABLY IS ACTING AS 3446 02:37:29,120 --> 02:37:29,640 PREDICTED. 3447 02:37:29,640 --> 02:37:35,400 SO JUST WANTED TO MENTION THAT I 3448 02:37:35,400 --> 02:37:37,440 DON'T KNOW IF I WOULD PUT IT 3449 02:37:37,440 --> 02:37:39,760 INTO A DIFFERENT CLASS -- IT 3450 02:37:39,760 --> 02:37:42,000 WILL COMPETE FOR PATIENTS. 3451 02:37:42,000 --> 02:37:46,640 THAT'S -- ONE OF THE CHALLENGES 3452 02:37:46,640 --> 02:37:49,280 WITH DRUG DEVELOPMENT. 3453 02:37:49,280 --> 02:37:52,640 IN GENERAL. 3454 02:37:52,640 --> 02:37:56,600 BUT IF I HAD TO PRIORITIZE I 3455 02:37:56,600 --> 02:37:59,080 DON'T KNOW WHAT I WOULD PUT AT 3456 02:37:59,080 --> 02:37:59,320 THE TOP. 3457 02:37:59,320 --> 02:38:02,720 THE NEW CHEMICAL ENTITY OR THE 3458 02:38:02,720 --> 02:38:04,320 ONE THAT IS BEING REPURPOSED. 3459 02:38:04,320 --> 02:38:11,800 7 -- 3460 02:38:11,800 --> 02:38:17,120 >> IS THERE ONE MORE? 3461 02:38:17,120 --> 02:38:20,040 WE'RE GOING TO THANK LIZ AND NOW 3462 02:38:20,040 --> 02:38:23,720 MOVE ON FOR PRECLINICAL STUDIES 3463 02:38:23,720 --> 02:38:25,440 INTO CLINICAL STUDIES. 3464 02:38:25,440 --> 02:38:27,400 WE HAVE A PRESENTATION FROM 3465 02:38:27,400 --> 02:38:29,520 BARBARA GOODMAN. 3466 02:38:29,520 --> 02:38:31,880 SHE IS THE PRESIDENT AND 3467 02:38:31,880 --> 02:38:33,560 SEYFERTH OF THE NONPROFIT CURES 3468 02:38:33,560 --> 02:38:34,200 WITHIN REACH. 3469 02:38:34,200 --> 02:38:36,880 THE TITLE OF HER PRESENTATION IS 3470 02:38:36,880 --> 02:38:39,560 CREATING VALUE THROUGH CLINICAL 3471 02:38:39,560 --> 02:38:39,880 REPURPOSING. 3472 02:38:39,880 --> 02:38:43,960 BAR BRANCH 3473 02:38:43,960 --> 02:38:44,560 >> WONDERFUL. 3474 02:38:44,560 --> 02:38:46,080 THANK YOU. 3475 02:38:46,080 --> 02:38:49,280 EVERYONE SEE ME AND MY SLIDES. 3476 02:38:49,280 --> 02:38:50,880 >> YEP. 3477 02:38:50,880 --> 02:38:51,440 >> WONDERFUL. 3478 02:38:51,440 --> 02:38:52,880 FIRST OF ALL THANK YOU SO MUCH 3479 02:38:52,880 --> 02:38:54,520 FOR HAVING ME. 3480 02:38:54,520 --> 02:38:56,680 IT'S AN HONOR TO SPEAK TO THIS 3481 02:38:56,680 --> 02:38:58,480 GROUP TODAY AND APPRECIATE THE 3482 02:38:58,480 --> 02:38:59,640 INVITATION TO SPEAK TO THE 3483 02:38:59,640 --> 02:39:01,520 MUSCULAR DYSTROPHY COORDINATING 3484 02:39:01,520 --> 02:39:02,240 COMMITTEE. 3485 02:39:02,240 --> 02:39:04,080 MY GOAL IS TO SHARE WITH THIS 3486 02:39:04,080 --> 02:39:05,520 PATIENT GROUP AND THIS COMMUNITY 3487 02:39:05,520 --> 02:39:10,680 A LITTLE BIT ABOUT CURES WITHIN 3488 02:39:10,680 --> 02:39:13,680 REACH AND TO PROVIDE YOU SOME 3489 02:39:13,680 --> 02:39:15,080 EXAMPLES AND SUCCESS STORIES 3490 02:39:15,080 --> 02:39:17,880 THAT HIGHLIGHT BOTH THE 3491 02:39:17,880 --> 02:39:19,280 OPPORTUNITIES THAT REPURPOSING 3492 02:39:19,280 --> 02:39:21,400 CAN BRING TO YOUR DISEASE AS 3493 02:39:21,400 --> 02:39:23,280 WELL AS LESSONS LEARNED FROM 3494 02:39:23,280 --> 02:39:24,680 OTHER AREAS. 3495 02:39:24,680 --> 02:39:29,000 SO FIRST -- A LITTLE BIT ABOUT 3496 02:39:29,000 --> 02:39:30,000 CURES WITHIN REACH. 3497 02:39:30,000 --> 02:39:34,080 WE ARE A CHICAGO BASED NONPROFIT 3498 02:39:34,080 --> 02:39:36,680 AND OUR GOAL IS TO LEVERAGE THE 3499 02:39:36,680 --> 02:39:38,760 SPEED, SAFETY AND COST 3500 02:39:38,760 --> 02:39:40,440 EFFECTIVENESS OF TESTING ALREADY 3501 02:39:40,440 --> 02:39:42,760 APPROVED THERAPIES FOR NEW 3502 02:39:42,760 --> 02:39:43,560 INDICATIONS. 3503 02:39:43,560 --> 02:39:46,440 THE COVID-19 PANDEMIC SHINED A 3504 02:39:46,440 --> 02:39:49,120 SPOTLIGHT ON WHAT WE HAVE KNOWN 3505 02:39:49,120 --> 02:39:51,640 IT AND CERTAINLY THOSE IN THE 3506 02:39:51,640 --> 02:39:53,400 RARE DISEASE COMMUNITY HAVE 3507 02:39:53,400 --> 02:39:54,880 KNOWN SINCE THEIR DIAGNOSIS AND 3508 02:39:54,880 --> 02:39:58,040 THAT IS THE FASTEST WAY TO A 3509 02:39:58,040 --> 02:39:59,840 PATIENT IMPACT IS BY USING 3510 02:39:59,840 --> 02:40:01,800 ALREADY APPROVED THERAPIES 3511 02:40:01,800 --> 02:40:06,280 THROUGH CONTROLLED CLINICAL 3512 02:40:06,280 --> 02:40:11,600 TRIALS. 3513 02:40:11,600 --> 02:40:15,000 AND SINCE WE'VE BEEN FOCUSING ON 3514 02:40:15,000 --> 02:40:16,480 REPURPOSING FOR OVER A DECADE 3515 02:40:16,480 --> 02:40:19,960 AND CLOSER TO 15 YEARS NOW WE'VE 3516 02:40:19,960 --> 02:40:21,880 PROVIDED OVER $7 MILLION IN 3517 02:40:21,880 --> 02:40:24,880 FUNDING OF MOSTLY CLINICAL SOME 3518 02:40:24,880 --> 02:40:28,600 PRECLINICAL TRIALS AND THAT HAS 3519 02:40:28,600 --> 02:40:31,880 LEVERAGED 76 CLOSER TO 3520 02:40:31,880 --> 02:40:34,440 79-$80 MILLION IN FOLLOW ON 3521 02:40:34,440 --> 02:40:36,880 FUNDING FOR THE RESEARCH AND' 3522 02:40:36,880 --> 02:40:38,120 THE RESEARCHERS THAT WE'VE 3523 02:40:38,120 --> 02:40:38,680 FUNDED. 3524 02:40:38,680 --> 02:40:41,560 IN TERMS OF WHEN WE FUND AND OUR 3525 02:40:41,560 --> 02:40:44,240 APPROACH A LOT OF THE SPEAKERS 3526 02:40:44,240 --> 02:40:48,040 TODAY HAVE HAD A SIMILAR LOOKING 3527 02:40:48,040 --> 02:40:50,320 DRUG DEVELOPMENT AND THERAPY 3528 02:40:50,320 --> 02:40:50,880 DEVELOPMENT. 3529 02:40:50,880 --> 02:40:53,800 MOST OF THE FUNDING THAT CURES 3530 02:40:53,800 --> 02:40:55,600 WITHIN REACH PROVIDES ARE SEED 3531 02:40:55,600 --> 02:40:56,080 FUNDS 3532 02:40:56,080 --> 02:40:59,160 AT THE PROOF OF CONCEPT THE 3533 02:40:59,160 --> 02:41:01,640 FIRST AND THAT PIVOTAL STUDY 3534 02:41:01,640 --> 02:41:02,160 STAGE. 3535 02:41:02,160 --> 02:41:05,080 WHAT WE CALL THE COME BACK 3536 02:41:05,080 --> 02:41:06,160 WEDNESDAY STAGE. 3537 02:41:06,160 --> 02:41:09,840 OUR GOAL IS TO DO THE IND 3538 02:41:09,840 --> 02:41:10,920 ENABLING STUDIES. 3539 02:41:10,920 --> 02:41:13,920 THAT FIRST STUDY THAT IF IT IS 3540 02:41:13,920 --> 02:41:16,160 SUCCESSFUL CATALYZES FOLLOW ON 3541 02:41:16,160 --> 02:41:19,280 FUNDING WHETHER FROM THE NIH OR 3542 02:41:19,280 --> 02:41:21,480 INDUSTRY OR OTHER LARGER 3543 02:41:21,480 --> 02:41:23,720 FOUNDATIONS FOR THE LARGER 3544 02:41:23,720 --> 02:41:25,880 CONFORM TORI AND THE LARGER 3545 02:41:25,880 --> 02:41:27,080 FOLLOW ON STUDIES. 3546 02:41:27,080 --> 02:41:29,080 WE FUND CLINICAL TRIALS WHETHER 3547 02:41:29,080 --> 02:41:32,280 OR NOT THEY HAVE COMMERCIAL OR 3548 02:41:32,280 --> 02:41:33,080 PHILANTHROPIC VALUE. 3549 02:41:33,080 --> 02:41:38,040 YOU CAN SEE SOMETIMES OUR 3550 02:41:38,040 --> 02:41:42,520 PROJECTS CAN GET INTO INDUSTRY 3551 02:41:42,520 --> 02:41:44,440 HANDS AND SUBMIT THEM. 3552 02:41:44,440 --> 02:41:47,280 OTHER TIMES IT'S A GENERIC AND 3553 02:41:47,280 --> 02:41:50,600 WHAT WE CALL PHILANTHROPIC 3554 02:41:50,600 --> 02:41:51,200 VALUE. 3555 02:41:51,200 --> 02:41:54,280 IT CERTAINLY HAS PATIENT VALUE 3556 02:41:54,280 --> 02:41:57,160 BUT THERE MAY NOT BE A 3557 02:41:57,160 --> 02:41:58,360 COMMERCIAL BENEFIT. 3558 02:41:58,360 --> 02:41:59,680 BUT WE ARE IN DIFFERENT. 3559 02:41:59,680 --> 02:42:01,480 WE LOOK AT PROJECTS REGARDLESS 3560 02:42:01,480 --> 02:42:03,080 OF WHETHER THEY HAVE FINANCIAL 3561 02:42:03,080 --> 02:42:05,120 VALUE OR COMMERCIAL VALUE WE'RE 3562 02:42:05,120 --> 02:42:07,120 LOOKING AT PATIENTS WITH UNMET 3563 02:42:07,120 --> 02:42:09,640 MEDICAL NEEDS. 3564 02:42:09,640 --> 02:42:12,000 A LOT OF CONVERSATION FROM 3565 02:42:12,000 --> 02:42:14,680 EARLIER SPEAKERS AND ELIZABETH 3566 02:42:14,680 --> 02:42:17,280 MENTIONED THIS. 3567 02:42:17,280 --> 02:42:21,200 CURES WITHIN REACH FOCUSES ON 3568 02:42:21,200 --> 02:42:23,200 REPURPOSING. 3569 02:42:23,200 --> 02:42:25,120 WHILE THERE IS AN OPPORTUNITY IN 3570 02:42:25,120 --> 02:42:27,560 REPOSITIONING THAT ARE ALREADY 3571 02:42:27,560 --> 02:42:29,360 SHELVED OR OTHER TREATMENTS THAT 3572 02:42:29,360 --> 02:42:33,160 HAVE NOT YET GOTTEN A REGULATORY 3573 02:42:33,160 --> 02:42:36,720 APPROVAL CURES WITHIN REACH 3574 02:42:36,720 --> 02:42:38,520 FOCUSES ON THOSE THAT HAVE BEEN 3575 02:42:38,520 --> 02:42:41,840 APPROVED BY EITHER THE FDA OR 3576 02:42:41,840 --> 02:42:43,880 ANOTHER REGULATORY AGENCY AROUND 3577 02:42:43,880 --> 02:42:45,480 THE WORLD. 3578 02:42:45,480 --> 02:42:47,440 WE ALSO AS THE PREVIOUS SLIDE 3579 02:42:47,440 --> 02:42:49,800 SAID WE LOOK AT BOTH COMMERCIAL 3580 02:42:49,800 --> 02:42:51,560 AND PHILANTHROPIC VALUE 3581 02:42:51,560 --> 02:42:52,480 PROJECTS. 3582 02:42:52,480 --> 02:42:54,000 SO, THAT IS JUST SOME DEFINITE 3583 02:42:54,000 --> 02:42:55,400 ANYTHINGS THAT'S I THINK WILL BE 3584 02:42:55,400 --> 02:42:57,000 HELPFUL FOR THE REST OF MY 3585 02:42:57,000 --> 02:43:03,480 CONVERSATION TODAY. 3586 02:43:03,480 --> 02:43:06,720 SO IN TERMS OF CURRENT RESEARCH. 3587 02:43:06,720 --> 02:43:09,480 FUNDING AT 36 DIFFERENT TRIALS 3588 02:43:09,480 --> 02:43:11,840 AT 31 DIFFERENT INSTITUTIONS IN 3589 02:43:11,840 --> 02:43:14,680 29 DISEASES AND 7 COUNTRIES. 3590 02:43:14,680 --> 02:43:16,120 MOSTLY IN THE UNITED STATES BUT 3591 02:43:16,120 --> 02:43:18,040 WE HAVE ONGOING TRIALS RIGHT NOW 3592 02:43:18,040 --> 02:43:21,480 IN GERMANY, INDIA, KEN Y 3593 02:43:21,480 --> 02:43:23,280 NIGERIA, UNITED KINGDOM AND 3594 02:43:23,280 --> 02:43:25,400 VIETNAM WITH ANOTHER COUNTRY OR 3595 02:43:25,400 --> 02:43:27,240 TWO COMING SOON. 3596 02:43:27,240 --> 02:43:29,920 AS THIS SLIDE POINTS OUT THREE 3597 02:43:29,920 --> 02:43:31,880 QUARTERS OF OUR PROJECTS ARE IN 3598 02:43:31,880 --> 02:43:33,680 THE ADULT POPULATION. 3599 02:43:33,680 --> 02:43:35,680 OVER 90% ARE IN THE CLINICAL 3600 02:43:35,680 --> 02:43:36,680 TRIAL STAGE. 3601 02:43:36,680 --> 02:43:39,320 MOSTLY PHASE I. 3602 02:43:39,320 --> 02:43:42,680 SOMETIMES PHASE 2A. 3603 02:43:42,680 --> 02:43:44,200 80% ARE IN THE UNITED STATES AND 3604 02:43:44,200 --> 02:43:49,840 OVER 80% ARE IN DRUGS WITH THE 3605 02:43:49,840 --> 02:43:53,800 REMAINING 20% IN DEVICES, 3606 02:43:53,800 --> 02:43:56,280 DIAGNOSTICS AND NEUTRA SUIT 3607 02:43:56,280 --> 02:43:58,280 CALLS AND WE * COVER A VARIETY 3608 02:43:58,280 --> 02:44:02,360 OF DISEASE AREAS AND 3609 02:44:02,360 --> 02:44:02,920 INDICATIONS. 3610 02:44:02,920 --> 02:44:05,280 THE RARE DISEASE SPACE -- WE'VE 3611 02:44:05,280 --> 02:44:08,600 HAD A LOT OF SPEAKERS TALK ABOUT 3612 02:44:08,600 --> 02:44:13,040 RARE DISEASES AND REPURPOSING. 3613 02:44:13,040 --> 02:44:17,800 IT IS VERY COMMON AND PREVALENT. 3614 02:44:17,800 --> 02:44:27,080 OVER 50% ARE IN OU OURS. 3615 02:44:27,080 --> 02:44:30,480 -- PROJECTS. 3616 02:44:30,480 --> 02:44:33,720 WE HAVE MANY DISEASES AND BLOOD 3617 02:44:33,720 --> 02:44:35,960 CANCERS' NOW FIVE OR SIX 3618 02:44:35,960 --> 02:44:39,000 DIFFERENT TYPES OF BLOOD 3619 02:44:39,000 --> 02:44:39,240 CANCERS. 3620 02:44:39,240 --> 02:44:40,400 OVARIAN CANCER. 3621 02:44:40,400 --> 02:44:42,680 BONE CANCER. 3622 02:44:42,680 --> 02:44:44,360 VASCULAR MALFORMATIONS. 3623 02:44:44,360 --> 02:44:47,640 HEARING LOSSES AND RARE LIVER 3624 02:44:47,640 --> 02:44:49,520 DISEASES AND OTHER IMMUNE 3625 02:44:49,520 --> 02:44:50,080 DISORDERS. 3626 02:44:50,080 --> 02:44:54,280 WE HAVE OVER -- FIVE FUNDING 3627 02:44:54,280 --> 02:44:56,960 OPPORTUNITIES RFPs 3628 02:44:56,960 --> 02:44:58,440 OPPORTUNITIES OPEN RIGHT NOW. 3629 02:44:58,440 --> 02:45:00,360 FOUR OF THE FIVE MUSCULAR 3630 02:45:00,360 --> 02:45:01,360 DYSTROPHY DISEASES WOULD BE 3631 02:45:01,360 --> 02:45:02,080 ELIGIBLE. 3632 02:45:02,080 --> 02:45:04,400 THOSE ARE ALL OPEN NOW WITH 3633 02:45:04,400 --> 02:45:07,120 PROPOSAL DEADLINES DUE AT THE 3634 02:45:07,120 --> 02:45:08,720 END OF -- THROUGHOUT THE MONTH 3635 02:45:08,720 --> 02:45:12,000 OF JUNE SO IF ANY OF THE PATIENT 3636 02:45:12,000 --> 02:45:13,400 GROUPS HAVE RESEARCHERS THAT 3637 02:45:13,400 --> 02:45:15,480 THEY HAVE PROJECTS IN THIS SPACE 3638 02:45:15,480 --> 02:45:17,600 OR ANY RESEARCHERS THAT ARE 3639 02:45:17,600 --> 02:45:18,920 LISTENING THAT ARE INTERESTED IN 3640 02:45:18,920 --> 02:45:22,960 OUR PROPOSALS AND/OR RFPs A 3641 02:45:22,960 --> 02:45:24,720 LOT OF THE INFORMATION IS ON OUR 3642 02:45:24,720 --> 02:45:25,760 WEBSITE. 3643 02:45:25,760 --> 02:45:28,080 THE REST OF MY TIME IS GOING TO 3644 02:45:28,080 --> 02:45:29,800 BE TALKING ABOUT THE DIFFERENT 3645 02:45:29,800 --> 02:45:32,680 WAYS TO CREATE VALUE FROM 3646 02:45:32,680 --> 02:45:33,480 REPURPOSING. 3647 02:45:33,480 --> 02:45:35,840 AS I SAID WE LOOK AT BOTH 3648 02:45:35,840 --> 02:45:38,280 COMMERCIAL VALUE AND 3649 02:45:38,280 --> 02:45:39,800 PHILANTHROPIC VALUE. 3650 02:45:39,800 --> 02:45:43,240 ON THE COMMERCIAL SIDE THIS 3651 02:45:43,240 --> 02:45:44,680 INCLUDES NEW INTELLECTUAL 3652 02:45:44,680 --> 02:45:45,320 PROPERTY. 3653 02:45:45,320 --> 02:45:47,400 IT COULD BE DOSING CHANGES, 3654 02:45:47,400 --> 02:45:49,200 FORMULATION CHANGES OR CHANGE OF 3655 02:45:49,200 --> 02:45:52,480 A NEW DELIVERY METHOD OR 3656 02:45:52,480 --> 02:45:53,080 MECHANISM. 3657 02:45:53,080 --> 02:45:54,760 WE HAVE DRUG COMBINATIONS. 3658 02:45:54,760 --> 02:45:56,320 WE HAVE OTHER PROJECT EXAMPLES 3659 02:45:56,320 --> 02:45:59,200 THAT DON'T HAVE ANY FORMULATION 3660 02:45:59,200 --> 02:46:01,880 CHANGES OR DOSING CHANGES. 3661 02:46:01,880 --> 02:46:04,680 IT'S THE SAME DRUG AND SAME 3662 02:46:04,680 --> 02:46:05,200 MOLECULE. 3663 02:46:05,200 --> 02:46:09,800 WE HAVE PROJECTS GOING AFTER 3664 02:46:09,800 --> 02:46:15,880 MARKET EXCLUSIVITY. 3665 02:46:15,880 --> 02:46:19,680 SIMILAR WITH PHILANTHROPIC 3666 02:46:19,680 --> 02:46:20,120 VALUE. 3667 02:46:20,120 --> 02:46:24,480 OFF LABEL USE. 3668 02:46:24,480 --> 02:46:27,280 AS WELL AS OTHER OPPORTUNITIES 3669 02:46:27,280 --> 02:46:29,480 IN THIS SPACE. 3670 02:46:29,480 --> 02:46:31,800 WHERE DO WE GET OUR IDEAS? 3671 02:46:31,800 --> 02:46:34,680 IT'S ONE OF THE QUESTIONS THAT I 3672 02:46:34,680 --> 02:46:36,080 WE GET ASKED ALL THE TIME. 3673 02:46:36,080 --> 02:46:38,520 VAST MAJORITY OF WHAT OUR WORK 3674 02:46:38,520 --> 02:46:42,480 ENTAILS IS INVESTIGATOR 3675 02:46:42,480 --> 02:46:44,160 INITIALED IDEAS. 3676 02:46:44,160 --> 02:46:45,280 THEY COME FROM RESEARCH 3677 02:46:45,280 --> 02:46:46,560 INSTITUTIONS AROUND THE WORLD. 3678 02:46:46,560 --> 02:46:48,920 THEY ARE COMING FROM CLINICIANS 3679 02:46:48,920 --> 02:46:52,040 WHO ARE SEEN REALLY INTERESTING 3680 02:46:52,040 --> 02:46:53,720 THINGS IN THEIR PATIENTS AND 3681 02:46:53,720 --> 02:46:56,440 WANT TO DO A CLINICAL STUDY. 3682 02:46:56,440 --> 02:46:58,760 FROM INDUSTRY AS WELL AS FROM 3683 02:46:58,760 --> 02:46:59,440 PATIENTS. 3684 02:46:59,440 --> 02:47:01,200 PATIENT GROUPS, DISEASE GROUPS 3685 02:47:01,200 --> 02:47:04,520 AND CAREGIVERS OF PATIENTS IN 3686 02:47:04,520 --> 02:47:05,520 PEDIATRIC SPACE. 3687 02:47:05,520 --> 02:47:08,560 AND AGAIN HOW DOES THIS LOOK FOR 3688 02:47:08,560 --> 02:47:10,120 THE PATIENTS THAT ARE RECEIVING 3689 02:47:10,120 --> 02:47:11,680 THESE TREATMENTS? 3690 02:47:11,680 --> 02:47:15,840 SOMETIMES THE END GAME IS AN OFF 3691 02:47:15,840 --> 02:47:17,920 LABEL USE WITHOUT REIMBURSEMENT. 3692 02:47:17,920 --> 02:47:21,800 SOMETIMES IT'S OFF LABEL WITH 3693 02:47:21,800 --> 02:47:22,720 REIMBURSEMENT AND SOMETIMES THE 3694 02:47:22,720 --> 02:47:24,440 DRUG REPURPOSING OR OTHER 3695 02:47:24,440 --> 02:47:26,520 INDICATIONS AND THERAPIES WILL 3696 02:47:26,520 --> 02:47:28,280 GET ON LABEL AND RECEIVE NOT 3697 02:47:28,280 --> 02:47:31,360 ONLY REGULATORY APPROVAL BUT 3698 02:47:31,360 --> 02:47:32,840 ALSO REIMBURSEMENT. 3699 02:47:32,840 --> 02:47:35,080 THE EXAMPLES HIGHLIGHT THE VAST 3700 02:47:35,080 --> 02:47:37,520 NUMBER OF STAKEHOLDERS THAT ARE 3701 02:47:37,520 --> 02:47:38,680 INVOLVED IN REPURPOSING TODAY. 3702 02:47:38,680 --> 02:47:41,320 IT INCLUDES INDUSTRY, RESEARCH, 3703 02:47:41,320 --> 02:47:45,520 PATIENT DISEASE GROUPS, 3704 02:47:45,520 --> 02:47:46,880 NONPROFITS, GOVERNMENT AGENCIES 3705 02:47:46,880 --> 02:47:48,520 LIKE THE NIH AND THE DEPARTMENT 3706 02:47:48,520 --> 02:47:50,360 OF DEFENSE. 3707 02:47:50,360 --> 02:47:52,720 WHETHER THESE STAKEHOLDERS ARE 3708 02:47:52,720 --> 02:47:55,120 FORMAL OR INFORMAL PARTNERSHIPS 3709 02:47:55,120 --> 02:47:56,280 THE REALLY IMPORTANT STEP IS 3710 02:47:56,280 --> 02:47:58,680 BRINGING ALL OF THE STAKEHOLDERS 3711 02:47:58,680 --> 02:48:00,120 TO THE TABLE. 3712 02:48:00,120 --> 02:48:02,240 WHETHER THEY ARE PRIVATE OR 3713 02:48:02,240 --> 02:48:02,880 PARTNERSHIP. 3714 02:48:02,880 --> 02:48:05,720 7 PRIVATE OR PUBLIC 3715 02:48:05,720 --> 02:48:06,440 PARTNERSHIPS. 3716 02:48:06,440 --> 02:48:08,280 THE FIRSTAR STAR EXAMPLE IS ONE 3717 02:48:08,280 --> 02:48:10,960 OF OUR EARLIEST EXAMPLES FROM 3718 02:48:10,960 --> 02:48:11,840 CURES WITHIN REACH. 3719 02:48:11,840 --> 02:48:15,040 THIS REALLY SHOWS THE POWER OF 3720 02:48:15,040 --> 02:48:16,720 REPURPOSE A IT HELPED US TO 3721 02:48:16,720 --> 02:48:19,000 SHIFT OUR MISSION TO SOLELY 3722 02:48:19,000 --> 02:48:21,720 FOCUS ON REPURPOSING BACK IN 3723 02:48:21,720 --> 02:48:21,920 2008. 3724 02:48:21,920 --> 02:48:25,280 IT WAS THE SUCCESS OF THIS 3725 02:48:25,280 --> 02:48:25,800 PROJECT. 3726 02:48:25,800 --> 02:48:28,360 ONE OF THE FIRST EXAMPLES IN A 3727 02:48:28,360 --> 02:48:31,480 RARE PEDIATRIC DISEASE CALLS 3728 02:48:31,480 --> 02:48:32,320 ALPS. 3729 02:48:32,320 --> 02:48:35,520 RESEARCH STARTED IN A MOUSE 3730 02:48:35,520 --> 02:48:37,360 MODEL. 3731 02:48:37,360 --> 02:48:39,040 THAT SUCCESSFUL MOUSE MODEL 3732 02:48:39,040 --> 02:48:41,040 ALLOWED US TO FUND A SMALL 3733 02:48:41,040 --> 02:48:42,280 CLINICAL TRIAL. 3734 02:48:42,280 --> 02:48:43,920 MOST OF THE KIDS IN THE TRIAL 3735 02:48:43,920 --> 02:48:48,080 IMPROVED USING THIS GENERIC DRUG 3736 02:48:48,080 --> 02:48:51,200 USED FOR TRANSPLANTS. 3737 02:48:51,200 --> 02:48:54,280 WE OFTEN JOKE THIS IS ONE OF THE 3738 02:48:54,280 --> 02:48:56,720 HANDFUL OF WONDERFUL REPURPOSING 3739 02:48:56,720 --> 02:48:57,200 DRUGS. 3740 02:48:57,200 --> 02:48:59,040 IT CHANGED THE QUALITY OF LIFE 3741 02:48:59,040 --> 02:49:01,960 FOR THESE PATIENTS AND THEIR 3742 02:49:01,960 --> 02:49:04,320 PARENTS AND CAREGIVERS QUALITY 3743 02:49:04,320 --> 02:49:05,880 OF LIFE. 3744 02:49:05,880 --> 02:49:08,160 NOT ONLY WERE THE RESULTS 3745 02:49:08,160 --> 02:49:10,680 PUBLISHED IN MANY PUBLICATIONS 3746 02:49:10,680 --> 02:49:12,560 OTHER DOCTORS BECAME AWARE OF 3747 02:49:12,560 --> 02:49:14,160 THIS TREATMENT AND IT BECAME THE 3748 02:49:14,160 --> 02:49:16,040 STANDARD OF CARE. 3749 02:49:16,040 --> 02:49:18,960 THEN DR. -- WITH SOME ADDITIONAL 3750 02:49:18,960 --> 02:49:21,440 FUNDS TESTED THIS IN OTHER 3751 02:49:21,440 --> 02:49:23,280 PEDIATRIC RARE DISEASES. 3752 02:49:23,280 --> 02:49:24,960 HE RECEIVED OVER A MILLION 3753 02:49:24,960 --> 02:49:26,640 DOLLARS FROM THE NIH FOR FOLLOW 3754 02:49:26,640 --> 02:49:27,360 ON TRIALS. 3755 02:49:27,360 --> 02:49:30,720 AND IT'S A GREAT EX OF SUCCESS. 3756 02:49:30,720 --> 02:49:33,680 ON THE NIH'S OWN WEBSITE YOU CAN 3757 02:49:33,680 --> 02:49:36,720 SEE -- IT IS CONSIDERED STANDARD 3758 02:49:36,720 --> 02:49:39,480 OF CARE AND IT'S AN EXAMPLE HAVE 3759 02:49:39,480 --> 02:49:41,680 THE PHILANTHROPIC VALUE. 3760 02:49:41,680 --> 02:49:44,000 THIS IS A GENERIC THAT IS 3761 02:49:44,000 --> 02:49:45,880 GETTING REIMBURSEMENT TODAY 3762 02:49:45,880 --> 02:49:47,360 USING OFF LABEL AS THE STANDARD 3763 02:49:47,360 --> 02:49:48,720 OF CARE. 3764 02:49:48,720 --> 02:49:50,880 ANOTHER EXAMPLE TO SHARE WITH 3765 02:49:50,880 --> 02:49:52,760 YOU TODAY IS ON THE MEDICAL 3766 02:49:52,760 --> 02:49:53,640 DEVICE SIDE. 3767 02:49:53,640 --> 02:49:58,520 IT'S ON MARKET TODAY IN THE 3768 02:49:58,520 --> 02:50:05,680 MULTIPLMULTIPLE -- MULTIPLE SCLEROSIS 3769 02:50:05,680 --> 02:50:07,320 CASE. 3770 02:50:07,320 --> 02:50:12,320 HERE IS AN EXAMPLE WHERE THE 3771 02:50:12,320 --> 02:50:14,480 UNIVERSITY OF WISCONSIN -- 3772 02:50:14,480 --> 02:50:16,400 SEVERAL YEARS GO BY AND FOLLOW 3773 02:50:16,400 --> 02:50:18,400 ON CLINICAL TRIALS AND 3774 02:50:18,400 --> 02:50:20,600 DEPARTMENT OF DEFENSE FUNDING 3775 02:50:20,600 --> 02:50:22,040 OVER $4 MILLION FOR FOLLOW ON 3776 02:50:22,040 --> 02:50:22,680 FUNDS. 3777 02:50:22,680 --> 02:50:25,880 THE DEVICE WAS APPROVED IN 3778 02:50:25,880 --> 02:50:27,840 CANADA FOR TRAUMATIC BRAIN 3779 02:50:27,840 --> 02:50:30,200 INJURY AND IT GOT APPROVED FOR 3780 02:50:30,200 --> 02:50:31,520 MULTIPLE SCLEROSIS HERE IN THE 3781 02:50:31,520 --> 02:50:33,240 UNITED STATES A COUPLE OF YEARS 3782 02:50:33,240 --> 02:50:34,000 AGO. 3783 02:50:34,000 --> 02:50:35,760 NEXT EXAMPLE IS THAT PERSON 3784 02:50:35,760 --> 02:50:39,280 EXAMPLE OF WHAT WE CALL THE COME 3785 02:50:39,280 --> 02:50:41,480 BACK WHEN FUNDING. 3786 02:50:41,480 --> 02:50:44,480 DR. -- AT PENN STATE HAD A 3787 02:50:44,480 --> 02:50:45,960 CONVERSATION WITH THE NATIONAL 3788 02:50:45,960 --> 02:50:47,880 INSTITUTE OF HEALTH ABOUT HIS 3789 02:50:47,880 --> 02:50:50,440 IDEA TO USE A GENERIC MULTIPLE 3790 02:50:50,440 --> 02:50:52,920 SCLEROSIS DRUG TO LOOK AT NERVE 3791 02:50:52,920 --> 02:50:57,000 DAMAGE IN SEVERE LIMB TRAUMA 3792 02:50:57,000 --> 02:50:59,560 PATIENTS WHETHER OR NOT NERVES 3793 02:50:59,560 --> 02:51:00,880 WERE CRUSHED OR SEVERED IN A 3794 02:51:00,880 --> 02:51:02,360 SEVERE ACCIDENT. 3795 02:51:02,360 --> 02:51:04,880 HE TOLD THE NIH ABOUT HIS IDEA. 3796 02:51:04,880 --> 02:51:08,400 THE NIH SAID THAT IS FANTASTIC. 3797 02:51:08,400 --> 02:51:10,400 COME BACK WHEN YOU HAVE 5-10 3798 02:51:10,400 --> 02:51:11,960 PATIENTS AND YOU KNOW IT WORKS 3799 02:51:11,960 --> 02:51:14,360 IN A SMALL NUMBER OF PATIENTS' 3800 02:51:14,360 --> 02:51:18,760 HE APPLIED TO CURES WITHIN REACH 3801 02:51:18,760 --> 02:51:21,000 AND HE WON AN EVENT AND IF I 3802 02:51:21,000 --> 02:51:22,440 RECALL HEATHER STONE THE NEXT 3803 02:51:22,440 --> 02:51:27,400 SPEAKER WAS AN EXPERT PANELISTS 3804 02:51:27,400 --> 02:51:28,520 AT THAT EVENT. 3805 02:51:28,520 --> 02:51:31,200 WE STARTED FUNDING DR. -- IN 3806 02:51:31,200 --> 02:51:31,680 2019. 3807 02:51:31,680 --> 02:51:35,680 THE DoD AND WALTER REED 3808 02:51:35,680 --> 02:51:39,640 STARTED USING THIS IN OFF LABEL 3809 02:51:39,640 --> 02:51:40,200 USE. 3810 02:51:40,200 --> 02:51:42,440 THE SIMILAR PROTOCOL BUT OFF 3811 02:51:42,440 --> 02:51:44,000 LABEL USE WITH SOME EARLY 3812 02:51:44,000 --> 02:51:44,560 SUCCESS. 3813 02:51:44,560 --> 02:51:46,840 THERE IS A SPINOUT CONNECTED TO 3814 02:51:46,840 --> 02:51:49,480 PENN STATE SO THERE IS POSSIBLE 3815 02:51:49,480 --> 02:51:50,600 COMMERCIAL OPPORTUNITY. 3816 02:51:50,600 --> 02:51:53,200 HE WON THE COMPETITION BACK IN 3817 02:51:53,200 --> 02:51:53,760 2018. 3818 02:51:53,760 --> 02:51:56,480 WE STARTED FUNDING IN 2019 AND 3819 02:51:56,480 --> 02:51:58,680 IN JANUARY OF THIS YEAR THE NIH 3820 02:51:58,680 --> 02:52:00,280 GAVE HIM A $2 MILLION GRANT FOR 3821 02:52:00,280 --> 02:52:02,880 THE LARGER FOLLOW-UP STUDY. 3822 02:52:02,880 --> 02:52:06,000 SO A GREAT EXAMPLE OF A LOT OF 3823 02:52:06,000 --> 02:52:10,080 PARTNERS INVOLVED -- THERE. 3824 02:52:10,080 --> 02:52:12,520 NEXT EXAMPLE IS ALSO A WINNER OF 3825 02:52:12,520 --> 02:52:15,080 ONE OF OUR COMPETITION. 3826 02:52:15,080 --> 02:52:17,960 THIS IS FOR AN IMMUNE DISORDER 3827 02:52:17,960 --> 02:52:20,480 IN CHILDREN CALLED HLH. 3828 02:52:20,480 --> 02:52:24,360 USING -- WHICH IS AN ON PATENT 3829 02:52:24,360 --> 02:52:24,880 DRUG TODAY. 3830 02:52:24,880 --> 02:52:29,040 AFTER THIS RESEARCHER 3831 02:52:29,040 --> 02:52:30,520 DR. NICHOLS WON THE COMPETITION. 3832 02:52:30,520 --> 02:52:32,680 SHE WAS SPEAKING TO THE 3833 02:52:32,680 --> 02:52:34,840 PHARMACEUTICAL PARTNER WHO 3834 02:52:34,840 --> 02:52:37,360 AGREED TO PROVIDE DRUG AND 3835 02:52:37,360 --> 02:52:38,280 FUNDING FOR THIS TRIAL. 3836 02:52:38,280 --> 02:52:41,840 THE TRIAL IS STILL ONGOING SO 3837 02:52:41,840 --> 02:52:43,400 TBD TO DETERMINE THE OPPORTUNITY 3838 02:52:43,400 --> 02:52:47,120 BUT IT'S A GREAT EXAMPLE OF HOW 3839 02:52:47,120 --> 02:52:48,680 INDUSTRY CAN BE ENGAGED THIS. 3840 02:52:48,680 --> 02:52:51,560 IS AN INVESTIGATOR INITIATED 3841 02:52:51,560 --> 02:52:54,320 TRIAL COMING FROM KIM NICHOLS. 3842 02:52:54,320 --> 02:52:55,480 A GREAT EXAMPLE AND WE CANNOT 3843 02:52:55,480 --> 02:52:57,080 WAIT FOR THE RESULTS TO COME 3844 02:52:57,080 --> 02:52:58,080 FROM THAT. 3845 02:52:58,080 --> 02:53:00,760 ANOTHER EXAMPLE -- WITH INDUSTRY 3846 02:53:00,760 --> 02:53:04,640 ENGAGEMENT AND PARTNERSHIPS IN 3847 02:53:04,640 --> 02:53:06,480 NONSMALL CELL LUNG CANCER. 3848 02:53:06,480 --> 02:53:07,960 THAT WAS COMPLETED AT THE 3849 02:53:07,960 --> 02:53:09,880 UNIVERSITY OF CHICAGO. 3850 02:53:09,880 --> 02:53:12,040 THIS IS REPURPOSING A COMBINES 3851 02:53:12,040 --> 02:53:14,480 OF TREATMENTS ALONG WITH 3852 02:53:14,480 --> 02:53:17,000 RADIATION FOR STAGE 4 NONSMALL 3853 02:53:17,000 --> 02:53:21,600 CELL LUNG CANCER PATIENTS I. IT 3854 02:53:21,600 --> 02:53:25,440 HAD POSITIVE OUTCOMES. 3855 02:53:25,440 --> 02:53:28,880 * OTHER PUBLICATIONS OVER THE 3856 02:53:28,880 --> 02:53:31,120 LAST SEVERAL YEARS THE MOST 3857 02:53:31,120 --> 02:53:32,880 RECENT WAS JUST SEVERAL MONTHS 3858 02:53:32,880 --> 02:53:33,360 AGO. 3859 02:53:33,360 --> 02:53:35,360 A MAJOR PHARMACEUTICAL COMPANY 3860 02:53:35,360 --> 02:53:37,800 HAS AGREED TO FUND SOME FOLLOW 3861 02:53:37,800 --> 02:53:39,680 ON STUDY THERE AS WELL. 3862 02:53:39,680 --> 02:53:42,280 ANOTHER EXAMPLE OF INDUSTRY 3863 02:53:42,280 --> 02:53:44,920 ENGAGEMENT PARTNERING WITH BOTH 3864 02:53:44,920 --> 02:53:48,040 INDUSTRY AS WELL AS RESEARCH 3865 02:53:48,040 --> 02:53:49,800 INSTITUTIONS AND PATIENTS IN 3866 02:53:49,800 --> 02:53:52,000 COMBINES OF DRUGS AND DEVICES. 3867 02:53:52,000 --> 02:53:56,160 AND THE FINAL EXAMPLE IS IN 3868 02:53:56,160 --> 02:53:57,720 HUNTINGTON'S DISEASE. 3869 02:53:57,720 --> 02:54:00,160 IT WAS -- THE ORIGINAL STUDY WAS 3870 02:54:00,160 --> 02:54:03,080 IN PARKINSON'S DISEASES AND THEY 3871 02:54:03,080 --> 02:54:07,960 WERE REPURPOSES A CANCER * DRUG 3872 02:54:07,960 --> 02:54:14,200 -- THAT WAS OWNED -- UNDER A 3873 02:54:14,200 --> 02:54:17,800 PATENTOR PROTECTION AT THE TIME. 3874 02:54:17,800 --> 02:54:20,440 PARKINSONS A TRIAL WAS GOING ON. 3875 02:54:20,440 --> 02:54:22,440 THE VERY FIRST PHASE OF THE 3876 02:54:22,440 --> 02:54:24,080 PARKINSON'S STUDY WAS 3877 02:54:24,080 --> 02:54:25,440 SUCCESSFUL. 3878 02:54:25,440 --> 02:54:27,440 SO GEORGETOWN APPLIED TO OUR 3879 02:54:27,440 --> 02:54:30,520 FUNDING TO SEE IF THEY COULD 3880 02:54:30,520 --> 02:54:37,880 TEST THAT SAME -- SIMILAR DRUG. 3881 02:54:37,880 --> 02:54:39,440 THIS TRIAL IS JUST COMPLETING 3882 02:54:39,440 --> 02:54:39,960 NOW. 3883 02:54:39,960 --> 02:54:42,000 IT'S TOO EARLY TO SHARE RESULTS 3884 02:54:42,000 --> 02:54:44,480 BUT WE'RE EXCITED TO SEE WHAT 3885 02:54:44,480 --> 02:54:46,360 THE RESULTS ARE GOING TO BE FROM 3886 02:54:46,360 --> 02:54:47,600 ANDERSON AND ANOTHER GREAT 3887 02:54:47,600 --> 02:54:49,800 EXAMPLE OF HOW INDUSTRY CAN 3888 02:54:49,800 --> 02:54:51,600 INVOLVE ITSELF IN THIS SPACE. 3889 02:54:51,600 --> 02:54:53,520 WITH THAT I'M GOING TO TAKE YOUR 3890 02:54:53,520 --> 02:54:56,320 QUESTIONS AND I LOOK FORWARD TO 3891 02:54:56,320 --> 02:54:57,640 CONVERSATIONS WITH THE OTHER 3892 02:54:57,640 --> 02:54:59,120 SPEAKERS AS WELL. 3893 02:54:59,120 --> 02:55:02,040 I THANK YOU AGAIN FOR HAVING 3894 02:55:02,040 --> 02:55:03,840 CURES WITHIN REACH AT THIS 3895 02:55:03,840 --> 02:55:04,040 EVENT. 3896 02:55:04,040 --> 02:55:04,280 7 3897 02:55:04,280 --> 02:55:06,640 >> THANK YOU SO MUCH, BARBARA. 3898 02:55:06,640 --> 02:55:08,240 THAT WAS VERY INSPIRATIONAL. 3899 02:55:08,240 --> 02:55:19,120 AND WE'RE LOOKING FOR QUESTIONS. 3900 02:55:19,120 --> 02:55:20,040 >> GREAT TALK. 3901 02:55:20,040 --> 02:55:20,600 BARBARA. 3902 02:55:20,600 --> 02:55:23,040 I'M CURIOUS OF THE TRIALS THAT 3903 02:55:23,040 --> 02:55:25,120 YOUR ORGANIZATION HAS SUPPORTED 3904 02:55:25,120 --> 02:55:27,360 HAVE YOU FOUND THAT THERE IS A 3905 02:55:27,360 --> 02:55:29,840 DIFFICULT IN ENROLLING? 3906 02:55:29,840 --> 02:55:31,840 IF THE PATIENTS COULD GET ACCESS 3907 02:55:31,840 --> 02:55:33,880 TO THE DRUGS OFF LABEL WITHOUT 3908 02:55:33,880 --> 02:55:35,680 HAVING TO BE IN THE TRIAL. 3909 02:55:35,680 --> 02:55:38,520 IF IT'S DESIGNED WITH A PLACEBO 3910 02:55:38,520 --> 02:55:39,160 CONTROL? 3911 02:55:39,160 --> 02:55:42,400 >> SO THE PANDEMIC CERTAINLY 3912 02:55:42,400 --> 02:55:44,480 IMPACTED ENROLLMENT AND CLINICAL 3913 02:55:44,480 --> 02:55:48,000 TRIALS CERTAINLY. 3914 02:55:48,000 --> 02:55:52,840 WE -- SEE DIFFERENT INSTITUTIONS 3915 02:55:52,840 --> 02:55:54,400 ENGAGED WITH PATIENT GROUPS IN 3916 02:55:54,400 --> 02:55:56,720 DIFFERENT WAYS AND WHEN YOU'RE 3917 02:55:56,720 --> 02:55:58,080 ENGAGE WE HAD A PATIENT 3918 02:55:58,080 --> 02:55:59,720 COMMUNITY AND THE DISEASE 3919 02:55:59,720 --> 02:56:02,840 COMMUNITY WHEN YOU'RE IN CERTAIN 3920 02:56:02,840 --> 02:56:03,840 INSTITUTIONS THAT ARE WELL 3921 02:56:03,840 --> 02:56:07,040 RESPECTED WITH CLINICAL TRIALS 3922 02:56:07,040 --> 02:56:08,640 IT'S OFTEN IMPACTS ENROLLMENT. 3923 02:56:08,640 --> 02:56:10,840 WE'VE SEEN HAVING BEEN DOING 3924 02:56:10,840 --> 02:56:12,440 THIS FOR A LONG TIME WE'VE SEEN 3925 02:56:12,440 --> 02:56:14,080 EXAMPLES ACROSS THE BOARD IN 3926 02:56:14,080 --> 02:56:17,280 EVERY SPACE. 3927 02:56:17,280 --> 02:56:22,400 YOU CAN OFTEN GET PHYSICIAN TO 3928 02:56:22,400 --> 02:56:23,960 PRESCRIBE SOMETHING OFF LABEL 3929 02:56:23,960 --> 02:56:24,560 BUT AT THE SAME TIME IF YOU 3930 02:56:24,560 --> 02:56:28,200 DON'T HAVE A CLINICAL TRIAL WITH 3931 02:56:28,200 --> 02:56:30,920 A REPUTABLE DATA THAT IS 3932 02:56:30,920 --> 02:56:32,840 REPRODUCIBLE AND PUBLISHED IT'S 3933 02:56:32,840 --> 02:56:34,840 REALLY YOU'RE MISSING THE 3934 02:56:34,840 --> 02:56:37,280 OPPORTUNITY TO MAKE AN IMPACT ON 3935 02:56:37,280 --> 02:56:38,120 PATIENTS BROADLY. 3936 02:56:38,120 --> 02:56:41,400 WE'VE SEEN EXAMPLES OF THAT IN 3937 02:56:41,400 --> 02:56:43,040 COVID-19 WITH OFF LABEL USE THAT 3938 02:56:43,040 --> 02:56:46,000 WHEN YOU DIDN'T HAVE A ROBUST 3939 02:56:46,000 --> 02:56:48,000 DATASET FROM A CLINICAL TRIAL 3940 02:56:48,000 --> 02:56:50,200 THINGS CHANGE AND I WOULD SAY 3941 02:56:50,200 --> 02:56:51,880 USING THAT AS A GREAT EXAMPLE OF 3942 02:56:51,880 --> 02:56:54,760 WHY YOU DO WANT TO HAVE NOT 3943 02:56:54,760 --> 02:56:57,000 NECESSARILY ON LABEL BUT 3944 02:56:57,000 --> 02:56:58,160 PARTICIPATING IN A CLINICAL 3945 02:56:58,160 --> 02:57:00,120 TRIAL SO YOUR DATA CAN BE 3946 02:57:00,120 --> 02:57:01,520 PUBLISHED AND YOU'RE ON A PATH 3947 02:57:01,520 --> 02:57:06,240 TO REIMBURSEMENT AND BROAD USE 3948 02:57:06,240 --> 02:57:09,080 IN PATIENTS WHICH FOR US AS A 3949 02:57:09,080 --> 02:57:13,680 NONPROFIT IS OUR GOAL CERTAINLY. 3950 02:57:13,680 --> 02:57:23,280 >> I HAVE A QUESTION. 3951 02:57:23,280 --> 02:57:28,160 HAVE YOU THOUGHT ABOUT -- GENE 3952 02:57:28,160 --> 02:57:44,960 THERAPIES -- -- AND THE SECOND 3953 02:57:44,960 --> 02:57:50,680 IS TO THE COMMITTEE -- TO THINK 3954 02:57:50,680 --> 02:57:51,680 ABOUT INVESTMENT IN MUSCULAR 3955 02:57:51,680 --> 02:57:55,360 DYSTROPHY -- DO I WANT TO INVEST 3956 02:57:55,360 --> 02:57:58,040 MONEY IN RESOURCES IN DRUGS OR 3957 02:57:58,040 --> 02:58:02,880 INVEST MONEY IN MECHANISTIC -- 3958 02:58:02,880 --> 02:58:05,480 OR OTHER ASPECTS OF RESEARCH AND 3959 02:58:05,480 --> 02:58:10,080 HOW DO WE PRIORITIZE THAT? 3960 02:58:10,080 --> 02:58:11,880 THOSE ARE THE TWO OF QUESTIONS. 3961 02:58:11,880 --> 02:58:15,360 >> SO MY ONE THOUGHT ON THE 3962 02:58:15,360 --> 02:58:17,520 SECOND PART OF YOUR QUESTION -- 3963 02:58:17,520 --> 02:58:20,160 WE FEEL THAT REPURPOSING SHOULD 3964 02:58:20,160 --> 02:58:23,720 BE ONE OF THE RESEARCH LEGS OF 3965 02:58:23,720 --> 02:58:24,400 THE STOOL. 3966 02:58:24,400 --> 02:58:28,480 CERTAINLY PROMOTING DEKNOW VOTE 3967 02:58:28,480 --> 02:58:32,280 RESEARCH WE'RE NOT ANTIDENOVO 3968 02:58:32,280 --> 02:58:32,560 RESEARCH. 3969 02:58:32,560 --> 02:58:34,280 WE THINK IT SHOULD BE RUN ASPECT 3970 02:58:34,280 --> 02:58:35,880 OF THE RESEARCH PORTFOLIO. 3971 02:58:35,880 --> 02:58:39,000 BUT IN TERMS OF GENE THERAPY I 3972 02:58:39,000 --> 02:58:40,880 BELIEVE WE HAVE ONE TODAY THAT 3973 02:58:40,880 --> 02:58:44,080 IS USING GENE THERAPY IT'S MODEL 3974 02:58:44,080 --> 02:58:45,680 IF I RECALL. 3975 02:58:45,680 --> 02:58:47,000 PERHAPS TWO OR MAYBE WE HAVE 3976 02:58:47,000 --> 02:58:49,280 ANOTHER ONE COMING SOON BECAUSE 3977 02:58:49,280 --> 02:58:50,960 WE HAVE 8 TRIALS COMING SOON. 3978 02:58:50,960 --> 02:58:53,080 WITH YOU WE HAVE ONE ONGOING NOW 3979 02:58:53,080 --> 02:58:55,680 USING A GENE THERAPY IF I'M 3980 02:58:55,680 --> 02:58:56,800 REMEMBERING THE SPECK ONE 3981 02:58:56,800 --> 02:58:59,320 CORRECTLY. 3982 02:58:59,320 --> 02:59:02,240 -- SPECIFICALLY ONE CORRECTLY. 3983 02:59:02,240 --> 02:59:02,760 >> WALTER, DO YOU HAVE A 3984 02:59:02,760 --> 02:59:03,160 QUESTION. 3985 02:59:03,160 --> 02:59:31,280 7 3986 02:59:31,280 --> 02:59:33,280 >> THANK YOU AND WE HAVE FOUR 3987 02:59:33,280 --> 02:59:33,960 OPEN CALLS. 3988 02:59:33,960 --> 02:59:36,000 MUSCULAR DYSTROPHY AS A DISEASE 3989 02:59:36,000 --> 02:59:39,040 IS I BELIEVE ELIGIBLE FOR FOUR 3990 02:59:39,040 --> 02:59:39,880 OF THE FIVE. 3991 02:59:39,880 --> 02:59:42,000 YOU'VE GOT TO FIND THE RIGHT 3992 02:59:42,000 --> 02:59:44,200 INVESTIGATOR AND PI OR CLINICIAN 3993 02:59:44,200 --> 02:59:46,120 TO MAKE AN APPLICATION BETWEEN 3994 02:59:46,120 --> 02:59:47,880 NOW AND MID- TO LATE JUNE AND IF 3995 02:59:47,880 --> 02:59:50,120 YOU HAVE ANY QUESTIONS PLEASE 3996 02:59:50,120 --> 02:59:53,360 FEEL FREE TO REACH OUT TO CURES 3997 02:59:53,360 --> 02:59:54,080 WITHIN REACH. 3998 02:59:54,080 --> 02:59:56,000 THANK YOU FOR HAVING ME. 3999 02:59:56,000 --> 02:59:56,760 >> THANK YOU. 4000 02:59:56,760 --> 02:59:58,680 WE'VE HEARD TWO OUTSTANDING 4001 02:59:58,680 --> 03:00:00,760 TALKS ON POTENTIAL RESOURCES 4002 03:00:00,760 --> 03:00:02,880 AVAILABLE TO RESEARCHERS LOOKING 4003 03:00:02,880 --> 03:00:04,480 FOR NEW THERAPIES FOR MUSCULAR 4004 03:00:04,480 --> 03:00:06,160 DYSTROPHY. 4005 03:00:06,160 --> 03:00:08,400 THE FINAL PRESENTATION FOR THIS 4006 03:00:08,400 --> 03:00:10,600 SESSION IS BY HEATHER STONE 4007 03:00:10,600 --> 03:00:14,600 WHO'S FROM THE OFFICE OF MEDICAL 4008 03:00:14,600 --> 03:00:19,280 POLICY AND. 4009 03:00:19,280 --> 03:00:21,680 TITLE IS REPURPOSING IN MUSCULAR 4010 03:00:21,680 --> 03:00:22,880 DYSTROPHY. 4011 03:00:22,880 --> 03:00:26,000 LESSONS FROM THE FDA AND NCATS 4012 03:00:26,000 --> 03:00:32,400 CURE ID AND THE CURE DRUG 4013 03:00:32,400 --> 03:00:34,280 REPURPOSING LABORATORY. 4014 03:00:34,280 --> 03:00:35,120 HEATHER. 4015 03:00:35,120 --> 03:00:36,200 I SAW YOU. 4016 03:00:36,200 --> 03:00:40,600 >> SHORT TITLES ARE NOT MY 4017 03:00:40,600 --> 03:00:41,240 STRONG SUIT 4018 03:00:41,240 --> 03:00:43,720 >> THANK YOU FOR THE OPPORTUNITY 4019 03:00:43,720 --> 03:00:45,840 TO PRESENT TO YOU TODAY. 4020 03:00:45,840 --> 03:00:47,360 * . 4021 03:00:47,360 --> 03:00:49,520 >> IT'S WONDERFUL TO BE HERE AND 4022 03:00:49,520 --> 03:00:52,360 SEE MANY FAMILIAR FACES AND 4023 03:00:52,360 --> 03:01:11,680 FRIENDS 4024 03:01:11,680 --> 03:01:13,680 I'LL BEGIN WITH A BRIEF OVERVIEW 4025 03:01:13,680 --> 03:01:16,600 OF THE FDA DRUG APPROVAL PROCESS 4026 03:01:16,600 --> 03:01:19,480 AS IT PERTAINS TO DRUG 4027 03:01:19,480 --> 03:01:22,440 REPURPOSING AND TALK ABOUT OUR 4028 03:01:22,440 --> 03:01:26,680 EFFORTS AND THE CURE ID PLATFORM 4029 03:01:26,680 --> 03:01:29,520 THAT WAS DEVELOPED BY FDA AND 4030 03:01:29,520 --> 03:01:30,520 NCATS. 4031 03:01:30,520 --> 03:01:32,200 AND THEN THE PUBLIC/PRIVATE 4032 03:01:32,200 --> 03:01:33,000 PARTNERSHIP THAT DEVELOPED OUT 4033 03:01:33,000 --> 03:01:34,480 OF THE MEETING THAT WAS 4034 03:01:34,480 --> 03:01:37,280 MENTIONED THE 2019 OFF PATENT 4035 03:01:37,280 --> 03:01:39,600 REPURPOSING MEETING AND THEN 4036 03:01:39,600 --> 03:01:42,320 DISCUSS OUR EXPANSION TO DATA 4037 03:01:42,320 --> 03:01:44,360 COMING FROM ELECTRONIC HEALTH 4038 03:01:44,360 --> 03:01:45,560 RECORDS AND THE OPPORTUNITY THAT 4039 03:01:45,560 --> 03:01:48,200 I SEE FOR THE PLATFORM IN RARE 4040 03:01:48,200 --> 03:01:50,400 DISEASES AND HOW WE'RE EXPANDING 4041 03:01:50,400 --> 03:01:51,960 IN THAT SPACE. 4042 03:01:51,960 --> 03:01:54,680 CURRENTLY FDA APPROVAL AND 4043 03:01:54,680 --> 03:01:56,960 MARKETING REQUIRES A SPONSOR TO 4044 03:01:56,960 --> 03:02:00,040 SUBMIT A NEW DRUG APPLICATION. 4045 03:02:00,040 --> 03:02:03,120 HOWEVER WHEN THERE IS NO 4046 03:02:03,120 --> 03:02:05,240 COMMERCIAL INCENTIVE DISEASES 4047 03:02:05,240 --> 03:02:07,320 WERE OFTEN NEGLECTED AND TRIALS 4048 03:02:07,320 --> 03:02:11,640 ARE NOT FUNDED AND DRUG 4049 03:02:11,640 --> 03:02:13,880 DISCOVERY IN SOME INSTANCES 4050 03:02:13,880 --> 03:02:14,080 CEASES. 4051 03:02:14,080 --> 03:02:15,280 *. 4052 03:02:15,280 --> 03:02:20,440 ONCE APPROVED -- THERE IS 4053 03:02:20,440 --> 03:02:23,280 SUBSTANTIAL EVIDENCE OF THE 4054 03:02:23,280 --> 03:02:25,280 EFFECTIVENESS -- ONCE A DRUG IS 4055 03:02:25,280 --> 03:02:27,080 APPROVED HOWEVER BASED ON 4056 03:02:27,080 --> 03:02:33,400 KNOWLEDGE AND PROFESSIONAL JUDGE 4057 03:02:33,400 --> 03:02:35,880 THEY MAY PRESCRIBE FOR DIFFERENT 4058 03:02:35,880 --> 03:02:39,920 POPULATIONS AND DOSES AND 4059 03:02:39,920 --> 03:02:41,160 DISEASES NOT PREVIOUSLY 4060 03:02:41,160 --> 03:02:42,080 DESCRIBED FOR. 4061 03:02:42,080 --> 03:02:49,080 THE PATHWAY INCLUDES THE 505 B-1 4062 03:02:49,080 --> 03:02:52,080 B-1. 4063 03:02:52,080 --> 03:02:53,880 505B2. 4064 03:02:53,880 --> 03:03:01,520 AND THEN THE 505J. 4065 03:03:01,520 --> 03:03:03,960 THE PATHWAYS THAT APPLY 4066 03:03:03,960 --> 03:03:08,320 TYPICALLY FOR REPURPOSED DRUGS 4067 03:03:08,320 --> 03:03:13,240 ARE EITHER A SUPPLEMENT MENTAL 4068 03:03:13,240 --> 03:03:14,600 INDICATION OR NDA. 4069 03:03:14,600 --> 03:03:16,360 THIS WOULD BE THE TRADITIONAL 4070 03:03:16,360 --> 03:03:18,880 PATHWAY THAT A COMMERCIAL 4071 03:03:18,880 --> 03:03:20,480 SPONSOR WOULD GO THROUGH TO ADD 4072 03:03:20,480 --> 03:03:22,960 AN INDICATION TO THEIR LABEL OR 4073 03:03:22,960 --> 03:03:30,080 MOST FREQUENTLY THE 505B2 IS AN 4074 03:03:30,080 --> 03:03:34,040 OFF PATENT DRUG -- AND NOT THE 4075 03:03:34,040 --> 03:03:34,840 HOLDER. 4076 03:03:34,840 --> 03:03:38,360 THERE ARE SOME EXCEPTIONS. 4077 03:03:38,360 --> 03:03:40,080 PARTICULARLY WHEN WE START 4078 03:03:40,080 --> 03:03:41,320 THINKING ABOUT DRUGS THAT MAY 4079 03:03:41,320 --> 03:03:42,760 HAVE BEEN APPROVED IN OTHER 4080 03:03:42,760 --> 03:03:45,520 COUNTRIES. 4081 03:03:45,520 --> 03:03:47,280 BUT THESE ARE THE STANDARD 4082 03:03:47,280 --> 03:03:47,720 PATHWAYS. 4083 03:03:47,720 --> 03:03:52,040 7 SO ONCE A DRUG IS APPROVED FDA 4084 03:03:52,040 --> 03:03:53,920 GENERALLY DOES NOT REGULATE HOW 4085 03:03:53,920 --> 03:03:57,560 AND FOR WHAT USES A PHYSICIAN 4086 03:03:57,560 --> 03:03:59,040 PRESCRIBE THAT'S DRUG SO IT CAN 4087 03:03:59,040 --> 03:04:01,240 BE PRESCRIBED OFF LABEL FOR 4088 03:04:01,240 --> 03:04:03,120 DIFFERENT TREATMENT REGIMENS OR 4089 03:04:03,120 --> 03:04:05,680 PATIENT POPULATIONS. 4090 03:04:05,680 --> 03:04:10,280 SO THINKING ABOUT WHAT FDA COULD 4091 03:04:10,280 --> 03:04:13,480 DO TO TRY TO FACILITATE DRUG 4092 03:04:13,480 --> 03:04:15,640 DEVELOPMENT FOR AREAS OF HIGH 4093 03:04:15,640 --> 03:04:18,320 UNMET MEDICAL NEED -- WE SAW AN 4094 03:04:18,320 --> 03:04:21,360 OPPORTUNITY FOR CAPTURE 4095 03:04:21,360 --> 03:04:24,240 INFORMATION DIRECTLY FROM 4096 03:04:24,240 --> 03:04:26,120 CLINICIANS WHO WERE USING DRUGS 4097 03:04:26,120 --> 03:04:27,120 OFF LABEL. 4098 03:04:27,120 --> 03:04:31,320 SO IN 2012 AND 2013 ESTABLISHED 4099 03:04:31,320 --> 03:04:34,440 CURE ID WHICH IS THIS PLATFORM 4100 03:04:34,440 --> 03:04:36,920 WHICH THE GOAL BEING TO VERY 4101 03:04:36,920 --> 03:04:39,440 RAPIDLY ENABLE CLINICIANS TO 4102 03:04:39,440 --> 03:04:41,360 SUBMIT AND OTHER HEALTH CARE 4103 03:04:41,360 --> 03:04:44,480 PROVIDERS GLOBALLY TO BE ABLE TO 4104 03:04:44,480 --> 03:04:46,680 SUBMIT A CASE TO TELL US WHAT 4105 03:04:46,680 --> 03:04:48,320 DISEASE DID YOUR PATIENT HAVE. 4106 03:04:48,320 --> 03:04:51,000 WHAT DRUGS DID YOU USE IN A NEW 4107 03:04:51,000 --> 03:04:53,120 WAY AND WHY AND WHAT WAS THE 4108 03:04:53,120 --> 03:04:53,360 OUTCOME? 4109 03:04:53,360 --> 03:04:55,760 WITH ADDITIONAL QUESTIONS ABOUT 4110 03:04:55,760 --> 03:04:57,760 ADVERSE EVENTS AND DEMO 4111 03:04:57,760 --> 03:04:58,280 GRAPHINGS. 4112 03:04:58,280 --> 03:04:59,960 AND ALL OF THAT. 4113 03:04:59,960 --> 03:05:03,280 THE DATA WOULD THEN BE MADE 4114 03:05:03,280 --> 03:05:04,640 OPENLY AVAILABLE TO HEALTH CARE 4115 03:05:04,640 --> 03:05:07,640 PROVIDERS TO EXPLORE. 4116 03:05:07,640 --> 03:05:10,400 AND IT WOULD ALSO INCLUDE A 4117 03:05:10,400 --> 03:05:11,880 DISCUSSION FORUM WHERE PEOPLE 4118 03:05:11,880 --> 03:05:13,680 COULD ASK TREATMENT QUESTIONS. 4119 03:05:13,680 --> 03:05:15,680 REFERENCE TO CLINICAL TRIALS SO 4120 03:05:15,680 --> 03:05:19,080 YOU COULD READILY SEARCH FOR 4121 03:05:19,080 --> 03:05:21,040 CLINICAL TRIALS AND OTHER 4122 03:05:21,040 --> 03:05:21,680 REGISTRIES. 4123 03:05:21,680 --> 03:05:23,920 AND OTHER FEATURES TO TRY TO 4124 03:05:23,920 --> 03:05:26,680 ENCOURAGE ENGAGEMENT. 4125 03:05:26,680 --> 03:05:28,200 AND THIS INCLUDES BOTH THE 4126 03:05:28,200 --> 03:05:29,680 WEBSITE AND THE THE MOBILE 4127 03:05:29,680 --> 03:05:30,800 APPLICATION. 4128 03:05:30,800 --> 03:05:33,120 WHICH I'LL SHARE THE LINK IN 4129 03:05:33,120 --> 03:05:34,040 JUST A MINUTE. 4130 03:05:34,040 --> 03:05:36,920 THE GOAL OF THE PLATFORM WAS TO 4131 03:05:36,920 --> 03:05:38,680 ENHANCE OUR UNDERSTANDING OF NEW 4132 03:05:38,680 --> 03:05:40,440 USES OF APPROVED MEDICAL 4133 03:05:40,440 --> 03:05:42,760 PRODUCTS TO FACILITATE CLINICAL 4134 03:05:42,760 --> 03:05:44,320 TRIALS AND DEVELOPMENT BASED ON 4135 03:05:44,320 --> 03:05:46,880 THE ENHANCED UNDERSTANDING WHILE 4136 03:05:46,880 --> 03:05:48,120 RECOGNIZING THAT IT COULD SERVE 4137 03:05:48,120 --> 03:05:50,440 AS A RESOURCE FOR PHYSICIANS TO 4138 03:05:50,440 --> 03:05:51,240 SHARE INFORMATION WHERE THERE 4139 03:05:51,240 --> 03:05:54,720 MAY BE NO FDA APPROVED PRODUCTS 4140 03:05:54,720 --> 03:05:57,520 THAT EXISTED FOR THE NEW USE 4141 03:05:57,520 --> 03:05:59,520 THAT WAS USED AND PROVEN TO BE 4142 03:05:59,520 --> 03:06:01,240 SAFE AND EFFECTIVE. 4143 03:06:01,240 --> 03:06:03,640 IT WAS CLEAR THIS WAS A 4144 03:06:03,640 --> 03:06:05,760 MULTI-STAKEHOLDER PROCESS AND IT 4145 03:06:05,760 --> 03:06:06,680 WOULD BENEFIT FROM THE 4146 03:06:06,680 --> 03:06:08,680 INVOLVEMENT OF EXTERNAL PARTIES. 4147 03:06:08,680 --> 03:06:11,120 IN PARTICULAR YOU CAN IMAGINE 4148 03:06:11,120 --> 03:06:15,440 THAT FOR FDA IT WAS A DIFFICULT 4149 03:06:15,440 --> 03:06:19,400 TIGHT ROPE TO WALK BETWEEN 4150 03:06:19,400 --> 03:06:20,520 WANTING TO COLLECT CASE REPORTS 4151 03:06:20,520 --> 03:06:22,960 AND SEE THE VALUE OF THE CASE 4152 03:06:22,960 --> 03:06:24,680 REPORTS WHILE RECOGNIZING THE 4153 03:06:24,680 --> 03:06:26,440 SIGNIFICANT LIMITATIONS OF CASE 4154 03:06:26,440 --> 03:06:28,080 REPORTS AS WELL AND THAT WE 4155 03:06:28,080 --> 03:06:29,960 REALLY WANTED TO DRIVE THIS 4156 03:06:29,960 --> 03:06:32,320 TOWARDS RANDOMIZED CONTROL 4157 03:06:32,320 --> 03:06:33,680 TRIALS WHERE POSSIBLE. 4158 03:06:33,680 --> 03:06:35,680 HOWEVER, REPRESENTING THAT THAT 4159 03:06:35,680 --> 03:06:36,920 IS NOT ALWAYS POSSIBLE. 4160 03:06:36,920 --> 03:06:39,600 BUT IT WAS IMPORTANT THAT WHERE 4161 03:06:39,600 --> 03:06:41,480 THAT WAS FEASIBLE THAT WE TRY TO 4162 03:06:41,480 --> 03:06:44,960 DRIVE IT FROM YOU ARE NOW 4163 03:06:44,960 --> 03:06:48,560 COLLECTING CASES SYSTEMATICALLY 4164 03:06:48,560 --> 03:06:52,240 SO IT'S USING STANDARDIZED 4165 03:06:52,240 --> 03:06:54,160 PROCESSES AND THEM TO PUSH IT 4166 03:06:54,160 --> 03:06:56,000 FURTHER TO HOW DO WE ACTUALLY 4167 03:06:56,000 --> 03:06:59,280 GENERATE EVIDENCE AND 4168 03:06:59,280 --> 03:07:00,120 DISSEMINATE THAT EVIDENCE IN THE 4169 03:07:00,120 --> 03:07:02,880 FORM OF CLINICAL TRIALS AND 4170 03:07:02,880 --> 03:07:05,880 ULTIMATELY REGULATORY APPROVAL. 4171 03:07:05,880 --> 03:07:09,640 SO IN JUNE OF 2020 AS A RESULT 4172 03:07:09,640 --> 03:07:11,720 OF THE 2019 MEETING THAT I 4173 03:07:11,720 --> 03:07:13,880 REFERRED TO WHICH IDENTIFIED THE 4174 03:07:13,880 --> 03:07:16,320 NEED FOR A PUBLIC/PRIVATE 4175 03:07:16,320 --> 03:07:18,080 PARTNERSHIP WE WERE ABLE TO WORK 4176 03:07:18,080 --> 03:07:20,120 WITH THE CRITICAL PATH INSTITUTE 4177 03:07:20,120 --> 03:07:22,880 AS WELL AS OUR PARTNERS AT NCATS 4178 03:07:22,880 --> 03:07:27,880 TO CONVENE A PUBLIC/PRIVATE 4179 03:07:27,880 --> 03:07:30,680 PARTNERSHIP -- CDRC. 4180 03:07:30,680 --> 03:07:34,280 IT BEGAN WITH A PILOT FOCUSED ON 4181 03:07:34,280 --> 03:07:36,920 DRUG REPURPOSING IN COVID WHICH 4182 03:07:36,920 --> 03:07:38,880 AS BARBARA POINTED OUT 4183 03:07:38,880 --> 03:07:41,400 HIGHLIGHTED BOTH THE PROS AND 4184 03:07:41,400 --> 03:07:43,000 CONS BUT IMPORTANTLY THE NEED 4185 03:07:43,000 --> 03:07:45,640 FOR A BETTER INFRASTRUCTURE WITH 4186 03:07:45,640 --> 03:07:47,880 WHICH TO CONDUCT REPURPOSING TO 4187 03:07:47,880 --> 03:07:51,280 BE ABLE TO CAPTURE DATA HAVE 4188 03:07:51,280 --> 03:07:53,920 THAT WHERE APPROPRIATE. 4189 03:07:53,920 --> 03:07:55,680 OUR GOAL HAS BEEN TO BUILD 4190 03:07:55,680 --> 03:07:57,360 INFRASTRUCTURE FOR COVID THAT 4191 03:07:57,360 --> 03:07:59,720 COULD BE USED FOR PARTICULARLY 4192 03:07:59,720 --> 03:08:01,440 NEGLECTED AND INFECTIOUS 4193 03:08:01,440 --> 03:08:03,960 DISEASES IN THE FUTURE BUT ALSO 4194 03:08:03,960 --> 03:08:06,400 FOR RARE DISEASES. 4195 03:08:06,400 --> 03:08:08,680 THEY HOPE TO DEMONSTRATE HOW 4196 03:08:08,680 --> 03:08:11,040 DATA SHARED FROM CLINICIANS IN 4197 03:08:11,040 --> 03:08:13,280 REALTIME COULD BE USED TO INFORM 4198 03:08:13,280 --> 03:08:15,080 ONGOING AND FUTURE CLINICAL 4199 03:08:15,080 --> 03:08:19,840 TRIALS AND BASED ON THE TRIAL 4200 03:08:19,840 --> 03:08:24,320 RESULTS DRUG LABELING. 4201 03:08:24,320 --> 03:08:25,880 I'LL JUST POINT OUT THAT THERE 4202 03:08:25,880 --> 03:08:27,880 ARE FOUR -- THIS IS THE ADVISORY 4203 03:08:27,880 --> 03:08:30,680 COMMITTEE THAT OVERSEES THE CDRC 4204 03:08:30,680 --> 03:08:32,480 AND THERE ARE FOUR TOPICAL 4205 03:08:32,480 --> 03:08:33,280 COMMITTEES. 4206 03:08:33,280 --> 03:08:34,520 THE INFECTIOUS DISEASE 4207 03:08:34,520 --> 03:08:35,080 COMMITTEE. 4208 03:08:35,080 --> 03:08:36,880 THE RARE DISEASE COMMITTEE. 4209 03:08:36,880 --> 03:08:39,800 SPECIAL POPULATIONS AND THEN AN 4210 03:08:39,800 --> 03:08:40,760 OPERATION. 4211 03:08:40,760 --> 03:08:44,280 FOR THIS GROUP THE INTEREST IS 4212 03:08:44,280 --> 03:08:47,080 PROBABLY MOSTLY IN THE RARE 4213 03:08:47,080 --> 03:08:50,080 DISEASE COMMITTEE. 4214 03:08:50,080 --> 03:08:51,920 WE'VE BEEN ABLE TO RECEIVE A 4215 03:08:51,920 --> 03:08:53,760 GOOD DEAL OF SUPPORT. 4216 03:08:53,760 --> 03:08:56,680 AND SO THEY HAVE FUNDED BOTH A 4217 03:08:56,680 --> 03:09:02,440 RARE ONCOLOGY AS WELL AS A RARE 4218 03:09:02,440 --> 03:09:04,880 NONONCOLOGY GROUP AND THE 4219 03:09:04,880 --> 03:09:08,160 NONONCOLOGY GROUP HAS CHOSEN AS 4220 03:09:08,160 --> 03:09:10,200 ITS FIRST PILOT. 4221 03:09:10,200 --> 03:09:11,880 BUT WE TALKED ABOUT THE 4222 03:09:11,880 --> 03:09:14,480 POTENTIAL OF THE MUSCULAR 4223 03:09:14,480 --> 03:09:15,840 DYSTROPHYs BEING ANOTHER 4224 03:09:15,840 --> 03:09:19,480 POTENTIAL PILOT AREA. 4225 03:09:19,480 --> 03:09:21,960 SO THE CDRC TRIES TO THINK ABOUT 4226 03:09:21,960 --> 03:09:26,040 THE SORT OF WHOLE SPECTRUM OF 4227 03:09:26,040 --> 03:09:30,560 DRUG REPURPOSING AND TO RATHER 4228 03:09:30,560 --> 03:09:32,360 THAN VIEWING INDIVIDUAL POCKETS 4229 03:09:32,360 --> 03:09:36,320 TRY TO THINK ABOUT THE FULL 4230 03:09:36,320 --> 03:09:38,480 SPECTRUM IN TOTALITY. 4231 03:09:38,480 --> 03:09:41,040 SO WE BEGIN BUT I THINKING ABOUT 4232 03:09:41,040 --> 03:09:45,280 WHICH DISEASES -- DRUG 4233 03:09:45,280 --> 03:09:47,400 REPURPOSING CAN BE HELPFUL THIS. 4234 03:09:47,400 --> 03:09:49,840 THERE ARE DISEASES THAT HAVE 4235 03:09:49,840 --> 03:09:52,680 EFFECTIVE TREATMENTS OR DRUG 4236 03:09:52,680 --> 03:09:55,120 REPURPOSING DOESN'T SEEM TO BE A 4237 03:09:55,120 --> 03:09:57,000 PROMISING OPTION OR IT'S JUST 4238 03:09:57,000 --> 03:09:59,520 NOT HAPPENING FOR OTHER REASONS. 4239 03:09:59,520 --> 03:10:02,440 BUT THERE ARE SOME DISEASES 4240 03:10:02,440 --> 03:10:04,120 WHERE IT'S BEEN SUCCESSFUL. 4241 03:10:04,120 --> 03:10:07,080 AND WHERE THERE APPEARS TO BE 4242 03:10:07,080 --> 03:10:08,760 OPPORTUNITIES SO WE'VE TRIED TO 4243 03:10:08,760 --> 03:10:10,760 DEFINE THOSE DISEASES AND 4244 03:10:10,760 --> 03:10:12,480 PRIORITIZE THEM. 4245 03:10:12,480 --> 03:10:15,240 WE THEN MOSTLY FOCUS ON THIS 4246 03:10:15,240 --> 03:10:19,280 REAL WORD DATA HYPOTHESIS 4247 03:10:19,280 --> 03:10:21,640 GENERATION -- AND THIS IS THE 4248 03:10:21,640 --> 03:10:24,080 CURE ID PLATFORM AS WELL AS 4249 03:10:24,080 --> 03:10:29,680 TREATMENT SURVEYS AND OTHER 4250 03:10:29,680 --> 03:10:31,240 MEANS OF COLLECTION. 4251 03:10:31,240 --> 03:10:33,560 AND THEN THE GOAL IS TO CONFIRM 4252 03:10:33,560 --> 03:10:36,000 THE HYPOTHESIS GENERATED IN 4253 03:10:36,000 --> 03:10:37,600 CLINICAL TRIALS AND TO THINK 4254 03:10:37,600 --> 03:10:41,480 ABOUT HOW TO DO THAT IN A MORE 4255 03:10:41,480 --> 03:10:49,280 STRATEGIC AND ROBUST MANNER AND 4256 03:10:49,280 --> 03:10:51,600 HOW DOES IT GET DISSEMINATED 4257 03:10:51,600 --> 03:10:54,160 INTO CLINICAL PRACTICE SO IT CAN 4258 03:10:54,160 --> 03:10:56,080 BENEFIT PATIENTS WHAT ARE THE 4259 03:10:56,080 --> 03:10:57,840 LEGISLATIVE AND POLICY CHANGES 4260 03:10:57,840 --> 03:11:00,280 THAT MAY BE NEEDED IN ORDER TO 4261 03:11:00,280 --> 03:11:02,600 FACILITATE THAT. 4262 03:11:02,600 --> 03:11:08,040 SO I'M NOT GOING TO LIE. 4263 03:11:08,040 --> 03:11:09,360 GETTING CASE REPORTS IS HARD IN 4264 03:11:09,360 --> 03:11:11,480 THE BEST OF TIMES AND DURING A 4265 03:11:11,480 --> 03:11:12,920 GLOBAL PANDEMIC HAS BEEN NEARLY 4266 03:11:12,920 --> 03:11:13,360 IMPOSSIBLE. 4267 03:11:13,360 --> 03:11:18,800 7 BUT IT DID PRESENT AN 4268 03:11:18,800 --> 03:11:20,400 OPPORTUNITY TO THINK OUTSIDE THE 4269 03:11:20,400 --> 03:11:23,480 BOX TO COME UP WITH ALTERNATIVE 4270 03:11:23,480 --> 03:11:25,360 WAYS ABOUT HOW DRUGS ARE BEING 4271 03:11:25,360 --> 03:11:26,360 REPURPOSED. 4272 03:11:26,360 --> 03:11:28,480 SO ACTUALLY TWO ASPECTS OF THIS 4273 03:11:28,480 --> 03:11:30,160 MAY BE OF INTEREST TO THIS 4274 03:11:30,160 --> 03:11:30,360 GROUP. 4275 03:11:30,360 --> 03:11:33,440 THE FIRST IS THAT SO WE WERE 4276 03:11:33,440 --> 03:11:38,480 AWARDED A LARGE GRANT FROM -- 4277 03:11:38,480 --> 03:11:38,680 HHS. 4278 03:11:38,680 --> 03:11:45,960 WHICH IS PART OF THE -- TO BUILD 4279 03:11:45,960 --> 03:11:56,480 AN AUTOMATED EXTRACTIONS TOOL. 4280 03:11:56,480 --> 03:12:01,240 INITIALLY FOR COVID BUT WITH THE 4281 03:12:01,240 --> 03:12:06,320 BEYOND CO COVID OBJECTIVE. 4282 03:12:06,320 --> 03:12:09,000 AND WE'VE PARTNERED WITH EMORY, 4283 03:12:09,000 --> 03:12:11,520 THE SOCIETY FOR CRITICAL CARE 4284 03:12:11,520 --> 03:12:14,960 MEDICINE VIRUS REGISTRY AND THE 4285 03:12:14,960 --> 03:12:26,600 OBASOHA--OBSERVATORY IN OXFORD. 4286 03:12:26,600 --> 03:12:30,120 IT WILL BE MADE OPEN AND 4287 03:12:30,120 --> 03:12:32,000 ACCESSIBLE AS WELL AS A LARGER 4288 03:12:32,000 --> 03:12:34,000 MORE DETAILED DATASET WHICH WILL 4289 03:12:34,000 --> 03:12:36,000 BE MADE AVAILABLE TO RESEARCHERS 4290 03:12:36,000 --> 03:12:39,440 WHO SUBMIT A RESEARCH PROPOSAL. 4291 03:12:39,440 --> 03:12:41,800 WE'RE NOW EVALUATING THE 4292 03:12:41,800 --> 03:12:43,480 POTENTIAL UTILITY THAT THIS TOOL 4293 03:12:43,480 --> 03:12:45,800 COULD HAVE IN DIFFERENT RARE 4294 03:12:45,800 --> 03:12:47,720 DISEASE SPACES AS WELL AS OTHER 4295 03:12:47,720 --> 03:12:49,720 SPACES BUT WE'RE EXCITED ABOUT 4296 03:12:49,720 --> 03:12:52,000 THE POTENTIAL IT HAS FOR RARE 4297 03:12:52,000 --> 03:12:52,520 DISEASES. 4298 03:12:52,520 --> 03:12:54,720 WE CURRENTLY HAVE ACCESS OR WILL 4299 03:12:54,720 --> 03:12:59,560 HAVE ACCESS TO SEVERAL UP 4300 03:12:59,560 --> 03:13:00,720 INSTITUTIONAL SITES HOSPITALS 4301 03:13:00,720 --> 03:13:02,880 AND HEALTH CARE SYSTEMS SO THE 4302 03:13:02,880 --> 03:13:05,880 ABILITY TO IDENTIFY CASES WITH 4303 03:13:05,880 --> 03:13:07,640 RARE DISEASES AND THEN 4304 03:13:07,640 --> 03:13:09,200 UNDERSTAND HOW THEY WERE TREATED 4305 03:13:09,200 --> 03:13:10,880 AND AGGREGATE THAT INFORMATIONS 4306 03:13:10,880 --> 03:13:13,160 VERY EXCITING. 4307 03:13:13,160 --> 03:13:16,000 MUCH TO WORK OUT IN TERMS OF THE 4308 03:13:16,000 --> 03:13:19,600 LOGISTICS OF HOW TO DO THAT. 4309 03:13:19,600 --> 03:13:22,240 WHILE OF COURSE BEING COGNIZANT 4310 03:13:22,240 --> 03:13:23,280 OF PRIVACY CONCERNS AND THE 4311 03:13:23,280 --> 03:13:26,720 OTHER ISSUES BUT I THINK IF WE 4312 03:13:26,720 --> 03:13:28,720 WERE ABLE TO DO THAT IT WOULD 4313 03:13:28,720 --> 03:13:31,080 HAVE GREAT UTILITY TO THE 4314 03:13:31,080 --> 03:13:32,440 RESEARCH COMMUNITY. 4315 03:13:32,440 --> 03:13:33,880 THE SECOND PART WHICH IS 4316 03:13:33,880 --> 03:13:35,440 IMPORTANT IS THAT THIS GRANT 4317 03:13:35,440 --> 03:13:37,080 ALSO INCLUDES THE DEVELOPMENT OF 4318 03:13:37,080 --> 03:13:40,880 A PATIENT PORTAL TO THE CURE 4319 03:13:40,880 --> 03:13:41,680 PLATFORM. 4320 03:13:41,680 --> 03:13:44,320 WE'RE PILOTING THIS WITH LONG 4321 03:13:44,320 --> 03:13:45,800 COVID. 4322 03:13:45,800 --> 03:13:47,600 THIS WOULD INCLUDE A CASE REPORT 4323 03:13:47,600 --> 03:13:50,040 FORM FOR PATIENTS INSTEAD OF 4324 03:13:50,040 --> 03:13:51,680 CLINICIANS TO FILL OUT AND THERE 4325 03:13:51,680 --> 03:13:53,600 IS GREAT EXCITEMENT ABOUT THE 4326 03:13:53,600 --> 03:13:55,840 POTENTIAL TO USE THIS IN THE 4327 03:13:55,840 --> 03:13:57,680 AREA DISEASE SPACE. 4328 03:13:57,680 --> 03:14:00,480 GIVEN THAT PATIENTS KNOW MORE 4329 03:14:00,480 --> 03:14:03,040 ABOUT THEIR DISEASES THAN THEIR 4330 03:14:03,040 --> 03:14:03,680 DOCTORS DO. 4331 03:14:03,680 --> 03:14:05,640 THE LAST THINGS THAT I MENTIONED 4332 03:14:05,640 --> 03:14:07,880 THE IMPORTANCE OF MOVING TOWARDS 4333 03:14:07,880 --> 03:14:10,640 AN INFRASTRUCTURE FOR BETTER 4334 03:14:10,640 --> 03:14:14,840 STUDYING POTENTIAL DRUG 4335 03:14:14,840 --> 03:14:18,040 REPURPOSING HYPOTHESIS IN 4336 03:14:18,040 --> 03:14:19,680 RANDOMIZED CONTROLLED TRIALS. 4337 03:14:19,680 --> 03:14:22,680 SO IN THE LAST YEAR AND A HALF 4338 03:14:22,680 --> 03:14:25,920 CDRC AND EMORY HAVE PARTNERED 4339 03:14:25,920 --> 03:14:27,680 TOGETHER AND ARE REVIEWING TWO 4340 03:14:27,680 --> 03:14:31,680 LARGE ADAPTIVE PLATFORM 4341 03:14:31,680 --> 03:14:32,440 RANDOMIZED DESIGNS. 4342 03:14:32,440 --> 03:14:34,880 INITIALLY FOR COVID BUT THINKING 4343 03:14:34,880 --> 03:14:36,760 ABOUT HOW THE TRIAL DESIGNS 4344 03:14:36,760 --> 03:14:39,440 COULD BE PIVOTS TO OTHER DISEASE 4345 03:14:39,440 --> 03:14:40,000 AREAS. 4346 03:14:40,000 --> 03:14:41,680 AND I'LL TALK ABOUT THAT MORE IN 4347 03:14:41,680 --> 03:14:43,240 A SECOND. 4348 03:14:43,240 --> 03:14:47,960 SO WE HAVE PREDICT WHICH IS AN 4349 03:14:47,960 --> 03:14:49,880 ACUTE IMPATIENT TRIAL EMBEDDED 4350 03:14:49,880 --> 03:14:56,360 IN CLINICAL PRACTICE AND USING 4351 03:14:56,360 --> 03:15:00,920 EHRS. 4352 03:15:00,920 --> 03:15:03,200 AND THEN TLC WHICH IS TREATMENT 4353 03:15:03,200 --> 03:15:07,200 OF LONG COVID WHICH IS A CHRONIC 4354 03:15:07,200 --> 03:15:09,120 LONG-TERM OUTPATIENT TRIAL USING 4355 03:15:09,120 --> 03:15:13,200 TELEMEDICINE AND ELECTRONIC 4356 03:15:13,200 --> 03:15:14,880 PATIENT OUTCOME MEASURES. 4357 03:15:14,880 --> 03:15:17,800 WITH THE FOCUS OF THE EVALUATION 4358 03:15:17,800 --> 03:15:24,600 OF EFFICACY OF REPURPOSED AGENTS 4359 03:15:24,600 --> 03:15:26,680 AGENTS. 4360 03:15:26,680 --> 03:15:29,280 SO THE POINT WAS IN THE SLIDE 4361 03:15:29,280 --> 03:15:31,600 THAT I APPARENTLY TOOK OUT WAS 4362 03:15:31,600 --> 03:15:34,160 WE'RE THINKING ABOUT THIS IN 4363 03:15:34,160 --> 03:15:40,960 TERMS OF CURRENT PANDEMIC AND 4364 03:15:40,960 --> 03:15:42,240 REEMERGING PANDEMIC AND HOW IT 4365 03:15:42,240 --> 03:15:44,440 COULD BE SCALED UP OR SCALED 4366 03:15:44,440 --> 03:15:46,920 DOWN AND PIVOTED IN SUCH WAYS SO 4367 03:15:46,920 --> 03:15:48,760 THAT THE INFRASTRUCTURE COULD BE 4368 03:15:48,760 --> 03:15:50,800 MUCH MORE SUSTAINABLE AND USED 4369 03:15:50,800 --> 03:15:52,280 IN AN ONGOING WAY. 4370 03:15:52,280 --> 03:15:55,240 IT'S A RATHER ELABORATE VISION 4371 03:15:55,240 --> 03:15:58,440 OF A NEW WAY OF DOING TRIALS BUT 4372 03:15:58,440 --> 03:16:00,320 WE'RE HOPING IT WILL BE OF 4373 03:16:00,320 --> 03:16:01,880 INTERESTED. 4374 03:16:01,880 --> 03:16:03,200 HERE IS SOME OF THE 4375 03:16:03,200 --> 03:16:06,640 OPPORTUNITIES I WONDER ABOUT FOR 4376 03:16:06,640 --> 03:16:10,160 USING THE CURE PLATFORM AS A 4377 03:16:10,160 --> 03:16:12,920 RARE DISEASE PLATFORM BOTH ON A 4378 03:16:12,920 --> 03:16:15,040 NATIONAL OR INTERNATIONAL LEVEL. 4379 03:16:15,040 --> 03:16:17,600 I WOULD LOVE TO EVALUATE WHETHER 4380 03:16:17,600 --> 03:16:19,840 IT MIGHT BE POSSIBLE TO USE THE 4381 03:16:19,840 --> 03:16:23,040 EH RUCKS EXTRACTION TOOL TO 4382 03:16:23,040 --> 03:16:25,280 ENABLE THE GATHERING OF CASES 4383 03:16:25,280 --> 03:16:27,080 AND WOULD LOVE TO HEAR MORE IF 4384 03:16:27,080 --> 03:16:28,920 THERE ARE FOLKS THAT YOU MIGHT 4385 03:16:28,920 --> 03:16:30,800 THINK WOULD BE INTERESTED IN 4386 03:16:30,800 --> 03:16:32,280 PARTICIPATING OR EITHER 4387 03:16:32,280 --> 03:16:34,080 CONTRIBUTING DATA OR BEING ON 4388 03:16:34,080 --> 03:16:36,920 OUR RARE DISEASE COORDINATING 4389 03:16:36,920 --> 03:16:39,040 COMMITTEE OR ONE OF THE WORKING 4390 03:16:39,040 --> 03:16:41,080 GROUPS, WE WOULD LOVE TO TALK. 4391 03:16:41,080 --> 03:16:42,000 THAT IS IT. 4392 03:16:42,000 --> 03:16:44,680 THANK YOU SO MUCH. 4393 03:16:44,680 --> 03:16:45,440 >> THANK YOU. 4394 03:16:45,440 --> 03:16:48,240 THANK YOU, HEATHER FOR THAT VERY 4395 03:16:48,240 --> 03:16:49,680 GREAT OVERVIEW. 4396 03:16:49,680 --> 03:16:52,760 WE CAN TAKE QUESTIONS. 4397 03:16:52,760 --> 03:16:55,000 WE HAVE A LITTLE LESS THAN FOUR 4398 03:16:55,000 --> 03:16:57,000 MINUTES NOW SO WE APPRECIATE YOU 4399 03:16:57,000 --> 03:17:01,480 STAYING ON TIME. 4400 03:17:01,480 --> 03:17:06,400 I SAW A FLASH OF A HAND. 4401 03:17:06,400 --> 03:17:08,880 WOULD YOU LIKE TO ASK YOUR 4402 03:17:08,880 --> 03:17:11,760 QUESTION OR A MAKE YOUR COMMENT 4403 03:17:11,760 --> 03:17:14,480 >> I THOUGHT ALL OF THESE WERE 4404 03:17:14,480 --> 03:17:16,480 VERY INFORMATIVE AND EXCELLENT. 4405 03:17:16,480 --> 03:17:17,680 SO THANK YOU VERY MUCH. 4406 03:17:17,680 --> 03:17:19,280 THIS WAS A GREAT SESSION. 4407 03:17:19,280 --> 03:17:21,720 AND BACK TO DAN'S COMMENT ABOUT 4408 03:17:21,720 --> 03:17:28,680 HOW TO BALANCE THE BASIC 4409 03:17:28,680 --> 03:17:30,320 MECHANISTIC STUDIES VERSUS THE 4410 03:17:30,320 --> 03:17:32,240 DRUG REPURPOSING. 4411 03:17:32,240 --> 03:17:34,240 AND OF COURSE IN A PERFECT WORLD 4412 03:17:34,240 --> 03:17:37,200 WE WANT BOTH AND THEY ARE 4413 03:17:37,200 --> 03:17:38,680 COMPLIMENTARY AND WE NEED BOTH 4414 03:17:38,680 --> 03:17:40,680 BUT THEY CAN BE MUTUALLY 4415 03:17:40,680 --> 03:17:42,120 INFORMATIVE IF WE'RE SMART ABOUT 4416 03:17:42,120 --> 03:17:44,080 IT. 4417 03:17:44,080 --> 03:17:47,680 SO NEW UNDERSTANDING ABOUT BASIC 4418 03:17:47,680 --> 03:17:49,880 MECHANISMs FOR DISEASE SUGGEST 4419 03:17:49,880 --> 03:17:51,680 OTHER DRUGS CAN BE REPURPOSED. 4420 03:17:51,680 --> 03:17:54,680 INFORMATION ABOUT SUCCESSFUL 4421 03:17:54,680 --> 03:17:58,920 REPURPOSING EXERCISES CAN GIVE 4422 03:17:58,920 --> 03:18:02,080 US CLUES ABOUT NEW MECHANISMS 4423 03:18:02,080 --> 03:18:03,760 FOR DISEASE THAT WE HAD NOT 4424 03:18:03,760 --> 03:18:04,440 CONSIDERED. 4425 03:18:04,440 --> 03:18:07,320 THE GOAL IS TO BE THINKING AND 4426 03:18:07,320 --> 03:18:09,000 COMMUNICATING ACROSS THESE 4427 03:18:09,000 --> 03:18:11,600 SPACES AND I'LL JUST THROW OUT 4428 03:18:11,600 --> 03:18:13,480 ONE EXAMPLE FROM MY AREA OF 4429 03:18:13,480 --> 03:18:18,440 RESEARCH WHICH WAS COM -- COMPELLING 4430 03:18:18,440 --> 03:18:20,280 AND DISCOVERING THROUGH 4431 03:18:20,280 --> 03:18:23,640 COMPARING GENETIC FINDINGS THAT 4432 03:18:23,640 --> 03:18:26,880 A DRUG HAD A THERE BE USED FOR 4433 03:18:26,880 --> 03:18:29,920 CANCER MIGHT BE AN ATTRACTIVE 4434 03:18:29,920 --> 03:18:32,360 DRUG FOR RHEUMATOID ARTHRITIS. 4435 03:18:32,360 --> 03:18:34,480 THE OPPORTUNITIES TO INFORM 4436 03:18:34,480 --> 03:18:36,400 THESE DIFFERENT PATHS ARE REALLY 4437 03:18:36,400 --> 03:18:40,600 TREMENDOUS. 4438 03:18:40,600 --> 03:18:42,760 >> IF YOU DON'T MIND MY 4439 03:18:42,760 --> 03:18:44,680 RESPONDING THANKS, LINDSEY. 4440 03:18:44,680 --> 03:18:47,080 SUCH AN IMPORTANT POINT AND 4441 03:18:47,080 --> 03:18:49,680 MAYBE I CAN SHARE TWO QUICK 4442 03:18:49,680 --> 03:18:52,320 EXAMPLES AS WELL THAT I FIND 4443 03:18:52,320 --> 03:18:53,760 ILLUMINATE CAN. 4444 03:18:53,760 --> 03:18:58,800 DAVID -- DR. DAVID FAGENBOMB 4445 03:18:58,800 --> 03:19:00,040 FROM THE UNIVERSITY OF 4446 03:19:00,040 --> 03:19:02,400 PENNSYLVANIA -- HE WAS CURIOUS 4447 03:19:02,400 --> 03:19:03,960 WHAT WE WERE DOING ON MUSCULAR 4448 03:19:03,960 --> 03:19:04,280 DYSTROPHY. 4449 03:19:04,280 --> 03:19:08,440 HE IS A RARE DISEASE ADVOCATE 4450 03:19:08,440 --> 03:19:12,120 WHO SAVED HIS OWN LIFE BY 4451 03:19:12,120 --> 03:19:14,480 FINDING A REPURPOSED DRUG THAT 4452 03:19:14,480 --> 03:19:18,120 HAS KEPT HIM IN REMISSION NOW 4453 03:19:18,120 --> 03:19:20,360 FOR 100 MONTHS. 4454 03:19:20,360 --> 03:19:23,040 HE DID THAT BY STUDYING HIS 4455 03:19:23,040 --> 03:19:24,920 BLOOD SAMPLES IN GREAT DETAIL 4456 03:19:24,920 --> 03:19:27,080 AND FOUND THAT THE WAY THAT THE 4457 03:19:27,080 --> 03:19:29,040 CASTLE MAN'S DISEASE OPERATED 4458 03:19:29,040 --> 03:19:30,960 THEY HAD IT BACKWARDS. 4459 03:19:30,960 --> 03:19:35,040 THE IDEA OF HOW IT WAS 4460 03:19:35,040 --> 03:19:35,280 OCCURRING. 4461 03:19:35,280 --> 03:19:38,240 SO BY UNDERSTANDING THE 4462 03:19:38,240 --> 03:19:40,360 MECHANISM OF THE DISEASE BASED 4463 03:19:40,360 --> 03:19:42,280 UPON THE SAMPLES THAT HE 4464 03:19:42,280 --> 03:19:45,360 PROCURED AND ANALYZING THEM 4465 03:19:45,360 --> 03:19:48,280 HIMSELF HE WAS ABLE TO IDENTIFY 4466 03:19:48,280 --> 03:19:50,600 -- AND BARBARA'S FAVORITE 4467 03:19:50,600 --> 03:19:52,400 EXAMPLE AS A CANDIDATE -- AND 4468 03:19:52,400 --> 03:19:55,280 THEN THE OTHER EXAMPLE I WOULD 4469 03:19:55,280 --> 03:19:57,200 TURN TO IS WHERE PEOPLE MAY HAVE 4470 03:19:57,200 --> 03:19:59,720 MULTIPLE DISEASES AND BEING ON 4471 03:19:59,720 --> 03:20:01,280 TREATMENT FOR ONE CAN LEAD TO 4472 03:20:01,280 --> 03:20:03,080 INFORMATION ABOUT THE OTHER. 4473 03:20:03,080 --> 03:20:05,600 FIBRO MYALGIA IS A GREAT 4474 03:20:05,600 --> 03:20:09,640 EXAMPLE. 4475 03:20:09,640 --> 03:20:11,880 PATIENTS WERE GETTING -- 4476 03:20:11,880 --> 03:20:15,640 SUDDENLY REALIZED THAT THEIR 4477 03:20:15,640 --> 03:20:16,520 FIBROMYALGIA SYMPTOMS WERE 4478 03:20:16,520 --> 03:20:17,520 GETTING BETTER. 4479 03:20:17,520 --> 03:20:21,760 IT WAS THOUGHT TO BE A MUSCULAR 4480 03:20:21,760 --> 03:20:24,760 SKELETAL CONDITION AND THIS IS 4481 03:20:24,760 --> 03:20:31,760 SUGGESTING THAT IT'S A COMBINES. 4482 03:20:31,760 --> 03:20:33,880 -- COMBINATION. 4483 03:20:33,880 --> 03:20:36,240 HOW THEY CAN BE MUTUALLY 4484 03:20:36,240 --> 03:20:38,280 COMPLIMENTARY IS IMPORTANT. 4485 03:20:38,280 --> 03:20:44,880 >> SLEPT POIN -- EXCELLENT POINT. 4486 03:20:44,880 --> 03:20:47,640 INITIALLY WE WERE FOCUSED ON 4487 03:20:47,640 --> 03:20:49,320 INDIVIDUAL SINGLE GENE DISORDERS 4488 03:20:49,320 --> 03:20:51,000 AND NOT THINKING ABOUT PATHWAYS 4489 03:20:51,000 --> 03:20:53,520 AND WHEN IT BECAME POSSIBLE TO 4490 03:20:53,520 --> 03:20:55,320 LOOK AT A CONDITION AND LOOK AT 4491 03:20:55,320 --> 03:20:57,240 GENE EXPRESSION AND SEE THE 4492 03:20:57,240 --> 03:20:59,360 MULTIPLE PATHWAYS THAT HAVE BEEN 4493 03:20:59,360 --> 03:21:01,600 DISRUPTED YOU CAN LOOK UPSTREAM 4494 03:21:01,600 --> 03:21:04,320 IN THOSE PATHWAYS AND THAT 4495 03:21:04,320 --> 03:21:07,160 BEGINS TO CONNECT THE DOTS IN 4496 03:21:07,160 --> 03:21:09,680 DIFFERENT NETWORKS WHICH SUGGEST 4497 03:21:09,680 --> 03:21:12,800 THAT A DRUG MIGHT BE USEFUL FOR 4498 03:21:12,800 --> 03:21:14,880 ONE CONDITION HAS RELEVANCE 4499 03:21:14,880 --> 03:21:16,560 DOWNSTREAM FOR ANOTHER ONE. 4500 03:21:16,560 --> 03:21:22,080 SO, ITS REALLY EXCITING. 4501 03:21:22,080 --> 03:21:22,280 OKAY. 4502 03:21:22,280 --> 03:21:29,920 OTHER QUESTIONS? 4503 03:21:29,920 --> 03:21:31,920 >> I HAVE A QUESTION. 4504 03:21:31,920 --> 03:21:36,640 >> SO HEATHER -- I'VE SEEN RARE 4505 03:21:36,640 --> 03:21:38,320 DISEASE COMMUNITIES USE SOCIAL 4506 03:21:38,320 --> 03:21:40,800 MEDIA TO KIND OF EXCHANGE 4507 03:21:40,800 --> 03:21:43,880 INFORMATION AND ALMOST CONDUCT 4508 03:21:43,880 --> 03:21:46,120 THEIR OWN CLINICAL TRIAL. 4509 03:21:46,120 --> 03:21:47,640 GO TO THE PHYSICIAN. 4510 03:21:47,640 --> 03:21:50,760 GET A SERUM CK MEASUREMENT. 4511 03:21:50,760 --> 03:21:52,720 START A DIETARY SUPPLEMENT. 4512 03:21:52,720 --> 03:21:55,320 SO YOU HAD MENTIONED A PATIENT 4513 03:21:55,320 --> 03:21:58,000 PORTAL IN -- WHITE BE THAT SORT 4514 03:21:58,000 --> 03:21:59,280 OF THING? 4515 03:21:59,280 --> 03:22:01,480 IS THERE SOME OTHER WAY TO 4516 03:22:01,480 --> 03:22:04,600 ACCESS THAT INFORMATION THAT IS 4517 03:22:04,600 --> 03:22:06,040 BEING SHARED WITHIN THE 4518 03:22:06,040 --> 03:22:07,640 COMMUNITY THROUGH SOCIAL MEDIA. 4519 03:22:07,640 --> 03:22:09,360 >> THERE ARE A COUPLE OF 4520 03:22:09,360 --> 03:22:09,880 OPTIONS. 4521 03:22:09,880 --> 03:22:11,440 WE THOUGHT ABOUT TRYING TO GET 4522 03:22:11,440 --> 03:22:13,480 IT FROM SOCIAL MEDIA. 4523 03:22:13,480 --> 03:22:15,680 IT'S HARD TO HAVE THE 4524 03:22:15,680 --> 03:22:17,160 INFORMATION BE STANDARDIZED IN 4525 03:22:17,160 --> 03:22:20,440 THAT PROCESS SO I THINK THERE IS 4526 03:22:20,440 --> 03:22:22,520 SOMETHING TO BE SAID FOR 4527 03:22:22,520 --> 03:22:24,640 PATIENTS REPORTING IT THROUGH 4528 03:22:24,640 --> 03:22:26,000 ITS STANDARD PLATFORM. 4529 03:22:26,000 --> 03:22:31,280 AS LONG AS IT'S SORT OF REALLY 4530 03:22:31,280 --> 03:22:33,600 MINIMALLY INVASIVE AND IS NOT 4531 03:22:33,600 --> 03:22:35,440 VERY TIM TIME-CONSUMING. 4532 03:22:35,440 --> 03:22:37,560 THE ALTERNATIVE OPTION THAT 4533 03:22:37,560 --> 03:22:39,000 WE'VE THOUGHT ABOUT AND THIS IS 4534 03:22:39,000 --> 03:22:40,800 ALSO LIKE I SAID BECAUSE I THINK 4535 03:22:40,800 --> 03:22:45,000 IN SOME CASES THERE IS A LOT OF 4536 03:22:45,000 --> 03:22:47,520 INFORMATION THAT ALL OF THE 4537 03:22:47,520 --> 03:22:48,840 PATIENTS HOLD SO, IT'S IMPORTANT 4538 03:22:48,840 --> 03:22:50,280 TO HEAR DIRECTLY FROM THEM AND 4539 03:22:50,280 --> 03:22:52,760 IN MANY CASES I THINK PATIENTS 4540 03:22:52,760 --> 03:22:54,520 VERY MUCH WANT TO SHARE WHAT HAS 4541 03:22:54,520 --> 03:22:56,160 WORKED AND NOT WORKED 4542 03:22:56,160 --> 03:22:58,840 PARTICULARLY WITH NIH OR FDA OR 4543 03:22:58,840 --> 03:23:03,480 OTHER BODIES THAT THEY SEE WHERE 4544 03:23:03,480 --> 03:23:06,120 IT WOULD BE GOOD TO SEE 4545 03:23:06,120 --> 03:23:06,600 RESPONSIVENESS. 4546 03:23:06,600 --> 03:23:12,280 I THINK THE OTHER OPPORTUNITY IS 4547 03:23:12,280 --> 03:23:16,080 TO IN SOME CASES IS TO GET THE 4548 03:23:16,080 --> 03:23:18,200 INFORMATION FROM EHRs BUT 4549 03:23:18,200 --> 03:23:20,480 RATHER THAN PARTNER WITH THE 4550 03:23:20,480 --> 03:23:21,600 INSTITUTIONS IT'S PARTNER WITH 4551 03:23:21,600 --> 03:23:24,120 THE PATIENTS TO GET IT SO WHAT 4552 03:23:24,120 --> 03:23:26,120 DAVID'S GROUP DOES FOR EXAMPLE 4553 03:23:26,120 --> 03:23:28,560 IS THAT PATIENTS FOR THEIR 4554 03:23:28,560 --> 03:23:32,200 REGISTRY PATIENTS SIGN A CONCEPT 4555 03:23:32,200 --> 03:23:34,080 TO PROVIDE ACCESS TO THEIR 4556 03:23:34,080 --> 03:23:34,480 MEDICAL RECORDS. 4557 03:23:34,480 --> 03:23:38,160 WHICH THE TEAM THEN GOES AND 4558 03:23:38,160 --> 03:23:40,720 PROCURES THE MEDICAL RECORDS AND 4559 03:23:40,720 --> 03:23:42,200 EXTRACTS THE PERTINENT 4560 03:23:42,200 --> 03:23:45,200 INFORMATION INTO THEIR REGISTRY. 4561 03:23:45,200 --> 03:23:48,000 THIS IS AN MANUALLY INTENSIVE 4562 03:23:48,000 --> 03:23:50,720 PROCESS AND HUMAN RESOURCE 4563 03:23:50,720 --> 03:23:52,080 INTENSIVE BUT IF YOU WANT TO GET 4564 03:23:52,080 --> 03:23:53,640 A LOT OF DETAILED INFORMATION 4565 03:23:53,640 --> 03:23:55,120 AND THINGS LIKE LABORATORY 4566 03:23:55,120 --> 03:23:57,880 VALUES OR IMAGING RESULTS THEN 4567 03:23:57,880 --> 03:24:00,200 IT PROVIDES A POTENTIAL 4568 03:24:00,200 --> 03:24:01,640 OPPORTUNITY WHERE IT'S STILL THE 4569 03:24:01,640 --> 03:24:04,640 PATIENT IS VERY MUCH IN CONTROL 4570 03:24:04,640 --> 03:24:06,760 AND IS CONCEPTING TO THE 4571 03:24:06,760 --> 03:24:08,080 PROVISION OF THEIR INFORMATION 4572 03:24:08,080 --> 03:24:17,280 AND HOW IT WILL BE USED. 4573 03:24:17,280 --> 03:24:20,240 >> WE HAVE A LITTLE BIT MORE 4574 03:24:20,240 --> 03:24:21,760 TIME IF ANYBODY WANTS TO MAKE 4575 03:24:21,760 --> 03:24:23,560 ANY ADDITIONAL COMMENTS ON THIS 4576 03:24:23,560 --> 03:24:25,760 WHOLE DRUG REPURPOSING SECTION. 4577 03:24:25,760 --> 03:24:27,400 NOT JUST ON HEATHER'S 4578 03:24:27,400 --> 03:24:28,520 PRESENTATION BUT ANY OF THE 4579 03:24:28,520 --> 03:24:30,960 PRESENTATIONS THAT WE HEARD IF 4580 03:24:30,960 --> 03:24:32,760 ANYBODY WOULD LIKE TO MAKE 4581 03:24:32,760 --> 03:24:40,000 COMMENTS? 4582 03:24:40,000 --> 03:24:41,000 >> BARBARA, I HAVE A QUESTION. 4583 03:24:41,000 --> 03:24:43,200 YOU GUYS HAVE DONE A FANTASTIC 4584 03:24:43,200 --> 03:24:45,880 JOB WITH REPURPOSING THE 4585 03:24:45,880 --> 03:24:46,880 APPROVED DRUGS. 4586 03:24:46,880 --> 03:24:50,280 WHAT IS YOUR COST RANGE FOR 4587 03:24:50,280 --> 03:24:52,560 SUPPORTING THESE STUDIES? 4588 03:24:52,560 --> 03:24:57,280 >> SO OUR FUNDING IS AS LITTLE 4589 03:24:57,280 --> 03:25:00,480 AS $50,000. 4590 03:25:00,480 --> 03:25:02,880 WE WITH A FUNDING PARTNER THAT 4591 03:25:02,880 --> 03:25:06,640 IS WILLING TO FUND IT CERTAINLY 4592 03:25:06,640 --> 03:25:12,520 HAVAND HAVE GONE HIGHER IN THE 4593 03:25:12,520 --> 03:25:13,480 $250,000 RANGE. 4594 03:25:13,480 --> 03:25:15,040 SOMETIMES THESE PROJECTS ARE 4595 03:25:15,040 --> 03:25:16,320 JUST $50,000. 4596 03:25:16,320 --> 03:25:18,440 AGAIN IT'S REPURPOSING. 4597 03:25:18,440 --> 03:25:19,960 5-10 PATIENTS. 4598 03:25:19,960 --> 03:25:22,520 IT'S A GENERIC DRUG. 4599 03:25:22,520 --> 03:25:25,760 OTHER TIMES THE PROJECTS THAT WE 4600 03:25:25,760 --> 03:25:30,480 FUND -- IT MIGHT BE LARGER 4601 03:25:30,480 --> 03:25:35,280 ONGOING STUDY BUT WITH $50,000 4602 03:25:35,280 --> 03:25:38,160 OR $100,000 MORE THEY CAN HIRE A 4603 03:25:38,160 --> 03:25:39,840 DEDICATED CLINICAL ENROLLMENT 4604 03:25:39,840 --> 03:25:40,440 PERSON. 4605 03:25:40,440 --> 03:25:43,600 THEY CAN OPEN UP ANOTHER TRIAL 4606 03:25:43,600 --> 03:25:45,160 SITE TO SPEED ENROLLMENT. 4607 03:25:45,160 --> 03:25:48,440 THEY CAN DO ADDITIONAL BIOMARKER 4608 03:25:48,440 --> 03:25:50,400 STUDY THAT WILL MAKE THE DATA 4609 03:25:50,400 --> 03:25:52,520 MORE CLEAR AND HIGHER LIKELY TO 4610 03:25:52,520 --> 03:25:54,440 GO ON TO THE NEXT STAGE SO WE'RE 4611 03:25:54,440 --> 03:25:57,320 WILLING TO FUND ADDITIONAL AN 4612 03:25:57,320 --> 03:25:58,640 ONGOING TRIAL OR FUNDS THAT ARE 4613 03:25:58,640 --> 03:26:02,400 ALREADY AROUND AND ONGOING THAT 4614 03:26:02,400 --> 03:26:04,760 WILL STRENGTHEN THE TRIAL AND 4615 03:26:04,760 --> 03:26:08,280 SPEED ENROLLMENT OR BE MORE 4616 03:26:08,280 --> 03:26:10,080 COMPELLING FROM A DATA 4617 03:26:10,080 --> 03:26:12,280 STANDPOINT. 4618 03:26:12,280 --> 03:26:13,960 WE ARE NOT GOING TO BE FIRST 4619 03:26:13,960 --> 03:26:16,160 MONEY IN THAT HAS TO RAISE 4620 03:26:16,160 --> 03:26:16,720 ANOTHER MILLION. 4621 03:26:16,720 --> 03:26:19,960 BUT WE'LL ADD TO ANOTHER STUDY 4622 03:26:19,960 --> 03:26:22,080 BUT A LOT OF OUR STUDIES ARE 4623 03:26:22,080 --> 03:26:24,480 STAN ALONE BECAUSE THEY ARE 4624 03:26:24,480 --> 03:26:27,120 SMALL, PROOF OF CONCEPT SO IT'S 4625 03:26:27,120 --> 03:26:30,520 A MIX OF BOTH. 4626 03:26:30,520 --> 03:26:32,200 DOES THAT ANSWER YOUR QUESTION. 4627 03:26:32,200 --> 03:26:32,480 >> YEAH. 4628 03:26:32,480 --> 03:26:33,200 THAT IS GREAT. 4629 03:26:33,200 --> 03:26:43,880 THANK YOU. 4630 03:26:43,880 --> 03:26:49,640 >> JUST A COMMENT. 4631 03:26:49,640 --> 03:26:51,000 CURRENTLY THERE IS AN ONGOING 4632 03:26:51,000 --> 03:26:52,840 ONE OF THE LARGEST ONGOING 4633 03:26:52,840 --> 03:27:12,040 TRIALS -- THAT -- -- TO SEE WHAT 4634 03:27:12,040 --> 03:27:20,880 HAPPENS -- AS OTHER -- COME INTO 4635 03:27:20,880 --> 03:27:23,560 FOCUS THE -- WHAT HAPPENS TO 4636 03:27:23,560 --> 03:27:26,040 THEM? 4637 03:27:26,040 --> 03:27:28,160 VERY INTERESTING CASE STUDY -- 4638 03:27:28,160 --> 03:27:33,920 AND LOOKING AT THE DRUG THAT WAS 4639 03:27:33,920 --> 03:27:49,640 CHOSEN -- AND THEN YOU'VE GOT -- 4640 03:27:49,640 --> 03:27:52,480 A GAZILLION DOWNSTREAM THAT YOU 4641 03:27:52,480 --> 03:27:53,560 CAN TARGET. 4642 03:27:53,560 --> 03:28:03,640 -- -- -- I WONDER WHAT THE 4643 03:28:03,640 --> 03:28:12,360 APPLICATION CAN BE LONG-TERM -- 4644 03:28:12,360 --> 03:28:17,320 THE DRUG GOES INTO PHASE III AND 4645 03:28:17,320 --> 03:28:19,360 THAT IS A CONCERN. 4646 03:28:19,360 --> 03:28:20,720 IT'S JUST WHAT I'M THINKING 4647 03:28:20,720 --> 03:28:26,840 ABOUT. 4648 03:28:26,840 --> 03:28:28,320 THAT'S ALL I HAVE TO SAY. 4649 03:28:28,320 --> 03:28:43,880 THANKS. 4650 03:28:43,880 --> 03:28:45,800 >> ANY OTHER COMMENTS? 4651 03:28:45,800 --> 03:28:46,800 OTHERWISE WE'LL BRING THE 4652 03:28:46,800 --> 03:28:48,480 DISCUSSION TO AN END AND THEN 4653 03:28:48,480 --> 03:28:51,000 I'LL MAKE SOME BRIEF CLOSING 4654 03:28:51,000 --> 03:28:51,240 REMARKS. 4655 03:28:51,240 --> 03:28:53,640 I KNOW ASKING PEOPLE TO SIT FOR 4656 03:28:53,640 --> 03:28:54,960 FOUR HOURS IS A CHALLENGE. 4657 03:28:54,960 --> 03:28:58,080 I DON'T THINK I CAN SIT FOR MORE 4658 03:28:58,080 --> 03:28:59,160 THAN FOUR HOURS. 4659 03:28:59,160 --> 03:29:01,240 WE HEARD TWO DIFFERENT BROAD 4660 03:29:01,240 --> 03:29:02,640 THEMES TODAY. 4661 03:29:02,640 --> 03:29:05,280 WE HEARD SOME REALLY INTERESTING 4662 03:29:05,280 --> 03:29:07,240 AND IMPORTANT TALKS FROM OUR NEW 4663 03:29:07,240 --> 03:29:11,640 MEMBERS WHO TOLD US ABOUT EITHER 4664 03:29:11,640 --> 03:29:14,640 THEIR LIVED EXPERIENCES AND/OR 4665 03:29:14,640 --> 03:29:18,000 THEIR PROFESSIONAL AFFILIATIONS 4666 03:29:18,000 --> 03:29:19,880 AND HOW THEIR EXPERIENCES OR 4667 03:29:19,880 --> 03:29:21,880 FAMILY EXPERIENCES GUIDED THEM 4668 03:29:21,880 --> 03:29:23,320 TO WHERE THEY ARE TODAY. 4669 03:29:23,320 --> 03:29:26,280 AND AT THE END OF THE DAY THAT 4670 03:29:26,280 --> 03:29:28,640 IS WHAT IS ALWAYS MEMORABLE FOR 4671 03:29:28,640 --> 03:29:29,120 ME. 4672 03:29:29,120 --> 03:29:31,080 I THINK AS A CLINICIAN. 4673 03:29:31,080 --> 03:29:33,760 BUT IMPORTANTLY BIG PICTURE WE 4674 03:29:33,760 --> 03:29:37,360 HEARD FOUR OUTSTANDING TALKS 4675 03:29:37,360 --> 03:29:40,400 ABOUT HOW TO REPURPOSE DRUGS AND 4676 03:29:40,400 --> 03:29:42,600 IT'S SOMETHING THAT I'M VERY 4677 03:29:42,600 --> 03:29:47,400 INTERESTED IN AS WELL FOR MY OWN 4678 03:29:47,400 --> 03:29:50,280 LABORATORY RESEARCH ON REBURNING 4679 03:29:50,280 --> 03:29:52,640 EXISTING THERAPIES IN DOWN 4680 03:29:52,640 --> 03:29:53,520 SYNDROME. 4681 03:29:53,520 --> 03:29:55,760 A LOT OF OF THE APPROACHES THAT 4682 03:29:55,760 --> 03:29:58,480 WE HEARD ARE ALSO VERY RELEVANT 4683 03:29:58,480 --> 03:30:01,000 IN OTHER FIELDS. 4684 03:30:01,000 --> 03:30:05,440 I THINK THAT ESPECIALLY HEARING 4685 03:30:05,440 --> 03:30:06,760 BARBARA'S PRESENTATION FROM 4686 03:30:06,760 --> 03:30:08,760 CURES WITHIN REACH IT HIGHLIGHTS 4687 03:30:08,760 --> 03:30:13,520 THE IMPORTANCE OF GETTING 4688 03:30:13,520 --> 03:30:14,640 FUNDING FOR PRELIMINARY DATA. 4689 03:30:14,640 --> 03:30:16,880 THAT IS THE HARDEST THING FOR 4690 03:30:16,880 --> 03:30:18,080 ANYBODY AND IT'S REALLY 4691 03:30:18,080 --> 03:30:20,400 DIFFICULT TO GET MAJOR NIH 4692 03:30:20,400 --> 03:30:23,600 FUNDING UNLESS YOU HAVE A WAY OF 4693 03:30:23,600 --> 03:30:25,080 DEMONSTRATING THE CORE PRINCIPLE 4694 03:30:25,080 --> 03:30:26,640 AND SO ARE WE IN INDUSTRY AS 4695 03:30:26,640 --> 03:30:29,120 WELL. 4696 03:30:29,120 --> 03:30:31,880 SO THANK YOU BARBARA FOR YOUR 4697 03:30:31,880 --> 03:30:35,480 COMMITMENT TO THAT. 4698 03:30:35,480 --> 03:30:37,160 BEFORE WE CLOSE WE DID WANT TO 4699 03:30:37,160 --> 03:30:39,360 BRING UP TWO ISSUES. 4700 03:30:39,360 --> 03:30:42,480 ONE IS SUGGESTIONS FOR FUTURE 4701 03:30:42,480 --> 03:30:43,080 TOPICS. 4702 03:30:43,080 --> 03:30:45,760 DAN PEREZ IS VERY EXCITED ABOUT 4703 03:30:45,760 --> 03:30:47,240 THE COMPLETION OF THE SEQUENCING 4704 03:30:47,240 --> 03:30:50,080 OF THE HUMAN GENOME AND THE FACT 4705 03:30:50,080 --> 03:30:53,400 THAT THEY USE FHSD AS AN EXAMPLE 4706 03:30:53,400 --> 03:30:55,840 AND WE'RE LUCKY THAT ONE OF THE 4707 03:30:55,840 --> 03:31:00,280 LEAD AUTHORS OF THAT STUDY IS 4708 03:31:00,280 --> 03:31:03,080 ADAM FILL PEE AND WE'RE GOING TO 4709 03:31:03,080 --> 03:31:07,360 ASK HIM TO SEE IF HE CAN * SPEAK 4710 03:31:07,360 --> 03:31:08,920 AT OUR NEXT MEETING. 4711 03:31:08,920 --> 03:31:11,240 THIS IS AN AMAZING COLLABORATION 4712 03:31:11,240 --> 03:31:15,000 BECAUSE ALL OF THE PEOPLE WHO 4713 03:31:15,000 --> 03:31:15,600 CONTRIBUTED TO THE SEQUENCING 4714 03:31:15,600 --> 03:31:17,360 HAD NEVER MET EACH OTHER IN 4715 03:31:17,360 --> 03:31:17,920 PERSON BEFORE. 4716 03:31:17,920 --> 03:31:21,600 SO THIS WAS MAJOR SEQUENCING 4717 03:31:21,600 --> 03:31:22,960 THAT WAS DONE DURING THE 4718 03:31:22,960 --> 03:31:25,640 PANDEMIC AT A TIME WHEN 4719 03:31:25,640 --> 03:31:28,640 EVERYBODY WAS WORKING VIRTUALLY 4720 03:31:28,640 --> 03:31:31,640 AND ARE IN ISOLATION IN THEIR 4721 03:31:31,640 --> 03:31:33,080 LABORATORIES SO SOME GOOD THINGS 4722 03:31:33,080 --> 03:31:34,720 CAME OUT OF THE PAST TWO YEARS. 4723 03:31:34,720 --> 03:31:37,840 SO WE HAVE THAT ON DECKING THEN 4724 03:31:37,840 --> 03:31:39,800 FOR THE NEXT MEETING. 4725 03:31:39,800 --> 03:31:43,520 WE DID HAVE A SUGGESTION FOR A 4726 03:31:43,520 --> 03:31:44,760 FUTURE TOPIC. 4727 03:31:44,760 --> 03:31:49,720 CAN WE GET BACK TO THAT? 4728 03:31:49,720 --> 03:31:51,320 I'M FORGETTING WHO MENTIONED 4729 03:31:51,320 --> 03:31:52,200 THAT. 4730 03:31:52,200 --> 03:31:55,360 WAS IT YOU GUSTAVO 4731 03:31:55,360 --> 03:31:57,240 >> I THINK IT WAS DAN'S 4732 03:31:57,240 --> 03:32:00,000 SUGGESTION THAT WE LOOK MORE AT 4733 03:32:00,000 --> 03:32:01,920 NIH FUNDING LEVELS. 4734 03:32:01,920 --> 03:32:02,360 7 4735 03:32:02,360 --> 03:32:03,280 >> NO. 4736 03:32:03,280 --> 03:32:04,360 THERE WAS SOMETHING EVEN BEFORE 4737 03:32:04,360 --> 03:32:10,120 THAT. 4738 03:32:10,120 --> 03:32:10,560 >> I 4739 03:32:10,560 --> 03:32:12,480 >> IT WAS A A PREVIOUS 4740 03:32:12,480 --> 03:32:14,360 SUGGESTION I DON'T REMEMBER WHO 4741 03:32:14,360 --> 03:32:16,280 IT WAS FROM THAT WE WERE 4742 03:32:16,280 --> 03:32:17,480 CONSIDERING FOR THE OCTOBER 4743 03:32:17,480 --> 03:32:18,120 MEETING. 4744 03:32:18,120 --> 03:32:19,640 THE NEXT MEETING IS 4745 03:32:19,640 --> 03:32:21,880 OCTOBER 3rd BY THE WAY. 4746 03:32:21,880 --> 03:32:25,960 AND CANDIDATE TOPIC IS NOVEL 4747 03:32:25,960 --> 03:32:27,920 FUNCTIONAL OUTCOME MEASURES SO 4748 03:32:27,920 --> 03:32:30,880 THIS COULD INCLUDE THINGS LIKE 4749 03:32:30,880 --> 03:32:33,360 VIDEO MEASUREMENTS OF REARABLE 4750 03:32:33,360 --> 03:32:36,360 WORK SPACE. 4751 03:32:36,360 --> 03:32:37,800 REMOTE DATA COLLECTION. 4752 03:32:37,800 --> 03:32:39,160 THERE IS SOME INTERESTING 4753 03:32:39,160 --> 03:32:42,280 STUDIES OF USING VIDEOS IN THE 4754 03:32:42,280 --> 03:32:45,040 HOME TO CAPTURE DATA OF PATIENT 4755 03:32:45,040 --> 03:32:45,600 MOVEMENT. 4756 03:32:45,600 --> 03:32:48,280 AND THEN HAVE THAT VIDEO 4757 03:32:48,280 --> 03:32:52,880 ANALYZED IN LAB FOR QUANTITATIVE 4758 03:32:52,880 --> 03:32:58,720 MEASURES OF FUNCTION. 4759 03:32:58,720 --> 03:32:59,960 >> I'M THINKING ABOUT THE 4760 03:32:59,960 --> 03:33:01,640 DIRECTOR OF THE NATIONAL LIBRARY 4761 03:33:01,640 --> 03:33:06,240 OF MEDICINE -- WHOSE RESEARCH IS 4762 03:33:06,240 --> 03:33:09,000 ON THE FUNCTIONAL HOME 4763 03:33:09,000 --> 03:33:11,560 ENVIRONMENT. 4764 03:33:11,560 --> 03:33:12,800 NOT SPECIFIC TO MUSCULAR 4765 03:33:12,800 --> 03:33:14,640 DYSTROPHY BUT SHE COMES FROM A 4766 03:33:14,640 --> 03:33:16,040 NEWSING AND ENGINEERING 4767 03:33:16,040 --> 03:33:16,640 BACKGROUND. 4768 03:33:16,640 --> 03:33:19,120 AND I BELIEVE SHE HAS SET UP A 4769 03:33:19,120 --> 03:33:21,640 VIRTUAL HOME IN THE CLINICAL 4770 03:33:21,640 --> 03:33:24,480 CENTER WHERE SHE CAN MONITOR 4771 03:33:24,480 --> 03:33:27,640 PEOPLE AND LOOKS AT HOW THEY 4772 03:33:27,640 --> 03:33:29,880 FUNCTION WITHIN THEIR HOME AND 4773 03:33:29,880 --> 03:33:31,800 WHETHER AND HOW THEY GET ACCESS 4774 03:33:31,800 --> 03:33:33,120 TO MEDICATIONS. 4775 03:33:33,120 --> 03:33:36,360 WHAT IS IN THE REFRIGERATOR ETC. 4776 03:33:36,360 --> 03:33:38,640 SO THAT MIGHT BE INTERESTING TO 4777 03:33:38,640 --> 03:33:42,400 SEE COULD THERE BE A CO-LAB TORI 4778 03:33:42,400 --> 03:33:44,840 -- WHERE SHE AND HER ASSOCIATES 4779 03:33:44,840 --> 03:33:46,560 WOULD TALK WITH US AND SEE 4780 03:33:46,560 --> 03:33:48,280 WHETHER IT'S RELEVANT TO 4781 03:33:48,280 --> 03:33:51,760 MUSCULAR DYSTROPHY. 4782 03:33:51,760 --> 03:33:53,560 >> AT THE MEETING THERE WAS A 4783 03:33:53,560 --> 03:33:55,480 DISCUSSION ABOUT THAT TYPE OF 4784 03:33:55,480 --> 03:34:00,760 POSSIBILITY FOR OUTCOME MEASURES 4785 03:34:00,760 --> 03:34:04,480 -- IN HOME VIDEO -- AND I WAS 4786 03:34:04,480 --> 03:34:10,440 REALLY IMPRESSED WITH THE PEOPLE 4787 03:34:10,440 --> 03:34:13,760 -- THE DECISION THAT THE PEOPLE 4788 03:34:13,760 --> 03:34:17,000 TOOK. 4789 03:34:17,000 --> 03:34:19,200 >> MAYBE YOU CAN SHARE WITH US 4790 03:34:19,200 --> 03:34:20,640 WHO WERE SOME OF THE SPEAKERS 4791 03:34:20,640 --> 03:34:21,960 AND WE CAN INVITE THEM TO 4792 03:34:21,960 --> 03:34:25,160 PRESENT. 4793 03:34:25,160 --> 03:34:26,920 >> THE THING THAT I WOULD LIKE 4794 03:34:26,920 --> 03:34:33,320 TO ADD TO OUTCOME MEASURES LIKE 4795 03:34:33,320 --> 03:34:38,480 GAIT ANALYSIS -- THE CONCEPTS -- 4796 03:34:38,480 --> 03:34:40,680 THAT -- THE DISEASE TODAY AND 4797 03:34:40,680 --> 03:34:43,680 THE DISEASE PROGRESSES FOR 4798 03:34:43,680 --> 03:34:47,640 ANOTHER FEW YEARS -- SO DO WE 4799 03:34:47,640 --> 03:34:52,680 NEED TO WAIT TWO YEARS BEFORE WE 4800 03:34:52,680 --> 03:34:57,040 KNOW IF THE DRUG IS NOT WORKING 4801 03:34:57,040 --> 03:35:00,040 -- INSTEAD OF WAITING FOR THE 4802 03:35:00,040 --> 03:35:03,800 TWO-YEAR CASCADE TO FINISH. 4803 03:35:03,800 --> 03:35:08,320 CAN WE TEASE OUT THE BIOMARKERS 4804 03:35:08,320 --> 03:35:10,840 OF DISEASE AND PROGRESS VERSUS 4805 03:35:10,840 --> 03:35:14,640 BIOMARKERS OF DISEASE -- THAT 4806 03:35:14,640 --> 03:35:18,000 HAS BEEN HALTED. 4807 03:35:18,000 --> 03:35:22,560 CAN WE -- -- AND WILL THAT HELP 4808 03:35:22,560 --> 03:35:25,000 US -- GET BETTER OUTCOME 4809 03:35:25,000 --> 03:35:27,640 MEASURES? 4810 03:35:27,640 --> 03:35:30,280 BECAUSE THERE MAY BE A LOT OF 4811 03:35:30,280 --> 03:35:32,680 DRUGS THAT ARE WORKING BUT YOU 4812 03:35:32,680 --> 03:35:34,040 DON'T KNOW FOR TWO YEARS. 4813 03:35:34,040 --> 03:35:45,480 7 IS THAT A LOT -- ARE UNDER 4814 03:35:45,480 --> 03:35:46,120 REACTING TO THAT 4815 03:35:46,120 --> 03:35:47,960 >> CAN I REPHRASE IT A LITTLE 4816 03:35:47,960 --> 03:35:48,120 BIT. 4817 03:35:48,120 --> 03:35:52,040 I THINK YOU'RE LOOKING FOR MAYBE 4818 03:35:52,040 --> 03:35:56,920 -- A BIOMARKER? 4819 03:35:56,920 --> 03:36:02,920 >> THE DRUGS -- TODAY ON MAY 9, 4820 03:36:02,920 --> 03:36:04,280 2022, IT MAY TAKE ANOTHER TWO 4821 03:36:04,280 --> 03:36:07,280 YEARS FOR THE DISEASE PROCESS -- 4822 03:36:07,280 --> 03:36:09,640 WHAT IS ONGOING? 4823 03:36:09,640 --> 03:36:12,560 IT'S GOING TO GO. 4824 03:36:12,560 --> 03:36:16,080 BUT THE MUSCLE CELL NEXT TO IT 4825 03:36:16,080 --> 03:36:23,880 IS NOT AFFECTED -- SO DOVE AN 4826 03:36:23,880 --> 03:36:30,400 ISSUE OF HAVING TO SHUT DOWN THE 4827 03:36:30,400 --> 03:36:33,760 -- GENE SUCCESSION AND IT HAS A 4828 03:36:33,760 --> 03:36:37,840 CASCADE ALONG CASCADE -- SO ONCE 4829 03:36:37,840 --> 03:36:41,720 YOU SHUT IT DOWN YOU MAY NOT 4830 03:36:41,720 --> 03:36:46,160 KNOW -- I GUESS IT'S NOT 4831 03:36:46,160 --> 03:36:49,000 TRANSLATING -- AND THEN THERE IS 4832 03:36:49,000 --> 03:36:56,960 A WAY TO MEASURE -- -- ---TODAY 4833 03:36:56,960 --> 03:36:59,840 YOU WOULD HAVE AN A 10 SITUATION 4834 03:36:59,840 --> 03:37:04,240 -- * OF THE DISEASE EFFECT. 4835 03:37:04,240 --> 03:37:08,880 FOR ANOTHER TWO YEARS. 4836 03:37:08,880 --> 03:37:12,960 SO, ITS IMPORTANT TO MEASURE -- 4837 03:37:12,960 --> 03:37:15,600 TO KNOW WHETHER OR NOT YOU HAVE 4838 03:37:15,600 --> 03:37:21,320 GOT STOPPED THE DISEASE 4839 03:37:21,320 --> 03:37:24,160 PROGRESSION. 4840 03:37:24,160 --> 03:37:28,640 -- -- THE WORK SPACE -- SURVEYS. 4841 03:37:28,640 --> 03:37:32,360 HOW ARE WE TRYING TO -- THE NIH 4842 03:37:32,360 --> 03:37:33,640 -- THINKING ABOUT THESE KIND OF 4843 03:37:33,640 --> 03:37:34,440 THINGS. 4844 03:37:34,440 --> 03:37:38,040 HOW DO YOU TAKE THAT 4845 03:37:38,040 --> 03:37:43,640 CONSIDERATION -- APART -- TRY TO 4846 03:37:43,640 --> 03:37:49,360 UNDERSTAND IT. 4847 03:37:49,360 --> 03:37:50,680 DO YOU UNDERSTAND WHAT I'M 4848 03:37:50,680 --> 03:37:51,920 TRYING TO SAY? 4849 03:37:51,920 --> 03:37:53,480 >> I THINK SO. 4850 03:37:53,480 --> 03:37:55,520 AND LEE IF HE IS STILL ON YOU 4851 03:37:55,520 --> 03:37:56,640 MAY WANT TO COMMENT HERE. 4852 03:37:56,640 --> 03:38:00,240 I THINK THE MAGNETIC RESONANCE 4853 03:38:00,240 --> 03:38:02,880 IMAGING BIOMARKERS HAVE BEEN 4854 03:38:02,880 --> 03:38:07,320 QUITE EFFECTIVE IN PREDICTING 4855 03:38:07,320 --> 03:38:11,880 SUBSEQUENT PHYSICAL FUNCTIONING 4856 03:38:11,880 --> 03:38:13,640 OUTCOMES IN ORDER TO BE ABLE TO 4857 03:38:13,640 --> 03:38:15,640 HAVE BIOMARKERS THAT ARE 4858 03:38:15,640 --> 03:38:17,320 SENSITIVE TO CHANGE OVER SHORTER 4859 03:38:17,320 --> 03:38:19,840 PERIOD OF TIME AND FIT INTO 4860 03:38:19,840 --> 03:38:24,040 TRIALS BETTER THAN I THINK THE 4861 03:38:24,040 --> 03:38:26,280 MR BIOMARKERS MAY BE DOING A 4862 03:38:26,280 --> 03:38:33,920 GOOD JOB FOR THAT. 4863 03:38:33,920 --> 03:38:36,120 >> TO SHUT DOWN THE DISEASE 4864 03:38:36,120 --> 03:38:38,400 TODAY IT HAS A CASCADE AND IT 4865 03:38:38,400 --> 03:38:41,760 GOES ON AND ON. 4866 03:38:41,760 --> 03:38:45,400 AND SO HAS NOTHING TO DO WITH -- 4867 03:38:45,400 --> 03:38:47,280 THE MUSCLES ARE DYING. 4868 03:38:47,280 --> 03:38:51,680 THE MUSCLES ARE TURN INTO FAT -- 4869 03:38:51,680 --> 03:38:56,880 AND THEN IT GOES. 4870 03:38:56,880 --> 03:38:59,080 >> BUT DAN IF YOU WERE TO SHUT 4871 03:38:59,080 --> 03:39:00,800 DOWN THE PROTECTION THEN YOU 4872 03:39:00,800 --> 03:39:02,760 WOULD ALLOW REGENERATION TO 4873 03:39:02,760 --> 03:39:04,640 START SO IT MAY NOT TAKE TWO 4874 03:39:04,640 --> 03:39:06,240 YEARS TO SEE THE IMPACT OF THE 4875 03:39:06,240 --> 03:39:07,560 DRUG BUT IT WOULD TAKE SOME 4876 03:39:07,560 --> 03:39:09,880 TIME. 4877 03:39:09,880 --> 03:39:12,520 >> I THINK WE'RE IN THE SPOT 4878 03:39:12,520 --> 03:39:14,640 WITH A LOT OF DISEASES SO WHAT 4879 03:39:14,640 --> 03:39:17,640 YOU'RE GETTING AT WHICH IS 4880 03:39:17,640 --> 03:39:19,600 ABSOLUTELY TRUE IS WHAT STARTS 4881 03:39:19,600 --> 03:39:21,480 OFF THE DISEASE MAY DRIVE IT FOR 4882 03:39:21,480 --> 03:39:23,400 A WHILE BUT THEN OTHER DRIVERS 4883 03:39:23,400 --> 03:39:26,920 THAT COME ON IN LATER STAGES. 4884 03:39:26,920 --> 03:39:28,920 ALZHEIMER'S IS THE GREAT EXAMPLE 4885 03:39:28,920 --> 03:39:33,880 NOW WHERE AMYLOID WAS THOUGHT TO 4886 03:39:33,880 --> 03:39:36,440 BE THE MAJOR DRIVER AND NOW WE 4887 03:39:36,440 --> 03:39:39,440 CAN TAKE IT OUT AND NOT SEEING A 4888 03:39:39,440 --> 03:39:41,280 BIG CLINICAL EFFECT. 4889 03:39:41,280 --> 03:39:44,320 THE THOUGHT WAS THAT IS TOO LATE 4890 03:39:44,320 --> 03:39:45,760 AND SOMETHING ELSE CAME ON IN 4891 03:39:45,760 --> 03:39:46,520 THE MEANTIME. 4892 03:39:46,520 --> 03:39:48,000 THE SAME DISEASE. 4893 03:39:48,000 --> 03:39:50,080 IT'S JUST THAT IT'S DIFFERENT 4894 03:39:50,080 --> 03:39:52,840 DRIVERS AND DIFFERENT STAGES OF 4895 03:39:52,840 --> 03:39:53,920 THE DISEASE. 4896 03:39:53,920 --> 03:39:55,720 SO, THAT IS WHAT YOU HAVE TO TRY 4897 03:39:55,720 --> 03:39:57,200 TO UNDERSTAND. 4898 03:39:57,200 --> 03:39:58,960 NOT EASY. 4899 03:39:58,960 --> 03:40:01,280 CERTAINLY IN ALZHEIMER'S. 4900 03:40:01,280 --> 03:40:03,480 THE COMPANY SPENT BILLIONS -- 4901 03:40:03,480 --> 03:40:05,640 BEFORE FIGURING THAT OUT. 4902 03:40:05,640 --> 03:40:09,640 >> I WOULD LIKE TO UNDERSTAND 4903 03:40:09,640 --> 03:40:14,400 BEFORE THE NEXT MEETING -- TO 4904 03:40:14,400 --> 03:40:19,240 UNDERSTAND HOW WE DO THIS T. IS 4905 03:40:19,240 --> 03:40:20,480 THIS OF INTEREST TO ANYONE ON 4906 03:40:20,480 --> 03:40:21,120 THE COMMITTEE? 4907 03:40:21,120 --> 03:40:24,480 >> I HAVE A QUESTION FOR YOU -- 4908 03:40:24,480 --> 03:40:28,960 COULD YOU TAKE A SERIES OF MRI'S 4909 03:40:28,960 --> 03:40:31,080 FROM PEOPLE AT VARIOUS STAGES OF 4910 03:40:31,080 --> 03:40:33,080 DISEASE AND COME UP WITH 4911 03:40:33,080 --> 03:40:35,160 OBJECTIVE MEASURES AND USE THOSE 4912 03:40:35,160 --> 03:40:37,800 AS A BASELINE AGAINST WHICH YOU 4913 03:40:37,800 --> 03:40:40,880 COULD MEASURE RESPONSE TO 4914 03:40:40,880 --> 03:40:41,520 THERAPY? 4915 03:40:41,520 --> 03:40:44,320 IN PEOPLE WHO ARE BEING TREATED 4916 03:40:44,320 --> 03:40:49,600 OR ARE THE -- IS THE DAMAGE -- 4917 03:40:49,600 --> 03:40:51,600 INDIVIDUAL TO EACH PERSON THAT E 4918 03:40:51,600 --> 03:40:57,160 YOU COULD NOT TAKE THAT APPROACH 4919 03:40:57,160 --> 03:40:58,480 >> OBVIOUSLY FROM THE GRAPH I 4920 03:40:58,480 --> 03:41:01,520 SHOWED YOU THE VERGE ANNES IS 4921 03:41:01,520 --> 03:41:03,120 PRETTY GREAT PATIENT TO PATIENT 4922 03:41:03,120 --> 03:41:04,640 BUT STILL IF YOU'VE BEEN 4923 03:41:04,640 --> 03:41:06,080 FOLLOWING A PATIENT AND THAT IS 4924 03:41:06,080 --> 03:41:08,880 WHAT WE TRY TO DO IN OUR STUDIES 4925 03:41:08,880 --> 03:41:11,040 IS FOLLOW THEM AT BASELINE 4926 03:41:11,040 --> 03:41:13,800 BEFORE YOU TREAT THEM SO YOU 4927 03:41:13,800 --> 03:41:20,440 UNDERSTAND THEIR PERSONNEL 4928 03:41:20,440 --> 03:41:23,000 PERSONAL TRAJECTORY AND I SEE 4929 03:41:23,000 --> 03:41:26,720 DAN'S POINT AND WE SEE IT IN OUR 4930 03:41:26,720 --> 03:41:28,200 DUCHENNE MOUSE MODELS WITH 4931 03:41:28,200 --> 03:41:31,080 TRYING TO DO GENE THERAPY AT 4932 03:41:31,080 --> 03:41:34,360 LATER STAGES OF DISEASES. 4933 03:41:34,360 --> 03:41:37,200 IT'S BEEN SO PREDOMINANT AT THAT 4934 03:41:37,200 --> 03:41:38,760 POINT THAT EVEN THOUGH YOU PUT 4935 03:41:38,760 --> 03:41:40,320 BACK WHAT IS MISSING THE MUSCLES 4936 03:41:40,320 --> 03:41:42,440 ARE NOT CORRECTING AND SO YOU DO 4937 03:41:42,440 --> 03:41:46,600 HAVE TO THEN GO DOWN AND TWEAK 4938 03:41:46,600 --> 03:41:48,920 THE PATH WAYS THAT HAVE BEEN 7 4939 03:41:48,920 --> 03:41:51,160 DISRUPTED BY WHATEVER THE EVENT 4940 03:41:51,160 --> 03:41:52,600 WAS. 4941 03:41:52,600 --> 03:41:57,040 SO IT MAY BE THAT TURNING OFF 4942 03:41:57,040 --> 03:41:58,080 DUCHENNE 4 IS NOT SUFFICIENT. 4943 03:41:58,080 --> 03:42:00,560 YOU HAVE TO DO THAT FIRST BUT 4944 03:42:00,560 --> 03:42:04,000 THEN DO THINGS THAT WILL RESET 4945 03:42:04,000 --> 03:42:07,400 THE NORMAL EQUILIBRIUM AS A 4946 03:42:07,400 --> 03:42:08,040 FOLLOW-UP TO THAT. 4947 03:42:08,040 --> 03:42:10,480 I DO GET YOUR POINT NOW THAT I 4948 03:42:10,480 --> 03:42:16,200 THINK BY THE A LITTLE BIT. 4949 03:42:16,200 --> 03:42:17,280 >> THANK YOU FOR THAT 4950 03:42:17,280 --> 03:42:20,280 SUGGESTION. 4951 03:42:20,280 --> 03:42:25,040 THE REMAINING FEW MINUTES ONE 4952 03:42:25,040 --> 03:42:28,040 THING THAT WE WANTED TO GET 4953 03:42:28,040 --> 03:42:30,600 FEEDBACK ON IS ARE WE READY FOR 4954 03:42:30,600 --> 03:42:31,280 INPERSON MEETINGS. 4955 03:42:31,280 --> 03:42:34,160 DO WE WANT TO CONTINUE WITH THE 4956 03:42:34,160 --> 03:42:35,280 VIRTUAL FORMAT. 4957 03:42:35,280 --> 03:42:38,960 A HYBRID FORMAT? 4958 03:42:38,960 --> 03:42:40,640 WE'RE OPEN FOR FEEDBACK. 4959 03:42:40,640 --> 03:42:43,640 I KNOW THAT AT THE DIRECTOR'S 4960 03:42:43,640 --> 03:42:50,360 LEVEL -- THE HYBRID APPROACH HAS 4961 03:42:50,360 --> 03:42:51,240 BEEN WORKING VERY WELL. 4962 03:42:51,240 --> 03:42:53,480 WE HAVE THE TECHNOLOGY AND AT 4963 03:42:53,480 --> 03:42:56,000 ANY GIVEN TIME A THIRD TO A HALF 4964 03:42:56,000 --> 03:42:59,440 OF INSTITUTE AND CENTER 4965 03:42:59,440 --> 03:43:00,480 DIRECTORS ARE IN THE ROOM AND 4966 03:43:00,480 --> 03:43:01,880 THE REST OF THE PEOPLE ARE ON 4967 03:43:01,880 --> 03:43:02,400 ZOOM. 4968 03:43:02,400 --> 03:43:04,080 7 AND THE PEOPLE WHO ARE IN THE 4969 03:43:04,080 --> 03:43:06,360 ROOM ARE USING THEIR LAPTOPS AND 4970 03:43:06,360 --> 03:43:10,640 IT IS EFFECTIVELY A LAPTOP 4971 03:43:10,640 --> 03:43:12,320 DRIVEN OR ZOOM DRIVEN MEETING 4972 03:43:12,320 --> 03:43:14,480 BUT THERE IS THE OPPORTUNITY TO 4973 03:43:14,480 --> 03:43:16,320 INTERACT OBVIOUSLY IN PERSON. 4974 03:43:16,320 --> 03:43:23,240 AS WELL. 4975 03:43:23,240 --> 03:43:23,480 COMMENTS? 4976 03:43:23,480 --> 03:43:25,360 A FEW PEOPLE ARE SMILING BUT I 4977 03:43:25,360 --> 03:43:27,040 DON'T KNOW WHAT THAT MEANS? 4978 03:43:27,040 --> 03:43:28,120 >> THERE WAS A SURVEY ABOUT 4979 03:43:28,120 --> 03:43:34,080 THAT. 4980 03:43:34,080 --> 03:43:37,040 I WOULD LIKE TO GO BACK TO 4981 03:43:37,040 --> 03:43:37,640 PERSON. 4982 03:43:37,640 --> 03:43:43,880 AND A HYBRID OPTION IS THE BEST. 4983 03:43:43,880 --> 03:43:45,640 >> GENE YOU CAME ON VIDEO. 4984 03:43:45,640 --> 03:43:47,080 DO YOU HAVE A COMMENT? 4985 03:43:47,080 --> 03:43:52,040 >> I JUST WANTED TO SMILE. 4986 03:43:52,040 --> 03:43:54,320 I THINK THE HYBRID COULD WORK 4987 03:43:54,320 --> 03:43:57,880 AND GIVE PEOPLE THE OPTION. 4988 03:43:57,880 --> 03:44:02,760 PERSONALLY I DON'T LOOK FORWARD 4989 03:44:02,760 --> 03:44:04,880 TO GOING FROM BALTIMORE TO 4990 03:44:04,880 --> 03:44:09,480 BETHESDA WITH A LOT OF 4991 03:44:09,480 --> 03:44:11,080 ENTHUSIASM BUT IT'S AN IMPORTANT 4992 03:44:11,080 --> 03:44:12,320 MEETING. 4993 03:44:12,320 --> 03:44:15,600 I KIND OF WANT TO SEE WHO'S 4994 03:44:15,600 --> 03:44:17,360 COMING WHICH IS NOT GOOD 4995 03:44:17,360 --> 03:44:18,400 ETIQUETTE. 4996 03:44:18,400 --> 03:44:20,120 BUT IF IT LOOKS LIKE WE WOULD 4997 03:44:20,120 --> 03:44:22,320 HAVE A GOOD TURN OUT I WOULD BE 4998 03:44:22,320 --> 03:44:28,640 GAME FOR DOING THAT. 4999 03:44:28,640 --> 03:44:33,080 >> ANY MORE COMMENTS? 5000 03:44:33,080 --> 03:44:35,280 AND PEOPLE COMING FROM FARTHER 5001 03:44:35,280 --> 03:44:37,040 AWAY. 5002 03:44:37,040 --> 03:44:39,920 LIKE ERIC COMING FROM FROM AND 5003 03:44:39,920 --> 03:44:42,520 DENISE COMING FROM IDAHO. 5004 03:44:42,520 --> 03:44:46,920 >> AND HERE I WAS WHINING. 5005 03:44:46,920 --> 03:44:48,600 >> MY CONCERN WOULD BE HOW WOULD 5006 03:44:48,600 --> 03:44:50,840 THE TAB GET PICKED UP FOR MY 5007 03:44:50,840 --> 03:44:54,040 TRAVEL COSTS BECAUSE I'M PRETTY 5008 03:44:54,040 --> 03:44:58,760 LIMITED IN EXTRA RESOURCES. 5009 03:44:58,760 --> 03:45:00,520 >> DENISE WE HAVE A BUDGET FOR 5010 03:45:00,520 --> 03:45:02,560 OUR COMMITTEE AND WE SUPPORT THE 5011 03:45:02,560 --> 03:45:04,560 TRAVEL FOR ALL OF OUR MEMBERS TO 5012 03:45:04,560 --> 03:45:06,360 THE MEETINGS. 5013 03:45:06,360 --> 03:45:08,440 >> EVEN FROM IDAHO? 5014 03:45:08,440 --> 03:45:11,880 >> EVEN FROM IDAHO! 5015 03:45:11,880 --> 03:45:14,120 >> IT'S JUST LIKE A PAIN IN THE 5016 03:45:14,120 --> 03:45:19,040 BUTT YOU DRIVE FOUR HOURS TO 5017 03:45:19,040 --> 03:45:22,040 SAVE MONEY TO FLY OUT OF SALT 5018 03:45:22,040 --> 03:45:22,480 LAKE. 5019 03:45:22,480 --> 03:45:24,280 I WOULD LOVE TO COME BACK TO THE 5020 03:45:24,280 --> 03:45:26,760 EAST COAST. 5021 03:45:26,760 --> 03:45:28,520 I'VE GOT FAMILY THAT I WOULD 5022 03:45:28,520 --> 03:45:30,640 LOVE TO HOOK UP WITH AGAIN AND 5023 03:45:30,640 --> 03:45:31,720 AN OLD FRIEND. 5024 03:45:31,720 --> 03:45:34,120 >> THERE YOU GO. 5025 03:45:34,120 --> 03:45:37,800 >> MANY OF US ARE OBVIOUSLY, 5026 03:45:37,800 --> 03:45:40,320 WE'VE HAD PLENTY OF ZOOM 5027 03:45:40,320 --> 03:45:42,800 MEETINGS TO FILL THE PAST COUPLE 5028 03:45:42,800 --> 03:45:45,560 OF YEARS SO, ITS A TREMENDOUS 5029 03:45:45,560 --> 03:45:47,240 VALUE TO MEET IN PERSON AND I 5030 03:45:47,240 --> 03:45:49,680 WOULD BE SUPPORTIVE OF THAT AND 5031 03:45:49,680 --> 03:45:52,840 IT'S A MATTER OF BALANCING ALL 5032 03:45:52,840 --> 03:45:55,800 OF THE COMMITMENTS. 5033 03:45:55,800 --> 03:45:59,640 I'M SUPPORTIVE OF IN PERSON 5034 03:45:59,640 --> 03:46:00,880 INTERACTIONS 5035 03:46:00,880 --> 03:46:02,600 >> NATALIE, DID YOUR VIDEO COME 5036 03:46:02,600 --> 03:46:03,080 ON? 5037 03:46:03,080 --> 03:46:08,120 >> I REALLY DO MISS THE IN 5038 03:46:08,120 --> 03:46:10,680 PERSON INTERACTION AND WOULD 5039 03:46:10,680 --> 03:46:12,360 VALUE ANOTHER IN PERSON MEETING 5040 03:46:12,360 --> 03:46:15,560 AND MAYBE HAVING AN AND I HYBRID 5041 03:46:15,560 --> 03:46:16,600 APPROACH BECAUSE NOT EVERYBODY 5042 03:46:16,600 --> 03:46:19,280 COULD MAKE IT BUT BEING IN 5043 03:46:19,280 --> 03:46:21,360 PERSON -- I THINK IT'S JUST 5044 03:46:21,360 --> 03:46:28,480 GREAT TO BE ABLE TO SEE PEOPLE. 5045 03:46:28,480 --> 03:46:30,280 >> DEFINITELY HEAR A MAJORITY 5046 03:46:30,280 --> 03:46:36,080 WOULD LIKE TO EVER AN IN PERSON 5047 03:46:36,080 --> 03:46:36,320 MEETING. 5048 03:46:36,320 --> 03:46:39,000 >> THE OCTOBER MEETING WE'RE 5049 03:46:39,000 --> 03:46:41,880 SCHEDULED FOR 1:00 TO 5:00 P.M. 5050 03:46:41,880 --> 03:46:43,240 SO IN PERSON MEETING WE WOULD 5051 03:46:43,240 --> 03:46:48,000 WANT TO GO BACK TO 8:5:00 OR 5052 03:46:48,000 --> 03:46:49,080 9:00 TO 5:00. 5053 03:46:49,080 --> 03:46:51,480 BUT PERHAPS WE COULD DO THE 5054 03:46:51,480 --> 03:46:53,240 OCTOBER MEETING HYBRID AND THEN 5055 03:46:53,240 --> 03:46:56,080 FOR THE SPRING MEETING OF 2023 5056 03:46:56,080 --> 03:46:58,880 ARRANGE IT FOR A FULL DAY AND IN 5057 03:46:58,880 --> 03:47:00,440 PERSON FOR WHOEVER CAN MAKE IT 5058 03:47:00,440 --> 03:47:03,080 AND HYBRID FOR THOSE WHO CANNOT. 5059 03:47:03,080 --> 03:47:06,200 DOES THAT SOUND GOOD? 5060 03:47:06,200 --> 03:47:07,880 >> THAT GIVES US TIME TO SEE 5061 03:47:07,880 --> 03:47:12,280 WHAT IS GOING ON WITH 5062 03:47:12,280 --> 03:47:14,800 CORONAVIRUS. 5063 03:47:14,800 --> 03:47:16,600 I THINK IT'S A WISE CHOICE. 5064 03:47:16,600 --> 03:47:19,640 I PERSONALLY WOULD LOVE TO BE 5065 03:47:19,640 --> 03:47:27,800 THERE IN PERSON -- -- IN TERMS 5066 03:47:27,800 --> 03:47:28,880 OF TRAVEL AND EQUIPMENT. 5067 03:47:28,880 --> 03:47:32,280 THERE IS A HAZARD FOR ME. 5068 03:47:32,280 --> 03:47:34,240 >> AND THAT IS THE WISDOM OF 5069 03:47:34,240 --> 03:47:36,800 HAVING THE HYBRID OPTION BECAUSE 5070 03:47:36,800 --> 03:47:38,520 IF WE PLAN THE MEETING SIX 5071 03:47:38,520 --> 03:47:41,280 MONTHS IN ADVANCE BUT MAYBE ONE 5072 03:47:41,280 --> 03:47:43,640 MONTH OR EVEN TWO WEEKS IN 5073 03:47:43,640 --> 03:47:45,640 ADVANCE OH, MY GOSH THE PANDEMIC 5074 03:47:45,640 --> 03:47:47,640 IS GETTING HOT ENOUGH THAT A LOT 5075 03:47:47,640 --> 03:47:51,160 OF PEOPLE WOULD BE UNCOMFORTABLE 5076 03:47:51,160 --> 03:47:53,160 FLYING OR WHAT HAVE YOU AND THEN 5077 03:47:53,160 --> 03:47:55,640 WE STILL HAVE THE VIDEO IN THE 5078 03:47:55,640 --> 03:47:58,640 BACK POCKET AND REFUNDABLE 5079 03:47:58,640 --> 03:48:01,680 TICKETS SINCE ALL THE GOVERNMENT 5080 03:48:01,680 --> 03:48:04,720 PURCHASED TICKETS ARE THAT WAY. 5081 03:48:04,720 --> 03:48:08,240 7 I THINK IT'S A GOOD WAY TO GO. 5082 03:48:08,240 --> 03:48:09,080 >> WALTER. 5083 03:48:09,080 --> 03:48:11,080 >> I WAS THINKING IF WE DO A 5084 03:48:11,080 --> 03:48:15,160 HYBRID IN TERMS OF THE TIMING 5085 03:48:15,160 --> 03:48:17,160 THE COUNCIL PUSH THE START TIME 5086 03:48:17,160 --> 03:48:20,920 TO LATER BECAUSE PEOPLE ON THE 5087 03:48:20,920 --> 03:48:25,800 WEST COAST TIME ZONES -- PEOPLE 5088 03:48:25,800 --> 03:48:28,880 I'M NOT SURE WHO'S FROM 5089 03:48:28,880 --> 03:48:31,800 CALIFORNIA. 5090 03:48:31,800 --> 03:48:34,240 BUT IF THERE ARE ARE MAYBE 5091 03:48:34,240 --> 03:48:35,360 STARTING LATER AND GOING A 5092 03:48:35,360 --> 03:48:37,080 LITTLE LATER 5093 03:48:37,080 --> 03:48:39,520 >> IF DENISE CHOOSES NOT TO COME 5094 03:48:39,520 --> 03:48:41,440 FOR WHATEVER REASON THAT SHE IS 5095 03:48:41,440 --> 03:48:43,120 IN THE WESTERN TIME ZONE. 5096 03:48:43,120 --> 03:48:44,320 SO I BELIEVE. 5097 03:48:44,320 --> 03:48:47,360 MAYBE SHE IS IN MOUNTAIN. 5098 03:48:47,360 --> 03:48:49,320 >> I BELIEVE MOUNTAIN. 5099 03:48:49,320 --> 03:48:50,440 >> STILL TWO HOURS. 5100 03:48:50,440 --> 03:48:53,600 >> I'M IN SAN DIEGO. 5101 03:48:53,600 --> 03:48:56,480 >> OBSERVES OKAY. 5102 03:48:56,480 --> 03:48:58,320 WELL LET'S GO OUT THERE. 5103 03:48:58,320 --> 03:49:01,680 >> WHY NOT? 5104 03:49:01,680 --> 03:49:03,400 >> THAT IS WHAT WE'VE DONE AS 5105 03:49:03,400 --> 03:49:03,920 WELL. 5106 03:49:03,920 --> 03:49:05,320 WE STARTED LATER WITH THE 5107 03:49:05,320 --> 03:49:10,400 HYBRID. 5108 03:49:10,400 --> 03:49:12,280 AND WE HAVE A COUNCILMEMBER 5109 03:49:12,280 --> 03:49:14,160 BASED IN THE UK SO WE'RE 5110 03:49:14,160 --> 03:49:17,640 COVERING A LOT OF TIME ZONES. 5111 03:49:17,640 --> 03:49:17,880 OKAY. 5112 03:49:17,880 --> 03:49:22,160 7 IF NO ONE ELSE HAS ANY OTHER 5113 03:49:22,160 --> 03:49:24,160 THINGS TO ADD I'M GOING TO HAND 5114 03:49:24,160 --> 03:49:26,920 THIS OVER TO GLEN TO CLOSE OUT 5115 03:49:26,920 --> 03:49:29,480 THIS VERY PRODUCTIVE MEETING. 5116 03:49:29,480 --> 03:49:31,080 SO THANK YOU ALL. 5117 03:49:31,080 --> 03:49:32,960 >> THANKS, DR. BIANCHI AND THANK 5118 03:49:32,960 --> 03:49:34,760 YOU TO ALL OF OUR PRESENTERS 5119 03:49:34,760 --> 03:49:37,080 TODAY FOR EXCELLENT 5120 03:49:37,080 --> 03:49:40,720 PRESENTATIONS AND EVERYTHING -- EVERYBODY 5121 03:49:40,720 --> 03:49:42,600 THAT ENGAGED. 5122 03:49:42,600 --> 03:49:44,160 AND THANK YOU FOR YOUR COMMENTS 5123 03:49:44,160 --> 03:49:46,280 ABOUT TOPIC MEETINGS AND FORMAT 5124 03:49:46,280 --> 03:49:47,360 OF MEETINGS. 5125 03:49:47,360 --> 03:49:49,720 WE'LL MAKE UP A PLAN AND LET YOU 5126 03:49:49,720 --> 03:49:50,200 KNOW. 5127 03:49:50,200 --> 03:49:51,960 SO AS I MENTIONED OUR NEXT 5128 03:49:51,960 --> 03:49:55,520 MEETING IS OCTOBER 3rd. 5129 03:49:55,520 --> 03:49:56,880 1:00 TO 5:00. 5130 03:49:56,880 --> 03:49:59,240 SOME SORT OF HYBRID FOR THAT. 5131 03:49:59,240 --> 03:50:01,000 PERHAPS LOCAL PEOPLE WILL WANT 5132 03:50:01,000 --> 03:50:02,880 TO TRAVEL TO BETHESDA AND 5133 03:50:02,880 --> 03:50:05,480 OTHERWISE GREAT MEETING. 5134 03:50:05,480 --> 03:50:08,080 THANKS TO EVERYBODY. 5135 03:50:08,080 --> 03:50:09,800 >> THANKS AND HAVE A HEALTHY 5136 03:50:09,800 --> 00:00:00,000 SUMMER WE HOPE.