1 00:00:05,920 --> 00:00:07,520 >>WELCOME, EVERYBODY, TO THE 2 00:00:07,520 --> 00:00:15,160 OCTOBER MEETING OF THE MUSCULAR 3 00:00:15,160 --> 00:00:15,920 DYSTROPHY COORDINATING 4 00:00:15,920 --> 00:00:16,760 COMMITTEE, I'M GLEN NUCKOLLS, 5 00:00:16,760 --> 00:00:19,200 AND I'M A PROGRAM DIRECTOR AT 6 00:00:19,200 --> 00:00:22,200 THE NATIONAL INSTITUTE OF 7 00:00:22,200 --> 00:00:22,920 NEUROLOGICAL DISORDERS AND 8 00:00:22,920 --> 00:00:23,160 STROKE. 9 00:00:23,160 --> 00:00:26,200 WHY DON'T WE GO AROUND AND 10 00:00:26,200 --> 00:00:27,160 INTRODUCE COMMITTEE MEMBERS AND 11 00:00:27,160 --> 00:00:32,560 WE'LL START WITH OUR CHAIR, DR. 12 00:00:32,560 --> 00:00:34,920 BIANCHI. 13 00:00:34,920 --> 00:00:39,000 >>I DON'T SEE HER ON YET, GLEN. 14 00:00:39,000 --> 00:00:39,640 >>HMM. 15 00:00:39,640 --> 00:00:39,840 OKAY. 16 00:00:39,840 --> 00:00:48,200 WE'LL COME BACK TO HER. 17 00:00:48,200 --> 00:00:49,840 LET'S INTRODUCE THERESA 18 00:00:49,840 --> 00:00:51,120 SANAGUSTIN. 19 00:00:51,120 --> 00:00:57,600 >>GOOD AFTERNOON, EVERYONE. 20 00:00:57,600 --> 00:00:58,680 I'M THERESA SAN AGUSTIN, 21 00:00:58,680 --> 00:00:59,680 NATIONAL INSTITUTE ON 22 00:00:59,680 --> 00:01:00,280 DISABILITY, INDEPENDENT LIVING 23 00:01:00,280 --> 00:01:06,880 AND REHABILITATION RESEARCH 24 00:01:06,880 --> 00:01:07,680 UNDER ADMINISTRATION OF 25 00:01:07,680 --> 00:01:10,400 COMMUNITY LIVING, HHS. 26 00:01:10,400 --> 00:01:15,120 THANK YOU, EVERYONE. 27 00:01:15,120 --> 00:01:18,240 >>THANKS. 28 00:01:18,240 --> 00:01:19,280 29 00:01:19,280 --> 00:01:21,120 >>HI, CHRIS ANSELMO, MUSCULAR 30 00:01:21,120 --> 00:01:24,280 DYSTROPHY ASSOCIATION, ALSO A 31 00:01:24,280 --> 00:01:34,040 MEMBER OF THE MUSCULAR DYSTROPHY 32 00:01:34,040 --> 00:01:34,560 COMMUNITY. 33 00:01:34,560 --> 00:01:36,400 >>LINDSEY CRISWELL. 34 00:01:36,400 --> 00:01:37,720 >>I'M DIRECTOR OF NATIONAL 35 00:01:37,720 --> 00:01:41,360 INSTITUTE OF ARTHRITIS AND 36 00:01:41,360 --> 00:01:42,360 MUSCULOSKELETAL AND SKIN 37 00:01:42,360 --> 00:01:44,440 DISEASE, NIAMS, HAPPY TO BE HERE 38 00:01:44,440 --> 00:01:52,080 WITH YOU TODAY. 39 00:01:52,080 --> 00:02:01,640 >>GUSTAVO DZIEWCZAPOLSKI. 40 00:02:01,640 --> 00:02:03,600 >>I'M SCIENTIFIC DIRECTOR AT 41 00:02:03,600 --> 00:02:11,080 CURE CMD. 42 00:02:11,080 --> 00:02:14,800 >>EMILY FREILICH? 43 00:02:14,800 --> 00:02:17,440 EMILY FREILICH? 44 00:02:17,440 --> 00:02:18,800 45 00:02:18,800 --> 00:02:23,360 WE'LL GIVE HER A MINUTE. 46 00:02:23,360 --> 00:02:24,840 DR. BIANCHI JUST JOINED. 47 00:02:24,840 --> 00:02:27,920 >>I WAS SENT THE WRONG LINK, MY 48 00:02:27,920 --> 00:02:28,280 APOLOGIES. 49 00:02:28,280 --> 00:02:31,840 I KEPT GETTING DOUBLE LINKS TO 50 00:02:31,840 --> 00:02:33,920 THIS MEETING, I SIGNED IN AS 51 00:02:33,920 --> 00:02:35,440 ATTENDEE, NOT PARTICIPANT, SO 52 00:02:35,440 --> 00:02:36,360 IT'S FRUSTRATING WHEN I'M TRYING 53 00:02:36,360 --> 00:02:38,400 TO GET ON AND I CAN HEAR YOU, 54 00:02:38,400 --> 00:02:41,920 BUT YOU CAN'T HEAR ME, BUT I'M 55 00:02:41,920 --> 00:02:42,880 HERE NOW, WELCOME, EVERYBODY. 56 00:02:42,880 --> 00:02:43,840 >>SORRY ABOUT THAT TROUBLE. 57 00:02:43,840 --> 00:02:47,600 WE'LL GET THAT STRAIGHTENED OUT 58 00:02:47,600 --> 00:02:49,800 FOR YOU. 59 00:02:49,800 --> 00:02:52,480 GENE FREUND? 60 00:02:52,480 --> 00:02:56,840 IS GENE ON THE LINE? 61 00:02:56,840 --> 00:02:57,080 OKAY. 62 00:02:57,080 --> 00:02:59,400 MICHAEL GOLDSTEIN. 63 00:02:59,400 --> 00:03:02,680 64 00:03:02,680 --> 00:03:06,320 I HOPE THEY DON'T ALL HAVE 65 00:03:06,320 --> 00:03:11,240 ATTENDEE LINKS AS WELL. 66 00:03:11,240 --> 00:03:12,440 67 00:03:12,440 --> 00:03:13,920 ALISHA KEEHN? 68 00:03:13,920 --> 00:03:15,760 >>HI, I'M HERE. 69 00:03:15,760 --> 00:03:18,000 >>WOULD YOU LIKE TO INTRODUCE 70 00:03:18,000 --> 00:03:22,280 YOURSELF? 71 00:03:22,280 --> 00:03:32,880 >>I'M ALISHA KEEHN, BRANCH CHI. 72 00:03:35,280 --> 00:03:37,280 >>JIM KILEY? 73 00:03:37,280 --> 00:03:38,760 >>HE'S AWAY. 74 00:03:38,760 --> 00:03:39,360 >>OKAY. 75 00:03:39,360 --> 00:03:42,200 THANK YOU. 76 00:03:42,200 --> 00:03:43,320 WALTER KOROSHETZ? 77 00:03:43,320 --> 00:03:45,840 >>GOOD AFTERNOON, I'M WALTER, 78 00:03:45,840 --> 00:03:46,360 DIRECTOR OF THE NATIONAL 79 00:03:46,360 --> 00:03:46,880 INSTITUTE OF NEUROLOGICAL 80 00:03:46,880 --> 00:03:51,640 DISORDERS AND STROKE. 81 00:03:51,640 --> 00:03:57,880 PLEASED TO BE HERE. 82 00:03:57,880 --> 00:04:01,200 >>MARIELENA McGUIRE? 83 00:04:01,200 --> 00:04:09,920 YOU'RE MUTED STILL. 84 00:04:09,920 --> 00:04:11,600 85 00:04:11,600 --> 00:04:17,400 CAN'T HEAR YOU. 86 00:04:17,400 --> 00:04:22,320 >>HOW ABOUT NOW? 87 00:04:22,320 --> 00:04:23,960 88 00:04:23,960 --> 00:04:25,560 >>YES, THAT WORKS. 89 00:04:25,560 --> 00:04:28,760 >>SORRY ABOUT THAT, FOLKS. 90 00:04:28,760 --> 00:04:32,880 I'M THE PROGRAM MANAGER FOR THE 91 00:04:32,880 --> 00:04:35,920 DEE DUCHENNE MUSCULAR DYSTROPHY 92 00:04:35,920 --> 00:04:37,840 RESEARCH PROGRAM, UNDER 93 00:04:37,840 --> 00:04:38,560 CONGRESSIONALLY DIRECTED MEDICAL 94 00:04:38,560 --> 00:04:40,880 RESEARCH PROGRAMS AT THE DoD. 95 00:04:40,880 --> 00:04:44,480 THANK YOU. 96 00:04:44,480 --> 00:04:44,960 >>THANKS. 97 00:04:44,960 --> 00:04:46,360 DEBRA MILLER? 98 00:04:46,360 --> 00:04:47,680 >>GOOD MORNING IN CALIFORNIA, 99 00:04:47,680 --> 00:04:49,760 DEBRA MILLER, CEO AND FOUNDER OF 100 00:04:49,760 --> 00:04:52,280 CURE DUCHENNE, I ALSO HAVE A 101 00:04:52,280 --> 00:04:53,240 25-YEAR-OLD SON WITH DUCHENNE 102 00:04:53,240 --> 00:04:53,960 MUSCULAR DYSTROPHY. 103 00:04:53,960 --> 00:04:55,440 HAPPY TO BE HERE. 104 00:04:55,440 --> 00:04:56,760 THANK YOU. 105 00:04:56,760 --> 00:05:00,600 >>THANK YOU. 106 00:05:00,600 --> 00:05:04,760 DENICE MILER? 107 00:05:04,760 --> 00:05:07,640 >>FROM IDAHO, DISABLED ADVOCATE 108 00:05:07,640 --> 00:05:12,880 HELPING THE IDAHO ENTER MOUNTAIN 109 00:05:12,880 --> 00:05:15,080 AGENCY, RENTAL ASSISTANCE, ALSO 110 00:05:15,080 --> 00:05:18,000 INVOLVED WITH IDAHO CAREGIVER 111 00:05:18,000 --> 00:05:18,280 ALLIANCE. 112 00:05:18,280 --> 00:05:21,000 I THINK THAT'S ENOUGH FOR NOW. 113 00:05:21,000 --> 00:05:21,400 >>OKAY. 114 00:05:21,400 --> 00:05:27,840 THANK YOU. 115 00:05:27,840 --> 00:05:30,240 DAN PEREZ? 116 00:05:30,240 --> 00:05:35,080 >>DAN PEREZ, CO-FOUNDER AND 117 00:05:35,080 --> 00:05:45,600 DIRECTOR FSHD CODE, IN GLOCKTON, 118 00:05:49,760 --> 00:05:50,400 MASSACHUSETTS. 119 00:05:50,400 --> 00:05:51,320 >>NATALIE STREET? 120 00:05:51,320 --> 00:05:55,760 >>HI, NATALIE STREET, HEALTH 121 00:05:55,760 --> 00:05:57,000 SCIENTIST, PROGRAM ACTIVITIES IN 122 00:05:57,000 --> 00:06:00,680 THE CENTERS FOR DISEASE CONTROL 123 00:06:00,680 --> 00:06:06,120 AND PREVENTION. 124 00:06:06,120 --> 00:06:08,640 >>ERIC LANG? 125 00:06:08,640 --> 00:06:10,280 126 00:06:10,280 --> 00:06:14,640 NOT ON YET. 127 00:06:14,640 --> 00:06:18,680 I SEE EMILY FREILICH JOINED US. 128 00:06:18,680 --> 00:06:20,960 >>HI, I'M A PEDIATRIC 129 00:06:20,960 --> 00:06:25,560 NEUROLOGIST FROM FDA WITH 130 00:06:25,560 --> 00:06:28,000 DIVISION OF NEUROLOGY. 131 00:06:28,000 --> 00:06:31,480 >>DID GENE, MICHAEL OR ERIC 132 00:06:31,480 --> 00:06:31,680 JOIN? 133 00:06:31,680 --> 00:06:33,440 >>HI, GLEN, IT'S MICHAEL 134 00:06:33,440 --> 00:06:37,040 GOLDSTEIN, I WAS UPGRADED TO 135 00:06:37,040 --> 00:06:37,720 PARTICIPANT, THANK YOU FOR 136 00:06:37,720 --> 00:06:39,040 HAVING ME. 137 00:06:39,040 --> 00:06:40,880 I'M THE DIRECTOR OF THE OFFICE 138 00:06:40,880 --> 00:06:44,720 OF MEDICAL POLICY AT THE SOCIAL 139 00:06:44,720 --> 00:06:48,000 SECURITY ADMINISTRATION, AND OUR 140 00:06:48,000 --> 00:06:50,720 OFFICE HOUSES MEDICAL LISTINGS, 141 00:06:50,720 --> 00:06:51,520 SPECIFICALLY NEUROLOGICAL 142 00:06:51,520 --> 00:06:54,600 LISTINGS, WHICH INCLUDE MUSCULAR 143 00:06:54,600 --> 00:06:57,760 DYSTROPHIES AND ALSO ANOTHER 144 00:06:57,760 --> 00:07:03,120 NUMBER OF POLICY AREAS, 145 00:07:03,120 --> 00:07:04,880 INCLUDING OUR COMPASSIONATE 146 00:07:04,880 --> 00:07:06,280 ALLOWANCE INITIATIVES COMPRISING 147 00:07:06,280 --> 00:07:07,560 SEVERAL MUSCULAR DYSTROPHIES. 148 00:07:07,560 --> 00:07:11,640 THANKS FOR HAVING ME. 149 00:07:11,640 --> 00:07:13,640 >>GENE FREUND OR ERIC LANG? 150 00:07:13,640 --> 00:07:15,160 >>HI, GLEN. 151 00:07:15,160 --> 00:07:17,680 THERE'S A NOTE FROM GENE IN THE 152 00:07:17,680 --> 00:07:20,200 Q&A HE WAS SENT AN ATTENDEE 153 00:07:20,200 --> 00:07:22,160 LINK, NOT PANELLEST LINK, THAT'S 154 00:07:22,160 --> 00:07:24,680 WHY HE HASN'T BEEN ABLE TO TALK. 155 00:07:24,680 --> 00:07:28,840 >>SHERRY, CAN YOU SEND 156 00:07:28,840 --> 00:07:29,720 APPROPRIATE LINK PLEASE? 157 00:07:29,720 --> 00:07:31,280 >>TAKING CARE OF IT RIGHT NOW. 158 00:07:31,280 --> 00:07:32,960 THANK YOU. 159 00:07:32,960 --> 00:07:33,520 >>OKAY. 160 00:07:33,520 --> 00:07:39,760 WELL, LET'S GET STARTED AND THEN 161 00:07:39,760 --> 00:07:41,200 GIVE THEM AN OPPORTUNITY TO JOIN 162 00:07:41,200 --> 00:07:41,400 LATER. 163 00:07:41,400 --> 00:07:43,680 THIS MEETING IS CONDUCTED IN 164 00:07:43,680 --> 00:07:45,360 COMPLIANCE WITH THE FEDERAL 165 00:07:45,360 --> 00:07:48,720 ADVISORY COMMITTEE ACT, TO 166 00:07:48,720 --> 00:07:50,800 ENSURE THAT THE COMMITTEE 167 00:07:50,800 --> 00:07:52,880 PROVIDES EXPERT ADVICE AND 168 00:07:52,880 --> 00:07:53,680 DIVERSE OPINIONS TO INFORM 169 00:07:53,680 --> 00:07:55,200 DECISION MAKING BY OFFICIALS AND 170 00:07:55,200 --> 00:07:56,800 AGENCIES IN THE EXECUTIVE BRANCH 171 00:07:56,800 --> 00:07:59,240 OF THE FEDERAL GOVERNMENT IN A 172 00:07:59,240 --> 00:08:01,560 MANNER THAT IS TRANSPARENT AND 173 00:08:01,560 --> 00:08:03,840 FREE OF CONFLICTS OF INTEREST. 174 00:08:03,840 --> 00:08:05,760 THERE IS NO CLOSED SESSION 175 00:08:05,760 --> 00:08:07,440 PLANNED FOR TODAY'S MEETING. 176 00:08:07,440 --> 00:08:10,520 WE DO NOT ANTICIPATE DISCUSSIONS 177 00:08:10,520 --> 00:08:12,280 OR ACTIONS SPECIFIC TO THE 178 00:08:12,280 --> 00:08:13,240 ORGANIZATIONS OR FINANCIAL 179 00:08:13,240 --> 00:08:14,160 INTERESTS OF OUR MEMBERS. 180 00:08:14,160 --> 00:08:17,640 SO THERE'S NO NEED FOR RECUSALS. 181 00:08:17,640 --> 00:08:19,840 WE DID POST NOTICE TO FEDERAL 182 00:08:19,840 --> 00:08:25,520 REGISTER ABOUT THIS MEETING, AND 183 00:08:25,520 --> 00:08:27,280 INVITE OPPORTUNITIES FOR PUBLIC 184 00:08:27,280 --> 00:08:27,480 COMMENT. 185 00:08:27,480 --> 00:08:29,280 WE DID NOT RECEIVE REQUESTS TO 186 00:08:29,280 --> 00:08:30,560 ADDRESS THE COMMITTEE AND DID 187 00:08:30,560 --> 00:08:31,440 NOT SCHEDULE PUBLIC COMMENTS 188 00:08:31,440 --> 00:08:33,000 DURING THIS MEETING. 189 00:08:33,000 --> 00:08:34,200 THE MEETING IS BEING STREAMED 190 00:08:34,200 --> 00:08:36,280 LIVE FOR PUBLIC VIEWING AND IS 191 00:08:36,280 --> 00:08:37,480 BEING RECORDED. 192 00:08:37,480 --> 00:08:41,400 THE STREAM AND RECORDING ARE 193 00:08:41,400 --> 00:08:43,480 AVAILABLE AT VIDEOCAST.NIH.GOV, 194 00:08:43,480 --> 00:08:48,800 WE'LL ALSO PROVIDE A LINK AT OUR 195 00:08:48,800 --> 00:08:50,640 WEBSITE, MDCCC.NIH.GOV. 196 00:08:50,640 --> 00:08:52,160 FOR THOSE CONNECTING THROUGH 197 00:08:52,160 --> 00:08:54,560 ZOOM, THE COMMITTEE MEMBERS AND 198 00:08:54,560 --> 00:08:55,320 OTHER DESIGNATED PANELISTS, YOU 199 00:08:55,320 --> 00:08:58,280 CAN RAISE YOUR HAND AND UNMUTE 200 00:08:58,280 --> 00:08:59,360 TO SPEAK, ASK QUESTIONS, IF 201 00:08:59,360 --> 00:09:02,120 YOU'RE NOT A COMMITTEE MEMBER OR 202 00:09:02,120 --> 00:09:04,720 DESIGNATED PANELIST, AND YOU'RE 203 00:09:04,720 --> 00:09:06,240 A DESIGNATED PARTICIPANT YOU CAN 204 00:09:06,240 --> 00:09:07,760 SUBMIT QUESTIONS VIA THE CHAT 205 00:09:07,760 --> 00:09:09,640 FEATURE AND WE'LL READ THOSE OUT 206 00:09:09,640 --> 00:09:10,160 FOR YOU. 207 00:09:10,160 --> 00:09:13,240 I WOULD LIKE TO THANK THE NIH 208 00:09:13,240 --> 00:09:17,560 AND CONTRACTOR STAFF THAT HAVE 209 00:09:17,560 --> 00:09:21,600 CONTRIBUTED TO HELP PREPARE FOR 210 00:09:21,600 --> 00:09:27,840 THIS MEETING. 211 00:09:27,840 --> 00:09:29,040 SO UNLESS THERE'S ANY QUESTIONS 212 00:09:29,040 --> 00:09:36,680 I'LL TURN IT OVER TO DR. BIANCHI 213 00:09:36,680 --> 00:09:37,280 FOR YOUR CHAIR PRESENTATION 214 00:09:37,280 --> 00:09:39,120 >>THANK YOU. 215 00:09:39,120 --> 00:09:40,760 I'M DIRECTOR OF NICHD SHALL 216 00:09:40,760 --> 00:09:45,640 SERVING RIGHT NOW AS CHAIR OF 217 00:09:45,640 --> 00:09:46,560 THE MDCC. 218 00:09:46,560 --> 00:09:52,120 IT'S FRUSTRATING WHEN YOU KNOW 219 00:09:52,120 --> 00:09:53,320 YOU ARE CHAIRING AND HOPEFULLY 220 00:09:53,320 --> 00:09:55,480 CAN'T GET INTO THE MEETING, 221 00:09:55,480 --> 00:09:58,560 HOPEFULLY WE'LL FIX THAT NEXT 222 00:09:58,560 --> 00:09:58,800 TIME. 223 00:09:58,800 --> 00:10:00,520 I'LL GO AHEAD AND SHARE MY 224 00:10:00,520 --> 00:10:03,480 SCREEN AND IT WILL BE MY GREAT 225 00:10:03,480 --> 00:10:05,760 PLEASURE TO UPDATE YOU ON WHAT'S 226 00:10:05,760 --> 00:10:12,480 BEEN HAPPENING SINCE WE LAST 227 00:10:12,480 --> 00:10:12,640 MET. 228 00:10:12,640 --> 00:10:23,160 CAN YOU SEE IT IN PRESENTATION 229 00:10:24,120 --> 00:10:24,320 MODE? 230 00:10:24,320 --> 00:10:26,600 WE'RE CELEBRATING OUR 60th 231 00:10:26,600 --> 00:10:27,800 ANNIVERSARY, WE'LL HAVE OUR 232 00:10:27,800 --> 00:10:38,280 60th ANNIVERSARY SYMPOSIUM IN 233 00:10:38,280 --> 00:10:39,080 A COUPLE WEEKS. 234 00:10:39,080 --> 00:10:41,320 WE'LL GIVE YOU AN UPDATE ON THE 235 00:10:41,320 --> 00:10:44,720 NIH BUDGET, TALK ABOUT CHANGES 236 00:10:44,720 --> 00:10:48,360 IN ARPA-H, AND SOME UPDATES ON 237 00:10:48,360 --> 00:10:50,320 THE NIH DATA MANAGEMENT AND 238 00:10:50,320 --> 00:10:53,920 SHARING POLICY, SOME NEWS ABOUT 239 00:10:53,920 --> 00:10:56,440 THE NIH COMMON FUND ARTIFICIAL 240 00:10:56,440 --> 00:10:58,000 INTELLIGENCE INITIATIVE, AND 241 00:10:58,000 --> 00:11:00,880 SELECTED ADVANCES IN MUSCULAR 242 00:11:00,880 --> 00:11:01,600 DYSTROPHY RESEARCH. 243 00:11:01,600 --> 00:11:04,440 I'D LIKE TO THANK STAFF MEMBERS 244 00:11:04,440 --> 00:11:05,880 WHO CONTRIBUTED WHAT THEY 245 00:11:05,880 --> 00:11:09,920 THOUGHT WERE THE HIGHLIGHTS IN 246 00:11:09,920 --> 00:11:11,280 MUSCULAR DYSTROPHY RESEARCH. 247 00:11:11,280 --> 00:11:14,080 THE HEADLINE IS OF COURSE AS OF 248 00:11:14,080 --> 00:11:18,680 FRIDAY, WE WERE GRANTED A 249 00:11:18,680 --> 00:11:21,320 CONTINUING RESOLUTION THROUGH 250 00:11:21,320 --> 00:11:24,480 DECEMBER 16, 2022. 251 00:11:24,480 --> 00:11:27,520 THAT WAS BETTER THAN ALTERNATIVE 252 00:11:27,520 --> 00:11:29,720 OF A GOVERNMENT SHUTDOWN, STILL 253 00:11:29,720 --> 00:11:31,720 NOT AS OPTIMAL AS HAVING A 254 00:11:31,720 --> 00:11:33,760 BUDGET ACT OF OCTOBER 1. 255 00:11:33,760 --> 00:11:36,400 WE'RE NOW ALREADY INTO FISCAL 256 00:11:36,400 --> 00:11:37,600 YEAR 2023, AND WHAT IT MEANS 257 00:11:37,600 --> 00:11:40,880 WHEN WE HAVE A CONTINUING 258 00:11:40,880 --> 00:11:43,400 RESOLUTION IS WE HAVE FUNDS THAT 259 00:11:43,400 --> 00:11:45,480 ALLOW US TO MOVE FORWARD BUT WE 260 00:11:45,480 --> 00:11:47,880 DON'T REALLY KNOW WHAT THE 261 00:11:47,880 --> 00:11:48,960 BUDGET IS. 262 00:11:48,960 --> 00:11:52,040 WE'RE AT A FLAT LINE, AND WE CAN 263 00:11:52,040 --> 00:11:55,920 DO OUR BEST TO ESTIMATE. 264 00:11:55,920 --> 00:11:57,600 BUT EVEN THOUGH WE GOT THE 265 00:11:57,600 --> 00:12:01,200 BUDGET FOR THIS PAST FISCAL 266 00:12:01,200 --> 00:12:05,240 YEAR, 22, ON MARCH 15, YOU CAN 267 00:12:05,240 --> 00:12:07,240 IMAGINE THE AMOUNT OF WORK IT 268 00:12:07,240 --> 00:12:09,080 INVOLVES TO GET WHAT WE WOULD 269 00:12:09,080 --> 00:12:12,840 NORMALLY DO IN 12 MONTHS DONE IN 270 00:12:12,840 --> 00:12:15,400 6 MONTHS. 271 00:12:15,400 --> 00:12:17,800 SO, I'D LIKE TO EXPRESS MY 272 00:12:17,800 --> 00:12:20,080 APPRECIATION FOR EVERYONE WHO 273 00:12:20,080 --> 00:12:21,440 MOVED MOUNTAINS, LITERALLY, TO 274 00:12:21,440 --> 00:12:23,840 GET TWICE AS MUCH WORK DONE IN 275 00:12:23,840 --> 00:12:27,640 HALF OF THE USUAL TIME. 276 00:12:27,640 --> 00:12:29,320 WHICH WAS SUCCESSFULLY DONE. 277 00:12:29,320 --> 00:12:31,720 WE'RE KINDING IN THE SAME 278 00:12:31,720 --> 00:12:32,920 SITUATION AGAIN, HOPEFUL THAT 279 00:12:32,920 --> 00:12:34,440 PERHAPS AFTER THE MIDTERM 280 00:12:34,440 --> 00:12:36,760 ELECTIONS THAT DECISIONS CAN BE 281 00:12:36,760 --> 00:12:36,960 MADE. 282 00:12:36,960 --> 00:12:39,480 AND THAT WE COULD GET A 283 00:12:39,480 --> 00:12:40,800 WONDERFUL HOLIDAY PRESENT OF 284 00:12:40,800 --> 00:12:44,720 HAVING A REAL BUDGET TO PLAN 285 00:12:44,720 --> 00:12:45,040 ACCORDINGLY. 286 00:12:45,040 --> 00:12:45,920 THERE ARE COMPONENTS OF THE 287 00:12:45,920 --> 00:12:49,240 BUDGET THAT HAVE BEEN WORKED ON 288 00:12:49,240 --> 00:12:51,800 ALREADY, THE PRESIDENT'S BUDGET 289 00:12:51,800 --> 00:12:56,640 PROPOSED $62.5 BILLION FOR NIH. 290 00:12:56,640 --> 00:13:04,000 CONSISTING OF $45.5 BILLION IN A 291 00:13:04,000 --> 00:13:07,800 BASE BUDGET, $5 BILLION FOR 292 00:13:07,800 --> 00:13:14,560 ARPA-H, AND SPECIFIC PROBLEMS. 293 00:13:14,560 --> 00:13:16,280 THE SENATE, $48 BILLION AND LESS 294 00:13:16,280 --> 00:13:20,320 FOR ARPA-H, HOUSE APPROACH 295 00:13:20,320 --> 00:13:21,720 R IGS -- APPROPRIATION BILL HAS 296 00:13:21,720 --> 00:13:23,040 NOT YET PASSED. 297 00:13:23,040 --> 00:13:25,480 ARPA-H WAS ESTABLISHED BY 298 00:13:25,480 --> 00:13:28,880 CONGRESS WITHIN HEALTH AND HUMAN 299 00:13:28,880 --> 00:13:32,040 SERVICES LAST SPRING, WITH THE 300 00:13:32,040 --> 00:13:33,240 BUDGET ACTIVITIES. 301 00:13:33,240 --> 00:13:35,560 SOME WHAT CONFUSING TO FOLKS, 302 00:13:35,560 --> 00:13:38,720 DOES IT -- IS IT AT NIH? 303 00:13:38,720 --> 00:13:40,800 HOW DOES IT RELATE TO NIH? 304 00:13:40,800 --> 00:13:43,320 THERE'S QUITE A BIT OF 305 00:13:43,320 --> 00:13:44,800 COMPETITION AMONGST DIFFERENT 306 00:13:44,800 --> 00:13:50,200 STATES AND CITIES TO HOUSE 307 00:13:50,200 --> 00:13:50,440 ARPA-H. 308 00:13:50,440 --> 00:13:54,280 IT IS AN INDEPENDENT ENTITY BUT 309 00:13:54,280 --> 00:13:55,880 IS RECEIVING INFRASTRUCTURE 310 00:13:55,880 --> 00:13:58,560 SUPPORT SUCH AS PERSONNEL 311 00:13:58,560 --> 00:14:00,600 MANAGEMENT, FOR EXAMPLE, ALL 312 00:14:00,600 --> 00:14:02,680 KINDS OF THINGS THAT NIH DOES, 313 00:14:02,680 --> 00:14:04,720 ALREADY DOES FOR OTHER AGENCIES 314 00:14:04,720 --> 00:14:05,200 AS WELL. 315 00:14:05,200 --> 00:14:10,200 SO RATHER THAN START UP A 316 00:14:10,200 --> 00:14:11,560 COMPLETELY NEW AGENCY, FROM 317 00:14:11,560 --> 00:14:13,040 SCRATCH, IT'S GETTING A LOT OF 318 00:14:13,040 --> 00:14:14,400 SUPPORT FROM NIH. 319 00:14:14,400 --> 00:14:17,760 IT'S HAD AN INTERIM DIRECTOR, 320 00:14:17,760 --> 00:14:19,080 DR. ADAM RUSSELL. 321 00:14:19,080 --> 00:14:26,440 BUT WITHIN THE PAST MONTH DR. 322 00:14:26,440 --> 00:14:28,360 RENEE WEGRYGN WAS APPOINTED THE 323 00:14:28,360 --> 00:14:30,160 FIRST PERMANENT DIRECTOR, SEEMS 324 00:14:30,160 --> 00:14:40,640 TO BE IDEALLY SUITED WITH A 325 00:14:41,040 --> 00:14:43,720 BIOTECH COMPANY IN BOSTON, 326 00:14:43,720 --> 00:14:45,560 WORKED AT DARPA AND IARPA, 327 00:14:45,560 --> 00:14:48,560 FAMILIAR WITH THIS CONCEPT OF 328 00:14:48,560 --> 00:14:50,200 HIGH RISK, HIGH REWARD RESEARCH, 329 00:14:50,200 --> 00:14:54,320 THAT IS EXPECTED TO FAIL FAST 330 00:14:54,320 --> 00:14:55,560 AND FAIL OFTEN. 331 00:14:55,560 --> 00:14:58,520 AND SOMETHING THE PRESIDENT IS 332 00:14:58,520 --> 00:15:01,680 HIGHLY SUPPORTIVE OF TO REALLY 333 00:15:01,680 --> 00:15:05,200 GET TO CHORES AND OTHER SORTS OF 334 00:15:05,200 --> 00:15:07,280 TRANSFORMATIVE SOLUTIONS FOR ALL 335 00:15:07,280 --> 00:15:07,680 PATIENTS. 336 00:15:07,680 --> 00:15:11,000 AND PROJECTS THAT ARE ASKING FOR 337 00:15:11,000 --> 00:15:13,160 SUPPORT MUST ALL INCLUDE EQUITY 338 00:15:13,160 --> 00:15:17,360 IN THEIR DESIGN, AND NIH HAS A 339 00:15:17,360 --> 00:15:20,360 WEBSITE DEDICATED TO ARPA-H, NOT 340 00:15:20,360 --> 00:15:30,880 SURE WHEN DR. DR.ER DR.WEGRYGN 341 00:15:39,080 --> 00:15:41,640 SHOULD START SOON ON CAMPUS. 342 00:15:41,640 --> 00:15:48,320 WE WANTED TO BEGINNING THIS UP, 343 00:15:48,320 --> 00:15:49,880 IT'S CAUSING CONCERN AMONGST 344 00:15:49,880 --> 00:15:52,360 INVESTIGATORS, THIS POLICY THAT 345 00:15:52,360 --> 00:15:56,760 WILL BECOME EFFECTIVE IN 346 00:15:56,760 --> 00:15:58,040 JANUARY,OF NEXT YEAR. 347 00:15:58,040 --> 00:15:59,080 IT'S NOT RETROACTIVE IN THE 348 00:15:59,080 --> 00:16:02,040 SENSE IF YOU HAVE A FUNDED 349 00:16:02,040 --> 00:16:03,440 GRANT, YOU'RE NOT GOING TO HAVE 350 00:16:03,440 --> 00:16:06,840 TO DO THIS FOR THE FUNDED GRANT. 351 00:16:06,840 --> 00:16:10,000 IT'S GOING TO AFFECT ALL 352 00:16:10,000 --> 00:16:12,000 APPLICATIONS GOING FORWARD. 353 00:16:12,000 --> 00:16:14,480 AND IT EXPECTS INVESTIGATORS AND 354 00:16:14,480 --> 00:16:17,120 INSTITUTIONS TO PLAN AND BUDGET 355 00:16:17,120 --> 00:16:19,960 FOR MANAGEMENT AND SHARING OF 356 00:16:19,960 --> 00:16:21,080 DATA. 357 00:16:21,080 --> 00:16:21,760 AND ALL INVESTIGATORS WILL NEED 358 00:16:21,760 --> 00:16:23,720 TO SUBMIT A DATA MANAGEMENT 359 00:16:23,720 --> 00:16:26,720 SHARING PLAN FOR REVIEW WHEN 360 00:16:26,720 --> 00:16:28,960 APPLYING FOR NIH FUNDING. 361 00:16:28,960 --> 00:16:34,000 AND IT WILL BE LOOKED AT AND 362 00:16:34,000 --> 00:16:36,640 EXAMINED FOR COMPLIANCE WITH THE 363 00:16:36,640 --> 00:16:39,280 OVERALL NIH APPROVED DATA 364 00:16:39,280 --> 00:16:40,440 MANAGEMENT AND SHARING PLAN. 365 00:16:40,440 --> 00:16:43,920 THE GOAL IS REALLY TO MAKE DATA, 366 00:16:43,920 --> 00:16:46,560 SCIENTIFIC DATA, AS ACCESSIBLE 367 00:16:46,560 --> 00:16:48,200 AS POSSIBLE, AS SOON AS 368 00:16:48,200 --> 00:16:51,280 POSSIBLE, BY THE EARLIEST OF TWO 369 00:16:51,280 --> 00:16:52,800 TIME POINTS. 370 00:16:52,800 --> 00:16:55,200 SO, BEFORE OR JUST BEFORE A 371 00:16:55,200 --> 00:16:56,640 PUBLICATION THAT IS ASSOCIATED 372 00:16:56,640 --> 00:16:59,280 WITH THIS GRANT FUNDING IS 373 00:16:59,280 --> 00:17:02,120 PUBLISHED, AND OR THE END OF THE 374 00:17:02,120 --> 00:17:03,640 PERFORMANCE PERIOD OF THE 375 00:17:03,640 --> 00:17:05,400 EXTRAMURAL AWARD THAT GENERATED 376 00:17:05,400 --> 00:17:05,960 THE DATA. 377 00:17:05,960 --> 00:17:16,360 AND THERE ARE MORE DETAILS ON 378 00:17:16,360 --> 00:17:26,320 THIS WEBSITE. 379 00:17:26,320 --> 00:17:27,680 HTTPS//SHARING.NIH.GOV. 380 00:17:27,680 --> 00:17:33,840 I'LL TELL YOU ABOUT AN EXCITING 381 00:17:33,840 --> 00:17:40,200 CHALLENGE THAT IS -- THAT HAS 382 00:17:40,200 --> 00:17:45,640 BEEN FUNDED NOW THAT IS -- WAS 383 00:17:45,640 --> 00:17:50,680 REALLY DEVELOPED BECAUSE 384 00:17:50,680 --> 00:17:52,200 CONCERNS WERE THAT MOST 385 00:17:52,200 --> 00:17:55,600 BIOMEDICAL MEDICAL IS NOT 386 00:17:55,600 --> 00:17:56,360 SUITABLE FOR MACHINE LEARNING 387 00:17:56,360 --> 00:18:05,920 BECAUSE THEY ARE INCOMPLETE AND 388 00:18:05,920 --> 00:18:08,640 GOAL OF BRIDGE2AI ADDRESSES 389 00:18:08,640 --> 00:18:09,880 BIOMEDICAL AND BEHAVIORAL 390 00:18:09,880 --> 00:18:12,040 RESEARCH GRANT CHALLENGES. 391 00:18:12,040 --> 00:18:15,120 SO, THE GOALS ARE TO GENERATE 392 00:18:15,120 --> 00:18:18,280 DATASETS THAT ARE ETHICALLY 393 00:18:18,280 --> 00:18:20,440 SOURCED, TRUSTWORTHY, WELL 394 00:18:20,440 --> 00:18:22,000 DEFINED, ACCESSIBLE. 395 00:18:22,000 --> 00:18:27,040 TO DEVELOP SOFTWARE AND 396 00:18:27,040 --> 00:18:32,520 STANDARDS ACROSS DATA SOURCES 397 00:18:32,520 --> 00:18:42,960 AND DATA TYPES, TO CREATE 398 00:18:44,920 --> 00:18:52,320 AUTOMATED TOOLS AND ETHICALLY 399 00:18:52,320 --> 00:18:53,040 RESOURCED DATASETS. 400 00:18:53,040 --> 00:18:55,840 ALSO, THE GOAL IS TO CREATE 401 00:18:55,840 --> 00:18:58,000 TRAINING MATERIALS AND 402 00:18:58,000 --> 00:18:59,360 ACTIVITIES FOR WORKFORCE 403 00:18:59,360 --> 00:19:04,320 DEVELOPMENT THAT BRIDGES A.I., 404 00:19:04,320 --> 00:19:08,560 BIOMEDICAL, BEHAVIORAL RESEARCH 405 00:19:08,560 --> 00:19:09,360 COMMUNITIES. 406 00:19:09,360 --> 00:19:12,160 THE FIRST AWARDS WERE MADE IN 407 00:19:12,160 --> 00:19:14,960 FISCAL YEAR 22 JUST LAST MONTH. 408 00:19:14,960 --> 00:19:16,400 THIS IS $130 MILLION PROGRAM 409 00:19:16,400 --> 00:19:25,640 THAT ISED -- IS ADMINISTERED 410 00:19:25,640 --> 00:19:26,360 THROUGH COMMON FUND. 411 00:19:26,360 --> 00:19:30,440 THE FIRST ARE THE BRIDGE AWARDS 412 00:19:30,440 --> 00:19:32,080 WHICH EMPHASIZE INTEGRATION, 413 00:19:32,080 --> 00:19:33,840 DISSEMINATION, EVALUATION. 414 00:19:33,840 --> 00:19:34,520 CENTER AWARDS. 415 00:19:34,520 --> 00:19:37,640 AND THE ONES THAT HAVE BEEN 416 00:19:37,640 --> 00:19:41,480 AWARDED SO FAR INCLUDE PRECISION 417 00:19:41,480 --> 00:19:44,080 PUBLIC HEALTH, THESE USE VOICE 418 00:19:44,080 --> 00:19:46,720 AS A BIOMARKER FOR HUMAN HEALTH 419 00:19:46,720 --> 00:19:49,680 REVEALING HOW GENOMIC VARIATION, 420 00:19:49,680 --> 00:19:50,640 BEHAVIORAL, AND ENVIRONMENTAL 421 00:19:50,640 --> 00:19:53,800 FACTORS AFFECT INDIVIDUAL AND 422 00:19:53,800 --> 00:19:55,160 POPULATION HEALTH. 423 00:19:55,160 --> 00:19:59,080 FUNCTIONAL GENOMICS, THIS IS 424 00:19:59,080 --> 00:20:00,160 MAPPING SPATIOTEMPORAL 425 00:20:00,160 --> 00:20:01,440 ARCHITECTURE OF HUMAN CELLS TO 426 00:20:01,440 --> 00:20:04,480 INTERPRET CELL STRUCTURE AND 427 00:20:04,480 --> 00:20:12,320 FUNCTION IN HEALTH AND DISEASE. 428 00:20:12,320 --> 00:20:17,720 SALUTEOGENESIS, UNCOVERING 429 00:20:17,720 --> 00:20:21,840 DETAILS RESTORING HEALTH AFTER 430 00:20:21,840 --> 00:20:28,080 DISEASE USING TYPE 2 DIABETES 431 00:20:28,080 --> 00:20:33,360 AS A MODEL, AND CLINICAL CARE 432 00:20:33,360 --> 00:20:34,440 INFORMATICS FOR DIAGNOSTIC AND 433 00:20:34,440 --> 00:20:35,480 RISK PREDICTION. 434 00:20:35,480 --> 00:20:38,640 LET'S GET TO ADVANCES IN 435 00:20:38,640 --> 00:20:41,720 MUSCULAR DYSTROPHY RESEARCH. 436 00:20:41,720 --> 00:20:45,040 SO, THE FIRST PAPER WAS 437 00:20:45,040 --> 00:20:46,960 PUBLISHED IN ACS CHEMICAL 438 00:20:46,960 --> 00:20:49,280 BIOLOGY RECENTLY, SUPPORTED BY 439 00:20:49,280 --> 00:20:49,520 NIAMS. 440 00:20:49,520 --> 00:20:56,040 AND THE TITLE WAS A SELECTIVE 441 00:20:56,040 --> 00:20:59,360 AGENT FOR CDG REPEATS IN 442 00:20:59,360 --> 00:21:03,000 MYOTONIC DYSTROPHY TYPE 1, THE 443 00:21:03,000 --> 00:21:06,520 RESEARCHERS CREATED A SELECTIVE 444 00:21:06,520 --> 00:21:08,240 DNA MODIFYING SMALL MOLECULE 445 00:21:08,240 --> 00:21:10,440 THAT TARGETS A DISEASE-SPECIFIC 446 00:21:10,440 --> 00:21:13,400 STRUCTURE AND MISMICHIGANS 447 00:21:13,400 --> 00:21:15,360 INVOLVED IN MYOTONIC DYSTROPHY 448 00:21:15,360 --> 00:21:17,600 TYPE 1. 449 00:21:17,600 --> 00:21:19,440 THEY INTRODUCED A SMALL MOLECULE 450 00:21:19,440 --> 00:21:22,720 IN CELLS WITH DM1, THAT LED TO 451 00:21:22,720 --> 00:21:26,400 DECREASED LEVELS OF THE TOXIC 452 00:21:26,400 --> 00:21:29,920 TRANSCRIPT R.C.UG EXPANDED. 453 00:21:29,920 --> 00:21:39,720 IT OFFERS A STRATEGY FOR ORIGINS 454 00:21:39,720 --> 00:21:46,680 RATHER THAN DOWNSTREAM PROCESS. 455 00:21:46,680 --> 00:21:50,720 THE NEXT STUDY INVOLVED 456 00:21:50,720 --> 00:21:51,280 LONG-TERM MAINTENANCE AND 457 00:21:51,280 --> 00:21:53,920 RESISTANCE TO INJURY OF SKELETAL 458 00:21:53,920 --> 00:21:57,400 MUSCLE IN DMD MICE THAT HAVE 459 00:21:57,400 --> 00:21:58,600 BEEN GENE EDITED. 460 00:21:58,600 --> 00:22:01,680 YOU CAN SEE HOW RECENTLY IT WAS 461 00:22:01,680 --> 00:22:02,760 PUBLISHED. 462 00:22:02,760 --> 00:22:04,280 THIS STUDY TESTED LONGEVITY AND 463 00:22:04,280 --> 00:22:08,560 DURABILITY OF CRISPR/CAS9 464 00:22:08,560 --> 00:22:19,000 EDITING IN YOUNG MICE WITH 465 00:22:19,240 --> 00:22:27,840 DUCHENNE, MONITORED LONGEVITY 466 00:22:27,840 --> 00:22:34,600 WITH DELETION OF DMD EXON 44, 467 00:22:34,600 --> 00:22:41,480 RESULTS SHOWS THERAPEUTIC 468 00:22:41,480 --> 00:22:49,120 EFFECTS FROM AAV CRISPR THERAPY, 469 00:22:49,120 --> 00:22:49,840 VERY HOPEFUL. 470 00:22:49,840 --> 00:22:53,920 IN ADDITION WITH REGARD TO 471 00:22:53,920 --> 00:22:54,720 DUCHENNE MUSCULAR DYSTROPHY 472 00:22:54,720 --> 00:22:59,480 CONCERN IS THAT PATIENTS WITH 473 00:22:59,480 --> 00:23:04,000 DMD CAN HAVE ARRHYTHMIAS, 474 00:23:04,000 --> 00:23:05,440 CARDIOMYOPATHY, SUDDEN CARDIAC 475 00:23:05,440 --> 00:23:11,880 DEATH, AND MECHANISMS 476 00:23:11,880 --> 00:23:14,000 RESPONSIBLE FOR CARDIAC 477 00:23:14,000 --> 00:23:16,840 COMORBIDITIES ARE POORLY 478 00:23:16,840 --> 00:23:27,360 UNDERSTOOD, SUPPORTED BY NHLBI, 479 00:23:31,320 --> 00:23:35,560 AND NIAMS, SNTA1 GENE RESCUES 480 00:23:35,560 --> 00:23:38,400 ION CHANNEL FUNCTION. 481 00:23:38,400 --> 00:23:40,360 PLURIPOTENT STEM CELLS 482 00:23:40,360 --> 00:23:41,800 TRANSFORMED INTO CARDIOMYOCYTES 483 00:23:41,800 --> 00:23:45,000 FROM PEOPLE WITH DUCHENNE 484 00:23:45,000 --> 00:23:54,160 MUSCULAR DYSTROPHY AND 485 00:23:54,160 --> 00:23:55,040 CARDIOMYOPATHY, DEMONSTRATING 486 00:23:55,040 --> 00:24:00,960 DMD MUTATIONS CONTROL CARDIAC 487 00:24:00,960 --> 00:24:02,800 EXCITABILITY AND CONDUCTION 488 00:24:02,800 --> 00:24:08,600 VELOCITY, DETECTS COULD BE 489 00:24:08,600 --> 00:24:14,160 RESCUED WITH SNTA1, PART OF 490 00:24:14,160 --> 00:24:15,400 DYSTROPHIN-ASSOCIATED PROTEIN 491 00:24:15,400 --> 00:24:15,760 COMPLEX. 492 00:24:15,760 --> 00:24:18,880 AND ALSO, WITH REGARD TO 493 00:24:18,880 --> 00:24:20,400 DUCHENNE MUSCULAR DYSTROPHY, A 494 00:24:20,400 --> 00:24:23,480 NEW THERAPY WAS TESTED IN THIS 495 00:24:23,480 --> 00:24:27,280 STUDY THAT WAS SUPPORTED BY 496 00:24:27,280 --> 00:24:33,760 NINDS, ENTITLED EFFICACY AND 497 00:24:33,760 --> 00:24:36,720 SAFETY OF VAMOROLONE VERSUS 498 00:24:36,720 --> 00:24:40,320 PLACEBO AND PREDNISONE AMONG 499 00:24:40,320 --> 00:24:43,520 BOYS WITH DUCHENNE. 500 00:24:43,520 --> 00:24:44,600 LONG-TERM CORTICOSTEROID USE 501 00:24:44,600 --> 00:24:47,440 SUCH AS TAKING PREDNISONE IS A 502 00:24:47,440 --> 00:24:50,400 STANDARD OF CARE FOR DUCHENNE 503 00:24:50,400 --> 00:24:53,600 MUSCULAR DYSTROPHY BUT HAS 504 00:24:53,600 --> 00:24:54,480 ADVERSE EFFECTS THAT DETRACT 505 00:24:54,480 --> 00:24:58,200 FROM QUALITY OF LIFE FROM PEOPLE 506 00:24:58,200 --> 00:25:00,320 WITH DMD. 507 00:25:00,320 --> 00:25:03,400 VAMOROLONE IS A STEROIDAL 508 00:25:03,400 --> 00:25:04,880 ANTI-INFLAMMATORY DRUG THAT 509 00:25:04,880 --> 00:25:11,920 BINDS THE SAME BUT SHOWS A 510 00:25:11,920 --> 00:25:14,960 DISTINCT CHEMICAL STRUCTURE. 511 00:25:14,960 --> 00:25:16,760 PARTICIPANTS RECEIVED A PLACEBO 512 00:25:16,760 --> 00:25:19,120 DRUG, PREDNISONE, OR ONE OF TWO 513 00:25:19,120 --> 00:25:20,960 DOSES OF VAMOROLONE, AND 514 00:25:20,960 --> 00:25:25,160 VAMOROLONE WAS ABLE TO REDUCE 515 00:25:25,160 --> 00:25:26,160 BONE MORBIDITIES COMPARED TO 516 00:25:26,160 --> 00:25:27,920 PREDNISONE, WHICH IS IMPORTANT 517 00:25:27,920 --> 00:25:29,840 BECAUSE PREDNISONE WILL THIN 518 00:25:29,840 --> 00:25:32,400 BONES, SO THERE WAS NO STUNTING 519 00:25:32,400 --> 00:25:37,080 OF GROWTH AND NO DELETERIOUS 520 00:25:37,080 --> 00:25:43,320 CHANGES IN BONE MARKERS. 521 00:25:43,320 --> 00:25:46,600 AND WITH FSHD PATIENTS, I KNOW 522 00:25:46,600 --> 00:25:48,680 WE'VE DISCUSSED PREVIOUSLY, IN 523 00:25:48,680 --> 00:25:49,480 PREVIOUS COMMITTEE MEETINGS, 524 00:25:49,480 --> 00:25:51,960 ABOUT SLEEP AND THE IMPORTANCE 525 00:25:51,960 --> 00:25:55,120 OF SLEEP, HIGH QUALITY SLEEP, SO 526 00:25:55,120 --> 00:26:00,240 HERE'S A STUDY SUPPORTED BY 527 00:26:00,240 --> 00:26:01,240 NINDS, SELF-REPORTED REDUCED 528 00:26:01,240 --> 00:26:06,200 SLEEP QUALITY AND EXCESSIVE 529 00:26:06,200 --> 00:26:09,000 DAYTIME SLEEPINESS IN FSHD. 530 00:26:09,000 --> 00:26:14,360 SO, WE'VE DISCUSSED QUITE A BIT 531 00:26:14,360 --> 00:26:24,920 THAT THE SECONDARY ASSOCIATIONS 532 00:26:28,800 --> 00:26:31,320 INCLUDE ABNORMAL SLEEP PATTERNS, 533 00:26:31,320 --> 00:26:31,800 INVESTIGATORS MEASURED 534 00:26:31,800 --> 00:26:33,760 SELF-REPORTED SLEEP QUALITY FROM 535 00:26:33,760 --> 00:26:38,680 THE PARTICIPANTS AND THEIR 536 00:26:38,680 --> 00:26:41,200 DAYTIME SLEEPINESS. 537 00:26:41,200 --> 00:26:43,280 66% SHOWED REDUCED SLEEP 538 00:26:43,280 --> 00:26:45,800 QUALITY, 15% EXCESSIVE DAYTIME 539 00:26:45,800 --> 00:26:46,120 SLEEPINESS. 540 00:26:46,120 --> 00:26:48,320 SO THE RECOMMENDATION WAS THAT 541 00:26:48,320 --> 00:26:50,840 CAREGIVERS SHOULD MONITOR SLEEP 542 00:26:50,840 --> 00:26:53,680 QUALITY OF PEOPLE WITH FSHD AS 543 00:26:53,680 --> 00:26:56,120 PART OF ROUTINE CARE, WITH 544 00:26:56,120 --> 00:27:00,440 SPECIAL ATTENTION TO POTENTIALLY 545 00:27:00,440 --> 00:27:04,400 MODIFIABLE FACTORS, SUCH AS 546 00:27:04,400 --> 00:27:07,120 CONSIDERATION OF BIPAP, AND 547 00:27:07,120 --> 00:27:12,400 ADDRESSED EFFECTS OF PAIN IN 548 00:27:12,400 --> 00:27:12,720 SLEEP. 549 00:27:12,720 --> 00:27:14,800 NOW, AN NIAID TRAINING GRANT 550 00:27:14,800 --> 00:27:18,200 SUPPORTED THIS STUDY WHICH WAS 551 00:27:18,200 --> 00:27:19,720 ENTITLED INVESTIGATING ATTITUDES 552 00:27:19,720 --> 00:27:21,560 TOWARDS PRENATAL DIAGNOSIS AND 553 00:27:21,560 --> 00:27:23,240 FETAL THERAPY FOR SPINAL 554 00:27:23,240 --> 00:27:24,200 MUSCULAR ATROPHY. 555 00:27:24,200 --> 00:27:27,000 I HAVE TO CONFESS I SELECTED 556 00:27:27,000 --> 00:27:32,440 THIS ARTICLE BECAUSE ONE OF MY 557 00:27:32,440 --> 00:27:33,040 PROFESSIONAL INTERESTS IS 558 00:27:33,040 --> 00:27:34,680 PRENATAL THERAPY. 559 00:27:34,680 --> 00:27:39,960 SO THIS WAS ACTUALLY A 560 00:27:39,960 --> 00:27:42,040 QUESTIONNAIRE-RELATED STUDY. 561 00:27:42,040 --> 00:27:43,800 LOOKING AT HOW AFFECTED FAMILIES 562 00:27:43,800 --> 00:27:47,280 WOULD RESPOND TO THE POSSIBILITY 563 00:27:47,280 --> 00:27:49,600 OF SPINAL MUSCULAR ATROPHY 564 00:27:49,600 --> 00:27:51,360 TREATMENT IN UTERO. 565 00:27:51,360 --> 00:27:54,720 AND IF TREATMENT IN UTERO CAN BE 566 00:27:54,720 --> 00:27:59,920 GIVEN, IT COULD IMPROVE SURVIVAL 567 00:27:59,920 --> 00:28:00,760 AND NEUROLOGIC OUTCOMES, 568 00:28:00,760 --> 00:28:02,160 INVESTIGATING ATTITUDES OF 569 00:28:02,160 --> 00:28:05,440 PATIENTS AND PARENTS WITH SMA 570 00:28:05,440 --> 00:28:07,360 REGARDING PRENATAL DIAGNOSIS, 571 00:28:07,360 --> 00:28:08,800 FETAL THERAPIES, CLINICAL 572 00:28:08,800 --> 00:28:09,200 TRIALS. 573 00:28:09,200 --> 00:28:13,240 AND THIS QUESTIONNAIRE WAS 574 00:28:13,240 --> 00:28:19,240 DISTRIBUTED BY CURE SMA, 114 575 00:28:19,240 --> 00:28:21,240 RESPONDENTS, 60% WERE PATIENTS 576 00:28:21,240 --> 00:28:27,120 OVER AGE 18, 40% CAREGIVERS. 577 00:28:27,120 --> 00:28:28,080 91 SUPPORTED PRENATAL TESTING, 578 00:28:28,080 --> 00:28:32,600 61% WOULD ENROLL IN FETAL GENE 579 00:28:32,600 --> 00:28:34,560 THERAPY TRIALS, 7% WOULD CHOOSE 580 00:28:34,560 --> 00:28:36,640 FETAL GENE THERAPIES. 581 00:28:36,640 --> 00:28:37,720 IT'S ALWAYS IMPORTANT TO 582 00:28:37,720 --> 00:28:40,160 CONSIDER BOTH ENDS OF THE 583 00:28:40,160 --> 00:28:42,800 LIFESPAN. 584 00:28:42,800 --> 00:28:45,600 SO THERE IS PARTICULARLY IN THE 585 00:28:45,600 --> 00:28:47,800 INTERNATIONAL SOCIETY FOR 586 00:28:47,800 --> 00:28:49,640 PRENATAL DIAGNOSIS, AND PRENATAL 587 00:28:49,640 --> 00:28:52,200 DIAGNOSIS, WHICH IS OFFICIAL 588 00:28:52,200 --> 00:28:54,600 JOURNAL OF THAT SOCIETY, A LOT 589 00:28:54,600 --> 00:28:55,880 OF INTEREST ABOUT EXPLORING 590 00:28:55,880 --> 00:28:58,200 PRENATAL THERAPIES. 591 00:28:58,200 --> 00:29:01,600 THE SENIOR AUTHOR HERE IS 592 00:29:01,600 --> 00:29:05,120 McKENZIE, DO A PRENATAL 593 00:29:05,120 --> 00:29:07,400 TREATMENT STUDY FOR ALPHA 594 00:29:07,400 --> 00:29:12,680 THALASSEMIA, QUITE EXPERIENCED 595 00:29:12,680 --> 00:29:13,320 WITH PRENATAL THERAPIES. 596 00:29:13,320 --> 00:29:15,520 I WANT TO CLOSE WITH SOMETHING 597 00:29:15,520 --> 00:29:15,960 VISUAL. 598 00:29:15,960 --> 00:29:20,480 I CAME ACROSS THESE PHOTOS IN AN 599 00:29:20,480 --> 00:29:21,440 AD FOR GENENTECH AND THOUGHT, 600 00:29:21,440 --> 00:29:23,320 YOU KNOW, I THOUGHT THEY WERE 601 00:29:23,320 --> 00:29:24,520 INCREDIBLY COOL. 602 00:29:24,520 --> 00:29:26,920 HERE WAS A FASHION SHOW THAT 603 00:29:26,920 --> 00:29:29,680 FOCUSED ON PEOPLE WITH SPINAL 604 00:29:29,680 --> 00:29:31,240 MUSCULAR ATROPHY, AND THEY JUST 605 00:29:31,240 --> 00:29:32,480 ARE ALL LOOKING FABULOUS. 606 00:29:32,480 --> 00:29:35,240 I JUST WANTED TO END WITH JUST 607 00:29:35,240 --> 00:29:38,200 SOMETHING VERY POSITIVE AND 608 00:29:38,200 --> 00:29:38,520 VISUAL. 609 00:29:38,520 --> 00:29:40,800 THAT'S THE END OF MY PREPARED 610 00:29:40,800 --> 00:29:41,240 REMARKS. 611 00:29:41,240 --> 00:29:42,600 I'M GOING TO STOP SHARING SO I 612 00:29:42,600 --> 00:29:51,000 CAN SEE YOU ALL AND I'M HAPPY TO 613 00:29:51,000 --> 00:29:51,800 TAKE QUESTIONS. 614 00:29:51,800 --> 00:29:54,080 WE'VE GOT TEN MINUTES FOR 615 00:29:54,080 --> 00:29:54,400 QUESTIONS. 616 00:29:54,400 --> 00:29:55,840 IF THERE ARE NO QUESTIONS I'LL 617 00:29:55,840 --> 00:30:01,760 GIVE YOU A POP QUIZ. 618 00:30:01,760 --> 00:30:04,920 619 00:30:04,920 --> 00:30:05,960 620 00:30:05,960 --> 00:30:08,960 THE NOBEL PRIZE WENT THIS 621 00:30:08,960 --> 00:30:12,480 MORNING FOR A ROLE IN 622 00:30:12,480 --> 00:30:16,080 PALEOGENOMICS, HE WAS ONE OF 623 00:30:16,080 --> 00:30:17,560 THREE PARENT-CHILD COMBOS WHO 624 00:30:17,560 --> 00:30:20,920 WON THE NOBEL PRIZE. 625 00:30:20,920 --> 00:30:23,200 SO HE AND HIS FATHER PREVIOUSLY 626 00:30:23,200 --> 00:30:24,440 WON THE NOBEL PRIZE. 627 00:30:24,440 --> 00:30:27,720 WHO ARE THE OTHER TWO? 628 00:30:27,720 --> 00:30:30,440 PARENT-CHILD COMBOS. 629 00:30:30,440 --> 00:30:36,400 630 00:30:36,400 --> 00:30:40,440 631 00:30:40,440 --> 00:30:43,600 I DON'T KNOW IF DR. KOROSHETZ OR 632 00:30:43,600 --> 00:30:45,120 DR. CRISWELL WOULD LIKE TO 633 00:30:45,120 --> 00:30:47,120 COMMENT ON THE STUDIES WE 634 00:30:47,120 --> 00:30:49,320 HIGHLIGHTED. 635 00:30:49,320 --> 00:30:55,120 636 00:30:55,120 --> 00:30:55,640 >>WELL -- 637 00:30:55,640 --> 00:30:56,080 >>GO AHEAD. 638 00:30:56,080 --> 00:30:57,400 >>WE WANT TO KNOW THE RESPONSE 639 00:30:57,400 --> 00:31:01,040 TO YOUR QUESTION. 640 00:31:01,040 --> 00:31:02,840 >>NO CHEATING ON GOOGLE. 641 00:31:02,840 --> 00:31:05,360 >>I'M JUST LOOKING. 642 00:31:05,360 --> 00:31:10,920 643 00:31:10,920 --> 00:31:12,480 >>ARE YOU GOING TO GIVE THE 644 00:31:12,480 --> 00:31:13,120 RESPONSE FIRST? 645 00:31:13,120 --> 00:31:14,680 >>I CAN, IF NOBODY HAS THE 646 00:31:14,680 --> 00:31:15,240 ANSWER. 647 00:31:15,240 --> 00:31:16,880 I'LL CHECK THE CHAT BOX TO SEE 648 00:31:16,880 --> 00:31:22,360 IF ANYBODY CAME UP WITH IT. 649 00:31:22,360 --> 00:31:23,760 >>ONE IMMEDIATELY, THE OTHER 650 00:31:23,760 --> 00:31:28,120 WAS HARDER. 651 00:31:28,120 --> 00:31:29,400 MARIE CURIE, HER DAUGHTER IRENE 652 00:31:29,400 --> 00:31:35,240 ALSO WON THE NOBEL PRIZE. 653 00:31:35,240 --> 00:31:38,960 AND THEN ARTHUR CORNBERG, I 654 00:31:38,960 --> 00:31:41,160 SHOULD HAVE KNOWN BECAUSE I WENT 655 00:31:41,160 --> 00:31:43,520 TO STANFORD, AND HIS SON ROGER 656 00:31:43,520 --> 00:31:50,920 ALSO WON THE NOBEL PRIZE. 657 00:31:50,920 --> 00:31:52,880 THAT WAS MEANT TO WAKE EVERYBODY 658 00:31:52,880 --> 00:31:54,320 UP SO YOU COULD HAVE QUESTIONS. 659 00:31:54,320 --> 00:31:57,240 >>I'M HAPPY TO TAKE YOUR OFFER 660 00:31:57,240 --> 00:31:59,320 TO SHARE A COUPLE OF EXPRESSIONS 661 00:31:59,320 --> 00:32:01,080 OF ENTHUSIASM FOR THE RESEARCH 662 00:32:01,080 --> 00:32:03,520 HIGHLIGHTED SO IT'S GREAT TO SEE 663 00:32:03,520 --> 00:32:05,200 ALL THE PROGRESS. 664 00:32:05,200 --> 00:32:06,200 WE WERE PLEASED YOU HIGHLIGHTED 665 00:32:06,200 --> 00:32:09,400 THE ARTICLE BY LEE AND THERE WAS 666 00:32:09,400 --> 00:32:12,040 ANOTHER BY KRUEGER, WITH STEPHEN 667 00:32:12,040 --> 00:32:15,680 ZIMMERMAN AS SENIOR AUTHOR ON 668 00:32:15,680 --> 00:32:18,160 SOME OF THE MYOTONIC DYSTROPHY 669 00:32:18,160 --> 00:32:19,800 RESEARCH AND REALLY HAPPY TO SEE 670 00:32:19,800 --> 00:32:21,760 KIND OF PROGRESS IN THAT AREA 671 00:32:21,760 --> 00:32:24,200 USING A SMALL MOLECULE THAT 672 00:32:24,200 --> 00:32:27,240 TARGET DNA SEQUENCE, THAT SEEMS 673 00:32:27,240 --> 00:32:28,560 REALLY EXCITING. 674 00:32:28,560 --> 00:32:31,840 AS WAS, AMONG OTHERS, THE 675 00:32:31,840 --> 00:32:33,160 ARTICLE THAT WAS HIGHLIGHTED 676 00:32:33,160 --> 00:32:41,320 ABOUT USING A microRNA TO 677 00:32:41,320 --> 00:32:43,080 INHIBIT DUX4 EXPRESSION IN 678 00:32:43,080 --> 00:32:43,320 CONTEXT. 679 00:32:43,320 --> 00:32:44,920 REALLY EXCITING TO SEE ALL THIS 680 00:32:44,920 --> 00:32:45,320 PROGRESS. 681 00:32:45,320 --> 00:32:48,240 THANK YOU FOR HIGHLIGHTING THOSE 682 00:32:48,240 --> 00:32:49,080 IN THIS SESSION. 683 00:32:49,080 --> 00:32:52,280 >>THANK YOU FOR YOUR COMMENTS. 684 00:32:52,280 --> 00:32:54,760 WALTER? 685 00:32:54,760 --> 00:32:55,080 686 00:32:55,080 --> 00:32:57,440 >>YES, THANK YOU, DIANA. 687 00:32:57,440 --> 00:32:59,520 I WONDERED, DO YOU HAVE ANY 688 00:32:59,520 --> 00:33:00,560 CHILDREN IN SCIENCE THAT COULD 689 00:33:00,560 --> 00:33:05,240 WIN THE NOBEL PRIZE WITH YOU? 690 00:33:05,240 --> 00:33:05,600 [LAUGHTER] 691 00:33:05,600 --> 00:33:06,600 >>I DO NOT. 692 00:33:06,600 --> 00:33:08,600 >>YES, SO I GUESS I JUST WANTED 693 00:33:08,600 --> 00:33:12,600 TO THROW OUT TO THE GROUP THIS 694 00:33:12,600 --> 00:33:15,240 PAPER POINTED OUT ABOUT THE 695 00:33:15,240 --> 00:33:17,440 LONG-TERM MAINTENANCE OF 696 00:33:17,440 --> 00:33:25,040 EXPRESSION AND RESISTANCE, 697 00:33:25,040 --> 00:33:26,360 INJURY. 698 00:33:26,360 --> 00:33:28,360 THIS IS USING THE CRISPR/CAS9 699 00:33:28,360 --> 00:33:29,760 GENE EDITING TOOLS THAT EXPLODED 700 00:33:29,760 --> 00:33:34,560 ON THE SCENE IN THE LAST TEN 701 00:33:34,560 --> 00:33:35,960 YEARS, ANOTHER NOBEL PRIZE WENT 702 00:33:35,960 --> 00:33:41,520 TO THE DISCOVERY THERE. 703 00:33:41,520 --> 00:33:43,000 AND THIS ENABLES -- REALLY WILL 704 00:33:43,000 --> 00:33:44,400 ENABLE A CORRECTION OF GENE 705 00:33:44,400 --> 00:33:47,240 DEFECTS BECAUSE YOU CAN GO IN 706 00:33:47,240 --> 00:33:49,080 PRECISELY FIND THE MUTATION AREA 707 00:33:49,080 --> 00:33:52,400 AND CUT IT OUT OR MAYBE EVEN 708 00:33:52,400 --> 00:33:57,080 REPLACE IT WITH NORMAL CODONS. 709 00:33:57,080 --> 00:33:59,920 AND SO, YOU KNOW, I THINK FOR A 710 00:33:59,920 --> 00:34:02,120 LOT OF GENETIC DISEASES IT'S 711 00:34:02,120 --> 00:34:03,320 KIND OF THE -- YOU KNOW, IT'S 712 00:34:03,320 --> 00:34:05,600 GETTING RIGHT AT THE TARGET OF 713 00:34:05,600 --> 00:34:09,680 THE PROBLEM, GOT TO BE DONE 714 00:34:09,680 --> 00:34:10,680 EARLY. 715 00:34:10,680 --> 00:34:14,360 BUT TO SAY THAT THE COMMON FUND 716 00:34:14,360 --> 00:34:16,480 AT NIH, WHICH IS MONEY THAT 717 00:34:16,480 --> 00:34:18,840 COMES TO THE DIRECTOR, ONE OF 718 00:34:18,840 --> 00:34:21,480 THE BIG PROGRAMS IN THE COMMON 719 00:34:21,480 --> 00:34:25,440 FUND IS ON SOMATIC CELL GENE 720 00:34:25,440 --> 00:34:28,040 EDITING, IT STARTED OUT FIVE 721 00:34:28,040 --> 00:34:29,680 YEARS AGO DEVELOPING THE TOOLS 722 00:34:29,680 --> 00:34:32,320 BUT AS I SAID, THE FIELD IS 723 00:34:32,320 --> 00:34:32,840 EXPLODING. 724 00:34:32,840 --> 00:34:34,400 AND THERE'S A SENSE THAT SOME OF 725 00:34:34,400 --> 00:34:36,720 THESE TOOLS WITHIN THE NEXT FIVE 726 00:34:36,720 --> 00:34:39,880 YEARS WILL BE READY TO BE USED 727 00:34:39,880 --> 00:34:40,720 IN PEOPLE. 728 00:34:40,720 --> 00:34:45,480 THERE HAVE BEEN SOME WORK DONE 729 00:34:45,480 --> 00:34:47,240 ON GENE EDITING OF CELLS TAKEN 730 00:34:47,240 --> 00:34:50,320 OUT OF THE PERSON, AND EDITED 731 00:34:50,320 --> 00:34:57,840 AND GIVEN BACK, OF COURSE WITH 732 00:34:57,840 --> 00:34:58,440 DUCHENNE'S MUSCULAR DYSTROPHY, 733 00:34:58,440 --> 00:35:01,800 IN THIS CASE IT WAS A VIRAL 734 00:35:01,800 --> 00:35:04,080 VECTOR TO DELIVER THE 735 00:35:04,080 --> 00:35:06,160 CRISPR/CAS9 MACHINERY, AND BUT 736 00:35:06,160 --> 00:35:07,880 IN SOMATIC GENE EDITING PROGRAM 737 00:35:07,880 --> 00:35:11,640 THERE'S A CALL FOR ACTUAL GRANTS 738 00:35:11,640 --> 00:35:13,920 THAT GO INTO PATIENTS BY THE END 739 00:35:13,920 --> 00:35:16,800 OF THE PROGRAM, SO THIS IS, YOU 740 00:35:16,800 --> 00:35:20,000 KNOW, KIND OF FUTURISTIC BUT THE 741 00:35:20,000 --> 00:35:22,680 FUTURE IS HERE ACTUALLY. 742 00:35:22,680 --> 00:35:23,400 743 00:35:23,400 --> 00:35:24,680 >>THANK YOU, WALTER. 744 00:35:24,680 --> 00:35:26,520 I SHOULD ALSO SAY THAT LATER 745 00:35:26,520 --> 00:35:29,840 THIS WEEK, I'LL BE ATTENDING AND 746 00:35:29,840 --> 00:35:32,200 PRESENTING AT THE FIRST 747 00:35:32,200 --> 00:35:34,520 INTERNATIONAL CONFERENCE ON 748 00:35:34,520 --> 00:35:35,760 NEWBORN SEQUENCING, WHICH IS 749 00:35:35,760 --> 00:35:39,960 GOING TO RAISE A LOT OF VERY 750 00:35:39,960 --> 00:35:43,080 INTERESTING QUESTIONS. 751 00:35:43,080 --> 00:35:44,920 NEWBORN SEQUENCING IS 752 00:35:44,920 --> 00:35:46,760 INCREASINGLY BEING USED 753 00:35:46,760 --> 00:35:48,120 CLINICALLY IN MANY NEWBORN 754 00:35:48,120 --> 00:35:50,360 ICUs AROUND THE COUNTRY. 755 00:35:50,360 --> 00:35:52,760 AND IN FACT I THINK THERE ARE 756 00:35:52,760 --> 00:35:54,440 NOW FIVE STATE MEDICAID PROGRAMS 757 00:35:54,440 --> 00:35:56,760 THAT ARE PAYING FOR IT. 758 00:35:56,760 --> 00:36:01,880 BUT THE QUESTION IS WHETHER TO 759 00:36:01,880 --> 00:36:04,040 OFFER THIS TO INFANTS WHO ARE 760 00:36:04,040 --> 00:36:06,120 HEALTHY AND WHETHER IT SHOULD BE 761 00:36:06,120 --> 00:36:10,320 ADDED ON TO EXISTING STATE-BASED 762 00:36:10,320 --> 00:36:11,880 NEWBORN SCREENING PROGRAMS. 763 00:36:11,880 --> 00:36:13,680 AND SO IT'S DEFINITELY GOING TO 764 00:36:13,680 --> 00:36:15,960 INTERSECT WITH THE WORLD OF 765 00:36:15,960 --> 00:36:17,120 MUSCULAR DYSTROPHY BECAUSE 766 00:36:17,120 --> 00:36:20,160 EITHER THERE WILL BE ADDITIONAL 767 00:36:20,160 --> 00:36:21,040 DIAGNOSES OF WELL-KNOWN 768 00:36:21,040 --> 00:36:23,680 CONDITIONS OR IT'S POSSIBLE THAT 769 00:36:23,680 --> 00:36:26,080 ADDITIONAL GENE MUTATIONS WILL 770 00:36:26,080 --> 00:36:29,600 BE FOUND THAT WILL BE ASSOCIATED 771 00:36:29,600 --> 00:36:30,880 EVENTUALLY CLINICALLY WITH 772 00:36:30,880 --> 00:36:33,200 CONDITIONS THAT PERHAPS ARE NOT 773 00:36:33,200 --> 00:36:35,640 RIGHT NOW RECOGNIZED AS BEING 774 00:36:35,640 --> 00:36:38,560 PART OF THE FAMILY OF MUSCULAR 775 00:36:38,560 --> 00:36:39,320 DYSTROPHIES. 776 00:36:39,320 --> 00:36:41,440 BUT THAT AREA IS PROGRESSING 777 00:36:41,440 --> 00:36:43,080 VERY QUICKLY AS WELL FROM 778 00:36:43,080 --> 00:36:45,480 RESEARCH THAT HAS BEEN SUPPORTED 779 00:36:45,480 --> 00:36:47,880 BY NIH TO CLINICAL 780 00:36:47,880 --> 00:36:49,320 IMPLEMENTATION IN MANY NEWBORN 781 00:36:49,320 --> 00:36:52,240 ICUs IN THE COUNTRY. 782 00:36:52,240 --> 00:36:57,160 783 00:36:57,160 --> 00:37:04,040 PEOPLE ARE A LITTLE QUIET TODAY. 784 00:37:04,040 --> 00:37:06,000 I DON'T KNOW, COLD WEATHER? 785 00:37:06,000 --> 00:37:07,880 WE'LL GIVE YOU THE THREE MINUTES 786 00:37:07,880 --> 00:37:08,880 BACK IF YOU DON'T HAVE 787 00:37:08,880 --> 00:37:09,360 QUESTIONS. 788 00:37:09,360 --> 00:37:11,720 ONE THING IN THE CHAT BOX? 789 00:37:11,720 --> 00:37:13,240 NO, JUST INSTRUCTIONS YOU CAN 790 00:37:13,240 --> 00:37:16,320 RAISE YOUR HAND OR ASK A 791 00:37:16,320 --> 00:37:17,200 QUESTION. 792 00:37:17,200 --> 00:37:20,040 >>WE'VE HAD TWO COMMITTEE 793 00:37:20,040 --> 00:37:22,000 MEMBERS THAT HAVE JOINED SINCE 794 00:37:22,000 --> 00:37:24,320 THE INTRODUCTION SO WE'LL GIVE 795 00:37:24,320 --> 00:37:25,560 ERIC LANG AND OPPORTUNITY TO 796 00:37:25,560 --> 00:37:30,200 INTRODUCE HIMSELF. 797 00:37:30,200 --> 00:37:32,800 >>THIS IS ERIC LANG, MY DAY JOB 798 00:37:32,800 --> 00:37:34,880 IS ASSOCIATE PROFESSOR AT 799 00:37:34,880 --> 00:37:36,440 UNIVERSITY OF FLORIDA, I STUDY 800 00:37:36,440 --> 00:37:38,080 ASPECTS OF GENE REGULATION AND 801 00:37:38,080 --> 00:37:40,480 NEUROMUSCULAR DISEASE WITH FOCUS 802 00:37:40,480 --> 00:37:41,920 ON MYOTONIC DYSTROPHY, AND WHAT 803 00:37:41,920 --> 00:37:43,800 INITIALLY BROUGHT ME INTO THIS 804 00:37:43,800 --> 00:37:46,400 SPACE WAS HAVING FAMILY MEMBERS 805 00:37:46,400 --> 00:37:49,280 AFFECTED AND SO I'M ALSO AN 806 00:37:49,280 --> 00:37:51,440 ADVOCATE FOR THE MYOTONIC 807 00:37:51,440 --> 00:37:54,480 DYSTROPHY FOUNDATION, SERVE ON 808 00:37:54,480 --> 00:37:56,120 SCIENTIFIC ADVISORY COMMITTEE 809 00:37:56,120 --> 00:37:58,960 WITH THEM AND PARTICIPATE IN A 810 00:37:58,960 --> 00:38:02,920 VARIETY OF WAYS TO PUSH MYOTONIC 811 00:38:02,920 --> 00:38:05,880 DYSTROPHY RESEARCH AND PATIENT 812 00:38:05,880 --> 00:38:07,440 CARE TOPICS FORWARD. 813 00:38:07,440 --> 00:38:11,320 >>YOU'RE IN GAINESVILLE, RIGHT? 814 00:38:11,320 --> 00:38:15,360 YOU WERE AFFECTED? 815 00:38:15,360 --> 00:38:16,000 >>COMPLETELY UNSCATHED 816 00:38:16,000 --> 00:38:18,000 FORTUNATELY, YEAH. 817 00:38:18,000 --> 00:38:20,080 >>THANKS, ERIC. 818 00:38:20,080 --> 00:38:21,840 GLAD YOU'RE WELL. 819 00:38:21,840 --> 00:38:23,160 GENE FREUND? 820 00:38:23,160 --> 00:38:24,680 >>GENE FREUND, MEDICAL OFFICER 821 00:38:24,680 --> 00:38:26,760 WITH THE CENTERS FOR MEDICARE 822 00:38:26,760 --> 00:38:28,920 AND MEDICAID SERVICES. 823 00:38:28,920 --> 00:38:31,920 ACTUALLY IN CINCINNATI VISITING 824 00:38:31,920 --> 00:38:33,720 MY MOM, THIS WEEK, SO BACK ON 825 00:38:33,720 --> 00:38:36,280 DUTY FOR AN AFTERNOON TO VISIT 826 00:38:36,280 --> 00:38:38,160 WITH YOU, AND HAPPY TO DO THAT. 827 00:38:38,160 --> 00:38:39,760 ONE OF THE NICE THINGS OR ONE OF 828 00:38:39,760 --> 00:38:41,320 THE SIDE EFFECTS OF HAVING A 829 00:38:41,320 --> 00:38:43,520 NAME LIKE GENE IS IF MY 830 00:38:43,520 --> 00:38:47,240 ATTENTION WANDERS I GET SNAPPED 831 00:38:47,240 --> 00:38:48,440 BACK INTO THE CONVERSATION 832 00:38:48,440 --> 00:38:50,800 FREQUENTLY WHEN YOU START 833 00:38:50,800 --> 00:38:53,440 TALKING ABOUT THAT. 834 00:38:53,440 --> 00:38:54,880 I'VE HAD COLLEAGUES DO GENE 835 00:38:54,880 --> 00:38:56,960 EDITING BUT NOT THE KIND DR. 836 00:38:56,960 --> 00:38:57,880 BIANCHI WAS TALKING ABOUT. 837 00:38:57,880 --> 00:38:59,480 GOOD TO BE HERE, ALWAYS 838 00:38:59,480 --> 00:39:06,160 INTERESTING TO HEAR WHAT'S GOING 839 00:39:06,160 --> 00:39:06,720 ON. 840 00:39:06,720 --> 00:39:08,600 >>GENE, YOU'VE BEEN EXTREMELY 841 00:39:08,600 --> 00:39:12,000 HELPFUL INFORMING US ABOUT CMS 842 00:39:12,000 --> 00:39:15,560 COVERAGE FOR BIPAP AND OTHER 843 00:39:15,560 --> 00:39:19,320 ASSOCIATIVE DEVICES FOR 844 00:39:19,320 --> 00:39:21,520 BREATHING. 845 00:39:21,520 --> 00:39:24,360 WE APPRECIATE YOUR PRESENCE AND 846 00:39:24,360 --> 00:39:24,640 ATTENTION. 847 00:39:24,640 --> 00:39:28,960 >>COVERAGE AND DRUG PROVISIONS 848 00:39:28,960 --> 00:39:31,080 OF DRUG REDUCTION ACT ARE BEING 849 00:39:31,080 --> 00:39:32,840 FORMED BUT AT A FUTURE 850 00:39:32,840 --> 00:39:33,800 PRESENTATION I SUSPECT THAT 851 00:39:33,800 --> 00:39:35,880 MIGHT BE -- FUTURE MEETING, THAT 852 00:39:35,880 --> 00:39:36,920 MIGHT BE SUBJECT FOR 853 00:39:36,920 --> 00:39:39,000 PRESENTATION TO THIS GROUP. 854 00:39:39,000 --> 00:39:40,720 >>THAT WOULD BE GREAT. 855 00:39:40,720 --> 00:39:43,160 >>QUITE PREMATURE NOW BUT IT 856 00:39:43,160 --> 00:39:45,440 COULD BE WORTH GOING TO THE 857 00:39:45,440 --> 00:39:45,960 GROUP LATER. 858 00:39:45,960 --> 00:39:48,960 >>RIGHT. 859 00:39:48,960 --> 00:39:50,680 THANK YOU SO MUCH. 860 00:39:50,680 --> 00:39:53,520 GLEN, WE'RE EXACTLY ON TIME, IF 861 00:39:53,520 --> 00:39:55,080 YOU'RE OKAY WITH MOVING FORWARD. 862 00:39:55,080 --> 00:39:57,600 >>PLEASE DO. 863 00:39:57,600 --> 00:39:58,240 THANK YOU. 864 00:39:58,240 --> 00:39:59,000 >>THANK YOU ALL. 865 00:39:59,000 --> 00:40:02,280 EACH OF THE NIH INSTITUTES AND 866 00:40:02,280 --> 00:40:05,120 CENTERS HAVE A STRATEGIC PLAN 867 00:40:05,120 --> 00:40:06,520 THAT IS PERIODICALLY REVIEWED 868 00:40:06,520 --> 00:40:08,280 AND UPDATED, ACTUALLY SPECIFIED 869 00:40:08,280 --> 00:40:10,360 IN THE 21ST CENTURY CURES ACT. 870 00:40:10,360 --> 00:40:13,120 THE NATIONAL INSTITUTE OF 871 00:40:13,120 --> 00:40:14,400 ARTHRITIS AND MUSCULOSKELETAL 872 00:40:14,400 --> 00:40:17,040 AND SKIN DISEASES WILL BE 873 00:40:17,040 --> 00:40:21,080 RENEWING THEIR STRATEGIC PLAN 874 00:40:21,080 --> 00:40:23,720 FOR 2025-2029 AND DR. LINDSEY 875 00:40:23,720 --> 00:40:24,800 CRISWELL, DIRECTOR OF NIAMS, 876 00:40:24,800 --> 00:40:27,560 WILL DESCRIBE TO US HOW YOU ALL 877 00:40:27,560 --> 00:40:30,200 CAN PROVIDE INPUT. 878 00:40:30,200 --> 00:40:31,640 LINDSEY, OVER TO YOU. 879 00:40:31,640 --> 00:40:32,880 >>THANK YOU. 880 00:40:32,880 --> 00:40:39,480 LET ME START BY SHARING MY 881 00:40:39,480 --> 00:40:40,040 SCREEN. 882 00:40:40,040 --> 00:40:43,720 CAN YOU ALL SEE THAT? 883 00:40:43,720 --> 00:40:44,160 OKAY. 884 00:40:44,160 --> 00:40:44,520 ALL RIGHT. 885 00:40:44,520 --> 00:40:45,800 SO THANK YOU FOR THE 886 00:40:45,800 --> 00:40:46,120 INTRODUCTION. 887 00:40:46,120 --> 00:40:48,520 IT'S REALLY A PLEASURE TO BE 888 00:40:48,520 --> 00:40:49,080 HERE. 889 00:40:49,080 --> 00:40:50,960 SO AS I JUST HEARD, YES, WE'RE 890 00:40:50,960 --> 00:40:52,680 BEGINNING TO THINK ABOUT OUR 891 00:40:52,680 --> 00:40:56,320 STRATEGIC PLAN FOR FISCAL YEARS 892 00:40:56,320 --> 00:40:59,400 2025 TO 29, I'D LIKE TO DESCRIBE 893 00:40:59,400 --> 00:41:01,320 HOW THIS WILL DIFFER FROM 894 00:41:01,320 --> 00:41:03,120 CURRENT PLAN AND HOW YOU CAN 895 00:41:03,120 --> 00:41:05,920 CONTRIBUTE. 896 00:41:05,920 --> 00:41:08,120 SO, AS YOU HEARD ALSO, ALL 897 00:41:08,120 --> 00:41:09,240 INSTITUTES ARE REQUIRED TO HAVE 898 00:41:09,240 --> 00:41:10,720 A STRATEGIC PLAN BY LAW, THEY 899 00:41:10,720 --> 00:41:13,280 HAVE GREAT VALUE AS A WAY OF 900 00:41:13,280 --> 00:41:14,360 COMMUNICATING THE INSTITUTE'S 901 00:41:14,360 --> 00:41:16,360 INTEREST TO THE PUBLIC, TO THE 902 00:41:16,360 --> 00:41:18,720 RESEARCH COMMUNITY, ALSO TO THE 903 00:41:18,720 --> 00:41:19,200 NIH. 904 00:41:19,200 --> 00:41:21,080 SO, THIS WILL REPRESENT A PUBLIC 905 00:41:21,080 --> 00:41:22,360 STATEMENT OF WHERE NIAMS CAN BE 906 00:41:22,360 --> 00:41:24,360 BEST POSITIONED OVER THE NEXT 907 00:41:24,360 --> 00:41:25,200 FIVE YEARS, TO MAKE A DIFFERENCE 908 00:41:25,200 --> 00:41:27,960 IN THE LIVES OF ALL AMERICANS. 909 00:41:27,960 --> 00:41:29,280 BUT IMPORTANTLY THE PLAN ALSO 910 00:41:29,280 --> 00:41:30,800 SERVES AS GUIDE POST FOR 911 00:41:30,800 --> 00:41:32,800 RESEARCHERS, SO IT NEEDS TO 912 00:41:32,800 --> 00:41:35,160 COVER THE INSTITUTE'S BROAD 913 00:41:35,160 --> 00:41:38,640 MISSION AREAS OF ARTHRITIS, 914 00:41:38,640 --> 00:41:40,440 MUSCULOSKELETAL AND SKIN 915 00:41:40,440 --> 00:41:40,880 DISEASES. 916 00:41:40,880 --> 00:41:42,840 SO, WITH INPUT FROM NIAMS 917 00:41:42,840 --> 00:41:44,720 ADVISORY COUNCIL AND PUBLIC, 918 00:41:44,720 --> 00:41:45,880 NIAMS DEVELOPED CURRENT 919 00:41:45,880 --> 00:41:47,880 STRATEGIC PLAN COVERING FISCAL 920 00:41:47,880 --> 00:41:51,920 YEARS 2020 TO 24, AND THE 921 00:41:51,920 --> 00:41:54,760 ULTIMATE GOAL TO DEVELOP PATIENT 922 00:41:54,760 --> 00:41:56,040 CENTERED WAYS TO IMPROVE 923 00:41:56,040 --> 00:42:00,480 OUTCOMES IN FIVE CORE AREAS, 924 00:42:00,480 --> 00:42:02,800 LISTED ON THE SLIDE, RHEUMATIC 925 00:42:02,800 --> 00:42:04,240 AND AUTOIMMUNE DISEASES, SKIN 926 00:42:04,240 --> 00:42:06,320 BIOLOGY AND DISEASES, BONE 927 00:42:06,320 --> 00:42:08,400 BIOLOGY AND DISEASES, MUSCLE 928 00:42:08,400 --> 00:42:10,040 BIOLOGY AND DISEASES, JOINT 929 00:42:10,040 --> 00:42:13,560 BIOLOGY AND DISEASES AS WELL AS 930 00:42:13,560 --> 00:42:14,320 ORTHOPEDICS. 931 00:42:14,320 --> 00:42:17,080 NOW, CONSIDERING THE AREAS 932 00:42:17,080 --> 00:42:18,760 WITHIN THIS BROAD PURVIEW, I 933 00:42:18,760 --> 00:42:25,560 UNDERSCORE THE TREMENDOUS 934 00:42:25,560 --> 00:42:26,400 BURDEN. 935 00:42:26,400 --> 00:42:29,120 , AFFECTING PEOPLE OF ALL AGES 936 00:42:29,120 --> 00:42:30,080 AND ALL BACKGROUNDS, COMBINED 937 00:42:30,080 --> 00:42:34,240 AFFECT TENS OF MILLIONS OF 938 00:42:34,240 --> 00:42:35,000 AMERICANS, CAUSE TREMENDOUS 939 00:42:35,000 --> 00:42:37,800 SUFFERING, AFFECT U.S. ECONOMY 940 00:42:37,800 --> 00:42:39,280 THROUGH IMPRESSIVE HEALTH CARE 941 00:42:39,280 --> 00:42:41,600 COSTS AS WELL AS LOST 942 00:42:41,600 --> 00:42:47,440 PRODUCTIVITY. 943 00:42:47,440 --> 00:42:48,400 MOST ARE CHRONIC, MANY 944 00:42:48,400 --> 00:42:50,640 ASSOCIATED WITH CHRONIC PAIN AND 945 00:42:50,640 --> 00:42:51,680 DISABILITY, MANY WHETHER COMMON 946 00:42:51,680 --> 00:42:54,400 OR RARE AFFECT WOMEN AND 947 00:42:54,400 --> 00:42:55,040 MINORITIES DISPROPORTIONATELY, 948 00:42:55,040 --> 00:42:57,600 BOTH IN TERMS OF INCREASED 949 00:42:57,600 --> 00:43:02,080 NUMBERS, AS WELL AS INCREASED 950 00:43:02,080 --> 00:43:02,400 SEVERITY. 951 00:43:02,400 --> 00:43:04,840 THE CURRENT STRATEGIC PLAN PLAN 952 00:43:04,840 --> 00:43:05,600 HIGHLIGHTS SEVERAL SCIENTIFIC 953 00:43:05,600 --> 00:43:10,080 THEMES THAT CUT ACROSS ALL OUR 954 00:43:10,080 --> 00:43:12,400 MISSION AREAS, NEW TECHNOLOGIES 955 00:43:12,400 --> 00:43:14,440 TO IDENTIFY SHARED MECHANISMS IN 956 00:43:14,440 --> 00:43:21,240 HEALTH AND AMONG DISEASES, 957 00:43:21,240 --> 00:43:21,800 ADVANCING PATIENT-CENTRIC 958 00:43:21,800 --> 00:43:22,680 APPROACHES, ENABLING PRECISION 959 00:43:22,680 --> 00:43:23,960 MEDICINE TO TAILOR CARE, 960 00:43:23,960 --> 00:43:26,280 ADDRESSING HEALTH NEEDS OF 961 00:43:26,280 --> 00:43:27,160 DIVERSE POPULATIONS. 962 00:43:27,160 --> 00:43:30,160 THESE WERE TOPICS THAT EMERGED 963 00:43:30,160 --> 00:43:31,480 FROM NIAMS LISTENING SESSIONS 964 00:43:31,480 --> 00:43:36,480 HELD IN EACH OF OUR DISEASE AND 965 00:43:36,480 --> 00:43:37,720 TISSUE-SPECIFIC AREAS. 966 00:43:37,720 --> 00:43:39,040 WHEREAS THE MAJORITY OF CURRENT 967 00:43:39,040 --> 00:43:41,360 PLAN CONSISTS OF OUR FIVE 968 00:43:41,360 --> 00:43:42,160 DISEASE AND TISSUE-SPECIFIC 969 00:43:42,160 --> 00:43:43,840 INTERESTS, THE NEW PLAN WILL 970 00:43:43,840 --> 00:43:46,000 FOCUS MORE ON THE CROSS-CUTTING 971 00:43:46,000 --> 00:43:46,600 THEMATIC RESEARCH OPPORTUNITIES 972 00:43:46,600 --> 00:43:49,120 THAT TOUCH ON ALL OF OUR DISEASE 973 00:43:49,120 --> 00:43:49,680 AREAS. 974 00:43:49,680 --> 00:43:52,280 WE STILL WANT TO REFERENCE OUR 975 00:43:52,280 --> 00:43:53,520 DISEASE AND TISSUE-SPECIFIC 976 00:43:53,520 --> 00:43:54,400 INTERESTS BUT WILL SHORTEN THIS 977 00:43:54,400 --> 00:43:57,040 PART OF THE PLAN AND MAKE SURE 978 00:43:57,040 --> 00:43:58,440 THE SECTIONS ARE CONSISTENT WITH 979 00:43:58,440 --> 00:44:00,640 REGARDS TO LEVEL OF DETAIL 980 00:44:00,640 --> 00:44:00,880 INCLUDED. 981 00:44:00,880 --> 00:44:03,520 BUT WE ALSO WANT TO HIGHLIGHT 982 00:44:03,520 --> 00:44:04,520 IMPORTANTLY OUR INTRAMURAL 983 00:44:04,520 --> 00:44:06,120 RESEARCH PROGRAM, SO IT WILL 984 00:44:06,120 --> 00:44:10,960 RECEIVE ITS OWN TWO-PAGE SECTION 985 00:44:10,960 --> 00:44:13,320 IN THIS NEW PLAN. 986 00:44:13,320 --> 00:44:14,720 NIAMS IS REQUESTING FEEDBACK ON 987 00:44:14,720 --> 00:44:16,600 TOPICS INCLUDED IN THE PLAN FROM 988 00:44:16,600 --> 00:44:19,440 INTERESTED MEMBERS OF THE PUBLIC 989 00:44:19,440 --> 00:44:20,400 INCLUDING RESEARCHERS IN 990 00:44:20,400 --> 00:44:23,920 ACADEMIA AND INDUSTRY, HEALTH 991 00:44:23,920 --> 00:44:26,560 CARE PROFESSIONALS, PATIENT 992 00:44:26,560 --> 00:44:27,640 ADVOCATES, HEALTH ADVOCACY 993 00:44:27,640 --> 00:44:28,640 ORGANIZATIONS, SCIENTIFIC OR 994 00:44:28,640 --> 00:44:30,240 PROFESSIONAL ORGANIZATIONS, AS 995 00:44:30,240 --> 00:44:32,160 WELL AS FEDERAL AGENTS. 996 00:44:32,160 --> 00:44:34,240 AND WE'RE SEEKING INPUT BOTH 997 00:44:34,240 --> 00:44:36,480 ABOUT CROSS-CUTTING THEMES AS 998 00:44:36,480 --> 00:44:38,080 WELL AS BOLD ASPIRATIONS. 999 00:44:38,080 --> 00:44:39,640 AND BROAD COMMUNITY INPUT IS 1000 00:44:39,640 --> 00:44:41,560 ESSENTIAL TO NOT ONLY IMPROVE 1001 00:44:41,560 --> 00:44:43,120 RESEARCH BUT ALSO TO ENSURE 1002 00:44:43,120 --> 00:44:44,640 REPRESENTATION FOR ALL AND 1003 00:44:44,640 --> 00:44:51,200 ADDRESS HEALTH DISPARITIES AND 1004 00:44:51,200 --> 00:44:51,520 HEALTH EQUITY. 1005 00:44:51,520 --> 00:44:53,480 I HOPE YOU'LL SHARE IDEAS AND 1006 00:44:53,480 --> 00:44:55,440 SHARE THIS ANNOUNCEMENT. 1007 00:44:55,440 --> 00:44:56,320 NOTICE THE VIDEO REFERENCE 1008 00:44:56,320 --> 00:44:58,280 NUMBER AT THE TOP OF THE PAGE, 1009 00:44:58,280 --> 00:45:01,720 WHERE YOU CAN FIND INFORMATION 1010 00:45:01,720 --> 00:45:02,600 ABOUT OUR RFI. 1011 00:45:02,600 --> 00:45:05,240 SO YOU CAN RESPOND TO THE RFI BY 1012 00:45:05,240 --> 00:45:06,440 SEARCHING ON THAT NOTICE NUMBER 1013 00:45:06,440 --> 00:45:08,920 THAT I SHARED WITH YOU, OR BY 1014 00:45:08,920 --> 00:45:11,120 GOING TO THE NIAMS HOME PAGE, 1015 00:45:11,120 --> 00:45:14,680 YOU'LL BE ASKED TO PROVIDE FOUR 1016 00:45:14,680 --> 00:45:16,760 PIECES OF INFORMATION. 1017 00:45:16,760 --> 00:45:17,880 CROSS-CUTTING RESEARCH 1018 00:45:17,880 --> 00:45:21,680 OPPORTUNITIES THAT NIAMS SHOULD 1019 00:45:21,680 --> 00:45:24,560 CONSIDER, SECOND, BOLD 1020 00:45:24,560 --> 00:45:25,880 ASPIRATIONS NIAMS-SUPPORTED 1021 00:45:25,880 --> 00:45:27,320 RESEARCH COULD ACCOMPLISH, YOUR 1022 00:45:27,320 --> 00:45:29,040 NAME AND YOUR ORGANIZATION. 1023 00:45:29,040 --> 00:45:30,760 INDIVIDUALS ARE WELCOME TO 1024 00:45:30,760 --> 00:45:31,560 RESPOND BUT ORGANIZATIONS ARE 1025 00:45:31,560 --> 00:45:32,880 ALSO WELCOME TO RESPOND ON 1026 00:45:32,880 --> 00:45:34,280 BEHALF OF THEIR GROUPS. 1027 00:45:34,280 --> 00:45:35,680 AND THE RESPONSES WILL BE DUE BY 1028 00:45:35,680 --> 00:45:40,640 THE END OF NOVEMBER OF THIS 1029 00:45:40,640 --> 00:45:41,080 YEAR. 1030 00:45:41,080 --> 00:45:44,480 AS THE NEXT STEP WE'LL ANALYZED 1031 00:45:44,480 --> 00:45:47,320 RESULTS AND PORTFOLIO ANALYSIS 1032 00:45:47,320 --> 00:45:48,400 THAT WE'RE ALSO UNDERTAKING. 1033 00:45:48,400 --> 00:45:51,240 WE WILL DISCUSS THE FINDINGS 1034 00:45:51,240 --> 00:45:54,640 WITH OUR ADVISORY COUNCIL, AND 1035 00:45:54,640 --> 00:45:56,920 HOLD LISTENING SESSIONS TO 1036 00:45:56,920 --> 00:45:58,440 REFINE THEMES WE HEARD, 1037 00:45:58,440 --> 00:45:59,640 INCLUDING REPRESENTATIVES FROM 1038 00:45:59,640 --> 00:46:01,200 BOTH SCIENTIFIC AND PATIENT 1039 00:46:01,200 --> 00:46:02,400 COMMUNITIES AND WILL BE HELD 1040 00:46:02,400 --> 00:46:04,160 DURING THE NEXT CALENDAR YEAR. 1041 00:46:04,160 --> 00:46:06,280 AS A FINAL STEP WE'LL POST NEAR 1042 00:46:06,280 --> 00:46:08,840 FINAL VERSION OF THE PLAN FOR 1043 00:46:08,840 --> 00:46:12,720 PUBLIC COMMENT AND AGAIN INVITE 1044 00:46:12,720 --> 00:46:14,080 FEEDBACK THROUGH RFI. 1045 00:46:14,080 --> 00:46:16,520 SO, IN CLOSING I'D LIKE TO THANK 1046 00:46:16,520 --> 00:46:18,160 YOU IN ADVANCE FOR YOUR 1047 00:46:18,160 --> 00:46:20,680 THOUGHTFUL INPUT ON WAYS TO 1048 00:46:20,680 --> 00:46:21,520 COVER EMERGING SCIENTIFIC 1049 00:46:21,520 --> 00:46:25,160 OPPORTUNITIES AND BE RELEVANT TO 1050 00:46:25,160 --> 00:46:28,160 RESEARCHERS ACROSS OUR NIAMS 1051 00:46:28,160 --> 00:46:36,240 MISSIONARIES, THANK -- MISSION 1052 00:46:36,240 --> 00:46:38,640 AREAS. 1053 00:46:38,640 --> 00:46:49,000 LET ME STOP SHARING. 1054 00:46:50,680 --> 00:46:52,560 >>THERE WAS A QUESTION ABOUT 1055 00:46:52,560 --> 00:46:54,400 SHARING THE NUMBER AGAIN. 1056 00:46:54,400 --> 00:46:55,720 >>ARE THESE SLIDES POSTED 1057 00:46:55,720 --> 00:46:56,560 FOLLOWING THE MEETING? 1058 00:46:56,560 --> 00:46:58,120 IF NOT I WILL PUT THAT NOTICE 1059 00:46:58,120 --> 00:46:59,960 NUMBER IN THE CHAT SO EVERYBODY 1060 00:46:59,960 --> 00:47:01,400 CAN SEE THAT. 1061 00:47:01,400 --> 00:47:03,040 SORRY THAT FLEW BY QUICKLY. 1062 00:47:03,040 --> 00:47:04,800 YOU CAN FIND THE INFORMATION AT 1063 00:47:04,800 --> 00:47:06,560 OUR HOME PAGE BUT I WILL PUT 1064 00:47:06,560 --> 00:47:08,400 THAT IN THE CHAT IN JUST A 1065 00:47:08,400 --> 00:47:08,640 MINUTE. 1066 00:47:08,640 --> 00:47:10,800 I HOPE THAT MEANS THERE WAS 1067 00:47:10,800 --> 00:47:14,680 SERIOUS INTEREST IN RESPONDING, 1068 00:47:14,680 --> 00:47:16,280 THAT'S A GOOD SIGN. 1069 00:47:16,280 --> 00:47:18,960 >>THANK YOU, LINDSEY. 1070 00:47:18,960 --> 00:47:27,560 OTHER QUESTIONS? 1071 00:47:27,560 --> 00:47:29,400 >>IS THIS PRETTY COMMON TO 1072 00:47:29,400 --> 00:47:32,720 SOLICIT INPUT FOR THESE INTEREST 1073 00:47:32,720 --> 00:47:32,960 PLANS? 1074 00:47:32,960 --> 00:47:36,320 AND HOW OFTEN DO YOU GET 1075 00:47:36,320 --> 00:47:38,320 FEEDBACK FROM -- HOW MUCH 1076 00:47:38,320 --> 00:47:41,040 FEEDBACK DO YOU GET FROM 1077 00:47:41,040 --> 00:47:41,400 INVESTIGATORS? 1078 00:47:41,400 --> 00:47:46,120 MAYBE FOR MYSELF, I'VE BEEN 1079 00:47:46,120 --> 00:47:47,200 UNAWARE OF THIS MECHANISM. 1080 00:47:47,200 --> 00:47:48,680 >>WE HAD A MEETING THAT TOUCHED 1081 00:47:48,680 --> 00:47:52,440 ON THAT A COUPLE HOURS AGO, AND 1082 00:47:52,440 --> 00:47:53,840 WE'RE TRYING TO -- THE SHORT 1083 00:47:53,840 --> 00:47:55,600 ANSWER IS YES, THIS IS VERY 1084 00:47:55,600 --> 00:47:55,840 COMMON. 1085 00:47:55,840 --> 00:47:58,920 I KNOW IT WAS A VERY IMPORTANT 1086 00:47:58,920 --> 00:48:00,640 COMPONENT OF THE LAST NIAMS 1087 00:48:00,640 --> 00:48:02,040 STRATEGIC PLAN, I WASN'T 1088 00:48:02,040 --> 00:48:02,720 INVOLVED IN THAT. 1089 00:48:02,720 --> 00:48:04,720 AND ONE OF THE THINGS WE'VE BEEN 1090 00:48:04,720 --> 00:48:06,120 THINKING ABOUT IS HOW EXACTLY TO 1091 00:48:06,120 --> 00:48:08,200 GET THE WORD OUT TO THE 1092 00:48:08,200 --> 00:48:10,200 INVESTIGATOR COMMUNITY. 1093 00:48:10,200 --> 00:48:12,920 IN GENERAL, IT'S A CHALLENGING 1094 00:48:12,920 --> 00:48:14,320 COMMUNICATION ENVIRONMENT RIGHT 1095 00:48:14,320 --> 00:48:14,640 NOW. 1096 00:48:14,640 --> 00:48:17,720 WE DON'T WANT TO OVERWHELM FOLKS 1097 00:48:17,720 --> 00:48:19,560 BUT WE'RE SERIOUSLY INTERESTED 1098 00:48:19,560 --> 00:48:20,880 IN RECEIVING FEEDBACK, AND WE 1099 00:48:20,880 --> 00:48:22,760 HOPE THAT YOU ALL CAN HELP US 1100 00:48:22,760 --> 00:48:24,960 SPREAD THE WORD SO WE'RE 1101 00:48:24,960 --> 00:48:26,680 CONTINUING TO THINK ABOUT HOW TO 1102 00:48:26,680 --> 00:48:28,480 BE EFFECTIVE ON THAT FRONT AND I 1103 00:48:28,480 --> 00:48:29,440 WOULD WELCOME ANY SUGGESTIONS 1104 00:48:29,440 --> 00:48:31,160 YOU HAVE OR OTHER INVESTIGATORS 1105 00:48:31,160 --> 00:48:32,640 THAT MIGHT BE LISTENING IN TO 1106 00:48:32,640 --> 00:48:35,720 THIS MEETING. 1107 00:48:35,720 --> 00:48:36,560 >>WALTER, DO YOU WANT TO 1108 00:48:36,560 --> 00:48:39,880 COMMENT ON YOUR EXPERIENCE WITH 1109 00:48:39,880 --> 00:48:41,520 RFIS AT NINDS? 1110 00:48:41,520 --> 00:48:43,880 >>SURE. 1111 00:48:43,880 --> 00:48:47,720 YES, I THINK IT'S PRETTY COMMON 1112 00:48:47,720 --> 00:48:50,360 THAT WE DO THAT, IN A WHOLE 1113 00:48:50,360 --> 00:48:52,000 BUNCH OF DIFFERENT AREAS WHERE 1114 00:48:52,000 --> 00:48:53,960 WE'RE LOOKING TO DEVELOP OUR 1115 00:48:53,960 --> 00:48:54,400 PLANNING. 1116 00:48:54,400 --> 00:48:59,760 RFIS OUT OF NIH ARE VERY COMMON. 1117 00:48:59,760 --> 00:49:06,760 AND, YOU KNOW, I THINK WE WANT 1118 00:49:06,760 --> 00:49:08,320 BROAD INPUT, SO THAT INDIVIDUALS 1119 00:49:08,320 --> 00:49:12,680 SENDING IN THEIR INFORMATION, WE 1120 00:49:12,680 --> 00:49:15,840 USUALLY, YOU KNOW, BUNDLE THEM 1121 00:49:15,840 --> 00:49:18,400 INTO CATEGORIES AND THEY INFORM 1122 00:49:18,400 --> 00:49:19,200 WHAT WE DO. 1123 00:49:19,200 --> 00:49:20,960 WE HAVE LIKE A WORKING GROUP OF 1124 00:49:20,960 --> 00:49:24,240 COUNCIL THAT'S WORKING ON THE 1125 00:49:24,240 --> 00:49:24,720 RECOMMENDATIONS. 1126 00:49:24,720 --> 00:49:27,440 THEY WILL BE SENT ALL THE RFI 1127 00:49:27,440 --> 00:49:27,840 COMMENTS. 1128 00:49:27,840 --> 00:49:31,400 AND THE LAST THING I'D SAY IS 1129 00:49:31,400 --> 00:49:36,040 THAT SOMETIMES THE MOST 1130 00:49:36,040 --> 00:49:37,400 THOUGHTFUL RFI RESPONSES COME 1131 00:49:37,400 --> 00:49:38,840 FROM ORGANIZATIONS THAT ARE 1132 00:49:38,840 --> 00:49:41,120 INTERESTED IN THE WORK THAT WE 1133 00:49:41,120 --> 00:49:44,280 DO HERE IN ONE OF THE 1134 00:49:44,280 --> 00:49:45,040 INSTITUTES, AND THEY OFTENTIMES 1135 00:49:45,040 --> 00:49:47,400 INFORM A LITTLE COMMITTEE AND 1136 00:49:47,400 --> 00:49:51,600 THEY WRITE OR, YOU KNOW, A SIX, 1137 00:49:51,600 --> 00:49:54,480 SEVEN-PAGE REPLY TO THE RFI. 1138 00:49:54,480 --> 00:49:56,800 THEY ARE VERY IMPORTANT IN 1139 00:49:56,800 --> 00:49:58,440 MAKING SURE THAT NIH IS 1140 00:49:58,440 --> 00:50:00,720 RESPONSIVE TO THE WIDER 1141 00:50:00,720 --> 00:50:03,600 COMMUNITY, BOTH PATIENTS AND 1142 00:50:03,600 --> 00:50:04,640 INVESTIGATORS AND CAREGIVERS. 1143 00:50:04,640 --> 00:50:08,720 >>I WOULD JUST ADD FROM THE 1144 00:50:08,720 --> 00:50:10,200 NICHD PERSPECTIVE, ERIC, THAT WE 1145 00:50:10,200 --> 00:50:11,680 DIDN'T HAVE A STRATEGIC PLAN FOR 1146 00:50:11,680 --> 00:50:12,800 20 YEARS. 1147 00:50:12,800 --> 00:50:16,560 SO THERE WAS A LOT OF PENT UP 1148 00:50:16,560 --> 00:50:18,960 DEMAND AND EMOTION FOR INPUT. 1149 00:50:18,960 --> 00:50:21,120 AND WHEN WE -- WE HAD A DRAFT 1150 00:50:21,120 --> 00:50:23,000 STRATEGIC PLAN, WHEN WE ASKED 1151 00:50:23,000 --> 00:50:26,160 FOR COMMENTS ON IT, WE DID GET 1152 00:50:26,160 --> 00:50:27,360 HUNDREDS, NOT ONLY FROM 1153 00:50:27,360 --> 00:50:29,640 INDIVIDUALS BUT FROM THE 1154 00:50:29,640 --> 00:50:30,960 PROFESSIONAL SOCIETIES AS WALTER 1155 00:50:30,960 --> 00:50:31,720 SAID. 1156 00:50:31,720 --> 00:50:34,440 IT'S A VERY USEFUL MECHANISM TO 1157 00:50:34,440 --> 00:50:36,240 REALLY PUT YOUR FINGER ON THE 1158 00:50:36,240 --> 00:50:37,880 PULSE, AS TO WHETHER WE'RE 1159 00:50:37,880 --> 00:50:41,680 GETTING IT RIGHT, DID WE FORGET 1160 00:50:41,680 --> 00:50:43,120 SOMETHING, AND WE ALSO PRESENTED 1161 00:50:43,120 --> 00:50:45,520 THE DRAFT TO OUR ADVISORY 1162 00:50:45,520 --> 00:50:48,480 COUNCIL, WHO ALSO HAD SOME VERY 1163 00:50:48,480 --> 00:50:51,120 IMPORTANT COMMENTS. 1164 00:50:51,120 --> 00:50:54,560 I'M GOING TO ACKNOWLEDGE 1165 00:50:54,560 --> 00:50:55,280 GUSTAVO'S HAND IS UP. 1166 00:50:55,280 --> 00:50:56,040 >>THANK YOU. 1167 00:50:56,040 --> 00:50:59,680 THIS IS A COMMENT AND QUESTION. 1168 00:50:59,680 --> 00:51:05,240 WE SHARED THE NEW IMPORTANT HIGH 1169 00:51:05,240 --> 00:51:12,880 FOCUS AND PATIENT-CENTRIC 1170 00:51:12,880 --> 00:51:15,960 HEALTH, PATIENT-CENTRIC 1171 00:51:15,960 --> 00:51:16,320 RESEARCH. 1172 00:51:16,320 --> 00:51:19,480 IN OUR ORGANIZATION, IT'S 1173 00:51:19,480 --> 00:51:21,480 IMPORTANT TO HELP EDUCATE 1174 00:51:21,480 --> 00:51:26,240 AFFECTED INDIVIDUALS AND 1175 00:51:26,240 --> 00:51:30,000 FAMILIES. 1176 00:51:30,000 --> 00:51:34,800 SO, THE QUESTION IS FUNDING 1177 00:51:34,800 --> 00:51:37,560 THROUGH NIH OR WE STILL SHOULD 1178 00:51:37,560 --> 00:51:41,680 GO THROUGH PCORI OR THOSE KINDS 1179 00:51:41,680 --> 00:51:43,120 OF ORGANIZATIONS. 1180 00:51:43,120 --> 00:51:44,680 >>WELL, ONE OF THE THINGS I'LL 1181 00:51:44,680 --> 00:51:46,520 SAY, MY COLLEAGUES MIGHT WONT TO 1182 00:51:46,520 --> 00:51:47,720 ADD, CERTAINLY SINCE I'VE 1183 00:51:47,720 --> 00:51:49,560 ARRIVED AT THE NIH THERE'S A LOT 1184 00:51:49,560 --> 00:51:51,560 OF DISCUSSION ABOUT THE 1185 00:51:51,560 --> 00:51:53,080 IMPORTANCE OF PATIENT 1186 00:51:53,080 --> 00:51:53,360 ENGAGEMENT. 1187 00:51:53,360 --> 00:51:54,480 AND EDUCATION AND HEALTH 1188 00:51:54,480 --> 00:51:56,360 LITERACY IS A VERY CRITICAL 1189 00:51:56,360 --> 00:51:58,120 PIECE OF THAT. 1190 00:51:58,120 --> 00:51:59,960 IT'S IMPORTANT FOR UNDERSTANDING 1191 00:51:59,960 --> 00:52:01,720 THE FULL IMPACT OF DISEASES AND 1192 00:52:01,720 --> 00:52:05,240 CONDITIONS ON INDIVIDUALS AND 1193 00:52:05,240 --> 00:52:08,400 THEIR FAMILIES, FOR ENSURING 1194 00:52:08,400 --> 00:52:10,280 THAT RESEARCH OF EFFORTS INVOLVE 1195 00:52:10,280 --> 00:52:11,960 PATIENTS AND FAMILIES IN 1196 00:52:11,960 --> 00:52:15,080 IMPACTFUL AND APPROPRIATE WAYS, 1197 00:52:15,080 --> 00:52:15,600 AND IN DISSEMINATING THE 1198 00:52:15,600 --> 00:52:16,680 RESEARCH, ET CETERA, ET CETERA. 1199 00:52:16,680 --> 00:52:19,920 I SEE A LOT OF EMPHASIS ON THAT. 1200 00:52:19,920 --> 00:52:22,000 I CAN'T SPEAK SPECIFICALLY TO 1201 00:52:22,000 --> 00:52:24,320 PATIENT EDUCATION EFFORTS AND 1202 00:52:24,320 --> 00:52:27,200 HOW THEY ARE FUNDED THROUGH THE 1203 00:52:27,200 --> 00:52:29,920 NIH, EXCEPT TO SAY IT'S CLEARLY 1204 00:52:29,920 --> 00:52:30,720 PART OF THE IMPORTANT MISSION OF 1205 00:52:30,720 --> 00:52:31,320 THE NIH. 1206 00:52:31,320 --> 00:52:33,600 I DON'T KNOW IF DIANA OR WALTER 1207 00:52:33,600 --> 00:52:42,000 WANT TO ADD TO THAT. 1208 00:52:42,000 --> 00:52:42,800 >>THERE ARE DEFINITELY SOME 1209 00:52:42,800 --> 00:52:44,760 THINGS THAT MIGHT BE OF 1210 00:52:44,760 --> 00:52:45,200 INTEREST. 1211 00:52:45,200 --> 00:52:50,320 SO THE NIH'S MISSION IS 1212 00:52:50,320 --> 00:52:51,320 RESEARCH. 1213 00:52:51,320 --> 00:52:53,160 SO, WE'VE SEEN RESEARCH GRANTS 1214 00:52:53,160 --> 00:52:58,880 TO TEST DIFFERENT WAYS OF 1215 00:52:58,880 --> 00:52:59,560 EDUCATING AT-RISK INDIVIDUALS, 1216 00:52:59,560 --> 00:53:05,880 SAY, ON HOW TO IMPROVE THEIR 1217 00:53:05,880 --> 00:53:07,320 HEALTH, WHERE THE OUTCOME IS, 1218 00:53:07,320 --> 00:53:08,800 YOU KNOW, TOOL AND IMPROVEMENT 1219 00:53:08,800 --> 00:53:09,480 IN HEALTH. 1220 00:53:09,480 --> 00:53:12,200 I THINK ON THE RESEARCH SIDE, 1221 00:53:12,200 --> 00:53:15,600 DEPENDING ON WHAT THE DISEASE 1222 00:53:15,600 --> 00:53:18,040 IS, AN INSTITUTE WOULD FUND -- 1223 00:53:18,040 --> 00:53:21,440 WOULD TAKE IN GRANTS THAT WOULD, 1224 00:53:21,440 --> 00:53:23,920 YOU KNOW, DO RESEARCH ON HOW 1225 00:53:23,920 --> 00:53:25,600 BEST TO EDUCATE. 1226 00:53:25,600 --> 00:53:29,200 I DON'T THINK THAT NIH FUNDS 1227 00:53:29,200 --> 00:53:34,920 ACTUAL, YOU KNOW, ONGOING 1228 00:53:34,920 --> 00:53:35,520 DAY-TO-DAY EDUCATION, IF THAT 1229 00:53:35,520 --> 00:53:38,360 WAS THE QUESTION. 1230 00:53:38,360 --> 00:53:41,880 DOES THAT MAKE SENSE? 1231 00:53:41,880 --> 00:53:48,040 >>YES, AND I THINK THAT AS WE 1232 00:53:48,040 --> 00:53:54,240 HAD COORDINATING COMMITTEE AMONG 1233 00:53:54,240 --> 00:53:57,120 ALL THE FEDERAL BRANCHES OF THE 1234 00:53:57,120 --> 00:54:05,720 GOVERNMENT RELATED TO THESE 1235 00:54:05,720 --> 00:54:06,880 PROGRAMS, IF IT'S NOT THROUGH 1236 00:54:06,880 --> 00:54:10,200 THE -- THROUGH WHAT WAS 1237 00:54:10,200 --> 00:54:11,560 MENTIONED, TO HAVE, I DON'T 1238 00:54:11,560 --> 00:54:16,080 KNOW, KIND OF A DOCUMENT OR 1239 00:54:16,080 --> 00:54:24,040 IDEAS PUT TOGETHER TO INCREASE 1240 00:54:24,040 --> 00:54:25,760 FUNDING FROM FEDERAL 1241 00:54:25,760 --> 00:54:26,800 ORGANIZATIONS, FOR EDUCATING 1242 00:54:26,800 --> 00:54:27,640 PATIENTS. 1243 00:54:27,640 --> 00:54:30,760 I THINK IT'S SOMETHING THAT IS 1244 00:54:30,760 --> 00:54:31,440 LACKING. 1245 00:54:31,440 --> 00:54:33,680 WE STRUGGLE WITH EDUCATING 1246 00:54:33,680 --> 00:54:36,000 PATIENTS AND FAMILIES. 1247 00:54:36,000 --> 00:54:39,400 >>I THINK -- ARE YOU TALKING 1248 00:54:39,400 --> 00:54:43,000 ABOUT INCREASING THE ROLE OF 1249 00:54:43,000 --> 00:54:44,760 AFFECTED INDIVIDUALS AND 1250 00:54:44,760 --> 00:54:46,760 FAMILIES INTO ADVOCATING FOR 1251 00:54:46,760 --> 00:54:48,480 MORE MONEY FOR RESEARCH ON THE 1252 00:54:48,480 --> 00:54:48,960 CONDITION? 1253 00:54:48,960 --> 00:54:50,640 IS THAT WHAT YOU'RE -- 1254 00:54:50,640 --> 00:54:56,240 >>NO, BECAUSE -- NO. 1255 00:54:56,240 --> 00:55:00,640 WE'RE TALKING ABOUT MAKING 1256 00:55:00,640 --> 00:55:03,920 PATIENT-CENTRIC RESEARCH OR 1257 00:55:03,920 --> 00:55:06,680 PATIENT-CENTRIC OUTCOMES, SO WE 1258 00:55:06,680 --> 00:55:09,000 NEED THE PATIENT INVOLVED, AND 1259 00:55:09,000 --> 00:55:13,080 FOR THE PATIENTS BEING INVOLVED 1260 00:55:13,080 --> 00:55:15,880 WE NEED THE EDUCATION. 1261 00:55:15,880 --> 00:55:17,000 >>I THINK THAT'S RECOGNIZED AS 1262 00:55:17,000 --> 00:55:19,960 A CRITICAL COMPONENT OF PATIENT 1263 00:55:19,960 --> 00:55:20,680 ENGAGEMENT, FOR SURE. 1264 00:55:20,680 --> 00:55:25,000 ANOTHER THING TO ADD, ALL OF THE 1265 00:55:25,000 --> 00:55:26,400 INSTITUTES HAVE COMMUNICATION 1266 00:55:26,400 --> 00:55:29,320 PROGRAMS AND DEPARTMENTS THAT 1267 00:55:29,320 --> 00:55:31,080 FOCUS ON, YOU KNOW, 1268 00:55:31,080 --> 00:55:32,280 APPROPRIATENESS OF LANGUAGE AND 1269 00:55:32,280 --> 00:55:34,840 OUTREACH AND DISSEMINATION. 1270 00:55:34,840 --> 00:55:37,680 I THINK WE GET AT MANY ASPECTS 1271 00:55:37,680 --> 00:55:39,880 OF WHAT YOU'RE RAISING, WHICH IS 1272 00:55:39,880 --> 00:55:41,840 VERY IMPORTANT OF COURSE. 1273 00:55:41,840 --> 00:55:44,440 >>I WOULD ADD THAT AT EACH 1274 00:55:44,440 --> 00:55:45,960 ADVISORY COUNCIL MEETING, WE 1275 00:55:45,960 --> 00:55:49,360 ALWAYS HAVE A SECTION CALLED THE 1276 00:55:49,360 --> 00:55:51,000 VOICE OF THE PARTICIPANT, IN 1277 00:55:51,000 --> 00:55:53,120 WHICH WE INVITE SOMEONE WHO IS 1278 00:55:53,120 --> 00:55:55,400 EITHER AN ACTIVE PARTICIPANT OR 1279 00:55:55,400 --> 00:55:57,280 PAST PARTICIPANT IN OUR 1280 00:55:57,280 --> 00:56:00,200 RESEARCH, TO TELL US, YOU KNOW, 1281 00:56:00,200 --> 00:56:01,680 HOW WAS THAT EXPERIENCE. 1282 00:56:01,680 --> 00:56:02,880 WHAT CAME OUT OF THE RESEARCH, 1283 00:56:02,880 --> 00:56:03,560 ET CETERA. 1284 00:56:03,560 --> 00:56:06,280 AND WHEN WE HAVE WORKSHOPS, 1285 00:56:06,280 --> 00:56:08,680 WHICH EFFECTIVELY PLAN FOR 1286 00:56:08,680 --> 00:56:11,200 FUTURE RESEARCH INITIATIVES, WE 1287 00:56:11,200 --> 00:56:12,880 FREQUENTLY INVOLVE PEOPLE WHO 1288 00:56:12,880 --> 00:56:14,240 HAVE THE LIVED EXPERIENCE. 1289 00:56:14,240 --> 00:56:15,800 SO, THAT'S ANOTHER WAY THAT WE 1290 00:56:15,800 --> 00:56:16,760 GET IT. 1291 00:56:16,760 --> 00:56:26,720 JUST SO WE DON'T GET TOO FAR OFF 1292 00:56:26,720 --> 00:56:29,360 SCHEDULE, DAN. 1293 00:56:29,360 --> 00:56:33,520 >>A GENERAL QUESTION. 1294 00:56:33,520 --> 00:56:39,520 I'VE PARTICIPATED MYSELF IN 1295 00:56:39,520 --> 00:56:49,760 (INDISCERNIBLE) TWO OR THREE 1296 00:56:49,760 --> 00:56:50,720 GENERATIONS OF MUSCULAR 1297 00:56:50,720 --> 00:56:53,840 DYSTROPHY. 1298 00:56:53,840 --> 00:56:58,240 I GUESS THE QUESTION IS, HOW IN 1299 00:56:58,240 --> 00:57:06,120 THE ANALYSIS, HOW DO YOU ANALYZE 1300 00:57:06,120 --> 00:57:15,400 OR DO YOU USE OR HOW MEASURED, 1301 00:57:15,400 --> 00:57:25,880 THE PROCESS, TALKING ABOUT 1302 00:57:27,520 --> 00:57:30,560 MUSCULAR DYSTROPHY PLAN, A 1303 00:57:30,560 --> 00:57:41,040 HUNDRED AREAS, GOOD AREAS TO 1304 00:57:43,600 --> 00:57:47,480 WORK ON, WHETHER YOU'RE 1305 00:57:47,480 --> 00:57:55,400 SUCCEEDED OR NOT, 100 1306 00:57:55,400 --> 00:57:56,920 RESEARCHERS, 600 PATIENTS, 1307 00:57:56,920 --> 00:57:57,560 THREE-YEAR PROCESS, 100 OTHERS, 1308 00:57:57,560 --> 00:58:02,720 DO YOU GO BACK AND SAY CHECK, 1309 00:58:02,720 --> 00:58:06,960 CHECK, CHECK, CHECK, WE 1310 00:58:06,960 --> 00:58:10,440 SUCCEEDED, WHERE DOES THIS GO? 1311 00:58:10,440 --> 00:58:15,600 HOW DO YOU AT THE NIH MEASURE 1312 00:58:15,600 --> 00:58:19,560 YOUR SUCCESS AGAINST THIS PLAN? 1313 00:58:19,560 --> 00:58:23,040 IS IT TIED TO PERFORMANCE REVIEW 1314 00:58:23,040 --> 00:58:26,520 AS A DIRECTOR, PROGRAM STAFF? 1315 00:58:26,520 --> 00:58:28,600 I MEAN, WHAT DOES IT DO? 1316 00:58:28,600 --> 00:58:30,920 >>I THINK THAT'S A GREAT 1317 00:58:30,920 --> 00:58:32,240 QUESTION, DAN. 1318 00:58:32,240 --> 00:58:34,960 AND CERTAINLY SINCE WE WILL 1319 00:58:34,960 --> 00:58:36,960 BEGIN OUR STRATEGIC PLANNING 1320 00:58:36,960 --> 00:58:38,040 PROCESS FOR THE NEXT ROUND, 1321 00:58:38,040 --> 00:58:40,440 THAT'S A BIG PART OF WHAT WE'RE 1322 00:58:40,440 --> 00:58:42,760 DOING IS WE'RE REVIEWING WHAT WE 1323 00:58:42,760 --> 00:58:45,520 SAID WE WERE GOING TO DO AND 1324 00:58:45,520 --> 00:58:47,040 LOOKING DID WE ACTUALLY DO IT. 1325 00:58:47,040 --> 00:58:48,600 ONE OF THE AREAS WHERE WE 1326 00:58:48,600 --> 00:58:50,760 SUCCEEDED AND ONE OF THE AREAS 1327 00:58:50,760 --> 00:58:54,360 WHERE EITHER WE FAILED OR WE 1328 00:58:54,360 --> 00:58:56,680 HAVEN'T APPROACHED IT YET, SO I 1329 00:58:56,680 --> 00:58:58,720 THINK FROM MY PERSPECTIVE IT'S 1330 00:58:58,720 --> 00:59:02,680 UP TO OUR INSTITUTE, IT'S NOT 1331 00:59:02,680 --> 00:59:04,200 LIKE NIH NECESSARILY IS LOOKING 1332 00:59:04,200 --> 00:59:06,560 AT WHETHER WE DID WHAT WE SAID 1333 00:59:06,560 --> 00:59:08,800 WE WERE GOING TO DO. 1334 00:59:08,800 --> 00:59:10,240 BUT CONGRESS DOES. 1335 00:59:10,240 --> 00:59:12,880 SO IF CONGRESS HAS APPROPRIATED 1336 00:59:12,880 --> 00:59:16,160 MONEY AND HAS SAID YOU MUST DO 1337 00:59:16,160 --> 00:59:18,440 A, B, C, D, ESSENTIALLY WE NEED 1338 00:59:18,440 --> 00:59:21,120 TO REPORT TO CONGRESS EITHER IN 1339 00:59:21,120 --> 00:59:22,760 PERSON OR IN WRITING THAT WE 1340 00:59:22,760 --> 00:59:24,280 HAVE DONE THAT. 1341 00:59:24,280 --> 00:59:26,440 AND IF NOT, THEY WANT TO KNOW 1342 00:59:26,440 --> 00:59:26,840 WHY. 1343 00:59:26,840 --> 00:59:29,240 AND THAT'S ANOTHER AREA WHERE 1344 00:59:29,240 --> 00:59:31,920 ADVOCACY GROUPS CAN GET INVOLVED 1345 00:59:31,920 --> 00:59:36,520 AND PUT THE PRESSURE ON NIH TO 1346 00:59:36,520 --> 00:59:37,680 ACCOMPLISH CERTAIN GOALS. 1347 00:59:37,680 --> 00:59:39,320 LINDSEY AND WALTER, DO YOU WANT 1348 00:59:39,320 --> 00:59:40,000 TO WEIGH IN? 1349 00:59:40,000 --> 00:59:41,120 >>I WOULD JUST SAY THANK YOU 1350 00:59:41,120 --> 00:59:43,680 VERY MUCH FOR THE QUESTION, DAN. 1351 00:59:43,680 --> 00:59:46,400 AND AT LEAST WITH RESPECT TO 1352 00:59:46,400 --> 00:59:48,160 STRATEGIC PLANNING, WE HAVE 1353 00:59:48,160 --> 00:59:49,800 TALKED ABOUT EXACTLY THE ISSUE 1354 00:59:49,800 --> 00:59:51,200 RAISED, THAT IS WHETHER YOU WANT 1355 00:59:51,200 --> 00:59:53,840 TO CALL IT STEWARDSHIP OR 1356 00:59:53,840 --> 00:59:55,040 ONGOING ASSESSMENT, AND WE'RE 1357 00:59:55,040 --> 00:59:57,320 DEFINITELY INTERESTED IN AS WE 1358 00:59:57,320 --> 00:59:59,560 DEVELOP THIS NEXT STRATEGIC PLAN 1359 00:59:59,560 --> 01:00:02,600 DEVELOPING METRICS OR HAVING A 1360 01:00:02,600 --> 01:00:03,360 SYSTEMATIC APPROACH TO 1361 01:00:03,360 --> 01:00:04,080 EVALUATING OUR PROGRESS. 1362 01:00:04,080 --> 01:00:07,480 THERE ARE TOOLS THAT CAN HELP US 1363 01:00:07,480 --> 01:00:10,360 DO THAT, IN THE OVERALL CATEGORY 1364 01:00:10,360 --> 01:00:12,120 OF PORTFOLIO ANALYSES, THOSE 1365 01:00:12,120 --> 01:00:14,160 AREN'T PERFECT, BUT WE ALSO 1366 01:00:14,160 --> 01:00:15,600 RECOGNIZE THAT WE CAN'T 1367 01:00:15,600 --> 01:00:18,120 ANTICIPATE TODAY SOME OF THE NEW 1368 01:00:18,120 --> 01:00:21,960 OPPORTUNITIES THAT WILL EMERGE 1369 01:00:21,960 --> 01:00:24,480 THREE YEARS FROM NOW SO WE NEED 1370 01:00:24,480 --> 01:00:26,240 TO MAKE SURE WE'RE NIMBLE, 1371 01:00:26,240 --> 01:00:27,760 OPPORTUNISTIC IN A SENSE LOOKING 1372 01:00:27,760 --> 01:00:30,160 FORWARD SO WE DON'T MISS NEW 1373 01:00:30,160 --> 01:00:31,320 OPPORTUNITIES THAT ARISE THAT WE 1374 01:00:31,320 --> 01:00:32,560 DIDN'T CONCEIVE OF AT THE OUTSET 1375 01:00:32,560 --> 01:00:34,200 OF THE PLAN. 1376 01:00:34,200 --> 01:00:37,600 SO, I THINK MOST IMPORTANTLY, 1377 01:00:37,600 --> 01:00:40,880 WHAT YOU'RE SPEAKING TO IS THE 1378 01:00:40,880 --> 01:00:43,400 TAKING SERIOUSLY THE PROCESS OF 1379 01:00:43,400 --> 01:00:45,160 SEEKING INPUT, DEVELOPING PLANS, 1380 01:00:45,160 --> 01:00:48,920 THEN MONITORING OUR PROGRESS 1381 01:00:48,920 --> 01:00:49,320 TOWARDS THOSE. 1382 01:00:49,320 --> 01:00:52,640 I DON'T KNOW IF WALTER HAS 1383 01:00:52,640 --> 01:00:58,280 ANYTHING ELSE TO SAY IN RESPONSE 1384 01:00:58,280 --> 01:01:01,200 FROM HIS EXPERIENCE. 1385 01:01:01,200 --> 01:01:01,480 >>OKAY. 1386 01:01:01,480 --> 01:01:02,120 THANK YOU. 1387 01:01:02,120 --> 01:01:02,760 >>THANK YOU. 1388 01:01:02,760 --> 01:01:04,560 >>THANKS FOR YOUR QUESTION, 1389 01:01:04,560 --> 01:01:05,760 DAN. 1390 01:01:05,760 --> 01:01:08,640 IN THE INTEREST OF TIME LET'S 1391 01:01:08,640 --> 01:01:09,880 MOVE ON. 1392 01:01:09,880 --> 01:01:12,680 WE'VE GOT TWO SUMMARY REPORTS, 1393 01:01:12,680 --> 01:01:20,360 TWO RECENT MUSCULAR DYSTROPHY 1394 01:01:20,360 --> 01:01:25,280 CONFERENCES, SO DR. GUSTAVO 1395 01:01:25,280 --> 01:01:26,720 DZIEWCZAPOLSKI WILL TELL US WILL 1396 01:01:26,720 --> 01:01:28,600 THE SCIENTIFIC AND FAMILY 1397 01:01:28,600 --> 01:01:31,800 CONFERENCE HELD IN JULY. 1398 01:01:31,800 --> 01:01:42,200 GUSTAVO, OVER TO YOU. 1399 01:01:45,840 --> 01:01:51,440 1400 01:01:51,440 --> 01:01:52,040 >>THANK YOU. 1401 01:01:52,040 --> 01:01:55,080 CAN YOU SEE MY SLIDES? 1402 01:01:55,080 --> 01:01:59,880 1403 01:01:59,880 --> 01:02:09,920 1404 01:02:22,480 --> 01:02:22,960 OKAY. 1405 01:02:22,960 --> 01:02:24,520 SO, WE THANK THE ORGANIZERS AND 1406 01:02:24,520 --> 01:02:25,840 FELLOW MEMBERS OF THE COMMITTEE 1407 01:02:25,840 --> 01:02:29,640 FOR GIVING US THIS CHANCE TO 1408 01:02:29,640 --> 01:02:31,760 SHARE WITH YOU. 1409 01:02:31,760 --> 01:02:35,240 OUR RECENT SCIENTIFIC AND FAMILY 1410 01:02:35,240 --> 01:02:37,600 CONFERENCE HOSTED IN NASHVILLE, 1411 01:02:37,600 --> 01:02:44,680 TENNESSEE, FOURTH OF JULY 1412 01:02:44,680 --> 01:02:48,480 WEEKEND, THE ONLY OPTION, IN THE 1413 01:02:48,480 --> 01:02:50,040 POST-PANDEMIC YEAR, POSTPONED 1414 01:02:50,040 --> 01:02:51,320 IN-PERSON MEETINGS, AND 1415 01:02:51,320 --> 01:02:57,040 EVERYBODY SEEMS TO WANT TO DO 1416 01:02:57,040 --> 01:02:58,040 THE CONFERENCES MISSED DURING 1417 01:02:58,040 --> 01:03:02,440 THE SUMMER, SAME AS US. 1418 01:03:02,440 --> 01:03:03,960 BUT IT WAS SUCCESSFUL. 1419 01:03:03,960 --> 01:03:10,960 IT WAS A FOUR-DAY MEETING, 1420 01:03:10,960 --> 01:03:12,480 TOTALLY HYBRID, MADE POSSIBLE IN 1421 01:03:12,480 --> 01:03:15,560 LARGE PART BY THE FINANCIAL 1422 01:03:15,560 --> 01:03:21,720 SUPPORT OF A FEW BUT VERY 1423 01:03:21,720 --> 01:03:22,480 IMPORTANT SPONSORS. 1424 01:03:22,480 --> 01:03:31,760 NIAMS WAS ONE OF THEM. 1425 01:03:31,760 --> 01:03:36,720 AND A PROGRAM BY THE INITIATIVE, 1426 01:03:36,720 --> 01:03:47,200 ONE COMPANY WORKING IN OUR 1427 01:03:50,280 --> 01:03:58,720 UMBRELLA, MDA, ANOTHER IN 1428 01:03:58,720 --> 01:04:09,200 FRANCE, A GROUP OF SPONSORS. 1429 01:04:14,800 --> 01:04:19,800 (INDISCERNIBLE) PATIENT 1430 01:04:19,800 --> 01:04:20,120 ORGANIZATIONS 1431 01:04:20,120 --> 01:04:29,280 [AUDIO DISTORTION] 1432 01:04:29,280 --> 01:04:32,160 COVERS FIVE DYSTROPHIES. 1433 01:04:32,160 --> 01:04:42,760 WE WERE JOINED BY A SMALL 1434 01:04:42,760 --> 01:04:49,360 ORGANIZATION JUST LAST WEEK, 1435 01:04:49,360 --> 01:04:59,880 NON-PROFIT ORGANIZATION STATUS 1436 01:05:05,080 --> 01:05:07,120 COVERING TITINOPATHYS. 1437 01:05:07,120 --> 01:05:13,040 WE JOINED FORCES, MADE STRONGER 1438 01:05:13,040 --> 01:05:23,440 FOR DIFFERENT PATIENTS. 1439 01:05:27,000 --> 01:05:34,680 ALL READINESS FOR ALL THESE RARE 1440 01:05:34,680 --> 01:05:38,280 CONDITIONS, COMMON STRATEGIES, 1441 01:05:38,280 --> 01:05:40,680 WHICH LEAVES POTENTIAL 1442 01:05:40,680 --> 01:05:41,680 ESTABLISHING RESEARCH PLATFORMS 1443 01:05:41,680 --> 01:05:43,240 WHERE MORE THAN ONE DISEASE 1444 01:05:43,240 --> 01:05:46,920 COULD BE MOVED FORWARD IN THE 1445 01:05:46,920 --> 01:05:55,040 SAME PIPELINE. 1446 01:05:55,040 --> 01:05:57,600 WE'VE BEEN COLLABORATING, 1447 01:05:57,600 --> 01:06:01,040 PROVIDING RESEARCH TO CMD 1448 01:06:01,040 --> 01:06:05,760 INTERNATIONAL REGISTRY, ALSO 1449 01:06:05,760 --> 01:06:06,160 TISSUE REPOSITORY. 1450 01:06:06,160 --> 01:06:11,760 THEN WE HAVE EXPERIENCE 1451 01:06:11,760 --> 01:06:17,000 ORGANIZING SCIENTIFIC CONFERENCE 1452 01:06:17,000 --> 01:06:20,200 IN 2019, SO WE'RE VERY FRIEND 1453 01:06:20,200 --> 01:06:20,640 ORGANIZATIONS. 1454 01:06:20,640 --> 01:06:24,680 ALSO THE FACT THAT MOST OF THE 1455 01:06:24,680 --> 01:06:35,200 EXPERTS IN THIS CMD SEE PATIENTS 1456 01:06:35,680 --> 01:06:36,800 (INDISCERNIBLE) SAVING TIME BY 1457 01:06:36,800 --> 01:06:39,640 ASKING THEM TO ATTEND ONLY ONE 1458 01:06:39,640 --> 01:06:43,680 CONFERENCE INSTEAD OF THREE 1459 01:06:43,680 --> 01:06:48,200 DIFFERENT ONES. 1460 01:06:48,200 --> 01:06:50,360 1461 01:06:50,360 --> 01:06:52,800 THE CONFERENCE THEMES WERE NOT 1462 01:06:52,800 --> 01:06:54,800 THAT ORIGINAL BUT VERY IMPORTANT 1463 01:06:54,800 --> 01:06:56,440 IN OFFERING RESEARCHERS AND 1464 01:06:56,440 --> 01:06:57,960 CLINICIANS THE OPPORTUNITY TO 1465 01:06:57,960 --> 01:07:05,680 LEARN FROM EACH OTHER AND 1466 01:07:05,680 --> 01:07:13,080 ENHANCE ONE ANOTHER'S WORK. 1467 01:07:13,080 --> 01:07:14,720 THIS ARENA ANALYZES POSSIBILITY 1468 01:07:14,720 --> 01:07:18,240 TO DETECT RESEARCH AND CARE 1469 01:07:18,240 --> 01:07:19,640 PRIORITIES FOR EACH SPECIFIC 1470 01:07:19,640 --> 01:07:24,480 DISEASE AS WELL AS POSSIBILITIES 1471 01:07:24,480 --> 01:07:25,560 FOR PLATFORMS OR PROJECTS 1472 01:07:25,560 --> 01:07:30,480 TOTALING MORE THAN ONE DISEASE 1473 01:07:30,480 --> 01:07:31,920 AT A TIME. 1474 01:07:31,920 --> 01:07:38,160 AND AS IT HAPPENED IN PAST 1475 01:07:38,160 --> 01:07:39,240 CONFERENCES, CONCLUSIONS OF THE 1476 01:07:39,240 --> 01:07:41,080 SCIENTIFIC CONFERENCE GUIDE US 1477 01:07:41,080 --> 01:07:43,960 TO FOCUS FUTURE FUNDING IN OUR 1478 01:07:43,960 --> 01:07:44,920 GRANT PROGRAMS. 1479 01:07:44,920 --> 01:07:48,080 AND LAST BUT NOT LEAST, 1480 01:07:48,080 --> 01:07:50,280 PROVIDING THE OPPORTUNITY OF 1481 01:07:50,280 --> 01:07:51,400 GATHERING AFFECTED INDIVIDUALS 1482 01:07:51,400 --> 01:07:54,480 WITH EACH OTHER AND WITH 1483 01:07:54,480 --> 01:07:58,520 CLINICIANS AND RESEARCHERS, 1484 01:07:58,520 --> 01:07:59,600 EXPERTS IN THESE DISEASES, THAT 1485 01:07:59,600 --> 01:08:02,200 HELP US IN OUR MISSION OF 1486 01:08:02,200 --> 01:08:11,760 CRAFTING THE MOST RELEVANT 1487 01:08:11,760 --> 01:08:22,360 PATIENT-FOCUSED APPROACHES POSS. 1488 01:08:24,400 --> 01:08:25,080 THE CONFERENCE STRUCTURE 1489 01:08:25,080 --> 01:08:30,760 THURSDAY AND FRIDAY MORNING WAS 1490 01:08:30,760 --> 01:08:35,480 SCIENTIFIC SYMPOSIUM. 1491 01:08:35,480 --> 01:08:37,560 RESEARCH FUNDING BY THE THREE 1492 01:08:37,560 --> 01:08:43,800 ORGANIZING GROUPS AS WELL AS 1493 01:08:43,800 --> 01:08:46,880 INVITED GRANTEES FROM 1494 01:08:46,880 --> 01:08:48,680 PATIENT-ADVOCACY PARTNERS, SO 1495 01:08:48,680 --> 01:08:50,600 CONSISTED OF 31 INDIVIDUAL TALKS 1496 01:08:50,600 --> 01:08:54,320 BY GRANTEES, GROUPED IN BLOCKS 1497 01:08:54,320 --> 01:08:55,920 BY POTENTIAL THERAPEUTIC 1498 01:08:55,920 --> 01:09:00,040 MODALITIES, AND OF COURSE ALSO 1499 01:09:00,040 --> 01:09:01,440 POSTER SESSIONS. 1500 01:09:01,440 --> 01:09:04,120 HERE AT DIFFERENT STAGES OF 1501 01:09:04,120 --> 01:09:09,520 PROJECT DEVELOPMENT, SOME OF 1502 01:09:09,520 --> 01:09:15,880 THEM HAVE PLANS, OTHERS WERE IN 1503 01:09:15,880 --> 01:09:20,280 BETWEEN, WE WANTED TO MAXIMIZE 1504 01:09:20,280 --> 01:09:24,240 CHANCES TO OBTAIN FEEDBACK FROM 1505 01:09:24,240 --> 01:09:29,960 PEERS AND OTHER STAKEHOLDERS, BY 1506 01:09:29,960 --> 01:09:35,320 HAVING PLATFORM TALKS, POSTERS 1507 01:09:35,320 --> 01:09:36,440 AND DISCUSSION. 1508 01:09:36,440 --> 01:09:46,200 ALL THE PRESENTERS, WE ASK THEM. 1509 01:09:46,200 --> 01:09:48,120 WE ALSO HAD ONE PRESENTATION BY 1510 01:09:48,120 --> 01:09:58,640 A COMPANY WORKING, A PLENARY 1511 01:10:07,000 --> 01:10:14,120 TALK, ONE OF THE HIGHLIGHTS WAS 1512 01:10:14,120 --> 01:10:15,920 TO HAVE THE PATIENT FOCUS WITH 1513 01:10:15,920 --> 01:10:17,120 FDA, PLANNING SIX MONTHS IN 1514 01:10:17,120 --> 01:10:22,000 ADVANCE, ALMOST A YEAR IN 1515 01:10:22,000 --> 01:10:24,760 ADVANCE, GRANTED THIS 1516 01:10:24,760 --> 01:10:26,280 OPPORTUNITY, THE FDA 1517 01:10:26,280 --> 01:10:33,720 INDIVIDUALLY AND ACCORDING TO 1518 01:10:33,720 --> 01:10:36,680 OUR FDA PROGRAM OFFICER 20 1519 01:10:36,680 --> 01:10:38,480 ATTENDED LIVE, OTHERS HAD THE 1520 01:10:38,480 --> 01:10:41,880 CHANCE OF WATCHING THE 1521 01:10:41,880 --> 01:10:43,360 RECORDINGS ON DEMAND. 1522 01:10:43,360 --> 01:10:46,880 TO US, IT WAS A SUCCESSFUL 1523 01:10:46,880 --> 01:10:50,360 MEETING. 1524 01:10:50,360 --> 01:10:53,000 WE WERE ABLE TO SHOW AND WE HAD 1525 01:10:53,000 --> 01:10:57,840 A LOT OF PATIENTS AND FAMILIES 1526 01:10:57,840 --> 01:11:02,680 AND WE CREATED A SHORT THEME TO 1527 01:11:02,680 --> 01:11:12,440 SHOW DAY-TO-DAY STRUGGLES AND 1528 01:11:12,440 --> 01:11:15,920 HOPES FOR PATIENTS, LAST TWO 1529 01:11:15,920 --> 01:11:19,440 DAYS DEDICATED TO THE DEEP 1530 01:11:19,440 --> 01:11:20,640 INTERACTION OF AFFECTED 1531 01:11:20,640 --> 01:11:25,760 COMMUNITIES, WITH RESEARCHERS 1532 01:11:25,760 --> 01:11:31,400 AND CLINICIANS, TOPICS AND ALSO 1533 01:11:31,400 --> 01:11:33,040 INDIVIDUAL BREAKOUT SESSIONS, 1534 01:11:33,040 --> 01:11:34,360 RESEARCH UPDATES, CARE UPDATES, 1535 01:11:34,360 --> 01:11:41,040 PROFESSIONAL SUPPORT, HOW TO AND 1536 01:11:41,040 --> 01:11:45,640 NAVIGATE LIFE WITH DISORDERS, 1537 01:11:45,640 --> 01:11:48,680 MENTAL HEALTH SUPPORT TO 1538 01:11:48,680 --> 01:11:50,880 UNAFFECTED SIBLINGS, NAVIGATED 1539 01:11:50,880 --> 01:11:53,160 CARE BENEFITS, FINANCIAL ADVICE, 1540 01:11:53,160 --> 01:11:55,560 ET CETERA. 1541 01:11:55,560 --> 01:12:01,360 1542 01:12:01,360 --> 01:12:02,960 1543 01:12:02,960 --> 01:12:10,800 FUNDED RESEARCH, IT WAS VERY 1544 01:12:10,800 --> 01:12:15,160 BROAD. 1545 01:12:15,160 --> 01:12:18,360 WE HAD VERY DIVERSE 1546 01:12:18,360 --> 01:12:20,440 PRESENTATIONS, THIS IS USING 1547 01:12:20,440 --> 01:12:25,920 DIFFERENT DISEASE MODELS FROM 1548 01:12:25,920 --> 01:12:28,720 ZEBRAFISH TO MOUSE MODELS, 1549 01:12:28,720 --> 01:12:36,960 ANIMAL MODELS. 1550 01:12:36,960 --> 01:12:38,800 AND PATIENT DERIVED iPSCs 1551 01:12:38,800 --> 01:12:43,520 AND 3D MUSCLES, ALSO VERY 1552 01:12:43,520 --> 01:12:48,880 DIVERSE EXPERIMENTAL THERAPEUTIC 1553 01:12:48,880 --> 01:12:54,480 MODALITIES, SOME PROJECTS 1554 01:12:54,480 --> 01:12:56,520 ATTEMPTING DRUG REPURPOSING, 1555 01:12:56,520 --> 01:12:58,960 USING HIGH-THROUGHPUT 1556 01:12:58,960 --> 01:13:03,960 METHODOLOGY, ALL KIND OF GENETIC 1557 01:13:03,960 --> 01:13:05,160 MANIPULATION, TRANSCRIPT 1558 01:13:05,160 --> 01:13:08,360 ACTIVATION, SPECIFIC GENE 1559 01:13:08,360 --> 01:13:09,480 ACTIVATION, SPECIFIC TRANSCRIPT 1560 01:13:09,480 --> 01:13:12,200 ACTIVATION, GENE EDITING AND 1561 01:13:12,200 --> 01:13:13,840 BASE EDITING. 1562 01:13:13,840 --> 01:13:20,960 ALSO SOME COUPLE OF GRANTEES 1563 01:13:20,960 --> 01:13:27,760 WORKING ON IMPROVING THE TOOLS. 1564 01:13:27,760 --> 01:13:31,520 >>WE HAVE ALLOTTED TEN MINUTES. 1565 01:13:31,520 --> 01:13:32,800 THERE'S A TIMER. 1566 01:13:32,800 --> 01:13:34,000 TEN MINUTES IS OVER, IF YOU 1567 01:13:34,000 --> 01:13:35,880 COULD BEGIN TO WRAP IT UP 1568 01:13:35,880 --> 01:13:37,720 BECAUSE WE'RE QUITE A BIT OVER 1569 01:13:37,720 --> 01:13:42,640 TIME RIGHT NOW. 1570 01:13:42,640 --> 01:13:43,640 >>YES. 1571 01:13:43,640 --> 01:13:44,280 SORRY. 1572 01:13:44,280 --> 01:13:48,080 ALSO MECHANISM OF POTENTIAL 1573 01:13:48,080 --> 01:13:49,800 TARGET DISCOVERIES. 1574 01:13:49,800 --> 01:13:56,880 SINCE I'M A MEMBER OF THE MBCC I 1575 01:13:56,880 --> 01:14:00,720 HAVEN'T SEEN A PAPER BEING 1576 01:14:00,720 --> 01:14:02,640 HIGHLIGHTED MAINLY BECAUSE 1577 01:14:02,640 --> 01:14:06,560 UMBRELLA USED TO BE BEHIND OTHER 1578 01:14:06,560 --> 01:14:11,800 MORE RARE, IT IS EXCITING TO SEE 1579 01:14:11,800 --> 01:14:13,360 HOW THE SAME TECHNOLOGY AND 1580 01:14:13,360 --> 01:14:23,040 APPROACHES ARE BEING USED IN 1581 01:14:23,040 --> 01:14:27,080 RESEARCH. 1582 01:14:27,080 --> 01:14:31,400 IT WAS ATTENDED BY 400 IN 1583 01:14:31,400 --> 01:14:32,680 PERSON, 150 ONLINE, 89 STATES, 1584 01:14:32,680 --> 01:14:38,480 IF YOU WANT TO LEARN MORE -- 18 1585 01:14:38,480 --> 01:14:39,520 COUNTRIES, 39 STATES. 1586 01:14:39,520 --> 01:14:43,080 THAT'S IT. 1587 01:14:43,080 --> 01:14:47,200 THANK YOU VERY MUCH. 1588 01:14:47,200 --> 01:14:49,480 [AUDIO DISTORTION] 1589 01:14:49,480 --> 01:14:50,360 >>THANK YOU. 1590 01:14:50,360 --> 01:14:52,840 IT WAS MY FAULT FOR NOT POINTING 1591 01:14:52,840 --> 01:14:53,520 IT OUT. 1592 01:14:53,520 --> 01:14:55,920 BUT WE THANK YOU FOR NOT ONLY 1593 01:14:55,920 --> 01:14:58,560 ORGANIZING THIS CONFERENCE BUT 1594 01:14:58,560 --> 01:15:00,960 GIVING US A THOROUGH REPORT ON 1595 01:15:00,960 --> 01:15:02,160 IT. 1596 01:15:02,160 --> 01:15:04,320 I THINK SINCE WE'RE ALREADY 11 1597 01:15:04,320 --> 01:15:05,320 MINUTES OVER SCHEDULE, I DON'T 1598 01:15:05,320 --> 01:15:08,200 WANT TO CUT INTO THE BREAK TOO 1599 01:15:08,200 --> 01:15:10,040 MUCH SO IF WE HAVE PRESSING 1600 01:15:10,040 --> 01:15:11,360 QUESTIONS PLEASE ASK THEM IN THE 1601 01:15:11,360 --> 01:15:12,760 Q&A OR THE CHAT BOX. 1602 01:15:12,760 --> 01:15:17,120 I ALSO WANT TO POINT OUT THAT 1603 01:15:17,120 --> 01:15:20,120 DR. CRISWELL PUT THE INFORMATION 1604 01:15:20,120 --> 01:15:22,080 ON THE REQUEST FOR INFORMATION 1605 01:15:22,080 --> 01:15:24,800 FOR THE NIAMS STRATEGIC PLAN IN 1606 01:15:24,800 --> 01:15:29,400 THE CHAT BOX. 1607 01:15:29,400 --> 01:15:36,800 NEXT A REPORT FROM DR. NANDI, HE 1608 01:15:36,800 --> 01:15:38,760 AND HIS COLLEAGUES HELPED 1609 01:15:38,760 --> 01:15:40,280 ORGANIZE A CONFERENCE ON CARDIAC 1610 01:15:40,280 --> 01:15:43,480 CARE FOR BOYS WITH DUCHENNE 1611 01:15:43,480 --> 01:15:45,720 MUSCULAR DYSTROPHY, SUPPORTED BY 1612 01:15:45,720 --> 01:15:47,720 PART PROJECT MUSCULAR DYSTROPHY. 1613 01:15:47,720 --> 01:15:48,720 WELCOME, DR. NANDI. 1614 01:15:48,720 --> 01:15:50,480 I DON'T SEE YOU BUT I'M HOPING 1615 01:15:50,480 --> 01:15:52,560 YOU'RE THERE. 1616 01:15:52,560 --> 01:15:53,320 >>I AM. 1617 01:15:53,320 --> 01:15:54,760 THANK YOU FOR HAVING ME. 1618 01:15:54,760 --> 01:15:57,920 I MUCH APPRECIATE IT. 1619 01:15:57,920 --> 01:16:01,840 I'LL BE PRESENTING ON ON BEHALF 1620 01:16:01,840 --> 01:16:03,400 OF THE ACTION NETWORK, A GROUP 1621 01:16:03,400 --> 01:16:05,360 THROUGHOUT THE NATION AND WORLD 1622 01:16:05,360 --> 01:16:07,320 GROWING IN SIZE, I'LL EXPLAIN 1623 01:16:07,320 --> 01:16:11,840 WHY, ON BEHALF OF MY CLEGS, 1624 01:16:11,840 --> 01:16:14,320 CHAT, AND LINDA, AND NETWORK OF 1625 01:16:14,320 --> 01:16:21,200 CARDIOLOGISTS TAKING CARE OF 1626 01:16:21,200 --> 01:16:21,520 FAMILIES. 1627 01:16:21,520 --> 01:16:27,440 AS WE HAVE DONE BETTER AND 1628 01:16:27,440 --> 01:16:30,440 BETTER WITH, THE BURDEN OF 1629 01:16:30,440 --> 01:16:32,080 MORTALITY HAS ACTUALLY SPREAD 1630 01:16:32,080 --> 01:16:34,480 INTO THE CARDIAC WORLD WHERE 1631 01:16:34,480 --> 01:16:35,680 CARDIAC DEATHS NOW MAKE A 1632 01:16:35,680 --> 01:16:38,240 MAJORITY OF WHERE WE LOSE OUR 1633 01:16:38,240 --> 01:16:40,280 PATIENTS, WHY OUR CARDIOLOGY 1634 01:16:40,280 --> 01:16:41,600 POPULATION HAD INCREASING CARE 1635 01:16:41,600 --> 01:16:44,000 TAKEN OVER IN THESE GROUPS. 1636 01:16:44,000 --> 01:16:52,640 SO, WITH THE HELP, WE 1637 01:16:52,640 --> 01:16:54,600 ORCHESTRATED A CONFERENCE IN 1638 01:16:54,600 --> 01:16:58,440 CHARLESTON THIS YEAR TAKING CARE 1639 01:16:58,440 --> 01:17:01,160 OF CARDIAC SIDE OF THINGS, WELL 1640 01:17:01,160 --> 01:17:02,760 ATTENDED FOR THE CARDIOLOGY 1641 01:17:02,760 --> 01:17:05,880 WORLD IN PEDIATRICS, 80 1642 01:17:05,880 --> 01:17:07,520 ATTENDEES, HALF WERE 1643 01:17:07,520 --> 01:17:09,040 CARDIOLOGISTS, ALSO 1644 01:17:09,040 --> 01:17:13,840 REPRESENTATIVES FROM 1645 01:17:13,840 --> 01:17:16,040 NEUROMUSCULAR, PULMONARY 1646 01:17:16,040 --> 01:17:16,840 COLLEAGUES, REPRESENTATIVES FROM 1647 01:17:16,840 --> 01:17:19,320 SEVERAL INDUSTRY FOLKS AS WELL 1648 01:17:19,320 --> 01:17:22,160 AS MEMBERS OF FDA AND NHLBI. 1649 01:17:22,160 --> 01:17:23,680 THE POINT OF THE CONFERENCE WAS 1650 01:17:23,680 --> 01:17:27,720 TO TRY TO MOVE FORWARD WITH 1651 01:17:27,720 --> 01:17:29,840 FURTHER CARE IN THE CARDIAC 1652 01:17:29,840 --> 01:17:33,120 SPEAR, LUCKY TO HAVE DR. SANTORE 1653 01:17:33,120 --> 01:17:36,160 FROM THE FDA SPEAK WITH US ON 1654 01:17:36,160 --> 01:17:39,040 ENDPOINTS AND CLINICAL TRIALS, 1655 01:17:39,040 --> 01:17:41,560 REITERATING FETAL FUNCTION, END 1656 01:17:41,560 --> 01:17:43,400 POINTS FOR RARE DISEASES. 1657 01:17:43,400 --> 01:17:45,600 TO SPEAK ON THOSE, THE FIELD IS 1658 01:17:45,600 --> 01:17:47,640 DIFFICULT IN CARDIAC PATIENTS 1659 01:17:47,640 --> 01:17:48,520 WITH NEUROMUSCULAR ISSUES, WHERE 1660 01:17:48,520 --> 01:17:51,160 WE HAVE A HARD TIME 1661 01:17:51,160 --> 01:17:52,880 UNDERSTANDING WHAT THEY MAY LOOK 1662 01:17:52,880 --> 01:17:57,080 LIKE, FUNCTION IS ALSO DIFFICULT 1663 01:17:57,080 --> 01:17:59,720 TO ASSESS, AND MORTALITY IS POOR 1664 01:17:59,720 --> 01:18:01,480 IMPROVED OUTCOME WHICH MANY 1665 01:18:01,480 --> 01:18:03,960 FAMILIES CANNOT WAIT, SURROGATE 1666 01:18:03,960 --> 01:18:05,600 ENDPOINTS ARE OFTEN NEEDED, 1667 01:18:05,600 --> 01:18:06,800 WHILE THEY ARE IMPORTANT AND CAN 1668 01:18:06,800 --> 01:18:08,920 BE BENEFICIAL THEY ARE ALSO 1669 01:18:08,920 --> 01:18:11,520 FRAUGHT WITH MANY ISSUES LEADING 1670 01:18:11,520 --> 01:18:14,960 TO CHOOSING IMPROPER ENDPOINTS 1671 01:18:14,960 --> 01:18:16,880 WHICH MAY BECOME PROBLEMATIC. 1672 01:18:16,880 --> 01:18:24,760 NOW, WE WERE VERY LUCKY TO 1673 01:18:24,760 --> 01:18:32,200 DISCUSS, DR. BOB SHATY AT USC, A 1674 01:18:32,200 --> 01:18:35,560 FORMER MENTOR OF MYSELF, THE 1675 01:18:35,560 --> 01:18:39,320 FIRST OF ITS KIND TRIAL OF A 1676 01:18:39,320 --> 01:18:42,040 MAJOR NEW DRUG WHICH APPROVED 1677 01:18:42,040 --> 01:18:48,200 FDA -- RECEIVED FDA APPROVAL IN 1678 01:18:48,200 --> 01:18:50,800 OCTOBER OF 2019, THE ONLY REAL 1679 01:18:50,800 --> 01:18:51,920 DRUG WE HAVE AN ACTUAL 1680 01:18:51,920 --> 01:18:53,240 INDICATION FOR. 1681 01:18:53,240 --> 01:18:59,920 IT'S VERY IMPORTANT TO NOTE 1682 01:18:59,920 --> 01:19:01,320 HOWEVER ENTRESTO WAS APPROVED ON 1683 01:19:01,320 --> 01:19:08,560 THE BASIS OF A SURROGATE MARKER, 1684 01:19:08,560 --> 01:19:10,760 ON PAR WITH WHAT WAS SEEN IN 1685 01:19:10,760 --> 01:19:12,760 ADULTS, HELPFUL TO FDA IN MAKING 1686 01:19:12,760 --> 01:19:15,160 APPROVAL DECISION WHICH AGAIN 1687 01:19:15,160 --> 01:19:17,360 SHOWS YOU THAT THE SURROGATE 1688 01:19:17,360 --> 01:19:18,880 MARKERS WHEN CHOSEN 1689 01:19:18,880 --> 01:19:21,280 APPROPRIATELY CAN BE THE KEY TO 1690 01:19:21,280 --> 01:19:22,320 GETTING THESE APPROVALS. 1691 01:19:22,320 --> 01:19:23,920 SO, KNOWING WE HAVE A GOAL OF 1692 01:19:23,920 --> 01:19:25,440 TRYING TO FIGURE OUT THOSE 1693 01:19:25,440 --> 01:19:27,320 MARKERS HOW DO WE UNDERSTAND 1694 01:19:27,320 --> 01:19:29,960 WITHIN THAT THE CURRENT CARDIAC 1695 01:19:29,960 --> 01:19:31,360 PROGNOSIS FOR OUR PATIENTS, AND 1696 01:19:31,360 --> 01:19:34,440 TREATMENTS THAT WE HAVE FOR 1697 01:19:34,440 --> 01:19:34,840 THEM. 1698 01:19:34,840 --> 01:19:39,120 THESE NEXT SLIDES ARE ON BEHALF 1699 01:19:39,120 --> 01:19:41,760 OF THE -- COURTESY OF A 1700 01:19:41,760 --> 01:19:44,280 PATHOLOGIST AT CINCINNATI 1701 01:19:44,280 --> 01:19:46,040 CHILDREN'S WHO WITH COLLEAGUES 1702 01:19:46,040 --> 01:19:47,680 UNDERTOOK PATHOLOGIC REVIEW OF 1703 01:19:47,680 --> 01:19:51,520 TEN PATIENTS, INCLUDING NINE 1704 01:19:51,520 --> 01:19:58,760 MALES, ONCE CINCINNATI CARRIER 1705 01:19:58,760 --> 01:19:59,040 FEMALE. 1706 01:19:59,040 --> 01:20:02,640 THIS IS THE HEART, YOUNG 1707 01:20:02,640 --> 01:20:04,200 36-YEAR-OLD MAN, DUCHENNE 1708 01:20:04,200 --> 01:20:06,680 MUSCULAR DYSTROPHY ON STEROIDS 1709 01:20:06,680 --> 01:20:09,600 FROM YOUNG AGE, FOUND DECEASED 1710 01:20:09,600 --> 01:20:09,920 AT HOME. 1711 01:20:09,920 --> 01:20:11,120 STRIKINGLY YOU CAN SEE HOW MUCH 1712 01:20:11,120 --> 01:20:14,120 FAT THERE IS THROUGHOUT THE 1713 01:20:14,120 --> 01:20:15,200 EPICARDIAL SURFACE, SIGNIFICANT 1714 01:20:15,200 --> 01:20:15,720 INFILTRATE. 1715 01:20:15,720 --> 01:20:18,800 LOOKING TO THE BACK, THIS IS THE 1716 01:20:18,800 --> 01:20:22,080 RIGHT ATRIUM, AND THE ATRIUM 1717 01:20:22,080 --> 01:20:24,000 THERE IS JUST VERY TRANSLUCENT 1718 01:20:24,000 --> 01:20:27,560 AND JUST SHOWS HOW MUCH 1719 01:20:27,560 --> 01:20:30,040 DESTRUCTION MYOCYTES OF GONE ON 1720 01:20:30,040 --> 01:20:30,600 THERE. 1721 01:20:30,600 --> 01:20:32,240 MOVING MORE CLOSELY, MICROSCOPIC 1722 01:20:32,240 --> 01:20:35,560 CLOSE-UP OF THE RIGHT ATRIUM, 1723 01:20:35,560 --> 01:20:37,200 WE'RE SEEING SIGNIFICANT 1724 01:20:37,200 --> 01:20:39,360 MYOCARDIAL REPLACEMENT, 5% OF 1725 01:20:39,360 --> 01:20:40,480 MYOCYTES REMAIN, AT INTEGRALS 1726 01:20:40,480 --> 01:20:44,200 YOU SEE ON THIS EPICARDIAL 1727 01:20:44,200 --> 01:20:46,720 SURFACE MORE INFILTRATE WITH 1728 01:20:46,720 --> 01:20:48,760 MORE REPLACEMENT OF MYOCYTES AS 1729 01:20:48,760 --> 01:20:48,960 WELL. 1730 01:20:48,960 --> 01:20:53,360 THIS HELD TRUE IN ALL OF THE 1731 01:20:53,360 --> 01:20:53,960 PATIENTS ESSENTIALLY, SAMPLES 1732 01:20:53,960 --> 01:20:59,720 TAKEN FROM AUTOPSY, SHOWING THAT 1733 01:20:59,720 --> 01:21:05,200 FAT AND INFILL INFILTRATE IS SO 1734 01:21:05,200 --> 01:21:06,200 DIFFICULT TO ASSESS WITH 1735 01:21:06,200 --> 01:21:07,480 EXTERNAL VIEWS OF OUR PATIENTS. 1736 01:21:07,480 --> 01:21:09,800 HOW DO WE UNDERSTAND THAT 1737 01:21:09,800 --> 01:21:14,800 DISEASE PROGRESSION WITHOUT AN 1738 01:21:14,800 --> 01:21:16,120 AUTOPSY, WITHOUT PATHOLOGIC 1739 01:21:16,120 --> 01:21:16,360 SAMPLES? 1740 01:21:16,360 --> 01:21:20,720 THE ANSWER IS, OF COURSE, VERY 1741 01:21:20,720 --> 01:21:20,960 POORLY. 1742 01:21:20,960 --> 01:21:24,000 THESE ARE FROM VANDERBILT 1743 01:21:24,000 --> 01:21:24,680 UNIVERSITY. 1744 01:21:24,680 --> 01:21:27,800 WE LOOKED AT BIOMARKERS, NO 1745 01:21:27,800 --> 01:21:29,560 CORRELATION WITH LD EJECTION 1746 01:21:29,560 --> 01:21:33,080 FRACTION, DO NOT CORRELATE 1747 01:21:33,080 --> 01:21:37,200 FUNCTION WITH THESE BLOOD 1748 01:21:37,200 --> 01:21:37,520 BIOMARKERS. 1749 01:21:37,520 --> 01:21:40,640 THIS IS -- WE BEGAN TO 1750 01:21:40,640 --> 01:21:42,840 UNDERSTAND BACK IN EARLY 2000s 1751 01:21:42,840 --> 01:21:46,760 HOW MRI COULD BE A SIGNIFICANT 1752 01:21:46,760 --> 01:21:51,520 BENEFIT, SPECIFICALLY WITH 1753 01:21:51,520 --> 01:21:55,680 REGARDS TO GADOLINIUM 1754 01:21:55,680 --> 01:21:57,160 ENHANCEMENT, CALLED FIBROSIS, 1755 01:21:57,160 --> 01:21:58,920 INCREASING ENHANCEMENT 1756 01:21:58,920 --> 01:21:59,800 ASSOCIATED WITH WORSE EJECTION 1757 01:21:59,800 --> 01:22:02,360 FRACTION AND INCREASED RATES OF 1758 01:22:02,360 --> 01:22:02,760 MORTALITY. 1759 01:22:02,760 --> 01:22:04,840 HOWEVER, IT'S A BLUNT TOOL. 1760 01:22:04,840 --> 01:22:10,560 ISN'T NECESSARILY THE MOST CLEAR 1761 01:22:10,560 --> 01:22:10,800 OUTCOME. 1762 01:22:10,800 --> 01:22:14,840 FIBROSIS IS SEEN ON THE LEFT 1763 01:22:14,840 --> 01:22:19,320 IMAGE, CORRELATING WITH PERHAPS 1764 01:22:19,320 --> 01:22:21,640 THE FATTY INFILTRATE, IT'S NOT 1765 01:22:21,640 --> 01:22:27,320 ALWAYS 1:1 AND CAN BE DIFFICULT 1766 01:22:27,320 --> 01:22:29,080 TO ASSESS. 1767 01:22:29,080 --> 01:22:35,080 THE LEADING NIH FUNDED R01 1768 01:22:35,080 --> 01:22:38,680 CENTER STUDY LOOKING AT MRI AT 1769 01:22:38,680 --> 01:22:41,560 LATE GADOLINIUM ENHANCEMENT, T1 1770 01:22:41,560 --> 01:22:43,600 TIMES, EXTRACELLULAR VOLUME, 1771 01:22:43,600 --> 01:22:54,040 NORMAL PATIENTS CAN HAVE 1772 01:22:55,640 --> 01:22:57,440 DIFFERENT LGE PATTERN, HOW DO WE 1773 01:22:57,440 --> 01:22:59,400 UNDERSTAND THIS AS ACTUALLY WHAT 1774 01:22:59,400 --> 01:23:04,640 IS GOING ON IN MYOCARDIAL 1775 01:23:04,640 --> 01:23:04,880 TISSUE. 1776 01:23:04,880 --> 01:23:09,440 THIS IS A THOUGHT EXPERIMENT HOW 1777 01:23:09,440 --> 01:23:10,680 YOU TAKE EXTRACELLULAR VOLUME, 1778 01:23:10,680 --> 01:23:14,960 NATIVE T1 AND PLACE THEM ON 1779 01:23:14,960 --> 01:23:20,000 DIFFERENT PARTS OF THE SCALE, 1780 01:23:20,000 --> 01:23:22,040 UNDERSTAND FIBROSIS, EDEMA, 1781 01:23:22,040 --> 01:23:24,760 FATTY INFILTRATE. 1782 01:23:24,760 --> 01:23:29,160 DR. SASLOW WAS ABLE TO EVALUATE 1783 01:23:29,160 --> 01:23:31,040 MANY PATIENTS, FINDS THIS 1784 01:23:31,040 --> 01:23:32,760 SETTLES OUT WHERE LARGER VOLUMES 1785 01:23:32,760 --> 01:23:35,720 SETTLE OUT AS LARGER DOTS AND 1786 01:23:35,720 --> 01:23:38,040 CAN SPREAD PATIENTS ALONG T2 AND 1787 01:23:38,040 --> 01:23:39,560 T1 TIMES. 1788 01:23:39,560 --> 01:23:43,320 BY TAKING THOSE TIMES WE CAN 1789 01:23:43,320 --> 01:23:46,920 POTENTIALLY LOOK AT EJECTION 1790 01:23:46,920 --> 01:23:48,200 FRACTIONS, CHARACTERIZING NORMAL 1791 01:23:48,200 --> 01:23:53,160 HEART, PERHAPS HIGHER EJECTION 1792 01:23:53,160 --> 01:23:55,040 FRACTION, TIMES INCREASE, 1793 01:23:55,040 --> 01:23:59,400 PERHAPS AS MORE FAT OCCUR, 1794 01:23:59,400 --> 01:24:00,920 DECREASING EJECTION FRACTURE. 1795 01:24:00,920 --> 01:24:02,800 THIS IS PRELIMINARY DATA. 1796 01:24:02,800 --> 01:24:06,840 IT DOES PROVIDE MECHANISM TO 1797 01:24:06,840 --> 01:24:09,920 POTENTIALLY HAVE A REALLY USEFUL 1798 01:24:09,920 --> 01:24:11,240 SURROGATE ENDPOINT TO FOLLOW 1799 01:24:11,240 --> 01:24:19,160 MUCH MORE CLOSELY THAN 1800 01:24:19,160 --> 01:24:19,600 MORTALITY. 1801 01:24:19,600 --> 01:24:22,200 HOW CAN WE ASSESS? 1802 01:24:22,200 --> 01:24:25,040 WHAT PREVENTIVE TREATMENTS ARE 1803 01:24:25,040 --> 01:24:25,960 BEING USED? 1804 01:24:25,960 --> 01:24:27,800 THIS IS A FRENCH STUDY 1805 01:24:27,800 --> 01:24:30,000 DEMONSTRATING USE OF ACE 1806 01:24:30,000 --> 01:24:33,800 INHIBITORS ON MORTALITY IN 1807 01:24:33,800 --> 01:24:37,640 DUCHENNE, INCREASED HAZARD OF 1808 01:24:37,640 --> 01:24:43,760 MORTALITY, DATA FROM OHIO FROM 1809 01:24:43,760 --> 01:24:53,720 MY COLLEAGUES, LOOKING AT USE ON 1810 01:24:53,720 --> 01:24:57,440 EJECTION FRACTION, IN WHICH 1811 01:24:57,440 --> 01:24:58,840 EPLERONONE TENDS TOWARD 1812 01:24:58,840 --> 01:24:59,880 DECREASED CHANGE IN EJECTION 1813 01:24:59,880 --> 01:25:01,040 FRACTION BASED ON STUDIES SUCH 1814 01:25:01,040 --> 01:25:03,320 AS THESE WE HAVE COMMENTS FROM 1815 01:25:03,320 --> 01:25:09,600 THE AMERICAN HEART ASSOCIATION, 1816 01:25:09,600 --> 01:25:20,040 TELLING US WE CAN USE WITH 1817 01:25:21,040 --> 01:25:22,760 DECREASED FUNCTION. 1818 01:25:22,760 --> 01:25:32,000 THIS DATA IS INCONSISTENTLY 1819 01:25:32,000 --> 01:25:35,240 USED AND HARD TO TEASE OUT. 1820 01:25:35,240 --> 01:25:37,640 WE HAVE QUERIED NUMBER OF 1821 01:25:37,640 --> 01:25:38,600 MUSCULAR DYSTROPHY CARDIOLOGISTS 1822 01:25:38,600 --> 01:25:43,920 IN THE UNITED STATES, AND 1823 01:25:43,920 --> 01:25:48,760 DEMONSTRATED GREAT VARIABILITY. 1824 01:25:48,760 --> 01:25:52,800 ON THE LEFT INDICATIONS, 68% 1825 01:25:52,800 --> 01:25:56,320 HAVE USED ACEs AND ARBs, 10 1826 01:25:56,320 --> 01:25:59,720 OR GREATER, MILD SYSTOLIC 1827 01:25:59,720 --> 01:26:04,200 DYSFUNCTION, 23% FOR THOSE WITH 1828 01:26:04,200 --> 01:26:06,640 LATE GADOLINIUM ENHANCEMENT, AND 1829 01:26:06,640 --> 01:26:08,560 MANY INDICATIONS AND NOT ALWAYS 1830 01:26:08,560 --> 01:26:11,280 PURELY BASED ON AGE RELATION. 1831 01:26:11,280 --> 01:26:15,680 SO, WITH ALL OF THAT IT'S 1832 01:26:15,680 --> 01:26:16,800 DIFFICULT TO DECONSTRUCT WHAT 1833 01:26:16,800 --> 01:26:19,000 PATIENTS ARE ON WHAT MEDICINES, 1834 01:26:19,000 --> 01:26:21,840 WHAT EFFECTS ON HEARTS, AND HOW 1835 01:26:21,840 --> 01:26:23,920 WE CAN ASSESS TREATMENT DOSE OR 1836 01:26:23,920 --> 01:26:24,760 NEW THERAPIES. 1837 01:26:24,760 --> 01:26:28,840 WHERE ARE WE GOING AND HOW DO WE 1838 01:26:28,840 --> 01:26:30,720 GET THAT DATA? 1839 01:26:30,720 --> 01:26:32,120 SO THE MEETING ACTUALLY SPENT A 1840 01:26:32,120 --> 01:26:33,560 FAIR AMOUNT OF TIME TALKING 1841 01:26:33,560 --> 01:26:36,280 ABOUT THIS AND WE ENDED FOCUSING 1842 01:26:36,280 --> 01:26:40,320 ON THREE ASPECTS OF CARE, 1843 01:26:40,320 --> 01:26:42,000 COLLABORATION THROUGH THE ACTION 1844 01:26:42,000 --> 01:26:43,720 NETWORK, HARMONIZATION OF CARE, 1845 01:26:43,720 --> 01:26:45,280 THE OTHER BEING ENDPOINTS, WHICH 1846 01:26:45,280 --> 01:26:48,000 WE NEED TO FOLLOW. 1847 01:26:48,000 --> 01:26:49,280 REGARDING COLLABORATION, SO 1848 01:26:49,280 --> 01:26:51,520 ACTION, THE ADVANCED CARDIAC 1849 01:26:51,520 --> 01:26:52,960 THERAPIES IMPROVING OUTCOMES 1850 01:26:52,960 --> 01:26:56,120 NETWORK FOUNDED IN 2017 WITH 1851 01:26:56,120 --> 01:26:57,720 IDEA THAT ARE DIVERSITIES IN 1852 01:26:57,720 --> 01:27:00,160 CHILDREN WITH NO STANDARD OF 1853 01:27:00,160 --> 01:27:00,360 CARE. 1854 01:27:00,360 --> 01:27:07,480 WE BEGAN WORKING WITH PATIENTS 1855 01:27:07,480 --> 01:27:08,400 WITH CARDIOMYOPATHY 1856 01:27:08,400 --> 01:27:14,440 MYOCART ITIS, THERE'S A 1857 01:27:14,440 --> 01:27:15,760 COMPONENT FOCUSED ON PATIENTS, 1858 01:27:15,760 --> 01:27:19,600 NOT STUDIED IN PEDIATRICS OR 1859 01:27:19,600 --> 01:27:21,040 DISEASES WE TREAT. 1860 01:27:21,040 --> 01:27:22,680 OUR DATA IS LARGELY EXTRAPOLATED 1861 01:27:22,680 --> 01:27:28,120 FROM ADULT STUDIES THAT THE 1862 01:27:28,120 --> 01:27:29,320 MEDICATIONS MONITORING 1863 01:27:29,320 --> 01:27:30,520 REHABILITATION, AND THUS WE HAVE 1864 01:27:30,520 --> 01:27:33,320 LESS KNOWLEDGE OF HOW TO USE 1865 01:27:33,320 --> 01:27:36,240 THESE DEVICES AND THERAPIES IN 1866 01:27:36,240 --> 01:27:39,120 THESE SPECIAL POPULATIONS. 1867 01:27:39,120 --> 01:27:44,600 SO, ACTION WAS FOUNDED IN 2017, 1868 01:27:44,600 --> 01:27:47,440 60 CENTERS NOW, THESE ARE ACROSS 1869 01:27:47,440 --> 01:27:49,840 NORTH AMERICA AND EUROPE, WHICH 1870 01:27:49,840 --> 01:27:51,040 HAVE SHOWN SIGNIFICANT ABILITY 1871 01:27:51,040 --> 01:27:53,560 TO GET WORK DONE. 1872 01:27:53,560 --> 01:27:58,120 FOR INSTANCE, WE USE COMMONLY 1873 01:27:58,120 --> 01:28:00,960 THE BERLIN X CORE, KEPT FOR 1874 01:28:00,960 --> 01:28:03,600 SMALL CHILDREN, AND INFANTS 1875 01:28:03,600 --> 01:28:05,240 UNABLE TO GET LARGER DEVICES, 1876 01:28:05,240 --> 01:28:06,560 HISTORICALLY HIGH STROKE RATE OF 1877 01:28:06,560 --> 01:28:08,560 25% IN PRIOR REGISTRY DATA. 1878 01:28:08,560 --> 01:28:18,200 HOWEVER, WITH HARMONIZING CARE 1879 01:28:18,200 --> 01:28:23,080 CUT TO 13% ALLOWS US TO USE THAT 1880 01:28:23,080 --> 01:28:24,880 DEVICE FOR MANY PATIENTS. 1881 01:28:24,880 --> 01:28:34,720 WE'RE ABLE TO ALSO COLLABORATE, 1882 01:28:34,720 --> 01:28:37,480 THE ONLY DISCHARGEABLE LVAD THAT 1883 01:28:37,480 --> 01:28:40,960 EXISTS, TWO-YEAR POST 1884 01:28:40,960 --> 01:28:42,960 SURVEILLANCE IN PEDIATRIC REALM, 1885 01:28:42,960 --> 01:28:45,440 DATA TO FDA COLLABORATION AND 1886 01:28:45,440 --> 01:28:47,400 HAVE A PEDIATRIC LABELING WHICH 1887 01:28:47,400 --> 01:28:49,280 IS VERY RARE IN PEDIATRICS THAT 1888 01:28:49,280 --> 01:28:54,320 WE'RE ABLE TO GET DATA LABELING 1889 01:28:54,320 --> 01:28:55,680 FOR OUR THERAPIES. 1890 01:28:55,680 --> 01:28:57,720 SO, ACTION ALREADY HAS AN IRB 1891 01:28:57,720 --> 01:29:00,960 AND INFRASTRUCTURE IN PLACE TO 1892 01:29:00,960 --> 01:29:02,320 CORRECT DATA, INHOUSE REVIEW, A 1893 01:29:02,320 --> 01:29:04,080 PROVEN RECORD WITH THE FDA 1894 01:29:04,080 --> 01:29:05,400 REGARDING DEVICES, BRANCHING OUT 1895 01:29:05,400 --> 01:29:15,960 MORE AND MORE SIGNIFICANTLY AND 1896 01:29:16,960 --> 01:29:17,200 BRANCHING OUT. 1897 01:29:17,200 --> 01:29:18,440 HARMONIZATION IS REALLY 1898 01:29:18,440 --> 01:29:23,120 IMPORTANT PART OF THIS. 1899 01:29:23,120 --> 01:29:27,720 WE HAVE GREAT GENETIC 1900 01:29:27,720 --> 01:29:28,360 HETEROGENEITY, PHENOTYPIC 1901 01:29:28,360 --> 01:29:32,200 HETEROGENEITY AND LACK OF 1902 01:29:32,200 --> 01:29:35,600 GUIDELINES, IT'S DIFFICULT TO 1903 01:29:35,600 --> 01:29:44,120 UNDERSTAND EFFECTS OF OTHER CARE 1904 01:29:44,120 --> 01:29:45,480 AND PROTOPLASM. 1905 01:29:45,480 --> 01:29:55,800 THERE IS NO DATA, LARGELY 1906 01:29:55,800 --> 01:29:58,200 CONSENSUS DRIVEN. 1907 01:29:58,200 --> 01:30:01,040 THESE THERAPIES HAVE ALSO BEEN 1908 01:30:01,040 --> 01:30:02,200 HARMONIZED WITH DUCHENNE 1909 01:30:02,200 --> 01:30:05,760 POPULATION, ON VERSE 3 OF THE 1910 01:30:05,760 --> 01:30:07,160 HARMONIZATION OF BOTH 1911 01:30:07,160 --> 01:30:08,400 PROPHYLACTIC AND THERAPEUTIC 1912 01:30:08,400 --> 01:30:11,000 TREATMENTS FOR THE MUSCULAR 1913 01:30:11,000 --> 01:30:12,120 DYSTROPHY POPULATION, WITH THESE 1914 01:30:12,120 --> 01:30:13,840 WE WERE HAVING INCREASING UPTAKE 1915 01:30:13,840 --> 01:30:17,480 ACROSS MANY CENTERS, NOT JUST 1916 01:30:17,480 --> 01:30:22,960 THE LARGEST CORE CENTERS OF THE 1917 01:30:22,960 --> 01:30:23,360 ACTION COMMUNITY. 1918 01:30:23,360 --> 01:30:25,800 HOWEVER, IN THE END WHAT WE 1919 01:30:25,800 --> 01:30:29,080 NEED, ENDS POINTS AND DATA, 1920 01:30:29,080 --> 01:30:30,240 MODERN HISTORY STUDY WITH 1921 01:30:30,240 --> 01:30:31,160 STANDARDIZED BACKGROUND 1922 01:30:31,160 --> 01:30:32,600 THERAPIES, NEED TO UNDERSTAND 1923 01:30:32,600 --> 01:30:34,440 CARDIAC MRI DATA BETTER AND 1924 01:30:34,440 --> 01:30:37,120 STILL NEED TO GO AFTER FUNCTION 1925 01:30:37,120 --> 01:30:41,000 AND HOW PATIENTS ARE FEELING, 1926 01:30:41,000 --> 01:30:45,720 WHAT ARE PATIENT-REPORTED 1927 01:30:45,720 --> 01:30:45,960 OUTCOMES. 1928 01:30:45,960 --> 01:30:49,760 SO IN PARTNERSHIP, WE HAVE BEEN 1929 01:30:49,760 --> 01:30:51,120 GRANTED A GRANT TO FACILITATE 1930 01:30:51,120 --> 01:30:53,840 BROAD DATA ENTRY, WE'RE HOPING 1931 01:30:53,840 --> 01:30:57,760 TO EXPLOIT THESE INCREASING 1932 01:30:57,760 --> 01:30:58,360 STANDARDIZATION OF BACKGROUND 1933 01:30:58,360 --> 01:31:00,200 THERAPIES, WE HAVE THE MEDICINES 1934 01:31:00,200 --> 01:31:01,400 AND WE'RE NOT SURE HOW MUCH THEY 1935 01:31:01,400 --> 01:31:02,920 ARE BENEFICIAL TO OUR PATIENT 1936 01:31:02,920 --> 01:31:09,480 POPULATION BUT FEEL THEY MAY BE. 1937 01:31:09,480 --> 01:31:10,440 WE HAVE ENTRESTO, INHIBITORS, 1938 01:31:10,440 --> 01:31:15,920 AND FURTHER USE OF GENE 1939 01:31:15,920 --> 01:31:17,680 THERAPIES, VECTOR DRIVEN OR EXON 1940 01:31:17,680 --> 01:31:19,520 SKIPPING, ALL OF THOSE HAVE 1941 01:31:19,520 --> 01:31:20,760 VARYING EFFECTS ON CARDIAC CARE 1942 01:31:20,760 --> 01:31:22,840 AND WE DO NOT UNDERSTAND THEM 1943 01:31:22,840 --> 01:31:24,920 FULLY BUT BY WORKING TO CREATE A 1944 01:31:24,920 --> 01:31:27,560 BETTER NATURAL HISTORY STUDY AS 1945 01:31:27,560 --> 01:31:29,520 WELL AS CARVEOUTS FOR 1946 01:31:29,520 --> 01:31:30,240 UNDERSTANDING NEW THERAPIES, WE 1947 01:31:30,240 --> 01:31:31,760 VERY MUCH HOPE TO UNDERSTAND 1948 01:31:31,760 --> 01:31:33,680 WHAT THAT CARDIAC PROCESS IS 1949 01:31:33,680 --> 01:31:36,080 GOING TO BE, WE'RE HOPING TO 1950 01:31:36,080 --> 01:31:37,920 LAUNCH BROAD DATA COLLECTION 1951 01:31:37,920 --> 01:31:40,440 STARTING NEXT QUARTER, AFTER 1952 01:31:40,440 --> 01:31:43,240 HAVING STARTED PILOT SITES 1953 01:31:43,240 --> 01:31:43,800 ALREADY. 1954 01:31:43,800 --> 01:31:46,080 MRI DATA IS GROSSLY NEEDING TO 1955 01:31:46,080 --> 01:31:46,560 BE STANDARDIZED. 1956 01:31:46,560 --> 01:31:48,040 WE BELIEVE THIS IS GOING TO BE 1957 01:31:48,040 --> 01:31:49,240 THE SURROGATE OUTCOME WITH WHICH 1958 01:31:49,240 --> 01:31:50,960 WE'LL BE ABLE TO UNDERSTAND MORE 1959 01:31:50,960 --> 01:31:53,720 READILY HOW OUR PATIENTS ARE 1960 01:31:53,720 --> 01:31:59,640 DOING. 1961 01:31:59,640 --> 01:32:01,360 WE'RE CONTRIBUTING TO MRI 1962 01:32:01,360 --> 01:32:02,160 STANDARDIZATION FOR DIFFERENT 1963 01:32:02,160 --> 01:32:04,240 DEVICES AND DATA ACQUISITION AS 1964 01:32:04,240 --> 01:32:05,680 WELL AS STANDARDIZING DATA 1965 01:32:05,680 --> 01:32:07,120 REPORT AND INTERPRETATION SO 1966 01:32:07,120 --> 01:32:09,080 WHEN THE MRI DATA BECOMES MORE 1967 01:32:09,080 --> 01:32:10,920 PRIME TIME FOR USE AS A 1968 01:32:10,920 --> 01:32:13,560 SURROGATE MARKER, WE'LL BE ABLE 1969 01:32:13,560 --> 01:32:14,760 TO QUICKLY ADOPT TO USING THEM 1970 01:32:14,760 --> 01:32:17,400 AS SUCH. 1971 01:32:17,400 --> 01:32:19,240 LASTLY PATIENT-REPORTED 1972 01:32:19,240 --> 01:32:21,080 OUTCOMES, ACTION HAS DEVELOPED 1973 01:32:21,080 --> 01:32:24,480 PEDIATRIC RELATED PATIENT 1974 01:32:24,480 --> 01:32:30,120 REPORTED OUTCOMES AND WE'RE 1975 01:32:30,120 --> 01:32:32,160 ROLLING THOSE OUT CURRENTLY. 1976 01:32:32,160 --> 01:32:37,840 WE'RE ACTUALLY WORKING THIS 1977 01:32:37,840 --> 01:32:43,000 COMING MONTH TO START 1978 01:32:43,000 --> 01:32:43,400 DYSTROPHINOPATHY. 1979 01:32:43,400 --> 01:32:45,120 WE'RE POISED WITH A ROAD MAP FOR 1980 01:32:45,120 --> 01:32:47,480 WHERE WE ARE AND WHERE WE'RE 1981 01:32:47,480 --> 01:32:49,440 GOING, IMPROVING CARDIAC CARE IN 1982 01:32:49,440 --> 01:32:50,360 THIS POPULATION. 1983 01:32:50,360 --> 01:32:54,680 WE THANK MY COLLEAGUES AND 1984 01:32:54,680 --> 01:32:56,320 COLLABORATORS CHET AND LINDA, 1985 01:32:56,320 --> 01:32:57,280 COLLEAGUES, FAMILIES, PATIENTS 1986 01:32:57,280 --> 01:32:58,480 WORLDWIDE. 1987 01:32:58,480 --> 01:33:03,080 WE DO THIS WORK THROUGH THE 1988 01:33:03,080 --> 01:33:05,280 UMBRELLA OF ACTION AND GENEROUS 1989 01:33:05,280 --> 01:33:08,440 FUNDING OF MUSCULAR DYSTROPHY TO 1990 01:33:08,440 --> 01:33:10,080 UNDERSTAND NEXT STEPS. 1991 01:33:10,080 --> 01:33:12,120 THANK YOU VERY MUCH, BACK ON 1992 01:33:12,120 --> 01:33:13,000 TIME. 1993 01:33:13,000 --> 01:33:15,800 >>A GREAT PRESENTATION. 1994 01:33:15,800 --> 01:33:19,640 REALLY IMPORTANT TO SHINE A 1995 01:33:19,640 --> 01:33:23,240 SPOTLIGHT ON THIS PROBLEM, NOW 1996 01:33:23,240 --> 01:33:24,640 THAT BOYS WITH MUSCULAR 1997 01:33:24,640 --> 01:33:26,880 DYSTROPHY ARE LIVING LONGER FROM 1998 01:33:26,880 --> 01:33:32,880 A RESPIRATORY PERSPECTIVE THAT 1999 01:33:32,880 --> 01:33:36,720 CARDIAC COMPLICATIONS ARE MORE 2000 01:33:36,720 --> 01:33:39,320 SIGNIFICANT, OR BEING 2001 01:33:39,320 --> 01:33:40,080 RECOGNIZED. 2002 01:33:40,080 --> 01:33:40,560 EXCELLENT WORK. 2003 01:33:40,560 --> 01:33:41,600 BECAUSE YOU WERE ON TIME WE'LL 2004 01:33:41,600 --> 01:33:43,920 LET YOU HAVE A COUPLE QUESTIONS 2005 01:33:43,920 --> 01:33:47,600 IF ANYBODY HAS QUESTIONS FOR DR. 2006 01:33:47,600 --> 01:33:47,800 NANDI? 2007 01:33:47,800 --> 01:33:51,640 DON'T BE SHY. 2008 01:33:51,640 --> 01:33:56,400 2009 01:33:56,400 --> 01:33:58,680 I GUESS YOU MUST HAVE ANSWERED 2010 01:33:58,680 --> 01:34:01,080 THEM OR ADDRESSED THEM ALL IN 2011 01:34:01,080 --> 01:34:03,080 YOUR PRESENTATION WHICH I 2012 01:34:03,080 --> 01:34:05,160 THOUGHT WAS VERY CLEAR. 2013 01:34:05,160 --> 01:34:09,080 IT'S GREAT TO HEAR ENTRESSTO IS 2014 01:34:09,080 --> 01:34:11,600 GETTING APPROVED FOR CHILDREN, I 2015 01:34:11,600 --> 01:34:13,360 KNOW SEVERAL ADULTS ON IT WHO 2016 01:34:13,360 --> 01:34:14,760 SAY IT'S A MIRACLE DRUG. 2017 01:34:14,760 --> 01:34:18,160 >>IT IS GREATLY HELPING 2018 01:34:18,160 --> 01:34:24,080 PATIENTS, IT WOULD BE NICE TO BE 2019 01:34:24,080 --> 01:34:24,960 MORE THAN ANECDOTAL. 2020 01:34:24,960 --> 01:34:32,160 >>WE'LL TAKE A SHORT BACK. 2021 01:34:32,160 --> 01:34:33,720 GLEN, 15-MINUTE BREAK OR STICK 2022 01:34:33,720 --> 01:34:34,720 TO SCHEDULE AND GIVE 10-MINUTE 2023 01:34:34,720 --> 01:34:35,120 BREAK. 2024 01:34:35,120 --> 01:34:36,240 >>UP TO YOU. 2025 01:34:36,240 --> 01:34:37,840 WE COULD AFFORD A 15-MINUTE 2026 01:34:37,840 --> 01:34:40,600 BREAK IF YOU'D LIKE. 2027 01:34:40,600 --> 01:34:42,160 WE'LL COME BACK AT 2:50. 2028 01:34:42,160 --> 01:34:45,640 >>WE'LL SEE YOU THEN. 2029 01:34:45,640 --> 01:34:46,160 THANK YOU. 2030 01:34:46,160 --> 01:34:48,640 >>WELCOME EVERYONE BACK. 2031 01:34:48,640 --> 01:34:51,040 THE MAIN TOPIC IS INNOVATIVE 2032 01:34:51,040 --> 01:34:54,040 OUTCOME MEASURES FOR MUSCULAR 2033 01:34:54,040 --> 01:34:54,360 DYSTROPHIES. 2034 01:34:54,360 --> 01:34:56,000 I'LL TURN THE MEETING OVER TO 2035 01:34:56,000 --> 01:34:59,480 GLEN TO INTRODUCE THIS SESSION. 2036 01:34:59,480 --> 01:35:01,480 >>I WANTED TO TAKE A COUPLE 2037 01:35:01,480 --> 01:35:04,320 MINUTES TO SET THE STAGE FOR OUR 2038 01:35:04,320 --> 01:35:07,800 NEXT SET OF PRESENTATIONS ON 2039 01:35:07,800 --> 01:35:09,440 INNOVATIVE OUTCOME MEASURES AND 2040 01:35:09,440 --> 01:35:11,080 INTRODUCE CONCEPTS THAT WE'RE 2041 01:35:11,080 --> 01:35:17,080 LIKELY TO BE DISCUSSING TODAY. 2042 01:35:17,080 --> 01:35:19,600 NIH SELECTED THIS TOPIC, IT DOES 2043 01:35:19,600 --> 01:35:21,240 NOT NECESSARILY MEAN THAT WE 2044 01:35:21,240 --> 01:35:24,240 INTEND TO DEVELOP NEW 2045 01:35:24,240 --> 01:35:27,920 INITIATIVES ON THIS TOPIC, AND 2046 01:35:27,920 --> 01:35:29,040 IF YOU'RE INTERESTED IN GUIDANCE 2047 01:35:29,040 --> 01:35:31,000 IN SUPPORTING RESEARCH IN THIS 2048 01:35:31,000 --> 01:35:32,720 AREA, PLEASE CONTACT ME, THERE 2049 01:35:32,720 --> 01:35:35,480 ARE A NUMBER OF EXISTING FUNDING 2050 01:35:35,480 --> 01:35:40,080 OPPORTUNITIES THAT I CAN SUGGEST 2051 01:35:40,080 --> 01:35:42,280 FOR YOU. 2052 01:35:42,280 --> 01:35:49,400 AS DEFINED IN THE RESOURCE ON 2053 01:35:49,400 --> 01:35:50,920 BIOMARKERS, ENDPOINTS AND TOOLS, 2054 01:35:50,920 --> 01:35:54,200 COAs ARE MEASURES THAT 2055 01:35:54,200 --> 01:35:59,240 DESCRIBE OR REFLECT HOW AN 2056 01:35:59,240 --> 01:36:00,640 INDIVIDUAL FEELS, FUNCTIONS, 2057 01:36:00,640 --> 01:36:04,680 SURVIVES, IN THE CATEGORIES OF 2058 01:36:04,680 --> 01:36:06,040 CLINICIAN OR OBSERVER REPORTED, 2059 01:36:06,040 --> 01:36:07,320 PATIENT REPORTED, ACTUALLY A 2060 01:36:07,320 --> 01:36:11,720 TOPIC WE DISCUSSED IN JUNE OF 2061 01:36:11,720 --> 01:36:14,760 2020 OR MORE OF WHAT WE TALK 2062 01:36:14,760 --> 01:36:16,280 ABOUT TODAY, PERFORMANCE 2063 01:36:16,280 --> 01:36:21,840 OUTCOMES, MEASUREMENTS OF 2064 01:36:21,840 --> 01:36:22,280 PATIENTS UNDERTAKING 2065 01:36:22,280 --> 01:36:24,200 STANDARDIZED TASKS. 2066 01:36:24,200 --> 01:36:26,000 SOME EXAMPLES ARE ALSO NOT SO 2067 01:36:26,000 --> 01:36:27,840 MUCH DIRECT MEASURES OF FUNCTION 2068 01:36:27,840 --> 01:36:30,120 BUT MORE INDIRECT MEASURES OF 2069 01:36:30,120 --> 01:36:34,400 FUNCTION, THEY MAY FIT INTO A 2070 01:36:34,400 --> 01:36:35,400 CATEGORY CALLED DIGITAL 2071 01:36:35,400 --> 01:36:37,760 BIOMARKERS RATHER THAN CLINICAL 2072 01:36:37,760 --> 01:36:38,760 OUTCOME MEASURES, MUCH OF THE 2073 01:36:38,760 --> 01:36:39,480 SAME APPLIES. 2074 01:36:39,480 --> 01:36:43,480 WE HEARD FROM DR. NANDI ABOUT 2075 01:36:43,480 --> 01:36:44,920 MEASURES OF CARDIAC PERFORMANCE 2076 01:36:44,920 --> 01:36:47,400 IN DUCHENNE, WHICH COULD BE USED 2077 01:36:47,400 --> 01:36:49,480 IN CLINICAL TRIALS THAT ARE 2078 01:36:49,480 --> 01:36:52,440 TARGETING THE HEART. 2079 01:36:52,440 --> 01:36:54,960 MANY OF THE CLINICAL TRIALS 2080 01:36:54,960 --> 01:36:56,160 TARGET SKELETAL MUSCLE. 2081 01:36:56,160 --> 01:36:59,120 I WANTED TO BRIEFLY REVIEW THE 2082 01:36:59,120 --> 01:37:02,040 CLINICAL OUTCOME ASSESSMENTS 2083 01:37:02,040 --> 01:37:06,320 USED IN THOSE TRIALS. 2084 01:37:06,320 --> 01:37:08,640 SO, COMMONLY USED MEASURES 2085 01:37:08,640 --> 01:37:12,360 INCLUDE MUSCLE STRENGTH TESTING, 2086 01:37:12,360 --> 01:37:13,440 TIMED FUNCTION TESTS, RATINGS 2087 01:37:13,440 --> 01:37:16,280 SCALES SUCH AS NORTH STAR AND 2088 01:37:16,280 --> 01:37:17,280 LET'S INCLUDE RESPIRATORY 2089 01:37:17,280 --> 01:37:20,120 OUTCOME MEASURES WHICH WE ALSO 2090 01:37:20,120 --> 01:37:24,040 DISCUSSED IN JUNE OF LAST YEAR 2091 01:37:24,040 --> 01:37:27,560 AT THE MDCC MEETING, AND 2092 01:37:27,560 --> 01:37:35,240 DIAPHRAGM AND MUSCLES OF 2093 01:37:35,240 --> 01:37:37,440 RESPIRATORY OR SKELETAL MUSCLES. 2094 01:37:37,440 --> 01:37:39,840 DIRECT MEASURES CAN STAND AS 2095 01:37:39,840 --> 01:37:40,640 PRIMARY OUTCOME MEASURE IN 2096 01:37:40,640 --> 01:37:42,480 CLINICAL TRIALS AND HAVE IN MANY 2097 01:37:42,480 --> 01:37:45,440 CASES, BUT CAN ALSO BE AFFECTED 2098 01:37:45,440 --> 01:37:47,840 BY OTHER FACTORS SUCH AS 2099 01:37:47,840 --> 01:37:52,200 MOTIVATION OF THE PATIENT OR 2100 01:37:52,200 --> 01:37:54,080 PERHAPS SUBCONSCIOUS BIAS OF 2101 01:37:54,080 --> 01:37:58,360 CLINICAL INVESTIGATOR, LEADING 2102 01:37:58,360 --> 01:37:59,960 TO VARIABILITY IN THE MEASURES. 2103 01:37:59,960 --> 01:38:01,840 ONE THING WE'RE STRIVING FOR IS 2104 01:38:01,840 --> 01:38:04,560 OUTCOME MEASURES THAT HAVE LESS 2105 01:38:04,560 --> 01:38:06,320 VARIABILITY AND ALSO ARE QUITE 2106 01:38:06,320 --> 01:38:06,720 SENSITIVE. 2107 01:38:06,720 --> 01:38:10,680 I WANTED TO POINT OUT THERE'S A 2108 01:38:10,680 --> 01:38:14,080 MEASURE THAT'S BEEN DEVELOPED, 2109 01:38:14,080 --> 01:38:16,720 DIGITAL MEASURE, THE FASTEST 5% 2110 01:38:16,720 --> 01:38:20,760 OF STREETS, MEASURED WITH A 2111 01:38:20,760 --> 01:38:21,760 VALIDATED ANKLE WORN 2112 01:38:21,760 --> 01:38:23,520 ACCELEROMETER, QUALIFIED BY THE 2113 01:38:23,520 --> 01:38:29,600 EMA AS A SECONDARY END POINT IN 2114 01:38:29,600 --> 01:38:32,280 DUCHENNE CLINICAL TRIALS, UNDER 2115 01:38:32,280 --> 01:38:33,560 REVIEW FOR QUALIFICATION FOR 2116 01:38:33,560 --> 01:38:34,680 FDA. 2117 01:38:34,680 --> 01:38:40,800 THIS IS A FIRST STEP TOWARDS 2118 01:38:40,800 --> 01:38:44,080 MORE TECHNOLOGICALLY ADVANCED 2119 01:38:44,080 --> 01:38:45,280 MEASURES AND BIOMARKERS FOR 2120 01:38:45,280 --> 01:38:46,480 PHYSICAL FUNCTION. 2121 01:38:46,480 --> 01:38:50,760 WE NEED OUTCOME MEASURES TO BE 2122 01:38:50,760 --> 01:38:52,880 ANALYTICALLY VALIDATED, KNOWN TO 2123 01:38:52,880 --> 01:38:55,080 MEASURE INTENDED FUNCTIONAL 2124 01:38:55,080 --> 01:38:57,360 STATE, NEED MEASUREMENTED THAT 2125 01:38:57,360 --> 01:38:59,040 COORDINATE WITH DISEASE STATE, 2126 01:38:59,040 --> 01:39:01,400 SENSITIVE TO CHANGES IN DISEASE 2127 01:39:01,400 --> 01:39:02,840 PROGRESSION THAT MAY OCCUR OVER 2128 01:39:02,840 --> 01:39:05,240 THE TRIAL DURATION WHICH IS 2129 01:39:05,240 --> 01:39:09,960 USUALLY ABOUT 1 TO 2 YEARS 2130 01:39:09,960 --> 01:39:10,800 DEPENDING ON THE TYPE OF 2131 01:39:10,800 --> 01:39:13,200 MUSCULAR DYSTROPHY. 2132 01:39:13,200 --> 01:39:14,880 WE NEED LOW VARIABILITY, LIMITED 2133 01:39:14,880 --> 01:39:17,480 EFFECT OF EXTERNAL FACTORS. 2134 01:39:17,480 --> 01:39:21,440 IF WE COMBINE SENSITIVITY AND 2135 01:39:21,440 --> 01:39:24,480 LOW VARIABILITY, IT MEANS THAT 2136 01:39:24,480 --> 01:39:27,080 WE CAN DESIGN CLINICAL TRIALS 2137 01:39:27,080 --> 01:39:28,440 THAT HAVE APPROPRIATE 2138 01:39:28,440 --> 01:39:33,520 STATISTICAL POWER WITH FEWER 2139 01:39:33,520 --> 01:39:38,120 PARTICIPANTS OR SHORTER 2140 01:39:38,120 --> 01:39:39,600 DURATION. 2141 01:39:39,600 --> 01:39:40,320 MEASUREMENTS NEED TO BE RELEVANT 2142 01:39:40,320 --> 01:39:47,880 TO QUALITY OF LIFE AND OVERALL 2143 01:39:47,880 --> 01:39:48,200 WELL-BEING. 2144 01:39:48,200 --> 01:39:50,680 IF THERE'S A LOT OF PATIENTS AND 2145 01:39:50,680 --> 01:39:53,560 A LOT OF SITES, THEN COST OF 2146 01:39:53,560 --> 01:39:54,880 EQUIPMENT AND AMOUNT OF TIME IT 2147 01:39:54,880 --> 01:39:57,480 TAKES TO TAKE THE MEASUREMENT 2148 01:39:57,480 --> 01:39:58,240 ALSO BECOME CONSIDERATIONS. 2149 01:39:58,240 --> 01:40:01,000 WE WOULD LIKE TO HAVE OUTCOME 2150 01:40:01,000 --> 01:40:04,040 MEASURES APPLICABLE TO BOTH 2151 01:40:04,040 --> 01:40:06,760 AMBULATORY AND NON-AMBULATORY 2152 01:40:06,760 --> 01:40:07,760 INDIVIDUALS WHEN POSSIBLE. 2153 01:40:07,760 --> 01:40:09,560 AND IF IT'S POSSIBLE WE WOULD 2154 01:40:09,560 --> 01:40:12,080 LIKE TO COLLECT DATA REMOTELY, 2155 01:40:12,080 --> 01:40:14,720 THIS CERTAINLY DECREASES THE 2156 01:40:14,720 --> 01:40:17,000 BURDEN OF STUDY PARTICIPATION 2157 01:40:17,000 --> 01:40:19,760 AND ALSO PROMOTES EQUITY, SO AS 2158 01:40:19,760 --> 01:40:21,920 WE DISCUSSED IN THE DECEMBER 2159 01:40:21,920 --> 01:40:24,120 MDCC MEETING LAST YEAR, BY 2160 01:40:24,120 --> 01:40:26,280 REDUCING THE BURDEN OF STUDY 2161 01:40:26,280 --> 01:40:30,800 PARTICIPATION WE CAN BETTER 2162 01:40:30,800 --> 01:40:32,560 ENSURE THE APPROPRIATE DIVERSITY 2163 01:40:32,560 --> 01:40:38,120 OF THE COHORTS. 2164 01:40:38,120 --> 01:40:42,600 SO, OUTCOME ASSESSMENTS CAN BE 2165 01:40:42,600 --> 01:40:44,800 USED AFTER THE DRUG IS APPROVED, 2166 01:40:44,800 --> 01:40:47,400 FOR EXAMPLE IN STUDIES OF 2167 01:40:47,400 --> 01:40:48,480 COMPARATIVE EFFECTIVENESS TO 2168 01:40:48,480 --> 01:40:51,960 DETERMINE IF ONE OF SEVERAL 2169 01:40:51,960 --> 01:40:55,240 TREATMENTS IS DESIRABLE OVER THE 2170 01:40:55,240 --> 01:40:55,560 OTHERS. 2171 01:40:55,560 --> 01:40:57,720 AND THEN IN SOME CASES WE FIND 2172 01:40:57,720 --> 01:40:58,400 INSURANCE COMPANIES REQUIRE 2173 01:40:58,400 --> 01:41:00,240 EVIDENCE THAT A DRUG CONTINUES 2174 01:41:00,240 --> 01:41:02,320 TO HAVE THE DESIRED EFFECT, FOR 2175 01:41:02,320 --> 01:41:04,200 THEM TO MAINTAIN COVERAGE FOR AN 2176 01:41:04,200 --> 01:41:05,320 INDIVIDUAL. 2177 01:41:05,320 --> 01:41:06,760 SO WE'D LIKE TO HAVE BIOMARKERS 2178 01:41:06,760 --> 01:41:08,440 OR OUTCOME MEASURES THAT COULD 2179 01:41:08,440 --> 01:41:12,680 ALSO BE USEFUL FOR THAT PURPOSE. 2180 01:41:12,680 --> 01:41:14,960 SO, A GOAL OF THE SESSION IS 2181 01:41:14,960 --> 01:41:18,160 REALLY TO HIGHLIGHT THE WORK 2182 01:41:18,160 --> 01:41:21,600 THAT'S AIMED AT IDENTIFYING, 2183 01:41:21,600 --> 01:41:22,920 VALIDATING, IMPLEMENTING 2184 01:41:22,920 --> 01:41:24,000 IMPROVED CLINICAL OUTCOME 2185 01:41:24,000 --> 01:41:26,280 ASSESSMENTS AND MEASURES TO 2186 01:41:26,280 --> 01:41:28,400 INCREASE THE EFFICIENCY AND 2187 01:41:28,400 --> 01:41:29,600 LIKELIHOOD OF SUCCESS IN 2188 01:41:29,600 --> 01:41:31,440 CLINICAL TRIALS AND POST 2189 01:41:31,440 --> 01:41:32,760 MARKETING DATA COLLECTION. 2190 01:41:32,760 --> 01:41:35,480 THE SESSION WILL BEGIN WITH A 2191 01:41:35,480 --> 01:41:36,800 PATIENT PERSPECTIVE, SO DONOVAN 2192 01:41:36,800 --> 01:41:41,400 DECKER IS A FORMER MEMBER OF 2193 01:41:41,400 --> 01:41:51,880 MDCC, LIVING WITH MUSCULAR 2194 01:41:58,440 --> 01:42:04,120 DYSTROPHY, LINDA AND MINDY AND 2195 01:42:04,120 --> 01:42:04,560 DAMIANO. 2196 01:42:04,560 --> 01:42:07,480 NOW I'LL TURN IT BACK TO YOU, 2197 01:42:07,480 --> 01:42:12,200 DR. BIANCHI, TO INTRODUCE 2198 01:42:12,200 --> 01:42:16,920 MR. DECKER. 2199 01:42:16,920 --> 01:42:23,160 >>GREAT JOB SETTING THE STAGE. 2200 01:42:23,160 --> 01:42:24,360 THE TOPIC OF IMPROVED OUTCOME 2201 01:42:24,360 --> 01:42:25,440 MEASURES WAS RECOMMENDED BY THE 2202 01:42:25,440 --> 01:42:32,000 NEXT SPEAKER, A A FORMER MEMBER 2203 01:42:32,000 --> 01:42:32,440 OF MDCC. 2204 01:42:32,440 --> 01:42:35,760 WE WELCOME YOU TO GIVE YOUR 2205 01:42:35,760 --> 01:42:37,200 PERSPECTIVES ON THIS TOPIC. 2206 01:42:37,200 --> 01:42:39,000 GOOD TO SEE YOU TODAY. 2207 01:42:39,000 --> 01:42:39,880 >>THANK YOU. 2208 01:42:39,880 --> 01:42:44,600 I'D LIKE TO THANK THE MUSCULAR 2209 01:42:44,600 --> 01:42:46,040 DYSTROPHY COORDINATING 2210 01:42:46,040 --> 01:42:47,000 COMMITTEE. 2211 01:42:47,000 --> 01:42:49,320 SINCE 1999 WHEN I STARTED GENE 2212 01:42:49,320 --> 01:42:55,440 THERAPY IN MUSCULAR DYSTROPHY, 2213 01:42:55,440 --> 01:42:56,720 THE OUTCOME MEASUREMENTS HAVE 2214 01:42:56,720 --> 01:42:57,960 NOT CHANGED. 2215 01:42:57,960 --> 01:42:59,360 ALL MUSCULAR DYSTROPHY GENE 2216 01:42:59,360 --> 01:43:04,400 THERAPY OUTCOMES ARE BASED ON 2217 01:43:04,400 --> 01:43:07,920 NORTH STAR ASSESSMENT; 2218 01:43:07,920 --> 01:43:11,080 SIX-MINUTE WALK, GETTING UP FROM 2219 01:43:11,080 --> 01:43:18,680 FLOOR, CLIMBING STAIRS, USED IN 2220 01:43:18,680 --> 01:43:23,040 MY JUNE CLINICAL TRIAL, SCORING 2221 01:43:23,040 --> 01:43:23,680 A 52. 2222 01:43:23,680 --> 01:43:25,520 OUTCOME MEASUREMENTS MIGHT BE 2223 01:43:25,520 --> 01:43:26,960 GOOD FOR SOME TYPES OF TRIALS 2224 01:43:26,960 --> 01:43:29,360 BUT NOT ALL TYPES OF TRIALS. 2225 01:43:29,360 --> 01:43:31,960 IF THERE WERE DIFFERENT OUTCOME 2226 01:43:31,960 --> 01:43:32,640 MEASUREMENTS APPROVED, THIS 2227 01:43:32,640 --> 01:43:34,720 WOULD INCREASE THE NUMBER OF 2228 01:43:34,720 --> 01:43:37,440 PATIENTS AVAILABLE TO BE USED IN 2229 01:43:37,440 --> 01:43:39,400 RESEARCH. 2230 01:43:39,400 --> 01:43:43,040 MANY PATIENT GROUPS ARE LIMITED 2231 01:43:43,040 --> 01:43:47,280 FROM TRIALS, SINCE MANY PATIENTS 2232 01:43:47,280 --> 01:43:48,880 ARE IN WHEELCHAIR RARELY MEETING 2233 01:43:48,880 --> 01:43:52,760 THE CRITERIA FOR NORTH STAR 2234 01:43:52,760 --> 01:43:53,680 ASSESSMENT. 2235 01:43:53,680 --> 01:43:55,840 I SEARCHED SEVERAL TRIALS ON 2236 01:43:55,840 --> 01:43:57,120 clinicaltrials.gov AND NONE 2237 01:43:57,120 --> 01:43:59,120 HAVE RESPIRATORY IMPROVEMENTS AS 2238 01:43:59,120 --> 01:44:01,200 AN OUTCOME MEASUREMENT. 2239 01:44:01,200 --> 01:44:02,840 SOME HAVE RESPIRATORY IMPAIRMENT 2240 01:44:02,840 --> 01:44:07,880 AS AN EXCLUSION FOR CRITERIA. 2241 01:44:07,880 --> 01:44:13,880 A COMPANY IN FRANCE HAS STARTED 2242 01:44:13,880 --> 01:44:16,840 TO DOSE PATIENTS IN A TRIAL, ONE 2243 01:44:16,840 --> 01:44:24,400 MAIN INCLUSIONS IS TO HAVE 2244 01:44:24,400 --> 01:44:25,360 MODERATE RESPIRATORY IMPAIRMENT, 2245 01:44:25,360 --> 01:44:26,840 BELOW 80%. 2246 01:44:26,840 --> 01:44:30,320 OTHER THAN SAFETY TRIALS, ALL 2247 01:44:30,320 --> 01:44:33,520 THE TILES USED THE PATIENT'S 2248 01:44:33,520 --> 01:44:36,480 BELOW THE AGE OF 18, WHEN I WAS 2249 01:44:36,480 --> 01:44:38,960 18 MY RESPIRATORY SYSTEM WAS NOT 2250 01:44:38,960 --> 01:44:39,320 AFFECTED. 2251 01:44:39,320 --> 01:44:41,360 I DID NOT START TO HAVE ISSUES 2252 01:44:41,360 --> 01:44:45,000 UNTIL I WAS 50 YEARS OLD. 2253 01:44:45,000 --> 01:44:49,640 SO TESTING CHILDREN BELOW AGE OF 2254 01:44:49,640 --> 01:44:56,280 18 MOST LIKELY WON'T SHOW 2255 01:44:56,280 --> 01:44:57,160 IMPAIRMENT. 2256 01:44:57,160 --> 01:44:59,480 HOWEVER, FOR GENE THERAPY 2257 01:44:59,480 --> 01:45:04,920 INCLUDING PATIENTS WHOSE LUNG 2258 01:45:04,920 --> 01:45:05,400 CAPACITY IS AFFECTED, 2259 01:45:05,400 --> 01:45:06,560 IMPROVEMENT COULD BE SHOWN. 2260 01:45:06,560 --> 01:45:09,160 I'VE BEEN TOLD THERE ARE 200 2261 01:45:09,160 --> 01:45:11,800 MUSCLES THAT ARE INVOLVED IN 2262 01:45:11,800 --> 01:45:12,480 WALKING. 2263 01:45:12,480 --> 01:45:15,560 AND 100 ARE CRITICAL FOR 2264 01:45:15,560 --> 01:45:16,320 WALKING. 2265 01:45:16,320 --> 01:45:20,160 IN CONTRAST, BREATHING INVOLVES 2266 01:45:20,160 --> 01:45:24,080 18 MUSCLES, ONE MAJOR IS 2267 01:45:24,080 --> 01:45:24,640 DIAPHRAGM. 2268 01:45:24,640 --> 01:45:28,280 SEEMS PUTTING EMPHASIS ON 2269 01:45:28,280 --> 01:45:29,120 IMPROVING FUNCTION IMPROVES 2270 01:45:29,120 --> 01:45:38,120 FEWER MUSCLES COULD BE 2271 01:45:38,120 --> 01:45:39,440 WORTHWHILE. 2272 01:45:39,440 --> 01:45:40,000 FOR LIMBGERDLE, RESPIRATORY 2273 01:45:40,000 --> 01:45:44,920 FAILURE IS CAUSE OF DEATH, MAYBE 2274 01:45:44,920 --> 01:45:48,320 RESEARCH SHOULD BE FOCUSED 2275 01:45:48,320 --> 01:45:49,520 TOWARD STRENGTHENING RESPIRATORY 2276 01:45:49,520 --> 01:45:51,480 MUSCLES IN ADULTS. 2277 01:45:51,480 --> 01:45:53,160 BY THE WAY I'VE READ ABOUT 2278 01:45:53,160 --> 01:45:57,520 TECHNOLOGY AVAILABLE TODAY THAT 2279 01:45:57,520 --> 01:46:00,440 ASSESSES DIAPHRAGM STRENGTH AS 2280 01:46:00,440 --> 01:46:05,400 PRESENTED IN 2016 ARTICLE, 2281 01:46:05,400 --> 01:46:12,720 VALIDATION OF ULTRASOUND FOR 2282 01:46:12,720 --> 01:46:16,400 NON-INVASIVE ASSESSMENT BY DR. 2283 01:46:16,400 --> 01:46:17,520 WHITEHEAD AND FRANER AND OTHERS 2284 01:46:17,520 --> 01:46:22,760 IN JOURNAL OF PHYSICAL -- 2285 01:46:22,760 --> 01:46:24,480 PHYSIOLOGY. 2286 01:46:24,480 --> 01:46:26,120 IF IT CAN RESULT IN STRONGER 2287 01:46:26,120 --> 01:46:27,560 MUSCLES AND DIAPHRAGM CAN BE 2288 01:46:27,560 --> 01:46:28,640 STRENGTHED WOULD HAVE A MAJOR 2289 01:46:28,640 --> 01:46:30,040 IMPACT ON QUALITY OF LIFE. 2290 01:46:30,040 --> 01:46:32,360 NOT ONLY WOULD I BE ABLE TO 2291 01:46:32,360 --> 01:46:36,960 BREATHE EASIER, BUT IT WOULD 2292 01:46:36,960 --> 01:46:39,880 ALLOW ME TO COMMUNICATE WITH A 2293 01:46:39,880 --> 01:46:42,000 STRONGER VOICE AND TALK LONGER 2294 01:46:42,000 --> 01:46:45,720 WITHOUT GETTING SO TIRED. 2295 01:46:45,720 --> 01:46:49,120 MY WEAKENED LUNG AND DIAPHRAGM 2296 01:46:49,120 --> 01:46:51,560 MUSCLES REDUCE LUNG CAPACITY, I 2297 01:46:51,560 --> 01:46:53,680 CANNOT LAY FLAT ON A BED ANYMORE 2298 01:46:53,680 --> 01:46:56,800 OR FLY ON AN AIRPLANE WITHOUT MY 2299 01:46:56,800 --> 01:46:57,080 VENTILATOR. 2300 01:46:57,080 --> 01:46:58,960 IF I DRINK TOO MUCH OR EAT A 2301 01:46:58,960 --> 01:47:02,120 LITTLE TOO MUCH, IT PUTS 2302 01:47:02,120 --> 01:47:05,840 PRESSURE ON MY DIAPHRAGM, HARDER 2303 01:47:05,840 --> 01:47:06,680 TO BREATHE. 2304 01:47:06,680 --> 01:47:12,560 THEN I NEED TO USE VENTILATOR TO 2305 01:47:12,560 --> 01:47:15,360 GIVE MUSCLES A REST. 2306 01:47:15,360 --> 01:47:18,240 WITH STRONGER BREATHING MUSCLES 2307 01:47:18,240 --> 01:47:21,120 COULD DECREASE OR DISCONTINUE 2308 01:47:21,120 --> 01:47:26,400 USING VENTILATOR THROUGH THE 2309 01:47:26,400 --> 01:47:28,280 DAY OR NIGHT, REDUCING INSURANCE 2310 01:47:28,280 --> 01:47:31,680 BURDEN OF COVERING COST OF 2311 01:47:31,680 --> 01:47:33,080 VENTILATOR AND ASSOCIATED MASK 2312 01:47:33,080 --> 01:47:36,160 AND FILTERS. 2313 01:47:36,160 --> 01:47:37,440 THE SPEAKERS FOLLOWING ME WILL 2314 01:47:37,440 --> 01:47:39,520 TALK ABOUT TECHNOLOGY THEY 2315 01:47:39,520 --> 01:47:42,080 DEVELOPED FOR ASSISTING OUTCOME 2316 01:47:42,080 --> 01:47:43,040 MEASUREMENTS. 2317 01:47:43,040 --> 01:47:44,480 BECAUSE OF CHANGING TECHNOLOGY, 2318 01:47:44,480 --> 01:47:49,840 I BELIEVE THE FDA SHOULD BE 2319 01:47:49,840 --> 01:47:51,320 WORKING TO HELP LIMBGERDLE 2320 01:47:51,320 --> 01:47:53,320 PATIENTS BEYOND JUST WALKING AN 2321 01:47:53,320 --> 01:47:57,000 BEING ABLE TO WALK IS EXTREMELY 2322 01:47:57,000 --> 01:48:01,160 IMPORTANT AND SHOULD BE 2323 01:48:01,160 --> 01:48:01,800 MEASURED, BUT HELPING SOMEBODY 2324 01:48:01,800 --> 01:48:03,240 BREATHE BETTER SHOULD BE A 2325 01:48:03,240 --> 01:48:03,600 TARGET. 2326 01:48:03,600 --> 01:48:05,120 TO ME BREATHING IS ACTUALLY MORE 2327 01:48:05,120 --> 01:48:08,520 IMPORTANT THAN WALKING FOR OLDER 2328 01:48:08,520 --> 01:48:08,800 PATIENTS. 2329 01:48:08,800 --> 01:48:10,720 RESEARCH IN THAT DIRECTION MAY 2330 01:48:10,720 --> 01:48:13,080 BE EASIER TO DO, IN MY OPINION, 2331 01:48:13,080 --> 01:48:18,600 AS YOU'RE NOT TRYING TO 2332 01:48:18,600 --> 01:48:19,720 STRENGTHEN SO MANY MUSCLES. 2333 01:48:19,720 --> 01:48:20,840 THANK YOU. 2334 01:48:20,840 --> 01:48:23,160 I'VE LOST TWO SISTERS TO THE 2335 01:48:23,160 --> 01:48:25,720 DISEASE FROM RESPIRATORY 2336 01:48:25,720 --> 01:48:25,920 FAILURE. 2337 01:48:25,920 --> 01:48:29,160 THANK YOU. 2338 01:48:29,160 --> 01:48:31,360 >>THANK YOU VERY MUCH, DONAVAN, 2339 01:48:31,360 --> 01:48:33,560 FOR YOUR LIVED AND FAMILY 2340 01:48:33,560 --> 01:48:33,920 EXPERIENCE. 2341 01:48:33,920 --> 01:48:41,560 ARE THERE QUESTIONS FOR 2342 01:48:41,560 --> 01:48:42,240 MR. DECKER? 2343 01:48:42,240 --> 01:48:48,120 >>GREAT TALK, DONAVAN. 2344 01:48:48,120 --> 01:48:50,800 >>THANK YOU, DAN. 2345 01:48:50,800 --> 01:48:57,480 2346 01:48:57,480 --> 01:49:01,880 >>IN THE CHAT BOX, EXCELLENT 2347 01:49:01,880 --> 01:49:02,360 POINTS. 2348 01:49:02,360 --> 01:49:07,600 OKAY. 2349 01:49:07,600 --> 01:49:09,400 THANK YOU VERY MUCH. 2350 01:49:09,400 --> 01:49:10,520 NEXT THREE MEASURES FOR 2351 01:49:10,520 --> 01:49:11,960 MEASURING PHYSICAL FUNCTION. 2352 01:49:11,960 --> 01:49:14,920 WE'LL TAKE QUESTIONS AFTER EACH 2353 01:49:14,920 --> 01:49:16,440 INDIVIDUAL PRESENTATION. 2354 01:49:16,440 --> 01:49:20,040 FIRST PRESENTER IS DR. LINDA 2355 01:49:20,040 --> 01:49:27,600 LOWE'S, PRINCIPAL INVESTIGATOR 2356 01:49:27,600 --> 01:49:30,960 AT THE WEXNER INSTITUTE, USING 2357 01:49:30,960 --> 01:49:33,960 COMPUTER VISION TO QUANTIFY AND 2358 01:49:33,960 --> 01:49:36,520 EVALUATE MOVEMENT. 2359 01:49:36,520 --> 01:49:37,520 WELCOME, DR. LOWES. 2360 01:49:37,520 --> 01:49:38,680 >>THANK YOU VERY MUCH. 2361 01:49:38,680 --> 01:49:41,200 THANK YOU FOR THE OPPORTUNITY TO 2362 01:49:41,200 --> 01:49:43,000 SHARE OUR WORK THAT'S GOING ON 2363 01:49:43,000 --> 01:49:43,840 IN MY LAB. 2364 01:49:43,840 --> 01:49:47,480 I THINK WE ALL AGREE THAT 2365 01:49:47,480 --> 01:49:49,040 FINDING OPTIMAL OUTCOME MEASURE 2366 01:49:49,040 --> 01:49:51,200 TO HAVE DEFINITIVE TRIAL ANSWERS 2367 01:49:51,200 --> 01:49:52,240 IS REALLY IMPORTANT. 2368 01:49:52,240 --> 01:49:54,800 SO HERE ARE TWO THINGS THAT 2369 01:49:54,800 --> 01:49:56,760 WE'RE WORKING ON. 2370 01:49:56,760 --> 01:49:59,720 THE FIRST ONE IS A SYSTEM WE 2371 01:49:59,720 --> 01:50:03,240 MADE THAT LOOKS AT MOVEMENT 2372 01:50:03,240 --> 01:50:05,760 COMPLEXITY, IT LOOKS AT THE 2373 01:50:05,760 --> 01:50:07,160 HEALTHINESS OF THE MOVEMENT, 2374 01:50:07,160 --> 01:50:09,360 HOPEFULLY THAT WE CAN IDENTIFY 2375 01:50:09,360 --> 01:50:10,960 INFANTS EARLY WHO HAVE ATYPICAL 2376 01:50:10,960 --> 01:50:14,080 MOVEMENT BUT ALSO THAT WE CAN 2377 01:50:14,080 --> 01:50:15,840 QUANTIFY IT AS IT CHANGES AND 2378 01:50:15,840 --> 01:50:20,640 BECOMES HEALTHIER. 2379 01:50:20,640 --> 01:50:25,440 THE GOAL WOULD BE TO USE IN 2380 01:50:25,440 --> 01:50:28,120 NEONATAL STUDIES, AND HOPEFULLY 2381 01:50:28,120 --> 01:50:29,960 WILL REDUCE THE TIME, THE 2382 01:50:29,960 --> 01:50:32,480 DURATION OF STUDIES BECAUSE WE 2383 01:50:32,480 --> 01:50:39,600 WON'T HAVE TO WAIT UNTIL THEY 2384 01:50:39,600 --> 01:50:44,680 DEVELOP MOTOR. 2385 01:50:44,680 --> 01:50:48,160 WE HAD TWO INFANTS, 26 DAYS OLD, 2386 01:50:48,160 --> 01:50:50,040 HEALTHY CONTROL INFANT 21 DAYS 2387 01:50:50,040 --> 01:50:50,240 OLD. 2388 01:50:50,240 --> 01:50:53,360 THEY HAD A SIMILAR SCORE ON THE 2389 01:50:53,360 --> 01:50:55,080 TEST THAT'S TYPICALLY USED IN 2390 01:50:55,080 --> 01:50:58,480 THESE KINDS OF TRIALS THAT 2391 01:50:58,480 --> 01:51:01,760 CHILDREN'S HOSPITAL PHILADELPHIA 2392 01:51:01,760 --> 01:51:05,120 INTEND PERFORMANCE BASED TEST, 2393 01:51:05,120 --> 01:51:07,320 THERAPIST ENCOURAGES THE INFANT 2394 01:51:07,320 --> 01:51:08,640 OR FACILITATES MOVEMENT AND THEY 2395 01:51:08,640 --> 01:51:17,600 SCORE BASED ON WHAT THEY SEE. 2396 01:51:17,600 --> 01:51:18,800 LOOKING AT COMPLEXITY, A 2397 01:51:18,800 --> 01:51:21,880 DIFFERENCE ABLE TO BE DISCERNED. 2398 01:51:21,880 --> 01:51:24,280 YOU'RE LOOKING AT HANDS AND 2399 01:51:24,280 --> 01:51:34,840 FEET, TWO DIMENSIONAL TRACING OF 2400 01:51:36,000 --> 01:51:36,200 MOVEMENTS. 2401 01:51:36,200 --> 01:51:38,240 MOVEMENT OF THE HEALTHY CHILD IS 2402 01:51:38,240 --> 01:51:39,480 MORE COMPLEX. 2403 01:51:39,480 --> 01:51:43,000 THOSE ARE THE TWO DIMENSIONAL. 2404 01:51:43,000 --> 01:51:49,160 WE LOOK AT THE OTHER SIDE, 2405 01:51:49,160 --> 01:51:49,960 ANTI-GRAVITY MOVEMENTS, INFANTS 2406 01:51:49,960 --> 01:51:51,920 WITH SMA WERE NOT MOVING AS HIGH 2407 01:51:51,920 --> 01:51:56,480 OR WERE NOT AS FREQUENTLY OFF 2408 01:51:56,480 --> 01:51:58,680 THE GROUND, NOT PICKED UP 2409 01:51:58,680 --> 01:52:00,800 BECAUSE BOTH SCORES ARE 2410 01:52:00,800 --> 01:52:03,280 RELATIVELY HIGH SCORE. 2411 01:52:03,280 --> 01:52:11,480 WE TRIED TO SEE IF WE COULD 2412 01:52:11,480 --> 01:52:13,680 ESTIMATE THERE SCORE AND TRIED 2413 01:52:13,680 --> 01:52:20,720 TO PREDICT IT USING OUR MOTOR 2414 01:52:20,720 --> 01:52:21,920 COMPLEXITY SYSTEM. 2415 01:52:21,920 --> 01:52:24,080 THIS FIGURE SHOWS YOU THAT WE 2416 01:52:24,080 --> 01:52:26,960 ENDED UP MAKING A MOVEMENT -- A 2417 01:52:26,960 --> 01:52:30,320 MOTOR FUNCTION SCORE, AND SO IF 2418 01:52:30,320 --> 01:52:40,760 YOU LOOK ON THE Y-AXIS, THAT'S 2419 01:52:40,760 --> 01:52:41,600 PROPENSITY CLASSIFIED AS 2420 01:52:41,600 --> 01:52:42,160 HEALTHY. 2421 01:52:42,160 --> 01:52:46,160 WE USED ARTIFICIAL INTELLIGENCE 2422 01:52:46,160 --> 01:52:47,360 TO QUANTIFY MOVEMENT COMPLEXITY. 2423 01:52:47,360 --> 01:52:50,680 TWO SETS ON EITHER END WITH 2424 01:52:50,680 --> 01:52:51,520 SPINAL MUSCULAR ATROPHY, AND 2425 01:52:51,520 --> 01:52:53,080 THEY HAD MOTOR FUNCTION SCORES 2426 01:52:53,080 --> 01:52:53,400 BELOW 10. 2427 01:52:53,400 --> 01:52:56,360 WHEN YOU LOOK AT THE HEALTHY 2428 01:52:56,360 --> 01:52:58,320 INDIVIDUALS WITH BLUE SQUARES, 2429 01:52:58,320 --> 01:53:02,160 THEY ALL HAD MOTOR FUNCTION 2430 01:53:02,160 --> 01:53:03,280 SCORES, COMPLEXITY WAS WELL 2431 01:53:03,280 --> 01:53:04,040 ABOVE 80. 2432 01:53:04,040 --> 01:53:06,200 WHAT WE THOUGHT WAS MOST UNIQUE 2433 01:53:06,200 --> 01:53:08,520 IS ON THE LAST GROUP, YOU CAN 2434 01:53:08,520 --> 01:53:15,000 SEE THAT THE INFANT STARTED OUT 2435 01:53:15,000 --> 01:53:17,960 LOW BELOW 10 BUT POST-GENE 2436 01:53:17,960 --> 01:53:21,240 THERAPY MOTOR FUNCTION SCORE 2437 01:53:21,240 --> 01:53:22,440 IMPROVED, SHOWING THAT MOVEMENT 2438 01:53:22,440 --> 01:53:27,040 BECAME MORE LIKE THEIR HEALTHY 2439 01:53:27,040 --> 01:53:27,400 PEERS. 2440 01:53:27,400 --> 01:53:31,160 WHEN THE GENE WAS APPROVED, WE 2441 01:53:31,160 --> 01:53:35,800 STARTED SEEING BABIES THAT WERE 2442 01:53:35,800 --> 01:53:37,680 IDENTIFIED AS THROUGH NEWBORN 2443 01:53:37,680 --> 01:53:38,560 SCREENING, WE SAW SOMETHING 2444 01:53:38,560 --> 01:53:40,760 REALLY INTERESTING THAT WE COULD 2445 01:53:40,760 --> 01:53:43,480 DETECT DIFFERENCES BETWEEN THE 2446 01:53:43,480 --> 01:53:46,440 HEALTHY BABY AND BABY WITH SMA 2447 01:53:46,440 --> 01:53:48,720 TYPE 1, AT ONE WEEK OF AGE. 2448 01:53:48,720 --> 01:53:51,320 SO THERE REALLY IS NO SUCH THING 2449 01:53:51,320 --> 01:53:52,560 AS SYMPTOMATIC PERIOD. 2450 01:53:52,560 --> 01:53:56,240 THE REASON THE BABY SEEMS 2451 01:53:56,240 --> 01:53:59,160 ASYMPTOMATIC OR PRESYMPTOMATIC 2452 01:53:59,160 --> 01:54:02,880 IS BECAUSE THE DEMANDS OF THE 2453 01:54:02,880 --> 01:54:04,320 TEST AREN'T MUCH. 2454 01:54:04,320 --> 01:54:05,760 WHEN YOU'RE A BABY ALL YOU HAVE 2455 01:54:05,760 --> 01:54:08,360 TO DO IS MOVE AROUND, THERE'S NO 2456 01:54:08,360 --> 01:54:09,480 FUNCTIONAL DEMAND. 2457 01:54:09,480 --> 01:54:12,200 SO YOU DON'T NOTICE DEFICITS 2458 01:54:12,200 --> 01:54:14,520 UNTIL THEY ARE ASKED TO DO 2459 01:54:14,520 --> 01:54:15,640 HARDER THINGS LIKE REACHING FOR 2460 01:54:15,640 --> 01:54:18,120 SOMETHING OR SITTING UP. 2461 01:54:18,120 --> 01:54:20,200 THE GRAPH AT THE BOTTOM SHOWS 2462 01:54:20,200 --> 01:54:23,920 THAT WE WERE ABLE TO DETECT 2463 01:54:23,920 --> 01:54:26,320 INFANTS WITH SMA EVEN THOSE THAT 2464 01:54:26,320 --> 01:54:29,320 HAVE THREE AND FOUR COPIES, AN 2465 01:54:29,320 --> 01:54:30,720 INFANT WITH FOUR COPIES MIGHT 2466 01:54:30,720 --> 01:54:33,680 NOT BE EXPECTED TO BE DIAGNOSED 2467 01:54:33,680 --> 01:54:35,960 WITHOUT NEWBORN SCREENING UNTIL 2468 01:54:35,960 --> 01:54:39,560 THEY WERE MUCH LETTER, ABLE TO 2469 01:54:39,560 --> 01:54:40,880 WALK, ELEMENTARY SCHOOL OR 2470 01:54:40,880 --> 01:54:41,520 LATER. 2471 01:54:41,520 --> 01:54:43,200 EVEN IN THIS VERY EARLY THEY DO 2472 01:54:43,200 --> 01:54:46,880 NOT HAVE THE SAME MOVEMENT 2473 01:54:46,880 --> 01:54:48,000 COMPLEXITY AS HEALTHY PEERS. 2474 01:54:48,000 --> 01:54:51,640 WE WENT ON TO SEE IF WE COULD 2475 01:54:51,640 --> 01:54:53,840 NOTICE ANY DIFFERENT KINDS OF 2476 01:54:53,840 --> 01:54:55,440 MOVEMENT DISORDERS, SO THESE ARE 2477 01:54:55,440 --> 01:54:57,440 TWO BABIES WE RECORDED AND THEY 2478 01:54:57,440 --> 01:54:59,480 LOOK VERY GOOD BUT WHEN YOU LOOK 2479 01:54:59,480 --> 01:55:01,040 AT THE MOVEMENT COMPLEXITY THE 2480 01:55:01,040 --> 01:55:04,520 ONE ON THE RIGHT HAS BEEN 2481 01:55:04,520 --> 01:55:05,720 DIAGNOSED WITH CEREBRAL PALSY, 2482 01:55:05,720 --> 01:55:08,600 LEFT IS A TYPICAL MOVEMENTS. 2483 01:55:08,600 --> 01:55:11,800 YOU CAN SEE BY THE TRACINGS ONCE 2484 01:55:11,800 --> 01:55:15,400 AGAIN MOVEMENT COMPLEXITY IS 2485 01:55:15,400 --> 01:55:15,640 LACKING. 2486 01:55:15,640 --> 01:55:18,600 HERE AGAIN ARE TWO BABIES THAT 2487 01:55:18,600 --> 01:55:21,880 WE HAVE RECORDED, ABOUT THE SAME 2488 01:55:21,880 --> 01:55:25,960 AGE, WE NOTICE ONE INFANT HAS 2489 01:55:25,960 --> 01:55:30,640 DUCHENNE MUSCULAR DYSTROPHY, THE 2490 01:55:30,640 --> 01:55:32,200 OTHER IS TYPICALLY DEVELOPING 2491 01:55:32,200 --> 01:55:33,080 INFANT. 2492 01:55:33,080 --> 01:55:35,480 THE DIFFERENCE BETWEEN THE 2493 01:55:35,480 --> 01:55:37,760 TRACINGS IS QUITE OBVIOUS THAT 2494 01:55:37,760 --> 01:55:40,080 INFANT WITH DUCHENNE MUSCULAR 2495 01:55:40,080 --> 01:55:42,680 DYSTROPHY IS NOT MOVING QUITE AS 2496 01:55:42,680 --> 01:55:44,760 COMPLEXITY BUT HE LOOKS LIKE A 2497 01:55:44,760 --> 01:55:49,360 HAPPY BABY WIGGLING ARMS AND 2498 01:55:49,360 --> 01:55:49,840 LEGS. 2499 01:55:49,840 --> 01:55:52,080 JUST A COUPLE MORE EXAMPLES OF 2500 01:55:52,080 --> 01:55:55,800 INFANTS WITH DMD WE'VE NOTICED. 2501 01:55:55,800 --> 01:55:58,000 SO, WE'RE IN THE MIDDLE OF A 2502 01:55:58,000 --> 01:56:01,920 RESEARCH STUDY, NOW WE HAVE 1400 2503 01:56:01,920 --> 01:56:02,680 RECORDINGS, AND WE'RE CONTINUING 2504 01:56:02,680 --> 01:56:05,000 TO LOOK AT DIFFERENT WAYS TO USE 2505 01:56:05,000 --> 01:56:07,280 COMPUTER VISION, RIGHT NOW WE'RE 2506 01:56:07,280 --> 01:56:08,440 LOOKING AT SKELETAL TRACKING, 2507 01:56:08,440 --> 01:56:10,680 CURRENTLY WE LOOK AT HANDS AND 2508 01:56:10,680 --> 01:56:15,320 FEET, WE'RE ABLE TO DO QUITE A 2509 01:56:15,320 --> 01:56:16,720 FEW MORE DATA POINTS. 2510 01:56:16,720 --> 01:56:18,440 WE'RE LOOKING AT DIFFERENT WAYS 2511 01:56:18,440 --> 01:56:21,400 TO QUANTIFY THE ARTIFICIAL 2512 01:56:21,400 --> 01:56:22,400 INTELLIGENCE. 2513 01:56:22,400 --> 01:56:24,400 ONE THING THAT WILL HELP IS 2514 01:56:24,400 --> 01:56:25,480 MOVEMENT TO NEWBORN SCREENING 2515 01:56:25,480 --> 01:56:27,600 AND ALL OF THESE HAVE BEEN UNDER 2516 01:56:27,600 --> 01:56:29,840 THE AGE OF 6 MONTHS BUT WE'RE 2517 01:56:29,840 --> 01:56:31,040 WORKING WITH A NEWBORN HOSPITAL 2518 01:56:31,040 --> 01:56:32,360 TRYING TO GET THEM WITHIN THE 2519 01:56:32,360 --> 01:56:34,000 FIRST COUPLE DAYS OF LIFE TO SEE 2520 01:56:34,000 --> 01:56:35,440 HOW EARLY WE COULD POSSIBLY DO 2521 01:56:35,440 --> 01:56:35,960 IT. 2522 01:56:35,960 --> 01:56:37,840 THEN OF COURSE WE NEED TO LOOK 2523 01:56:37,840 --> 01:56:42,400 AT LONGITUDINAL CHANGE OF THE 2524 01:56:42,400 --> 01:56:43,720 INFANTS WITH DISABILITIES, 2525 01:56:43,720 --> 01:56:45,600 ESPECIALLY PROGRESSIVE ONES LIKE 2526 01:56:45,600 --> 01:56:46,920 DUCHENNE OR SPINAL MUSCULAR 2527 01:56:46,920 --> 01:56:47,120 ATROPHY. 2528 01:56:47,120 --> 01:56:49,440 THERE ARE A NUMBER OF DATA 2529 01:56:49,440 --> 01:56:51,280 POINTS WE COULD TRACK INCLUDING 2530 01:56:51,280 --> 01:56:53,000 EYE GAZE, HEAD TURNING, SO WE 2531 01:56:53,000 --> 01:56:54,760 CAN EXPAND THIS TO BE MORE 2532 01:56:54,760 --> 01:56:58,280 COMPLEX IF WE NEED TO. 2533 01:56:58,280 --> 01:57:00,360 THE OTHER THING WE'RE USING 2534 01:57:00,360 --> 01:57:02,640 COMPUTER VISION FOR TO MEASURE 2535 01:57:02,640 --> 01:57:04,320 WORKSPACE VOLUME, THE AREA THAT 2536 01:57:04,320 --> 01:57:06,640 YOU CAN INTERACT WITH, WITH YOUR 2537 01:57:06,640 --> 01:57:07,240 ARMS AROUND YOURSELF. 2538 01:57:07,240 --> 01:57:08,440 AND THIS IS THIS HAS BEEN GOING 2539 01:57:08,440 --> 01:57:10,800 ON FOR A WHILE. 2540 01:57:10,800 --> 01:57:14,200 THESE ARE ALL OF OUR GAMES, 2541 01:57:14,200 --> 01:57:15,080 GAME-BASED SYSTEM. 2542 01:57:15,080 --> 01:57:17,720 AS IT PROGRESSES WE'RE DOWN TO 2543 01:57:17,720 --> 01:57:19,000 THE AVATAR WITH THE ROCKET BUT 2544 01:57:19,000 --> 01:57:21,520 THE BOYS WANT US TO END UP CAN 2545 01:57:21,520 --> 01:57:23,480 CALL OF DUTY BUT YOU'RE NOT 2546 01:57:23,480 --> 01:57:25,120 ALLOWED TO KILL THINGS IN 2547 01:57:25,120 --> 01:57:26,360 CHILDREN'S HOSPITALS SO WE'RE 2548 01:57:26,360 --> 01:57:29,520 STUCK WITH THE AVATAR. 2549 01:57:29,520 --> 01:57:35,680 EVERY TASK YOU DO HAS A DISCRETE 2550 01:57:35,680 --> 01:57:38,600 AMOUNT OF SPACE YOU NEED. 2551 01:57:38,600 --> 01:57:41,240 TO PUT A SHIRT ON, LIFT A CUP TO 2552 01:57:41,240 --> 01:57:51,680 DRINK, TO USE A COMPUTER. 2553 01:57:53,160 --> 01:57:56,240 HERE IS OUR SYSTEM. 2554 01:57:56,240 --> 01:58:03,160 2555 01:58:03,160 --> 01:58:04,200 THE AVATAR TELLS THE PLAYER 2556 01:58:04,200 --> 01:58:05,000 WHERE TO REACH. 2557 01:58:05,000 --> 01:58:06,520 IT GIVES ALL OF THE 2558 01:58:06,520 --> 01:58:11,480 INSTRUCTIONS, ALL OF THE 2559 01:58:11,480 --> 01:58:11,840 ENCOURAGEMENT. 2560 01:58:11,840 --> 01:58:14,840 YOU DON'T NEED TO BE A SKILLED 2561 01:58:14,840 --> 01:58:15,880 THERAPIST TO ADMINISTER THIS, 2562 01:58:15,880 --> 01:58:18,600 YOU TURN THE GAME ON AND THEN 2563 01:58:18,600 --> 01:58:20,320 EVERYONE GETS THE SAME 2564 01:58:20,320 --> 01:58:20,960 INSTRUCTIONS. 2565 01:58:20,960 --> 01:58:24,360 AND WHAT WE GET FROM THAT IS 2566 01:58:24,360 --> 01:58:26,040 WORKSPACE VOLUME IN DIFFERENT 2567 01:58:26,040 --> 01:58:26,240 AREAS. 2568 01:58:26,240 --> 01:58:30,840 YOU CAN CHOOSE RIGHT VERSUS 2569 01:58:30,840 --> 01:58:36,760 LEFT, CHOOSE TEN DIFFERENT 2570 01:58:36,760 --> 01:58:41,160 QUADRANTS. 2571 01:58:41,160 --> 01:58:43,520 IT CAN SPAN AMBULENT AND 2572 01:58:43,520 --> 01:58:44,040 NON-AMBULENT. 2573 01:58:44,040 --> 01:58:45,200 IF THEY HAVE WEAKNESS IN THE 2574 01:58:45,200 --> 01:58:46,920 TRUNK THEY DON'T GET AS HIGH A 2575 01:58:46,920 --> 01:58:47,240 SCORE. 2576 01:58:47,240 --> 01:58:49,560 WE ALLOW THEM TO REACH AND 2577 01:58:49,560 --> 01:58:50,120 COMPENSATE. 2578 01:58:50,120 --> 01:58:54,600 IT'S ABLE TO DETECT VERY SUBTLE 2579 01:58:54,600 --> 01:58:56,120 CHANGES IN INDIVIDUALS 2580 01:58:56,120 --> 01:58:57,760 AMBULATORY AS WELL AS DOWN TO 2581 01:58:57,760 --> 01:59:00,280 INDIVIDUALS WHO CAN JUST MOVE 2582 01:59:00,280 --> 01:59:01,120 THEIR HANDS. 2583 01:59:01,120 --> 01:59:03,480 WE THINK IT WILL ELIMINATE THE 2584 01:59:03,480 --> 01:59:08,880 FLOOR AND CEILING EFFECT THAT A 2585 01:59:08,880 --> 01:59:10,240 LOT OF ASSESSMENTS HAVE. 2586 01:59:10,240 --> 01:59:17,600 WE TRIED IT IN A NUMBER OF 2587 01:59:17,600 --> 01:59:20,000 DIAGNOSES, SPINAL MUSCULAR 2588 01:59:20,000 --> 01:59:21,040 ATROPHY, LIMBGERDLE, DUCHENNE 2589 01:59:21,040 --> 01:59:21,640 AND BECKER. 2590 01:59:21,640 --> 01:59:24,040 THUS FAR IT'S WORKING WELL ON 2591 01:59:24,040 --> 01:59:25,040 ALL OF THOSE. 2592 01:59:25,040 --> 01:59:27,520 WE WANT TO MAKE SURE IT WAS 2593 01:59:27,520 --> 01:59:29,720 RELATED TO FUNCTION, WE NOTICE 2594 01:59:29,720 --> 01:59:32,760 IT IS CORRELATED TO A NUMBER OF 2595 01:59:32,760 --> 01:59:34,080 TYPICAL ASSESSMENTS. 2596 01:59:34,080 --> 01:59:35,720 MOST IMPORTANTLY, I THINK, IT'S 2597 01:59:35,720 --> 01:59:36,960 ALSO RELATED TO WALKING TESTS 2598 01:59:36,960 --> 01:59:38,480 BECAUSE IF YOU THINK ABOUT IT, I 2599 01:59:38,480 --> 01:59:41,440 A BUILT TO WALK IS HIGH -- 2600 01:59:41,440 --> 01:59:46,400 ABILITY TO WALK IS DEPENDENT ON 2601 01:59:46,400 --> 01:59:47,920 STABILITY OF TRUNK. 2602 01:59:47,920 --> 01:59:49,080 IN LIMBGERDLE OR DUCHENNE THE 2603 01:59:49,080 --> 01:59:50,120 TRUNK GOES FIRST. 2604 01:59:50,120 --> 01:59:52,200 WE'RE ABLE TO PICK UP DEFICITS 2605 01:59:52,200 --> 01:59:52,560 THERE. 2606 01:59:52,560 --> 01:59:55,680 THE OTHER THING WE THINK IS 2607 01:59:55,680 --> 01:59:57,320 UNIQUE, WE'VE HAD KIDS PLAY AS 2608 01:59:57,320 --> 02:00:00,400 YOUNG AS 3 YEARS OLD. 2609 02:00:00,400 --> 02:00:01,840 THE AVERAGE 3-YEAR-OLD MY NOT BE 2610 02:00:01,840 --> 02:00:04,680 ABLE TO BUT WE'VE HAD SUCCESS 2611 02:00:04,680 --> 02:00:14,000 WITH 4 AND UP, AS OLD AS '90s 2612 02:00:14,000 --> 02:00:14,200 PLAY. 2613 02:00:14,200 --> 02:00:16,360 WE MEASURED CHANGE, AND WE WERE 2614 02:00:16,360 --> 02:00:20,800 ABLE TO DEMONSTRATE THAT PEOPLE 2615 02:00:20,800 --> 02:00:22,680 WITHOUT TREATMENT WOULD DECLINE 2616 02:00:22,680 --> 02:00:24,000 IN THEIR WORKSPACE VOLUME BUT 2617 02:00:24,000 --> 02:00:31,320 PEOPLE WHO ARE TREATED WOULD 2618 02:00:31,320 --> 02:00:32,080 INCREASE. 2619 02:00:32,080 --> 02:00:35,680 IT DOES NOT HAVE THE SAME 2620 02:00:35,680 --> 02:00:37,120 CEILING EFFECT. 2621 02:00:37,120 --> 02:00:40,720 THE PERFORMANCE OF UPPER LIMB, 2622 02:00:40,720 --> 02:00:42,520 SOME PEOPLE ABLE TO GET A 2623 02:00:42,520 --> 02:00:43,960 PERFECT SCORE EVEN THOUGH THE 2624 02:00:43,960 --> 02:00:44,920 WORKSPACE VOLUME COULD CONTINUE 2625 02:00:44,920 --> 02:00:50,480 TO GO UP. 2626 02:00:50,480 --> 02:00:53,320 SO HERE'S YOUR PERCENTAGE OF 2627 02:00:53,320 --> 02:00:54,760 PUL, ROOM TO IMPROVE ON 2628 02:00:54,760 --> 02:00:56,000 WORKSPACE VOLUME BUT THEY HIT 2629 02:00:56,000 --> 02:00:58,240 THE CEILING EFFECT. 2630 02:00:58,240 --> 02:01:01,760 WE SAW TWO DIFFERENT PROBLEMS 2631 02:01:01,760 --> 02:01:03,640 WITH HAMMERSMITH, A NUMBER OF 2632 02:01:03,640 --> 02:01:04,160 INDIVIDUALS THAT COULD NOT 2633 02:01:04,160 --> 02:01:05,600 PERFORM BECAUSE YOU HAVE TO BE 2634 02:01:05,600 --> 02:01:15,960 ABLE TO ROLL AND SIT. 2635 02:01:19,760 --> 02:01:21,800 THERE IS A TEST TO SPANS FROM 2636 02:01:21,800 --> 02:01:32,280 MINIMAL USE WITH HANDS TO 2637 02:01:32,280 --> 02:01:32,520 WALKING. 2638 02:01:32,520 --> 02:01:36,720 WE ASKED PEOPLE TO YOUR THE 2639 02:01:36,720 --> 02:01:38,960 PROMIS SCALE, IT CORRELATED WELL 2640 02:01:38,960 --> 02:01:40,280 WITH THAT. 2641 02:01:40,280 --> 02:01:44,000 PEOPLE RATED THEMSELVES HIGHER, 2642 02:01:44,000 --> 02:01:50,000 GOT A HIGHER WORKSPACE FOLLOW, 2643 02:01:50,000 --> 02:01:51,360 WE TOOK ANSWERS FROM THE PROMIS, 2644 02:01:51,360 --> 02:01:53,920 THOSE ABLE TO DO IT AND UNABLE 2645 02:01:53,920 --> 02:01:55,880 TO DO AN ACTIVITY. 2646 02:01:55,880 --> 02:01:58,720 WE HAVE AN ESTIMATE OF WHAT IS 2647 02:01:58,720 --> 02:02:00,440 THE MINIMUM WORKSPACE VOLUME YOU 2648 02:02:00,440 --> 02:02:02,840 NEED TO PULL YOUR SHIRT OVER, TO 2649 02:02:02,840 --> 02:02:05,400 YOUR HEAD, ZIP UP CLOTHES OR PUT 2650 02:02:05,400 --> 02:02:09,640 TOOTHPASTE ON A TOOTHBRUSH. 2651 02:02:09,640 --> 02:02:11,800 THAT WE CAN TELL, THE PAYERS AND 2652 02:02:11,800 --> 02:02:15,920 REGULATORS, IF YOU DO IMPROVE BY 2653 02:02:15,920 --> 02:02:17,720 A SCORE OF 10 IT'S GOING TO MEAN 2654 02:02:17,720 --> 02:02:21,880 YOU CAN DO THESE DIFFERENT 2655 02:02:21,880 --> 02:02:22,120 ABILITIES. 2656 02:02:22,120 --> 02:02:23,760 AND THAT IS ALL I HAD. 2657 02:02:23,760 --> 02:02:29,400 I'M HAPPY TO ANSWER ANY 2658 02:02:29,400 --> 02:02:30,040 QUESTIONS. 2659 02:02:30,040 --> 02:02:40,480 >>THANK YOU VERY MUCH. 2660 02:02:43,080 --> 02:02:43,280 QUESTIONS? 2661 02:02:43,280 --> 02:02:50,120 >>DAN PEREZ, I HAVE A QUESTION. 2662 02:02:50,120 --> 02:02:57,440 >>YES. 2663 02:02:57,440 --> 02:03:00,080 >>WITH THESE TECHNOLOGIES, 2664 02:03:00,080 --> 02:03:02,600 YOU'RE ABLE TO VISUALIZE THE 2665 02:03:02,600 --> 02:03:04,600 DATA VERY WELL, AND I GUESS THE 2666 02:03:04,600 --> 02:03:11,040 QUESTION I HAVE IS, HOW DO WE 2667 02:03:11,040 --> 02:03:14,080 RELATE THAT TO A FUNCTION OF 2668 02:03:14,080 --> 02:03:15,040 CHANGE. 2669 02:03:15,040 --> 02:03:24,840 IN TERMS OF REACHING THE 2670 02:03:24,840 --> 02:03:35,360 WORKSPACE, AS I LOOK, WHAT DOES 2671 02:03:57,960 --> 02:04:00,440 THIS MEAN? 2672 02:04:00,440 --> 02:04:05,080 2673 02:04:05,080 --> 02:04:06,720 >>I'M NOT SURE YOU'RE FINISHED. 2674 02:04:06,720 --> 02:04:08,120 YOU'RE MUTED NOW. 2675 02:04:08,120 --> 02:04:10,000 I THINK THE QUESTION IS, WHAT'S 2676 02:04:10,000 --> 02:04:11,400 REALLY IMPORTANT, JUST LIKE 2677 02:04:11,400 --> 02:04:12,800 PEOPLE DON'T KNOW WHAT ONE POINT 2678 02:04:12,800 --> 02:04:15,200 ON THE NORTH STAR MEANS, WHAT 2679 02:04:15,200 --> 02:04:17,440 DOES IT MEAN TO IMPROVE IN YOUR 2680 02:04:17,440 --> 02:04:21,680 WORKSPACE VOLUME. 2681 02:04:21,680 --> 02:04:23,000 A COUPLE THINGS. 2682 02:04:23,000 --> 02:04:24,520 WE TRIED TO SET MINIMUM AMOUNTS 2683 02:04:24,520 --> 02:04:26,920 OF VOLUME YOU NEED FOR AN 2684 02:04:26,920 --> 02:04:27,680 ACTIVITY. 2685 02:04:27,680 --> 02:04:31,120 SO, IF YOU COULD GO FROM 14 TO 2686 02:04:31,120 --> 02:04:34,160 20 PERHAPS THAT WOULD MEAN THAT 2687 02:04:34,160 --> 02:04:35,640 YOU COULD BRUSH YOUR TEETH OR 2688 02:04:35,640 --> 02:04:36,800 LIFT YOUR OWN CUP. 2689 02:04:36,800 --> 02:04:39,320 THAT'S WHAT WE'RE WORKING ON 2690 02:04:39,320 --> 02:04:40,840 NOW, IT DOES CORRELATE WELL TO 2691 02:04:40,840 --> 02:04:43,160 PATIENT REPORTS OF CHANGE BUT WE 2692 02:04:43,160 --> 02:04:45,440 NEED TO GET A DISTINCT AMOUNT 2693 02:04:45,440 --> 02:04:46,640 THAT IS MEANINGFUL. 2694 02:04:46,640 --> 02:04:49,280 IT REALLY DEPENDS ON THE 2695 02:04:49,280 --> 02:04:49,560 ACTIVITY. 2696 02:04:49,560 --> 02:04:51,920 IF YOU NEED CERTAIN AMOUNT OF 2697 02:04:51,920 --> 02:04:53,640 WORKSPACE TO USE YOUR COMPUTER, 2698 02:04:53,640 --> 02:04:55,520 YOU NEED A DIFFERENT AMOUNT TO 2699 02:04:55,520 --> 02:04:58,360 LIFT THE CUP TO DRINK, SO THAT 2700 02:04:58,360 --> 02:04:59,320 IS OBVIOUSLY VERY IMPORTANT, 2701 02:04:59,320 --> 02:05:01,000 THAT'S WHAT WE'RE WORKING ON. 2702 02:05:01,000 --> 02:05:02,720 I THINK ONE THING THAT'S 2703 02:05:02,720 --> 02:05:04,360 DIFFERENT ABOUT OUR SYSTEM IS 2704 02:05:04,360 --> 02:05:06,720 THAT WE SCALE FOR YOUR HEIGHT, 2705 02:05:06,720 --> 02:05:10,080 SO IT DOESN'T MATTER THE CUBIC 2706 02:05:10,080 --> 02:05:10,880 CENTIMETERS, THEY DON'T MATTER, 2707 02:05:10,880 --> 02:05:12,480 IT'S THE AMOUNT YOU CAN REACH. 2708 02:05:12,480 --> 02:05:14,120 IT'S SCALED SO IF YOU CAN JUST 2709 02:05:14,120 --> 02:05:15,880 LIFT YOUR ARMS UP OVER YOUR HEAD 2710 02:05:15,880 --> 02:05:16,720 YOU GET A 100. 2711 02:05:16,720 --> 02:05:18,800 IF YOU CAN LEAN AND REACH, 2712 02:05:18,800 --> 02:05:21,000 YOU'LL GET A HIGHER SCORE THAN 2713 02:05:21,000 --> 02:05:21,280 100. 2714 02:05:21,280 --> 02:05:23,240 SO WE'RE TRYING, AGAIN, TO MAKE 2715 02:05:23,240 --> 02:05:24,760 IT SO WE CAN COMPARE DIFFERENT 2716 02:05:24,760 --> 02:05:31,880 PEOPLE AND THAT IT'S NOT JUST A 2717 02:05:31,880 --> 02:05:33,400 THREE CUBIC CENTIMETERS, IT'S AN 2718 02:05:33,400 --> 02:05:34,960 AMOUNT TOWARDS BEING ABLE TO DO 2719 02:05:34,960 --> 02:05:36,720 AN ACTIVITY. 2720 02:05:36,720 --> 02:05:44,000 DOES THAT ANSWER YOUR QUESTION? 2721 02:05:44,000 --> 02:05:52,880 >>YOU'RE SAYING YOU CAN DO IT, 2722 02:05:52,880 --> 02:06:03,600 FOR AN INDIVIDUAL, A GROUP OF I? 2723 02:06:14,520 --> 02:06:16,440 2724 02:06:16,440 --> 02:06:18,640 TWO UPPER QUADRANTS, ONE IN 2725 02:06:18,640 --> 02:06:29,120 THREE, NOT TWO AND FOUR, THE 2726 02:06:31,040 --> 02:06:33,720 OTHER QUADRANTS, IT CAN BE QUITE 2727 02:06:33,720 --> 02:06:36,920 A DIFFERENT PERSON TO PERSON, 2728 02:06:36,920 --> 02:06:39,200 AND BASED ON COMPENSATION, THEY 2729 02:06:39,200 --> 02:06:49,560 ARE GOING TO FUNCTION 2730 02:06:57,000 --> 02:06:57,240 DIFFERENTLY. 2731 02:06:57,240 --> 02:07:00,200 THERE MUST BE SOME NATURAL 2732 02:07:00,200 --> 02:07:00,400 HISTORY. 2733 02:07:00,400 --> 02:07:03,040 >>OH, RIGHT, RIGHT. 2734 02:07:03,040 --> 02:07:05,040 2735 02:07:05,040 --> 02:07:07,400 SO, I'M NOT SURE IF WE -- WHAT 2736 02:07:07,400 --> 02:07:10,560 WE DO IS WE PUT IT BASED ON YOUR 2737 02:07:10,560 --> 02:07:11,520 SIZE, DANIEL. 2738 02:07:11,520 --> 02:07:13,480 IF YOU'RE A CHILD, THEN YOU 2739 02:07:13,480 --> 02:07:16,360 GROW, WE CAN STILL SAY YOU 2740 02:07:16,360 --> 02:07:18,440 IMPROVED COMPARED TO YOUR FORMER 2741 02:07:18,440 --> 02:07:18,760 SELF. 2742 02:07:18,760 --> 02:07:21,200 AND THAT WAY WE DON'T NEED TO 2743 02:07:21,200 --> 02:07:25,760 KNOW, IT'S A 10% CHANGE OR 4% 2744 02:07:25,760 --> 02:07:27,880 CHANGE, FOR YOU BASED ON YOUR 2745 02:07:27,880 --> 02:07:29,960 SIZE, THE SAME AS A 4% CHANGE 2746 02:07:29,960 --> 02:07:32,240 BASED ON SOMEONE FROM ANOTHER 2747 02:07:32,240 --> 02:07:32,600 SIZE. 2748 02:07:32,600 --> 02:07:34,640 SO YOU'RE CORRECT, IT DOES 2749 02:07:34,640 --> 02:07:41,640 MATTER HOW MUCH TO GET THE 2750 02:07:41,640 --> 02:07:43,880 HURDLE, TYPING TAKES 12% OF 2751 02:07:43,880 --> 02:07:45,120 WORKSPACE VOLUME, DRINKING A CUP 2752 02:07:45,120 --> 02:07:46,600 IS MORE LIKE 22. 2753 02:07:46,600 --> 02:07:48,480 YOU'RE RIGHT, IT DOES MATTER 2754 02:07:48,480 --> 02:07:56,120 WHERE IN THAT CHANGE YOU GAIN. 2755 02:07:56,120 --> 02:07:57,880 SO THAT YOU KNOW WHAT MEANINGFUL 2756 02:07:57,880 --> 02:08:00,080 DIFFERENCE THAT'S GOING TO MAKE. 2757 02:08:00,080 --> 02:08:10,640 >>THE OTHER QUESTION I HAVE, 2758 02:08:21,200 --> 02:08:24,080 ARE THERE PHONE LINK, UNLIKE 2759 02:08:24,080 --> 02:08:31,600 CHANGES IN LENGTH, FOR WHICH 2760 02:08:31,600 --> 02:08:34,440 CASE GROWING QUICK ENOUGH 2761 02:08:34,440 --> 02:08:36,880 CAUSING QUICK CHANGES IN GROWTH? 2762 02:08:36,880 --> 02:08:41,000 >>YES, THAT'S WHY YOU WOULD PUT 2763 02:08:41,000 --> 02:08:42,560 IN THE PERSON'S HEIGHT. 2764 02:08:42,560 --> 02:08:44,760 THAT'S HOW WE KNOW, WE CAN 2765 02:08:44,760 --> 02:08:46,240 ESTIMATE YOUR WING SPAN, THE 2766 02:08:46,240 --> 02:08:47,400 LENGTH OF YOUR ARMS. 2767 02:08:47,400 --> 02:08:51,200 IF YOU DO GROW, YOU STILL WOULD 2768 02:08:51,200 --> 02:08:52,120 SAY PROPORTIONAL, 100 IS THE 2769 02:08:52,120 --> 02:08:53,880 LENGTH OF YOUR ARMS. 2770 02:08:53,880 --> 02:08:56,920 SO IT WOULD ACCOMMODATE GROWTH. 2771 02:08:56,920 --> 02:08:58,720 NO MATTER WHAT SIZE YOU ARE, IF 2772 02:08:58,720 --> 02:09:00,560 YOU HAVE FULL RANGE OF MOTION 2773 02:09:00,560 --> 02:09:09,120 YOU STAY A 100. 2774 02:09:09,120 --> 02:09:09,760 >>THANK YOU. 2775 02:09:09,760 --> 02:09:10,600 >>MICHELE HAS A QUESTION AND 2776 02:09:10,600 --> 02:09:12,600 THERE'S A QUESTION IN THE Q&A 2777 02:09:12,600 --> 02:09:15,720 BOX AND WALTER HAS A QUESTION. 2778 02:09:15,720 --> 02:09:17,960 MICHELE CAMPBELL. 2779 02:09:17,960 --> 02:09:18,320 >>THANK YOU. 2780 02:09:18,320 --> 02:09:21,680 THANK YOU FOR YOUR PRESENTATION. 2781 02:09:21,680 --> 02:09:23,000 THIS IS MICHELE CAMPBELL FROM 2782 02:09:23,000 --> 02:09:24,520 OFFICE OF NEUROSCIENCE AT FDA, 2783 02:09:24,520 --> 02:09:33,880 GREAT TO SEE THE UPDATED WORK. 2784 02:09:33,880 --> 02:09:36,600 FOLLOWING THE QUESTION, WE 2785 02:09:36,600 --> 02:09:38,240 TALKED ABOUT MINIMAL GAINS. 2786 02:09:38,240 --> 02:09:44,000 MINIMAL AND MEANINGFUL AREN'T 2787 02:09:44,000 --> 02:09:46,640 ALWAYS ALIGNED, HOW DO YOU 2788 02:09:46,640 --> 02:09:47,320 COLLECT INFORMATION TO DETERMINE 2789 02:09:47,320 --> 02:09:49,280 THAT, THAT YOU'RE ABLE TO 2790 02:09:49,280 --> 02:09:50,840 MONITOR AN COLLECT DATA OVER 2791 02:09:50,840 --> 02:09:53,240 TIME WHEN YOU'RE SEEING 2792 02:09:53,240 --> 02:09:54,440 PATIENTS, YOU'D BE ABLE TO 2793 02:09:54,440 --> 02:09:58,800 DEVELOP WHAT WOULD BE A 2794 02:09:58,800 --> 02:10:00,320 MEANINGFUL CHANGE, FOR OUR 2795 02:10:00,320 --> 02:10:02,880 INTERPRETATION OF OUR RESULT OF 2796 02:10:02,880 --> 02:10:04,920 A TRIAL, WAS THAT CHANGE 2797 02:10:04,920 --> 02:10:06,480 MEANINGFUL, I CAN'T REMEMBER, 2798 02:10:06,480 --> 02:10:08,520 LINDA, I THOUGHT YOU COLLECTED 2799 02:10:08,520 --> 02:10:10,400 CONTROL DATA AND THE ACTIVE 2800 02:10:10,400 --> 02:10:13,440 STUDY TO HELP INFORM THAT, 2801 02:10:13,440 --> 02:10:14,880 WONDERING HAVE YOU MORE 2802 02:10:14,880 --> 02:10:17,400 ADDITIONAL THOUGHTS TO THIS 2803 02:10:17,400 --> 02:10:19,360 DISCUSSION? 2804 02:10:19,360 --> 02:10:20,160 >>RIGHT. 2805 02:10:20,160 --> 02:10:22,080 THAT'S ABSOLUTELY MEANINGFUL AND 2806 02:10:22,080 --> 02:10:23,440 STATISTICAL ARE TWO DIFFERENT 2807 02:10:23,440 --> 02:10:23,760 THINGS. 2808 02:10:23,760 --> 02:10:29,120 WE DID LOOK AT MINIMUM 2809 02:10:29,120 --> 02:10:32,480 CLINICALLY IMPORTANT DIFFERENCE 2810 02:10:32,480 --> 02:10:34,280 USING THE TEST/ RETEST 2811 02:10:34,280 --> 02:10:36,560 RELIABILITY, FOUR SCALE POINTS, 2812 02:10:36,560 --> 02:10:37,520 USING ONE-THIRD STANDARD 2813 02:10:37,520 --> 02:10:40,960 DEVIATION FOR THE COHORT WITH 2814 02:10:40,960 --> 02:10:43,560 SMA IT WAS TEN SCALED POINTS. 2815 02:10:43,560 --> 02:10:45,160 BUT WHAT YOU'RE TALKING ABOUT 2816 02:10:45,160 --> 02:10:46,360 MORE IS WHETHER THEY FEEL LIKE 2817 02:10:46,360 --> 02:10:48,080 THEY ARE HAVING A CHANGE. 2818 02:10:48,080 --> 02:10:51,040 LOOKING AT THAT BY COMPARING TO 2819 02:10:51,040 --> 02:10:52,200 THE PROMIS, SO IF SOMEONE SAYS 2820 02:10:52,200 --> 02:10:53,920 THEY ARE BETTER ON THE PROMIS, 2821 02:10:53,920 --> 02:10:55,320 WE SAY THEY ARE BETTER, THAT 2822 02:10:55,320 --> 02:10:59,600 WOULD SEEM LIKE IT WOULD BE A 2823 02:10:59,600 --> 02:11:01,240 MEANINGFUL DIFFERENCE. 2824 02:11:01,240 --> 02:11:03,760 WE HAVE MORE WORK TO DO, BASED 2825 02:11:03,760 --> 02:11:04,880 ON PATIENT-REPORTED OUTCOMES TO 2826 02:11:04,880 --> 02:11:07,080 PROMIS TO SEE IF WE CAN TELL IF 2827 02:11:07,080 --> 02:11:09,320 WE'RE GOING IN THE RIGHT 2828 02:11:09,320 --> 02:11:10,880 DIRECTION AND HOW MUCH WE NEED 2829 02:11:10,880 --> 02:11:21,520 TO CHANGE SO THEY NOTICE A 2830 02:11:21,520 --> 02:11:22,560 DIFFERENCE. 2831 02:11:22,560 --> 02:11:23,920 >>THANK YOU. 2832 02:11:23,920 --> 02:11:24,200 >>UH-HUH. 2833 02:11:24,200 --> 02:11:26,440 >>NEXT QUESTION IS IN THE CHAT 2834 02:11:26,440 --> 02:11:28,960 BOX FROM AMY BROWER, HOW DO YOU 2835 02:11:28,960 --> 02:11:32,440 ENVISION THIS WORKING WITH 2836 02:11:32,440 --> 02:11:33,360 NEWBORN SCREENING? 2837 02:11:33,360 --> 02:11:37,280 ALSO WITH THE BIRTHING HOSPITAL 2838 02:11:37,280 --> 02:11:38,400 OR PEDIATRICIAN? 2839 02:11:38,400 --> 02:11:41,560 >>WE'RE WORKING TOWARDS BOTH. 2840 02:11:41,560 --> 02:11:43,640 RIGHT NOW WE'RE DOING INFANTS 2841 02:11:43,640 --> 02:11:47,560 THAT COME IN TO NATIONWIDE 2842 02:11:47,560 --> 02:11:50,080 EITHER AS PRIMARY CARE OR AS 2843 02:11:50,080 --> 02:11:50,960 SPECIALTY CLINIC. 2844 02:11:50,960 --> 02:11:53,160 SO WE'RE COLLECTING FROM BIRTH 2845 02:11:53,160 --> 02:11:55,200 TO SIX MONTHS OF AGE. 2846 02:11:55,200 --> 02:11:57,200 BUT WHAT WE REALLY LOVE TO DO IS 2847 02:11:57,200 --> 02:11:59,400 TO BE ABLE TO SCREEN EVERY 2848 02:11:59,400 --> 02:12:00,800 SINGLE INFANT, NOT JUST THOSE 2849 02:12:00,800 --> 02:12:03,000 THAT WE CAN CATCH IN THE 2850 02:12:03,000 --> 02:12:04,720 PEDIATRICIAN, THAT'S WHAT WE'RE 2851 02:12:04,720 --> 02:12:06,040 WORKING ON NEXT BUT WE KNOW IN 2852 02:12:06,040 --> 02:12:09,440 THE FIRST TWO DAYS OF LIFE YOU 2853 02:12:09,440 --> 02:12:10,880 HAVE ANESTHETIC AND YOU HAVE 2854 02:12:10,880 --> 02:12:12,000 CHANGES TO THE ENVIRONMENT, SO 2855 02:12:12,000 --> 02:12:14,280 WE'RE NOT SURE IF WE CAN DO IT 2856 02:12:14,280 --> 02:12:16,920 IN THE FIRST COUPLE DAYS OF 2857 02:12:16,920 --> 02:12:17,240 LIFE. 2858 02:12:17,240 --> 02:12:19,120 ALL OF OUR DATA NOW HAVE BEEN A 2859 02:12:19,120 --> 02:12:20,360 WEEK OLDER OR MORE. 2860 02:12:20,360 --> 02:12:22,560 IT'S SOMETHING WE NEED TO MOVE 2861 02:12:22,560 --> 02:12:24,360 TOWARDS AND SEE, IT WOULD BE 2862 02:12:24,360 --> 02:12:25,760 GREAT IF EVERY BABY COULD BE 2863 02:12:25,760 --> 02:12:27,600 SCREENED BEFORE THEY LEFT THE 2864 02:12:27,600 --> 02:12:30,680 HOSPITAL, AND THEN ALERTED TO 2865 02:12:30,680 --> 02:12:32,200 THOSE THAT SHOULD SEEK FURTHER 2866 02:12:32,200 --> 02:12:36,240 CARE OR SHOULD GET MORE 2867 02:12:36,240 --> 02:12:36,920 DIAGNOSTIC TESTING. 2868 02:12:36,920 --> 02:12:45,560 >>IF YOU COULD COORDINATE WITH 2869 02:12:45,560 --> 02:12:46,920 AUDITORY SCREENING, GOING TO THE 2870 02:12:46,920 --> 02:12:48,680 SOUNDS THEY ARE HEARING. 2871 02:12:48,680 --> 02:12:50,920 >>THAT'S A GREAT IDEA. 2872 02:12:50,920 --> 02:12:52,120 THAT'S THE THING. 2873 02:12:52,120 --> 02:12:53,920 A HEALTHY BABY CAN LOOK LIKE 2874 02:12:53,920 --> 02:12:55,920 SOMEONE WHO HAS GOT MOVEMENT 2875 02:12:55,920 --> 02:12:57,280 DISORDERS, IF THEY ARE JUST 2876 02:12:57,280 --> 02:12:58,520 BEING LAZY. 2877 02:12:58,520 --> 02:12:59,800 BUT AN UNHEALTHY BABY CAN NEVER 2878 02:12:59,800 --> 02:13:04,000 LOOK LIKE A HEALTHY BABY. 2879 02:13:04,000 --> 02:13:06,280 SOMETIMES WE DO NEED TO JIGGLE 2880 02:13:06,280 --> 02:13:07,000 SOMETHING, RANGE A BELL, A GREAT 2881 02:13:07,000 --> 02:13:10,240 IDEA TO DO IT WITH THE HEARING. 2882 02:13:10,240 --> 02:13:12,240 >>LOGISTICALLY THAT WOULD HELP 2883 02:13:12,240 --> 02:13:13,520 TOO THAT YOU'RE NOT LIKE DOING 2884 02:13:13,520 --> 02:13:16,520 TWO SEPARATE SETS OF SCREENS. 2885 02:13:16,520 --> 02:13:17,240 ANYWAY, VERY INTERESTING 2886 02:13:17,240 --> 02:13:20,360 PRESENTATION. 2887 02:13:20,360 --> 02:13:23,520 THANK YOU SO MUCH. 2888 02:13:23,520 --> 02:13:27,320 >>REAL QUICK QUESTION. 2889 02:13:27,320 --> 02:13:30,960 CAN YOU MEASURE MOVEMENT OF THE 2890 02:13:30,960 --> 02:13:31,240 DIAPHRAGM? 2891 02:13:31,240 --> 02:13:32,240 >>GLAD YOU ASKED. 2892 02:13:32,240 --> 02:13:36,400 WE STARTED DOING THAT IN INFANTS 2893 02:13:36,400 --> 02:13:38,400 WITH SMA. 2894 02:13:38,400 --> 02:13:39,800 THAT DOESN'T -- WE HAVEN'T 2895 02:13:39,800 --> 02:13:42,200 GOTTEN FAR WITH DATA BUT WHAT WE 2896 02:13:42,200 --> 02:13:46,480 LOOKED AT WAS DIAPHRAGMATIC 2897 02:13:46,480 --> 02:13:47,560 BREATHING, PUTTING COLORED 2898 02:13:47,560 --> 02:13:49,160 MARKERS ON THE STERNUM, 2899 02:13:49,160 --> 02:13:51,480 BELLYBUTTON, HALFWAY IN BETWEEN. 2900 02:13:51,480 --> 02:13:53,880 SO, I'M NOT -- WE'RE A LONG WAY 2901 02:13:53,880 --> 02:13:56,360 FROM TRYING IT IN GROWNUPS, BUT 2902 02:13:56,360 --> 02:14:00,320 WE WERE LOOKING TO SEE IF WE 2903 02:14:00,320 --> 02:14:02,920 COULD QUANTIFY BREATHING, IN THE 2904 02:14:02,920 --> 02:14:06,200 INFANTS WITH SMA THEY GO INTO 2905 02:14:06,200 --> 02:14:16,000 SEVERE BREATHING PATTERN. 2906 02:14:16,000 --> 02:14:17,480 >>THANK YOU. 2907 02:14:17,480 --> 02:14:21,440 >>NEXT MEANTER IS MINDY 2908 02:14:21,440 --> 02:14:22,840 LEFFLER, PRESIDENT OF CASIMIR, 2909 02:14:22,840 --> 02:14:24,480 TO IMPROVE THE QUALITY OF DATA 2910 02:14:24,480 --> 02:14:27,360 IN CLINICAL TRIALS BY FOCUSING 2911 02:14:27,360 --> 02:14:28,440 ON THE PATIENT EXPERIENCE, ALSO 2912 02:14:28,440 --> 02:14:32,840 THE MOTHER OF A YOUNG MAN WHO 2913 02:14:32,840 --> 02:14:38,240 LIVES WITH DUCHENNE MUSCULAR 2914 02:14:38,240 --> 02:14:39,760 DYSTROPHY. 2915 02:14:39,760 --> 02:14:46,120 THE TITLE IS QUANTITATIVE 2916 02:14:46,120 --> 02:14:47,200 NEUROMUSCULAR ASSESSMENT IN 2917 02:14:47,200 --> 02:14:47,920 NEUROMUSCULAR CONDITIONS. 2918 02:14:47,920 --> 02:14:49,640 WE NEED YOUR SLIDES IN 2919 02:14:49,640 --> 02:14:50,720 PRESENTATION MODE. 2920 02:14:50,720 --> 02:14:51,040 >>AWESOME. 2921 02:14:51,040 --> 02:14:51,360 THANK YOU. 2922 02:14:51,360 --> 02:14:53,800 THANK YOU FOR THE OPPORTUNITY TO 2923 02:14:53,800 --> 02:14:54,400 PRESENT. 2924 02:14:54,400 --> 02:14:55,720 I APPRECIATE IT. 2925 02:14:55,720 --> 02:14:57,280 I APPRECIATE THE OPPORTUNITY TO 2926 02:14:57,280 --> 02:14:58,480 FIELD QUESTIONS AND HOPEFULLY 2927 02:14:58,480 --> 02:15:01,080 GET FEEDBACK FROM THIS GROUP. 2928 02:15:01,080 --> 02:15:05,720 I'M THE MOTHER OF A NOW 2929 02:15:05,720 --> 02:15:06,320 19-YEAR-OLD WITH DUCHENNE 2930 02:15:06,320 --> 02:15:07,320 MUSCULAR DYSTROPHY. 2931 02:15:07,320 --> 02:15:09,320 HE PARTICIPATED IN HIS FIRST 2932 02:15:09,320 --> 02:15:14,240 STUDY WHEN HE WAS 8 YEARS OLD, 2933 02:15:14,240 --> 02:15:15,120 OUTCOME MEASURE WAS 6-MINUTE 2934 02:15:15,120 --> 02:15:15,480 WALK. 2935 02:15:15,480 --> 02:15:17,760 AT THE TIME, HE ASKED ME, WHY DO 2936 02:15:17,760 --> 02:15:19,800 THEY THINK THAT I CARE SO MUCH 2937 02:15:19,800 --> 02:15:20,600 ABOUT SPEED WALKING? 2938 02:15:20,600 --> 02:15:23,840 WHICH IS A GREAT QUESTION FOR AN 2939 02:15:23,840 --> 02:15:25,040 8-YEAR-OLD TO ASK, AND REALLY 2940 02:15:25,040 --> 02:15:31,440 ILLUSTRATES THE FACT THERE WAS A 2941 02:15:31,440 --> 02:15:32,480 SIGNIFICANT DISCONNECT BETWEEN 2942 02:15:32,480 --> 02:15:33,920 CONCEPT OF INTEREST BEING 2943 02:15:33,920 --> 02:15:36,480 MEASURED, OUTCOME TO MEASURE 2944 02:15:36,480 --> 02:15:39,400 EXERCISE INTOLERANCE, AND WHAT 2945 02:15:39,400 --> 02:15:40,720 WAS MEANINGFUL TO HIM, 2946 02:15:40,720 --> 02:15:41,360 FUNCTIONAL WALKING. 2947 02:15:41,360 --> 02:15:43,160 THAT REALLY WAS THE BEGINNING OF 2948 02:15:43,160 --> 02:15:45,040 OUR CLINICAL TRIAL JOURNEY. 2949 02:15:45,040 --> 02:15:50,400 AND IT'S BEEN FOR A DECADE OF 2950 02:15:50,400 --> 02:15:56,960 SUBMERGES IN CLINICAL TRIAL 2951 02:15:56,960 --> 02:15:57,240 LANDSCAPE. 2952 02:15:57,240 --> 02:15:58,160 THIS IS A LIST OF CLINICAL 2953 02:15:58,160 --> 02:16:04,400 TRIALS THAT FAILED TO REACH 2954 02:16:04,400 --> 02:16:05,160 STATISTICAL SIGNIFICANCE. 2955 02:16:05,160 --> 02:16:06,800 EACH REPRESENTS A GROUP OF 2956 02:16:06,800 --> 02:16:09,280 FAMILIES LIKE MY OWN WHO HAVE 2957 02:16:09,280 --> 02:16:15,760 GIVEN UP BLOOD, SWEAT, LITERAL 2958 02:16:15,760 --> 02:16:16,120 FLESH. 2959 02:16:16,120 --> 02:16:17,840 WE DON'T TRULY KNOW IF IT WAS 2960 02:16:17,840 --> 02:16:18,880 OUTCOME MEASURE THAT WAS THE 2961 02:16:18,880 --> 02:16:20,480 ISSUE OR WHETHER IT WAS THE 2962 02:16:20,480 --> 02:16:23,120 STUDY DESIGN THAT WAS THE ISSUE 2963 02:16:23,120 --> 02:16:25,800 AND OUTCOME MEASURE IN USE. 2964 02:16:25,800 --> 02:16:27,600 THAT'S DRIVING US TO TRY TO 2965 02:16:27,600 --> 02:16:29,320 ADDRESS THAT ISSUE WHICH IS 2966 02:16:29,320 --> 02:16:31,200 POTENTIAL FOR FAMILIES TO COME 2967 02:16:31,200 --> 02:16:32,400 AWAY WITH TRIAL PARTS 2968 02:16:32,400 --> 02:16:42,760 PARTICIPATION WITHOUT 2969 02:16:44,440 --> 02:16:46,320 DEFINITIVE ANSWERS. 2970 02:16:46,320 --> 02:16:49,600 WE ASKED WHAT IS MORE MEANINGFUL 2971 02:16:49,600 --> 02:16:50,560 CONCEPT OF INTEREST. 2972 02:16:50,560 --> 02:16:51,920 IT BECOMES MORE DIFFICULT TO 2973 02:16:51,920 --> 02:16:53,200 MOVE UNTIL PATIENTS ARE UNABLE 2974 02:16:53,200 --> 02:16:54,080 TO MOVE. 2975 02:16:54,080 --> 02:16:56,920 WE THOUGHT THE MOST MEANINGFUL 2976 02:16:56,920 --> 02:17:00,560 OF INTEREST IS EASE OF MOVEMENT. 2977 02:17:00,560 --> 02:17:03,640 WE THOUGHT ABOUT WAYS TO DO 2978 02:17:03,640 --> 02:17:03,920 THAT. 2979 02:17:03,920 --> 02:17:05,800 TALKED TO PARENTS. 2980 02:17:05,800 --> 02:17:07,320 WE NOTICED WHAT PARENTS 2981 02:17:07,320 --> 02:17:08,480 ARTICULATE IS THAT THEY TRACK 2982 02:17:08,480 --> 02:17:11,120 WHETHER THE CHILDREN ARE GETTING 2983 02:17:11,120 --> 02:17:12,880 WORSE, BETTER, STAYING STABLE BY 2984 02:17:12,880 --> 02:17:14,520 LOOKING AT COMPENSATORY MOVEMENT 2985 02:17:14,520 --> 02:17:16,400 PATTERNS THE BOYS ADOPT TO WORK 2986 02:17:16,400 --> 02:17:17,400 AROUND PROGRESSIVE WEAKNESS. 2987 02:17:17,400 --> 02:17:19,600 WE TOOK A LOOK HOW PATIENTS WERE 2988 02:17:19,600 --> 02:17:20,560 FUNCTIONING AS THEY WITHDREW 2989 02:17:20,560 --> 02:17:24,280 FROM TREATMENT IN A STUDY PUT ON 2990 02:17:24,280 --> 02:17:26,480 CLINICAL HOLD AND SAW 2991 02:17:26,480 --> 02:17:27,040 ACQUISITION OF COMPENSATIONS 2992 02:17:27,040 --> 02:17:30,520 SIMILAR TO THE WAY PARENTS TALK 2993 02:17:30,520 --> 02:17:31,280 ABOUT IT. 2994 02:17:31,280 --> 02:17:36,880 THE FIRST TIME POINT RIGHT 2995 02:17:36,880 --> 02:17:41,800 BEFORE, AND BY THE SECOND, 2996 02:17:41,800 --> 02:17:43,000 ALTERNATING LEGS TO A PATTERN 2997 02:17:43,000 --> 02:17:44,320 WHERE HE CLEARS THE STAIRS, 2998 02:17:44,320 --> 02:17:46,920 USING ONE ARM ON THE RAIL, ONE 2999 02:17:46,920 --> 02:17:50,080 ARM OFF THE THIGH, PICKING UP 3000 02:17:50,080 --> 02:17:50,880 COMPENSATIONS, THE FIRST CLUE 3001 02:17:50,880 --> 02:17:52,520 THIS COULD BE A RIGOROUS AND 3002 02:17:52,520 --> 02:17:54,040 VALID WAY TO QUANTIFY WHAT WE 3003 02:17:54,040 --> 02:17:57,480 TALK ABOUT AS PARENTS WITH 3004 02:17:57,480 --> 02:17:58,880 DUCHENNE, CHILDREN WITH 3005 02:17:58,880 --> 02:17:59,320 DUCHENNE. 3006 02:17:59,320 --> 02:18:01,360 WHEN MY SON EATS HIS MEAL LIKE 3007 02:18:01,360 --> 02:18:04,800 THIS, AND I START SEEING HIM DO 3008 02:18:04,800 --> 02:18:06,080 THIS, SUPPORTING ONE ARM WITH 3009 02:18:06,080 --> 02:18:07,800 THE OTHER ARM AT THE WRIST, I 3010 02:18:07,800 --> 02:18:09,000 KNOW WHAT THAT MEANS AS A 3011 02:18:09,000 --> 02:18:09,360 PARENT. 3012 02:18:09,360 --> 02:18:11,240 IT MEANS THE EATING IS GETTING 3013 02:18:11,240 --> 02:18:12,640 HARD FOR HIM. 3014 02:18:12,640 --> 02:18:14,480 IT WAS THAT FUNDAMENTAL SENSE OF 3015 02:18:14,480 --> 02:18:15,600 DISEASE PROGRESSION WE WERE 3016 02:18:15,600 --> 02:18:19,360 HOPING TO QUANTIFY IN A VALID 3017 02:18:19,360 --> 02:18:21,080 RELIABLE AND RIGOROUS FASHION. 3018 02:18:21,080 --> 02:18:25,120 SO, THE WAY IT WORKS, WE HAVE 3019 02:18:25,120 --> 02:18:27,200 VALIDATED APP, THAT PARENTS 3020 02:18:27,200 --> 02:18:29,600 DOWNLOAD FROM GOOGLE PLAY OR APP 3021 02:18:29,600 --> 02:18:31,560 STORE, AND THEY REGISTER FOR THE 3022 02:18:31,560 --> 02:18:32,840 APP, WATCH TRAINING VIDEOS, CAN 3023 02:18:32,840 --> 02:18:34,880 TAKE THEIR OWN VIDEOS. 3024 02:18:34,880 --> 02:18:38,400 WE HAVE SCORE CARDS TO TRAIN AND 3025 02:18:38,400 --> 02:18:40,000 CERTIFY CENTRAL READERS TO USE, 3026 02:18:40,000 --> 02:18:46,040 WHAT THAT MEANS TO QUANTIFY, WE 3027 02:18:46,040 --> 02:18:48,440 CAN BLIND RATERS TO TIME POINT 3028 02:18:48,440 --> 02:18:50,560 TO ELIMINATE TIME POINT BIAS. 3029 02:18:50,560 --> 02:18:52,200 IT'S DONE AT HOME WHICH WE FELT 3030 02:18:52,200 --> 02:18:54,360 WAS NOT ONLY IMPORTANT DUE TO 3031 02:18:54,360 --> 02:18:56,760 REDUCTION OF BURDEN ON FAMILIES 3032 02:18:56,760 --> 02:18:59,200 BUT ALSO I THINK THAT 3033 02:18:59,200 --> 02:19:00,640 REMOTE-BASED CAPTURE IS A MATTER 3034 02:19:00,640 --> 02:19:02,280 OF DATA INTEGRITY JUST BECAUSE 3035 02:19:02,280 --> 02:19:04,920 WE ALL KNOW HOW MUCH TRAVEL 3036 02:19:04,920 --> 02:19:06,960 FATIGUE CAN INFLUENCE DATA 3037 02:19:06,960 --> 02:19:09,160 COLLECTION AND DUCHENNE AND 3038 02:19:09,160 --> 02:19:10,240 OTHER NEUROMUSCULAR DISEASES. 3039 02:19:10,240 --> 02:19:12,600 ONE MAIN THING TO QUANTIFY IS 3040 02:19:12,600 --> 02:19:14,720 THE FACT THAT LOSS OF FUNCTION 3041 02:19:14,720 --> 02:19:16,360 IS AN EXTRAORDINARILY MEANINGFUL 3042 02:19:16,360 --> 02:19:18,360 STEP IN DISEASE PROGRESSION, FOR 3043 02:19:18,360 --> 02:19:19,080 PATIENTS WITH NEUROMUSCULAR 3044 02:19:19,080 --> 02:19:22,840 DISEASE, SO WE WANTED TO 3045 02:19:22,840 --> 02:19:24,080 QUANTIFY INCREMENTAL STEPS 3046 02:19:24,080 --> 02:19:25,320 TOWARDS THAT LOSS OF FUNCTION, 3047 02:19:25,320 --> 02:19:28,640 SUCH THAT WHEN THEY FINALLY DO 3048 02:19:28,640 --> 02:19:35,200 LOSE FUNCTION, IT ISN'T -- IT'S 3049 02:19:35,200 --> 02:19:35,720 WEIGHTED APPROPRIATELY. 3050 02:19:35,720 --> 02:19:40,960 BY THE TIME BOYS ACQUIRED EVERY 3051 02:19:40,960 --> 02:19:42,200 MOVEMENT, IT'S SO COMPENSATED 3052 02:19:42,200 --> 02:19:43,960 THE LAST STEP OF LOSS OF 3053 02:19:43,960 --> 02:19:46,040 FUNCTION IS WEIGHTED MORE 3054 02:19:46,040 --> 02:19:47,200 EQUALLY WITH EACH ACQUISITION OF 3055 02:19:47,200 --> 02:19:49,200 A COMPENSATION SO HERE IS A 3056 02:19:49,200 --> 02:19:50,200 COUPLE VIDEOS TO DEMONSTRATE. 3057 02:19:50,200 --> 02:19:51,720 THIS IS A PATIENT GETTING OFF 3058 02:19:51,720 --> 02:19:53,360 THE FLOOR AT ONE TIME POINT. 3059 02:19:53,360 --> 02:19:57,120 YOU CAN SEE HE'S HIGHLY, HIGHLY 3060 02:19:57,120 --> 02:19:59,160 COMPENSATED, LEGS ARE FAR APART, 3061 02:19:59,160 --> 02:20:02,240 WALKING WITH HANDS ALONG SIDES, 3062 02:20:02,240 --> 02:20:02,880 PUSHING OFF. 3063 02:20:02,880 --> 02:20:03,920 THAT MEANS GETTING OFF THE 3064 02:20:03,920 --> 02:20:07,480 GROUND IS SUCH A HIGHLY 3065 02:20:07,480 --> 02:20:08,120 COMPENSATED AND DIFFICULT 3066 02:20:08,120 --> 02:20:11,440 ACTIVITY THAT BY THE TIME THAT 3067 02:20:11,440 --> 02:20:14,600 LOSS OF FUNCTION HAPPENED, 3068 02:20:14,600 --> 02:20:15,240 SHORTLY AFTERWARDS, THERE'S NOT 3069 02:20:15,240 --> 02:20:21,200 A LOT OF CHANGE IN THAT MOVEMENT 3070 02:20:21,200 --> 02:20:23,440 PATTERN. 3071 02:20:23,440 --> 02:20:29,280 IT'S JUST BALANCED MORE EQUALLY. 3072 02:20:29,280 --> 02:20:31,160 WHEN WE WERE TAKING A LOOK THE A 3073 02:20:31,160 --> 02:20:33,080 DESIGNING SCORE CARDS, THERE ARE 3074 02:20:33,080 --> 02:20:34,960 TWO WAYS, LOTS OF ACTIVITY ON 3075 02:20:34,960 --> 02:20:37,280 THE A.I. FRONT AND THERE'S TWO 3076 02:20:37,280 --> 02:20:39,440 WAYS TO COME AT THAT PROCESS OF 3077 02:20:39,440 --> 02:20:39,880 AUTOMATION. 3078 02:20:39,880 --> 02:20:42,640 ONE ON THE RIGHT IS TO FEED DATA 3079 02:20:42,640 --> 02:20:44,080 INTO THE BLACK BOX, IDENTIFY 3080 02:20:44,080 --> 02:20:45,680 CONCEPT OF INTEREST AND PATTERNS 3081 02:20:45,680 --> 02:20:47,200 THAT EMERGE. 3082 02:20:47,200 --> 02:20:50,280 AND TO ANCHOR THAT CHANGE, BASED 3083 02:20:50,280 --> 02:20:52,240 ON AN ANCHORING INSTRUMENT TO 3084 02:20:52,240 --> 02:20:55,000 DETERMINE HOW MUCH CHANGE IS 3085 02:20:55,000 --> 02:20:55,640 MEANINGFUL. 3086 02:20:55,640 --> 02:21:00,120 WE WENT A DIFFERENT ROUTE, TO 3087 02:21:00,120 --> 02:21:03,200 TALK TO PATIENTS AND FAMILIES, 3088 02:21:03,200 --> 02:21:04,400 DETERMINE AMOUNT OF CLINICALLY 3089 02:21:04,400 --> 02:21:05,600 MEANINGFUL CHANGE THROUGH 3090 02:21:05,600 --> 02:21:07,280 PATIENTS OR CLINICIAN REPORT, 3091 02:21:07,280 --> 02:21:09,240 CHOSE TO USE CLINICIANS BECAUSE 3092 02:21:09,240 --> 02:21:12,080 IT WAS REALLY AN N OF 100 3093 02:21:12,080 --> 02:21:14,480 SITUATION RATHER THAN N-of-1, 3094 02:21:14,480 --> 02:21:15,320 TO LIFT MOVEMENT PATTERNS 3095 02:21:15,320 --> 02:21:16,320 UTILIZED ACROSS THE POPULATION 3096 02:21:16,320 --> 02:21:18,080 RATHER THAN MAYBE SOMETHING 3097 02:21:18,080 --> 02:21:19,080 UNIQUE TO A PARTICULAR PATIENT 3098 02:21:19,080 --> 02:21:22,160 AND OUR HOPE IS TO AUTOMATE BY 3099 02:21:22,160 --> 02:21:24,320 TRAINING ALGORITHM TO RECOGNIZE 3100 02:21:24,320 --> 02:21:25,680 THOSE MOVEMENT PATTERNS. 3101 02:21:25,680 --> 02:21:28,120 DIFFERENT WAYS TO APPROACH THAT, 3102 02:21:28,120 --> 02:21:30,800 ONE OF THE MAIN REASONS IS THAT 3103 02:21:30,800 --> 02:21:32,440 THIRD STEP ON THE RIGHT, EVERY 3104 02:21:32,440 --> 02:21:35,680 TIME WE TRIED TO DIG IN, WHAT 3105 02:21:35,680 --> 02:21:36,680 ANCHORING INSTRUMENTS COULD WE 3106 02:21:36,680 --> 02:21:39,440 USE THAT MIGHT BE APPROPRIATE TO 3107 02:21:39,440 --> 02:21:41,720 DUCHENNE, WE RAN AGAINST 3108 02:21:41,720 --> 02:21:42,960 PROBLEMS OF PERHAPS ANCHORING 3109 02:21:42,960 --> 02:21:44,680 INSTRUMENT MIGHT NOT BE 3110 02:21:44,680 --> 02:21:45,760 SUFFICIENTLY SENSITIVE TO DETECT 3111 02:21:45,760 --> 02:21:46,960 CHANGE, MIGHT STILL BE 3112 02:21:46,960 --> 02:21:48,280 MEANINGFUL TO PATIENTS AND 3113 02:21:48,280 --> 02:21:48,600 FAMILIES. 3114 02:21:48,600 --> 02:21:50,480 AND THEN I THINK IT'S WORTH 3115 02:21:50,480 --> 02:21:53,120 NOTING THAT THE USE OF PROs IN 3116 02:21:53,120 --> 02:21:56,360 THIS POPULATION CAN BE 3117 02:21:56,360 --> 02:21:56,680 PROBLEMATIC. 3118 02:21:56,680 --> 02:21:58,800 SOME PATIENTS KNOW MORE ABOUT 3119 02:21:58,800 --> 02:22:00,280 THEIR PROGNOSIS AND PROGRESSION 3120 02:22:00,280 --> 02:22:00,640 THAN OTHERS. 3121 02:22:00,640 --> 02:22:03,960 AND SO THEY ARE GOING TO ANSWER 3122 02:22:03,960 --> 02:22:05,040 DIFFERENTLY ON INSTRUMENTS WHEN 3123 02:22:05,040 --> 02:22:06,120 THEY KNOW MORE ABOUT WHAT'S 3124 02:22:06,120 --> 02:22:08,200 GOING TO HAPPEN TO THEM THAN IF 3125 02:22:08,200 --> 02:22:09,040 THEY DON'T KNOW THE 3126 02:22:09,040 --> 02:22:10,280 MEANINGFULNESS OF WHAT'S 3127 02:22:10,280 --> 02:22:10,640 HAPPENING. 3128 02:22:10,640 --> 02:22:11,400 THAT'S SOMETHING WE NEED TO TAKE 3129 02:22:11,400 --> 02:22:13,800 INTO ACCOUNT WHEN WE LOOK AT 3130 02:22:13,800 --> 02:22:14,800 ANCHORING WITH PRO INSTRUMENTS. 3131 02:22:14,800 --> 02:22:17,200 THE OTHER PIECE I SEE IN MY OWN 3132 02:22:17,200 --> 02:22:20,240 SON, HE USES DENIAL AS A COPING 3133 02:22:20,240 --> 02:22:20,520 MECHANISM. 3134 02:22:20,520 --> 02:22:25,960 IT'S NOT UP TO ME TO SAY THAT'S 3135 02:22:25,960 --> 02:22:28,480 NOT APPROPRIATE BUT IT DOES 3136 02:22:28,480 --> 02:22:33,080 PRESENT ISSUES UTILIZING PATIENT 3137 02:22:33,080 --> 02:22:40,600 REPORTED OUTCOMES OF ANCHORS, 3138 02:22:40,600 --> 02:22:41,720 THE DENIAL INTRODUCES 3139 02:22:41,720 --> 02:22:42,040 COMPLEXITY. 3140 02:22:42,040 --> 02:22:45,360 JUST TO TALK ABOUT THE PROCESS 3141 02:22:45,360 --> 02:22:46,760 TO DEVELOP THIS OUTCOME, THE 3142 02:22:46,760 --> 02:22:48,560 FIRST THING WE DID WAS IDENTIFY 3143 02:22:48,560 --> 02:22:49,720 THE TASK LIST OF THINGS THAT WE 3144 02:22:49,720 --> 02:22:52,600 WERE GOING TO PUT ON VIDEO, TO 3145 02:22:52,600 --> 02:22:59,160 MAKE SURE WE WEREN'T USING 3146 02:22:59,160 --> 02:23:05,280 STANDIN TASKS, BUT TASKS THAT 3147 02:23:05,280 --> 02:23:06,480 INDICATED INDEPENDENCE LIKE 3148 02:23:06,480 --> 02:23:09,400 GETTING OFF THE FLOOR, A CELL 3149 02:23:09,400 --> 02:23:11,520 PHONE, FROM VERY YOUNGER MORE 3150 02:23:11,520 --> 02:23:13,400 FUNCTIONAL AGES UP TO 3151 02:23:13,400 --> 02:23:15,840 NON-AMBULATORY PATIENTS WITH 3152 02:23:15,840 --> 02:23:17,120 LESS FUNCTION. 3153 02:23:17,120 --> 02:23:20,400 WE DESIGNED CAPTURE PROCEDURES 3154 02:23:20,400 --> 02:23:21,280 AND VALIDATED CAPTURE 3155 02:23:21,280 --> 02:23:22,600 PROCEDURES, PUBLISHED IN THE 3156 02:23:22,600 --> 02:23:25,320 MANUSCRIPT, DETAILED ON THE 3157 02:23:25,320 --> 02:23:25,520 SLIDE. 3158 02:23:25,520 --> 02:23:27,720 NEXT STEP IS THAT WE GOT TO 3159 02:23:27,720 --> 02:23:29,920 UTILIZE THE SERVICES OF THESE 3160 02:23:29,920 --> 02:23:37,160 WONDERFUL LADIES, AND THIS WHOLE 3161 02:23:37,160 --> 02:23:38,320 GROUP AT THE NATIONWIDE 3162 02:23:38,320 --> 02:23:39,560 CHILDREN'S HOSPITAL, BUILDING A 3163 02:23:39,560 --> 02:23:41,200 LIST OF COMPENSATIONS WE SHOULD 3164 02:23:41,200 --> 02:23:41,840 CONSIDER QUANTIFYING. 3165 02:23:41,840 --> 02:23:43,720 THIS IS A GROUP OF PEOPLE THAT 3166 02:23:43,720 --> 02:23:46,440 SEE HUNDREDS OF PATIENTS IN THE 3167 02:23:46,440 --> 02:23:48,200 CONTEXT OF CLINIC ASSESSMENT AND 3168 02:23:48,200 --> 02:23:48,840 CLINICAL TRIAL ASSESSMENT. 3169 02:23:48,840 --> 02:23:51,280 WE FELT IT WAS THE PERFECT 3170 02:23:51,280 --> 02:23:54,000 GROUP, EXTRAORDINARILY GENEROUS 3171 02:23:54,000 --> 02:23:54,560 WITH TIME. 3172 02:23:54,560 --> 02:23:57,720 WE BUILT A LIST OF 3173 02:23:57,720 --> 02:23:58,920 COMPENSATIONS, WHEN WE BUILT 3174 02:23:58,920 --> 02:24:01,160 SCORE CARDS NEEDED TO MAKE SURE 3175 02:24:01,160 --> 02:24:02,760 IF ONE LEVEL TRANSITIONED INTO A 3176 02:24:02,760 --> 02:24:04,080 COMPENSATION THAT WAS MORE 3177 02:24:04,080 --> 02:24:06,600 SEVERE, IT WAS QUANTIFIED THAT 3178 02:24:06,600 --> 02:24:08,040 WAY, AND SO FOR EXAMPLE WHEN 3179 02:24:08,040 --> 02:24:12,840 KIDS GO UP THE STAIRS THEY USE A 3180 02:24:12,840 --> 02:24:14,480 WIDE BASE OF SUPPORT, THEY 3181 02:24:14,480 --> 02:24:18,000 COLLAPSE THAT WIDE BASE AND IN 3182 02:24:18,000 --> 02:24:19,720 TURN SIDE STEP UP THE STAIRS. 3183 02:24:19,720 --> 02:24:22,600 WE WANTED TO MAKE SURE THE 3184 02:24:22,600 --> 02:24:23,760 DISAPPEARANCE DIDN'T LOOK LIKE 3185 02:24:23,760 --> 02:24:25,560 IMPROVEMENT, SO SOME CRITERIA 3186 02:24:25,560 --> 02:24:28,080 ARE NESTED, SOME CRITERIA ARE 3187 02:24:28,080 --> 02:24:29,480 LISTED INDEPENDENTLY FOR THAT 3188 02:24:29,480 --> 02:24:30,080 REASON. 3189 02:24:30,080 --> 02:24:34,320 NEXT THING WE DID, LIKE I 3190 02:24:34,320 --> 02:24:36,160 MENTIONED BEFORE, TO ESTABLISH 3191 02:24:36,160 --> 02:24:37,720 EACH COMPENSATORY ACQUISITION ON 3192 02:24:37,720 --> 02:24:39,440 OUR SCORE CARDS AS CLINICALLY 3193 02:24:39,440 --> 02:24:41,400 MEANINGFUL IN THE OPINION OF A 3194 02:24:41,400 --> 02:24:48,200 GROUP OF EXPERTS IN DUCHENNE 3195 02:24:48,200 --> 02:24:49,080 MUSCULAR DYSTROPHY. 3196 02:24:49,080 --> 02:24:58,720 THESE ARE P.T.s THAT ASSESS 3197 02:24:58,720 --> 02:24:59,280 PATIENTS. 3198 02:24:59,280 --> 02:25:00,240 WE REACHED AGREEMENT ON THE 3199 02:25:00,240 --> 02:25:03,080 SCORE CARDS AS THEY MOVE 3200 02:25:03,080 --> 02:25:04,120 FORWARD, REMOVED COMPENSATIONS 3201 02:25:04,120 --> 02:25:06,600 THAT THIS GROUP OF EXPERTS FELT 3202 02:25:06,600 --> 02:25:08,120 REPRESENTED LESS THAN CLINICALLY 3203 02:25:08,120 --> 02:25:11,640 MEANINGFUL AMOUNT OF CHANGE. 3204 02:25:11,640 --> 02:25:14,120 THAT WAS PUBLISHED RECENTLY IN 3205 02:25:14,120 --> 02:25:20,400 THE MANUSCRIPT INDICATED ON THE 3206 02:25:20,400 --> 02:25:21,200 SLIDE. 3207 02:25:21,200 --> 02:25:23,600 NEXT WAS ESTABLISH PSYCHOMETRIC 3208 02:25:23,600 --> 02:25:27,000 PROPERTIES ON THE SCORE CARD, 3209 02:25:27,000 --> 02:25:29,400 GREAT RELIABILITY AND STRONG 3210 02:25:29,400 --> 02:25:30,800 CONCEPT VALIDITY, LET US KNOW 3211 02:25:30,800 --> 02:25:33,520 THIS COULD BE DONE RELIABLY, 3212 02:25:33,520 --> 02:25:35,840 MEASURING WHAT WE INTENDED TO 3213 02:25:35,840 --> 02:25:36,560 MEASURE. 3214 02:25:36,560 --> 02:25:38,120 THE LAST STAGE, PUTTING THIS 3215 02:25:38,120 --> 02:25:40,200 TOGETHER, LIKE I MENTIONED OUR 3216 02:25:40,200 --> 02:25:44,240 APPROACH TO IDENTIFYING AMOUNT 3217 02:25:44,240 --> 02:25:47,120 OF CHANGE THAT WOULD CONSTITUTE 3218 02:25:47,120 --> 02:25:49,760 MEANINGFUL CHANGE WAS ESTABLISH 3219 02:25:49,760 --> 02:25:51,800 EACH PATTERN AS A CLINICALLY 3220 02:25:51,800 --> 02:25:54,200 MEANINGFUL AMOUNT OF DISEASE 3221 02:25:54,200 --> 02:25:55,840 PROGRESSION, MOUNTING OUR LAST 3222 02:25:55,840 --> 02:26:00,760 STUDY IN THE DUCHENNE 3223 02:26:00,760 --> 02:26:01,400 POPULATION, 150-PATIENT STUDY, 3224 02:26:01,400 --> 02:26:03,720 SPANS FROM YOUNG BOYS TO ADULT 3225 02:26:03,720 --> 02:26:04,840 PATIENTS WITH DUCHENNE, ANYONE 3226 02:26:04,840 --> 02:26:05,600 CAN PARTICIPATE. 3227 02:26:05,600 --> 02:26:07,880 WE'RE GOING TO DO A TEST-RETEST 3228 02:26:07,880 --> 02:26:10,960 AT BASELINE TO ALLOW US TO 3229 02:26:10,960 --> 02:26:12,160 ESTABLISH INHERENT INSTRUMENT 3230 02:26:12,160 --> 02:26:14,480 VARIABILITY, OUR APPROACH TO 3231 02:26:14,480 --> 02:26:16,120 DEFINING CHANGE IS TO TAKE A 3232 02:26:16,120 --> 02:26:19,280 LOOK AT AMOUNT OF CHANGE IN 3233 02:26:19,280 --> 02:26:21,160 EXCESS OF THAT INSTRUMENT 3234 02:26:21,160 --> 02:26:22,040 VARIABILITY AND WE'RE GOING TO 3235 02:26:22,040 --> 02:26:26,760 TAKE A LOOK HOW THIS MEASURE 3236 02:26:26,760 --> 02:26:28,160 MOVES ALONGSIDE GOLD STANDARDS 3237 02:26:28,160 --> 02:26:30,200 CURRENTLY IN USE IN DUCHENNE 3238 02:26:30,200 --> 02:26:36,880 MUSCULAR DYSTROPHY, SO IN THE 3239 02:26:36,880 --> 02:26:37,800 INTEREST OF FAMILIES WITH 3240 02:26:37,800 --> 02:26:40,240 DUCHENNE THAT MIGHT BE WILLING 3241 02:26:40,240 --> 02:26:41,800 TO PARTICIPATE, I'LL LEAVE THIS 3242 02:26:41,800 --> 02:26:41,960 UP. 3243 02:26:41,960 --> 02:26:43,360 WE'RE GOING TO RECRUIT THIS 3244 02:26:43,360 --> 02:26:43,560 WEEK. 3245 02:26:43,560 --> 02:26:45,720 WE HAVE A LOT OF AMBITION IN 3246 02:26:45,720 --> 02:26:47,200 TERMS OF NUMBER OF PATIENTS 3247 02:26:47,200 --> 02:26:48,440 WE'RE TRYING TO REACH, HOPEFULLY 3248 02:26:48,440 --> 02:26:50,600 WE GET SOME INTEREST FROM THAT. 3249 02:26:50,600 --> 02:26:52,960 WE'RE ALSO LOOKING TO ADAPT THIS 3250 02:26:52,960 --> 02:26:55,960 APPROACH TO OTHER NEUROMUSCULAR 3251 02:26:55,960 --> 02:26:57,080 DISEASES, SO CRITERIA FOR THAT 3252 02:26:57,080 --> 02:26:58,880 IS THAT THAT OTHER NEUROMUSCULAR 3253 02:26:58,880 --> 02:27:02,400 DISEASE ALSO HAS AN UNMET NEED, 3254 02:27:02,400 --> 02:27:04,840 THE LAST THING WE WANT TO DO IS 3255 02:27:04,840 --> 02:27:06,240 WASTE TIME, EFFORT, MONEY. 3256 02:27:06,240 --> 02:27:07,240 WE'RE LOOKING AT OTHER DISEASE 3257 02:27:07,240 --> 02:27:09,600 AREAS THAT WOULD BE WELL SERVED 3258 02:27:09,600 --> 02:27:11,440 WITH SAME SHARED CONCEPT OF 3259 02:27:11,440 --> 02:27:12,080 INTEREST. 3260 02:27:12,080 --> 02:27:14,680 IN THOSE EVENTS WE EVALUATE 3261 02:27:14,680 --> 02:27:15,520 DEGREE OF TASK OVERLAP, SHOULD 3262 02:27:15,520 --> 02:27:18,640 WE PUT THE SAME THINGS ON VIDEO, 3263 02:27:18,640 --> 02:27:20,400 IF THERE ARE ADDITIONAL TASKS 3264 02:27:20,400 --> 02:27:21,360 SPECIFIC TO THE NEW DISEASE AREA 3265 02:27:21,360 --> 02:27:26,280 WE HAVE TO IDENTIFY CAPTURE 3266 02:27:26,280 --> 02:27:27,120 PROCEDURES, COLLECTED SOURCE 3267 02:27:27,120 --> 02:27:27,760 MATERIAL. 3268 02:27:27,760 --> 02:27:30,600 THEN WE LOOK AT ADAPTING SCORE 3269 02:27:30,600 --> 02:27:30,800 CARDS. 3270 02:27:30,800 --> 02:27:32,480 ARE THERE MOVEMENT PATTERNS THAT 3271 02:27:32,480 --> 02:27:33,880 WERE NOT REFLECTING IN OUR 3272 02:27:33,880 --> 02:27:35,240 EXISTING SCORE CARDS? 3273 02:27:35,240 --> 02:27:36,760 ARE THERE COMPENSATIONS IN THE 3274 02:27:36,760 --> 02:27:38,040 EXISTING SCORE CARDS NOT 3275 02:27:38,040 --> 02:27:39,920 MANIFESTING IN THIS NEW DISEASE 3276 02:27:39,920 --> 02:27:40,360 AREA? 3277 02:27:40,360 --> 02:27:41,800 SO WE'LL ADAPT THOSE SCORE CARDS 3278 02:27:41,800 --> 02:27:44,880 TO THIS DISEASE AREA IN A WAY 3279 02:27:44,880 --> 02:27:47,160 THAT'S SPECIFIC AND AVOIDS SOME 3280 02:27:47,160 --> 02:27:48,280 PITFALLS OF BORROWING MEASURES 3281 02:27:48,280 --> 02:27:50,480 FROM ONE DISEASE TO THE NEXT 3282 02:27:50,480 --> 02:27:53,120 WITHOUT THAT VALIDATION EFFORT. 3283 02:27:53,120 --> 02:27:54,280 WE'LL REESTABLISH THE 3284 02:27:54,280 --> 02:27:55,600 PSYCHOMETRIC PROPERTIES OF THOSE 3285 02:27:55,600 --> 02:27:57,440 NEW SCORE CARDS TO MAKE SURE 3286 02:27:57,440 --> 02:28:01,720 THEY ARE PERFORMING THE WAY WE 3287 02:28:01,720 --> 02:28:04,120 HOPE THEY WILL. 3288 02:28:04,120 --> 02:28:05,000 WE'RE COLLECTING DATA THAT WILL 3289 02:28:05,000 --> 02:28:07,760 GET US TO THE LAST STEP, THE 3290 02:28:07,760 --> 02:28:09,120 DEFINITION OF WITHIN PATIENT 3291 02:28:09,120 --> 02:28:11,840 MEANINGFUL CHANGE WITHIN TWO 3292 02:28:11,840 --> 02:28:13,480 ARMS, THROUGH OUR STUDY WE'RE 3293 02:28:13,480 --> 02:28:16,280 RECRUITING THIS WEEK AND A 3294 02:28:16,280 --> 02:28:18,160 NUMBER OF OTHER DISEASES WE'RE 3295 02:28:18,160 --> 02:28:20,360 COLLECTING THIS MATERIAL TO LOOK 3296 02:28:20,360 --> 02:28:27,560 AT ADAPTATION OF SCORE CARDS 3297 02:28:27,560 --> 02:28:30,480 INCLUDING PMM, VWP, AND 3298 02:28:30,480 --> 02:28:30,760 LIMB-GIRDLE. 3299 02:28:30,760 --> 02:28:31,960 AND ALWAYS INTERESTED IN 3300 02:28:31,960 --> 02:28:33,320 ADAPTING THIS APPROACH TO OTHER 3301 02:28:33,320 --> 02:28:36,160 DISEASES IN WHICH IT MIGHT SERVE 3302 02:28:36,160 --> 02:28:36,920 THAT POPULATION. 3303 02:28:36,920 --> 02:28:38,320 AND JUST LAST TO MENTION WE'RE 3304 02:28:38,320 --> 02:28:43,440 TAKING A LOOK AT THIS APPROACH, 3305 02:28:43,440 --> 02:28:44,120 VARIOUS NEURODEVELOPMENTAL 3306 02:28:44,120 --> 02:28:44,760 DISEASES. 3307 02:28:44,760 --> 02:28:48,040 THIS SLIDE IS IN REFERENCE TO 3308 02:28:48,040 --> 02:28:48,240 THAT. 3309 02:28:48,240 --> 02:28:49,240 AND OBVIOUSLY ACTIVITIES LOOKING 3310 02:28:49,240 --> 02:28:53,120 BEYOND JUST EASE OF MOVEMENT IN 3311 02:28:53,120 --> 02:28:55,840 OTHER CONCEPTS OF INTEREST SUCH 3312 02:28:55,840 --> 02:28:56,400 AS COMMUNICATION. 3313 02:28:56,400 --> 02:28:58,320 LASTLY, I WANT TO ACKNOWLEDGE 3314 02:28:58,320 --> 02:28:59,520 THAT WE'VE HAD AN EXTRAORDINARY 3315 02:28:59,520 --> 02:29:01,880 AMOUNT OF HELP TO GET US TO THIS 3316 02:29:01,880 --> 02:29:04,600 POINT, ARE GRATEFUL FOR ALL OF 3317 02:29:04,600 --> 02:29:05,120 IT. 3318 02:29:05,120 --> 02:29:12,880 HAPPY TO ANSWER ANY QUESTIONS. 3319 02:29:12,880 --> 02:29:13,240 >>THANK YOU. 3320 02:29:13,240 --> 02:29:15,720 THIS IS GREAT AND SHOWS INTEREST 3321 02:29:15,720 --> 02:29:17,280 AND COMMITMENT. 3322 02:29:17,280 --> 02:29:18,240 WE'RE HAPPY TO TAKE QUESTIONS. 3323 02:29:18,240 --> 02:29:21,960 THERE MIGHT BE ONE IN THE Q&A 3324 02:29:21,960 --> 02:29:22,520 BOX. 3325 02:29:22,520 --> 02:29:24,040 DOUGLAS ALBRECHT ASKING HOW MANY 3326 02:29:24,040 --> 02:29:29,840 OF THE ASSESSMENTS ARE FOR 3327 02:29:29,840 --> 02:29:30,880 NON-AMBULENT PARTICIPANTS? 3328 02:29:30,880 --> 02:29:31,920 >>THE HOPE WE'RE EVENTUALLY 3329 02:29:31,920 --> 02:29:36,440 ABLE TO BUILD A COMPOSITE TO 3330 02:29:36,440 --> 02:29:37,880 SPAN PROGRESSION OF DUCHENNE. 3331 02:29:37,880 --> 02:29:39,760 IN TERMS OF TASKS, ABOUT HALF 3332 02:29:39,760 --> 02:29:44,040 FOR NON-AM BLAH AMBULATORY 3333 02:29:44,040 --> 02:29:44,480 PATIENTS. 3334 02:29:44,480 --> 02:29:47,280 WE NEED TO LOOK AT ARISE DATA, 3335 02:29:47,280 --> 02:29:49,440 WHETHER WE CAN COUNTER THE 3336 02:29:49,440 --> 02:29:51,320 FLOOR-CEILING EFFECTS BY 3337 02:29:51,320 --> 02:29:53,200 DETERMINING WHICH SCORE CARDS -- 3338 02:29:53,200 --> 02:29:54,720 WHICH INABILITIES CORRESPOND TO 3339 02:29:54,720 --> 02:29:57,680 100% SEVERITY ON ONE END AND 3340 02:29:57,680 --> 02:29:59,360 WHICH ABILITY IN DUCHENNE 3341 02:29:59,360 --> 02:30:01,160 CORRESPOND TO ZERO PERCENT ON 3342 02:30:01,160 --> 02:30:09,080 THE OTHER END TO MINIMIZE BURDEN 3343 02:30:09,080 --> 02:30:11,040 ON PATIENTS. 3344 02:30:11,040 --> 02:30:12,720 IF WE'RE SUCCESSFUL AT THAT, AND 3345 02:30:12,720 --> 02:30:15,000 THAT HAPPENS TO BE RELIABLE DATA 3346 02:30:15,000 --> 02:30:16,840 IN OUR ARISE STUDY, THE HOPE IS 3347 02:30:16,840 --> 02:30:19,600 WE CAN BUILD A COMPOSITE ACROSS 3348 02:30:19,600 --> 02:30:22,440 THE ENTIRE SPECTRUM OF DUCHENNE 3349 02:30:22,440 --> 02:30:22,760 PROGRESSION. 3350 02:30:22,760 --> 02:30:26,840 IF NOT WE HAVE BRANCHED 3351 02:30:26,840 --> 02:30:29,640 COMPOSITES FOR AMBULATORY VERSUS 3352 02:30:29,640 --> 02:30:31,680 NON-AMBULATORY, HALF THE TASKS 3353 02:30:31,680 --> 02:30:33,000 ARE TARGETED TO NON-AMBULATORY. 3354 02:30:33,000 --> 02:30:34,920 WE GOT QUITE A BIT OF INPUT 3355 02:30:34,920 --> 02:30:36,680 ABOUT WHAT TASKS TO PUT ON VIDEO 3356 02:30:36,680 --> 02:30:38,640 THAT MADE SENSE FOR THEM, 3357 02:30:38,640 --> 02:30:40,600 CORRESPONDING TO A DEGREE OF 3358 02:30:40,600 --> 02:30:41,400 INDEPENDENCE THAT THEY WERE 3359 02:30:41,400 --> 02:30:44,360 HOPING TO MAINTAIN. 3360 02:30:44,360 --> 02:30:45,360 FOR EXAMPLE, GETTING THE ARM 3361 02:30:45,360 --> 02:30:47,320 BACK UP ON THE WHEELCHAIR ARM 3362 02:30:47,320 --> 02:30:49,120 REST WHICH SEEMS SIMPLE TO US 3363 02:30:49,120 --> 02:30:50,920 BUT FOR THOSE YOUNG MEN WITH 3364 02:30:50,920 --> 02:30:52,440 DUCHENNE THE AMOUNT OF 3365 02:30:52,440 --> 02:30:53,320 COMPENSATION TO GET THAT ARM 3366 02:30:53,320 --> 02:30:56,600 BACK ON IF IT COMES OFF WAS 3367 02:30:56,600 --> 02:30:57,680 REALLY IMPACTFUL BECAUSE THAT 3368 02:30:57,680 --> 02:30:59,760 ARM REST GIVES THEM LEVERAGE TO 3369 02:30:59,760 --> 02:31:02,240 GET THAT ARM HIGHER, LINKED TO 3370 02:31:02,240 --> 02:31:03,920 EATING, GETTING YOUR CLASSES ON 3371 02:31:03,920 --> 02:31:05,680 AND OFF, MAKING SURE THE HAIR 3372 02:31:05,680 --> 02:31:07,320 LOOKS NICE, ALL THESE THINGS THE 3373 02:31:07,320 --> 02:31:10,600 YOUNG MEN CARE ABOUT, 3374 02:31:10,600 --> 02:31:11,800 CORRESPONDING TO A CERTAIN 3375 02:31:11,800 --> 02:31:12,640 DEGREE OF INDEPENDENCE. 3376 02:31:12,640 --> 02:31:14,000 THANKS FOR THE QUESTION. 3377 02:31:14,000 --> 02:31:15,400 >>I SEE ONE FROM MICHELE 3378 02:31:15,400 --> 02:31:15,800 CAMPBELL. 3379 02:31:15,800 --> 02:31:17,760 WOULD YOU LIKE TO ASK YOUR 3380 02:31:17,760 --> 02:31:18,160 QUESTION? 3381 02:31:18,160 --> 02:31:20,760 AND THEN WALTER. 3382 02:31:20,760 --> 02:31:21,560 >>YES. 3383 02:31:21,560 --> 02:31:22,080 THANK YOU. 3384 02:31:22,080 --> 02:31:24,720 MINDY, THANK YOU FOR A GREAT 3385 02:31:24,720 --> 02:31:26,080 PRESENTATION AND GLAD TO SEE 3386 02:31:26,080 --> 02:31:28,200 PROGRESSION ON THIS EFFORT. 3387 02:31:28,200 --> 02:31:29,400 TWO QUESTIONS. 3388 02:31:29,400 --> 02:31:32,680 ONE IS SIMPLE, IF YOU CAN REMIND 3389 02:31:32,680 --> 02:31:36,440 ME TWO YOUR CENTRAL RATERS ARE 3390 02:31:36,440 --> 02:31:39,880 FOR THE VIDEO, AND SECOND MY 3391 02:31:39,880 --> 02:31:41,600 THEME FOR TODAY, MEANINGFUL 3392 02:31:41,600 --> 02:31:41,920 CHANGE. 3393 02:31:41,920 --> 02:31:43,320 IT'S CONVERSATION THAT COMES UP 3394 02:31:43,320 --> 02:31:46,560 ALL THE TIME IN RARE DISEASES, 3395 02:31:46,560 --> 02:31:48,480 SOMETHING WE WANT TO FULLY 3396 02:31:48,480 --> 02:31:50,680 UNDERSTAND WHEN HAVING TO MAKE 3397 02:31:50,680 --> 02:31:53,160 AND INFORM A REGULATORY 3398 02:31:53,160 --> 02:31:53,760 DECISION. 3399 02:31:53,760 --> 02:31:59,320 YOU SAID SOMETHING, HOW THE BOYS 3400 02:31:59,320 --> 02:32:01,400 WITH DUCHENNE HELPED IN 3401 02:32:01,400 --> 02:32:02,800 SELECTING ITEMS WHICH SOUNDS 3402 02:32:02,800 --> 02:32:06,160 LIKE THEY THOUGHT THEY WERE 3403 02:32:06,160 --> 02:32:08,000 MEANINGFUL TO THEY WILL, TO HAVE 3404 02:32:08,000 --> 02:32:09,440 THOSE ABILITIES. 3405 02:32:09,440 --> 02:32:12,080 CAN YOU CONTINUED WITH 3406 02:32:12,080 --> 02:32:14,280 QUALITATIVE WORK ON CLINICALLY 3407 02:32:14,280 --> 02:32:19,320 MEANINGFUL FOR THEM? 3408 02:32:19,320 --> 02:32:23,120 I UNDERSTAND YOUR CLINICIANS ARE 3409 02:32:23,120 --> 02:32:24,760 PROBABLY UNDERSTANDING WHAT THAT 3410 02:32:24,760 --> 02:32:29,280 LOOKS LIKE BUT HAVE YOU 3411 02:32:29,280 --> 02:32:31,440 CONSIDERED SOME TYPE OF 3412 02:32:31,440 --> 02:32:32,800 ASSESSMENT ASKING ABOUT FUNCTION 3413 02:32:32,800 --> 02:32:34,400 AND WHAT NOT, TO HELP CONTINUE I 3414 02:32:34,400 --> 02:32:36,800 GUESS WHAT I LOOK AT THIS, THIS 3415 02:32:36,800 --> 02:32:41,200 IS A STORY WHERE MORE 3416 02:32:41,200 --> 02:32:42,280 INFORMATION IS ACTUALLY BETTER, 3417 02:32:42,280 --> 02:32:45,880 A BETTER STORY, A BETTER LIVED 3418 02:32:45,880 --> 02:32:46,240 EXPERIENCE. 3419 02:32:46,240 --> 02:32:47,640 JUST WONDERING HOW THIS 3420 02:32:47,640 --> 02:32:49,400 PROGRESSED OVER TIME SINCE 3421 02:32:49,400 --> 02:32:51,520 INITIAL WORK, REALLY TRYING TO 3422 02:32:51,520 --> 02:32:53,240 PUT THIS CONCEPT TOGETHER. 3423 02:32:53,240 --> 02:32:54,960 >>YEAH, ABSOLUTELY. 3424 02:32:54,960 --> 02:32:59,040 YOU'LL NOT BE SURPRISED I HAVE 3425 02:32:59,040 --> 02:33:00,360 STRONG OPINIONS. 3426 02:33:00,360 --> 02:33:03,400 FIRST THE CENTRAL RATERS, IN OUR 3427 02:33:03,400 --> 02:33:05,800 PHASE 1 ESTABLISHING THE 3428 02:33:05,800 --> 02:33:09,560 PSYCHOMETRIC PROPERTIES OF THE 3429 02:33:09,560 --> 02:33:11,400 SCORE CARDS WHETHER RELIABILITY 3430 02:33:11,400 --> 02:33:14,600 IS HIGHER IN OUR PHYSICAL 3431 02:33:14,600 --> 02:33:17,320 THERAPISTS WITH SPECIFIC 3432 02:33:17,320 --> 02:33:17,960 DUCHENNE EXPERTISE VERSUS 3433 02:33:17,960 --> 02:33:20,280 GENERALISTS, WE DID NOT SEE A 3434 02:33:20,280 --> 02:33:22,240 DIFFERENCE BETWEEN THE GROUP 3435 02:33:22,240 --> 02:33:27,080 WITH DUCHENNE SPECIFIC EXPERTISE 3436 02:33:27,080 --> 02:33:28,800 AND GENERALISTS. 3437 02:33:28,800 --> 02:33:31,960 WE'RE UTILIZING RATERS THAT PASS 3438 02:33:31,960 --> 02:33:32,600 OUR CERTIFICATION REQUIREMENTS, 3439 02:33:32,600 --> 02:33:35,960 AN EXTENSIVE TRAINING PROGRAM 3440 02:33:35,960 --> 02:33:36,720 AND CERTIFICATION REQUIREMENTS 3441 02:33:36,720 --> 02:33:39,400 TO SERVE AS CENTRAL RATER, 3442 02:33:39,400 --> 02:33:41,960 MINIMUM AMOUNT OF EXPERIENCE 3443 02:33:41,960 --> 02:33:43,000 REQUIRED THAT'S BEEN 3444 02:33:43,000 --> 02:33:45,640 ESTABLISHED, DOES NOT INCLUDE 3445 02:33:45,640 --> 02:33:47,080 DUCHENNE EXPERTISE WHICH WE'RE 3446 02:33:47,080 --> 02:33:48,480 EXCITED ABOUT BECAUSE IT ALLOWS 3447 02:33:48,480 --> 02:33:50,040 US TO EXPAND THE POOL BEYOND A 3448 02:33:50,040 --> 02:33:53,680 GROUP OF P.T.s THAT ARE VERY 3449 02:33:53,680 --> 02:33:54,760 MUCH IN DEMAND, GIVEN THE NUMBER 3450 02:33:54,760 --> 02:33:56,920 OF CLINICAL TRIALS THAT ARE 3451 02:33:56,920 --> 02:33:58,120 PROGRESSING IN DUCHENNE. 3452 02:33:58,120 --> 02:34:01,960 WE ALSO HAVE RECENTLY HIRED A 3453 02:34:01,960 --> 02:34:03,520 RATER MANAGER WHO IS SOLELY 3454 02:34:03,520 --> 02:34:04,480 RESPONSIBLE FOR TRAINING AND 3455 02:34:04,480 --> 02:34:12,360 CERTIFYING THAT GROUP OF PEOPLE, 3456 02:34:12,360 --> 02:34:13,800 SOLE RESPONSIBILITY IS ENSURING 3457 02:34:13,800 --> 02:34:17,800 THAT ACCURACY OF THOSE RATERS. 3458 02:34:17,800 --> 02:34:22,320 WE'RE STILL -- ICCs WERE QUITE 3459 02:34:22,320 --> 02:34:25,480 HIGH, WE'RE STILL UTILIZING A 3460 02:34:25,480 --> 02:34:28,440 PLAN OF HAVING SCORE BY THREE 3461 02:34:28,440 --> 02:34:32,600 ADJUDICATING DIFFERENCES AT THE 3462 02:34:32,600 --> 02:34:34,560 CRITERIA LEVEL SO DISCREPANCIES 3463 02:34:34,560 --> 02:34:36,120 BUILDING AN OVERALL SEVERITY 3464 02:34:36,120 --> 02:34:36,440 PERCENTAGE. 3465 02:34:36,440 --> 02:34:38,080 YOU CAN LET ME KNOW IF THAT 3466 02:34:38,080 --> 02:34:39,480 DIDN'T FULLY ANSWER YOUR 3467 02:34:39,480 --> 02:34:40,680 QUESTION ON RATERS. 3468 02:34:40,680 --> 02:34:42,640 IN TERMS OF MEANINGFUL CHANGE, 3469 02:34:42,640 --> 02:34:45,960 SO, IN TERMS OF MEANINGFUL 3470 02:34:45,960 --> 02:34:48,000 CHANGE, WE UTILIZED PATIENT 3471 02:34:48,000 --> 02:34:49,920 FEEDBACK TO SELECT THE TASKS AND 3472 02:34:49,920 --> 02:34:51,240 THE WAY THE CAPTURE PROCEDURES 3473 02:34:51,240 --> 02:34:54,640 WERE DEVELOPED BECAUSE WE WANTED 3474 02:34:54,640 --> 02:34:57,440 TO MAKE SURE THAT THE TASKS WE 3475 02:34:57,440 --> 02:35:04,680 INCLUDED REPRESENTED A 3476 02:35:04,680 --> 02:35:05,360 MEANINGFUL AMOUNT OF 3477 02:35:05,360 --> 02:35:07,880 INDEPENDENCE, SO LET ME GIVE 3478 02:35:07,880 --> 02:35:09,520 EXAMPLES. 3479 02:35:09,520 --> 02:35:10,360 THE BOYS WITH DUCHENNE 3480 02:35:10,360 --> 02:35:11,920 ARTICULATE THEY NEED TO REACH 3481 02:35:11,920 --> 02:35:14,240 ACROSS THE TABLE TO PULL CELL 3482 02:35:14,240 --> 02:35:15,560 PHONES TOWARD THEM, THEY WANT TO 3483 02:35:15,560 --> 02:35:16,960 STORE THE CELL PHONE SCREEN DOWN 3484 02:35:16,960 --> 02:35:18,280 BECAUSE THEY DIDN'T WANT ANYBODY 3485 02:35:18,280 --> 02:35:19,960 IN THEIR BUSINESS. 3486 02:35:19,960 --> 02:35:21,280 IT'S MEANINGFUL TO THEM, PULLING 3487 02:35:21,280 --> 02:35:23,840 IT TOWARDS THEM AND FLIPPING IT 3488 02:35:23,840 --> 02:35:25,440 WHICH REQUIRES A CERTAIN -- 3489 02:35:25,440 --> 02:35:26,520 LARGER AMOUNT OF MUSCLE FUNCTION 3490 02:35:26,520 --> 02:35:29,480 THAN JUST PULLING IT TOWARDS 3491 02:35:29,480 --> 02:35:29,800 THEM. 3492 02:35:29,800 --> 02:35:32,120 ANOTHER EXAMPLE OF THAT WAS 3493 02:35:32,120 --> 02:35:36,280 GETTING OFF OF THE FLOOR, IN 3494 02:35:36,280 --> 02:35:37,840 DUCHENNE WE UTILIZE THE 3495 02:35:37,840 --> 02:35:39,720 INABILITY TO GET OFF THE FLOOR 3496 02:35:39,720 --> 02:35:40,920 WITHOUT THE ASSISTANCE OF 3497 02:35:40,920 --> 02:35:42,320 FURNITURE AS A DEFINITION OF 3498 02:35:42,320 --> 02:35:44,760 INABILITY IN THIS TASK BECAUSE 3499 02:35:44,760 --> 02:35:48,440 BOYS WITH DUCHENNE ARE 3500 02:35:48,440 --> 02:35:49,960 SUSCEPTIBLE TO FALLS, WHEN 3501 02:35:49,960 --> 02:35:51,400 THERE'S NO FURNITURE AROUND. 3502 02:35:51,400 --> 02:35:53,200 THE MEANINGFULNESS OF THE TASK 3503 02:35:53,200 --> 02:35:55,240 IS ONCE THEY LOSE ABILITY TO DO 3504 02:35:55,240 --> 02:35:56,440 THAT WITHOUT ASSISTANCE OF 3505 02:35:56,440 --> 02:35:57,040 FURNITURE. 3506 02:35:57,040 --> 02:35:59,400 NOW THAT WE'RE WORKING IN PMM, 3507 02:35:59,400 --> 02:36:00,840 THOSE PATIENTS DON'T TAKE THE 3508 02:36:00,840 --> 02:36:03,880 TYPES OF FALLS KIDS WITH 3509 02:36:03,880 --> 02:36:08,280 DUCHENNE DO AND THEY WON'T DO 3510 02:36:08,280 --> 02:36:10,800 THAT UNLESS THERE'S FURNITURE 3511 02:36:10,800 --> 02:36:12,080 AROUND, WE'RE CONSIDERING 3512 02:36:12,080 --> 02:36:14,400 ALLOWED FURNITURE TO GET OFF THE 3513 02:36:14,400 --> 02:36:17,000 FLOOR, SO EVEN SOME LITTLE 3514 02:36:17,000 --> 02:36:18,800 NUANCES, IT'S IMPORTANT TO HAVE 3515 02:36:18,800 --> 02:36:20,800 THEM REFLECTIVE OF REALITY OF 3516 02:36:20,800 --> 02:36:22,160 THAT PARTICULAR DISEASE AREA. 3517 02:36:22,160 --> 02:36:24,600 SO IN TERMS OF THE COMPENSATORY 3518 02:36:24,600 --> 02:36:27,800 PATTERNS, THAT IS A PIECE IN 3519 02:36:27,800 --> 02:36:30,280 WHICH WE ARE NOT ASKING PATIENTS 3520 02:36:30,280 --> 02:36:32,160 ABOUT THE MEANINGFULNESS OF 3521 02:36:32,160 --> 02:36:33,040 THOSE COMPENSATORY ACQUISITIONS, 3522 02:36:33,040 --> 02:36:38,520 AND THE REASON FOR THAT IS 3523 02:36:38,520 --> 02:36:38,880 MULTI-FOLD. 3524 02:36:38,880 --> 02:36:41,840 ONE IS THAT WE ARE QUANTIFYING 3525 02:36:41,840 --> 02:36:43,080 THESE COMPENSATORY MOVEMENT 3526 02:36:43,080 --> 02:36:46,600 PATTERNS BECAUSE THEY ARE TO A 3527 02:36:46,600 --> 02:36:47,440 CERTAIN EXTENT SUBCONSCIOUSLY 3528 02:36:47,440 --> 02:36:48,920 ACQUIRED. 3529 02:36:48,920 --> 02:36:52,680 WE WANTED TO QUANTIFY SOMETHING, 3530 02:36:52,680 --> 02:36:54,520 BOYS WITH DUCHENNE AREN'T ALWAYS 3531 02:36:54,520 --> 02:36:56,360 AWARE THEY ARE ACQUIRING 3532 02:36:56,360 --> 02:36:57,240 COMPENSATIONS, THEY ARE DOING 3533 02:36:57,240 --> 02:36:59,280 WHAT THEY NEED TO DO TO MAINTAIN 3534 02:36:59,280 --> 02:37:00,640 INDEPENDENCE THROUGHOUT THEIR 3535 02:37:00,640 --> 02:37:01,000 DAY. 3536 02:37:01,000 --> 02:37:03,280 THAT'S WHAT MADE IT AN 3537 02:37:03,280 --> 02:37:05,200 ATTRACTIVE TARGET TO QUANTIFY, 3538 02:37:05,200 --> 02:37:06,800 OBSERVERS SEE THE BOYS MOVING 3539 02:37:06,800 --> 02:37:13,560 AROUND THAT ARE VERY AWARE OF 3540 02:37:13,560 --> 02:37:14,960 COMPENSATORY ACQUISITIONS. 3541 02:37:14,960 --> 02:37:16,080 THE BOYS DON'T NOTICE THEM 3542 02:37:16,080 --> 02:37:18,080 BECAUSE IT'S A COPING MECHANISM. 3543 02:37:18,080 --> 02:37:20,480 THEY DON'T WANT TO THINK ABOUT 3544 02:37:20,480 --> 02:37:22,520 THE ACQUISITION OF THESE 3545 02:37:22,520 --> 02:37:23,720 COMPENSATIONS BECAUSE IT PUTS IT 3546 02:37:23,720 --> 02:37:25,960 UP IN THEIR FACE, THE DEGREE OF 3547 02:37:25,960 --> 02:37:29,400 PROGRESSION THAT THEY ARE GOING 3548 02:37:29,400 --> 02:37:29,880 THROUGH. 3549 02:37:29,880 --> 02:37:31,160 LIKE I MENTIONED BEFORE, IT'S 3550 02:37:31,160 --> 02:37:34,360 TEMPTING TO THINK IF WE SPEAK TO 3551 02:37:34,360 --> 02:37:35,640 PATIENTS, WE'VE CHECKED THAT 3552 02:37:35,640 --> 02:37:37,520 BOX, WE'VE SPOKEN TO THE 3553 02:37:37,520 --> 02:37:40,480 PATIENT, BUT YOU'RE NOT GOING TO 3554 02:37:40,480 --> 02:37:42,120 GET, LIKE I MENTIONED, THE SAME 3555 02:37:42,120 --> 02:37:43,760 INFORMATION FROM A BOY WHO KNOWS 3556 02:37:43,760 --> 02:37:45,080 WHAT'S GOING TO HAPPEN TO HIM AS 3557 02:37:45,080 --> 02:37:46,640 YOU ARE FROM A BOY WHO DOESN'T 3558 02:37:46,640 --> 02:37:49,920 KNOW WHAT'S GOING TO HAPPEN TO 3559 02:37:49,920 --> 02:37:50,080 HIM. 3560 02:37:50,080 --> 02:37:54,160 AND SO EVEN THAT LEVEL OF 3561 02:37:54,160 --> 02:37:54,840 AWARENESS ABOUT THEIR 3562 02:37:54,840 --> 02:37:57,360 PROGRESSION AND PROGNOSIS IS A 3563 02:37:57,360 --> 02:37:59,760 HUGE CONFOUNDER, WHEN YOU'RE 3564 02:37:59,760 --> 02:38:01,080 TRYING TO QUANTIFY 3565 02:38:01,080 --> 02:38:03,120 MEANINGFULNESS FROM THE 3566 02:38:03,120 --> 02:38:03,800 PATIENT'S PERSPECTIVE. 3567 02:38:03,800 --> 02:38:07,080 AND SO THAT WAS ONE OF THE 3568 02:38:07,080 --> 02:38:11,360 ISSUES WE WERE TRYING TO -- 3569 02:38:11,360 --> 02:38:13,320 WELL, THERE WERE LARGER REASONS 3570 02:38:13,320 --> 02:38:16,040 WE CHOSE P.T.s AS OUR 3571 02:38:16,040 --> 02:38:17,040 REPORTERS OF COMPENSATORY 3572 02:38:17,040 --> 02:38:17,920 ACQUISITION BUT THOSE ARE A FEW. 3573 02:38:17,920 --> 02:38:20,320 IN THE INTEREST OF TIME I'LL END 3574 02:38:20,320 --> 02:38:20,880 WITH THAT. 3575 02:38:20,880 --> 02:38:22,400 >>MINDY, YOU'VE GOT A COUPLE 3576 02:38:22,400 --> 02:38:24,160 MORE QUESTIONS. 3577 02:38:24,160 --> 02:38:26,120 NOW THREE MORE. 3578 02:38:26,120 --> 02:38:27,000 WALTER, JACKIE, DAN. 3579 02:38:27,000 --> 02:38:32,360 WE'LL MOVE TO THE NEXT SPEAKER. 3580 02:38:32,360 --> 02:38:36,720 >>A GENERAL COMMENT AND 3581 02:38:36,720 --> 02:38:37,560 QUESTION. 3582 02:38:37,560 --> 02:38:39,240 GENERAL COMMENT THAT GOOD NEWS 3583 02:38:39,240 --> 02:38:41,480 IN OUR AREAS THERE'S MORE AND 3584 02:38:41,480 --> 02:38:46,600 MORE TARGETS COMING TO THE 3585 02:38:46,600 --> 02:38:47,920 FOREFRONT WHICH PUTS PREMIUM 3586 02:38:47,920 --> 02:38:49,880 BEING ABLE TO DETECT WHETHER A 3587 02:38:49,880 --> 02:38:51,640 THERAPY AIMED AT CERTAIN TARGET 3588 02:38:51,640 --> 02:38:55,000 IS ACTUALLY HAVING AN EFFECT IN 3589 02:38:55,000 --> 02:38:57,080 THE KIDS. 3590 02:38:57,080 --> 02:39:00,280 SO THE BIOMARKER IN THAT 3591 02:39:00,280 --> 02:39:03,240 SITUATION, THE KEY FEATURE IS 3592 02:39:03,240 --> 02:39:06,080 KNOWING HOW SOON DOES YOUR 3593 02:39:06,080 --> 02:39:15,800 MEASURE GET YOU A STATISTICALLY 3594 02:39:15,800 --> 02:39:17,000 SIGNIFICANT INDICATOR OF 3595 02:39:17,000 --> 02:39:21,240 WORSENING OR WHAT TIME FRAME TO 3596 02:39:21,240 --> 02:39:24,760 PREDICT A SLOPE, TO PREDICT IF 3597 02:39:24,760 --> 02:39:26,080 THEY DON'T INTERVENE, ANY 3598 02:39:26,080 --> 02:39:29,080 LONGITUDINAL INFO? 3599 02:39:29,080 --> 02:39:30,800 >>YEAH, WE DO HAVE A SET OF 3600 02:39:30,800 --> 02:39:32,800 STUDY RESULTS FROM INITIAL 3601 02:39:32,800 --> 02:39:36,160 SOURCE MATERIAL STUDY, 30 WEEKS 3602 02:39:36,160 --> 02:39:36,800 APART, SIGNIFICANT COMPENSATORY 3603 02:39:36,800 --> 02:39:39,680 ACQUISITION OVER THE 30 WEEKS. 3604 02:39:39,680 --> 02:39:41,200 AND SO THIS NEXT STUDY THAT 3605 02:39:41,200 --> 02:39:43,800 WE'RE ABOUT TO GET STARTED WITH 3606 02:39:43,800 --> 02:39:47,200 IS A 2-YEAR STUDY, IT WILL 3607 02:39:47,200 --> 02:39:48,200 CAPTURE DUCHENNE VIDEO 3608 02:39:48,200 --> 02:39:52,680 ASSESSMENT EVERY SIX MONTHS, AND 3609 02:39:52,680 --> 02:39:56,000 WE'LL DO STANDARD -- GOLD 3610 02:39:56,000 --> 02:39:57,320 STANDARD ASSESSMENT AT BASELINE, 3611 02:39:57,320 --> 02:39:59,920 12 AND 24 MONTHS, WE'LL BE ABLE 3612 02:39:59,920 --> 02:40:03,880 TO SEE HOW THE DBA MOVES 3613 02:40:03,880 --> 02:40:04,600 ALONGSIDE GOLD STANDARD 3614 02:40:04,600 --> 02:40:06,280 ASSESSMENTS TO GET INFORMATION 3615 02:40:06,280 --> 02:40:07,320 ABOUT ABILITY TO DETECT 3616 02:40:07,320 --> 02:40:09,560 LONGITUDINAL CHANGE ACROSS A 3617 02:40:09,560 --> 02:40:10,880 LARGER PATIENT POPULATION, THE 3618 02:40:10,880 --> 02:40:13,200 ORIGINAL SOURCE MATERIAL STUDY 3619 02:40:13,200 --> 02:40:16,680 WAS IN 60 PATIENTS BUT WE DID 3620 02:40:16,680 --> 02:40:17,880 SEE SIGNIFICANT COMPENSATORY 3621 02:40:17,880 --> 02:40:19,400 ACQUISITION DEPENDING ON AGE 3622 02:40:19,400 --> 02:40:21,040 GROUP, SOME ARE MORE STABLE THAN 3623 02:40:21,040 --> 02:40:23,680 OTHERS BUT WE SAW COMPENSATORY 3624 02:40:23,680 --> 02:40:24,360 ACQUISITION WITHIN THAT 30 WEEKS 3625 02:40:24,360 --> 02:40:28,160 FOR A LARGE AMOUNT OF THOSE 3626 02:40:28,160 --> 02:40:28,440 PATIENTS. 3627 02:40:28,440 --> 02:40:29,360 >>THANK YOU. 3628 02:40:29,360 --> 02:40:31,240 >>THANK YOU. 3629 02:40:31,240 --> 02:40:32,880 >>JACKIE, YOU'RE NEXT. 3630 02:40:32,880 --> 02:40:35,840 >>THANK YOU, MINDY, FOR A GREAT 3631 02:40:35,840 --> 02:40:36,280 PRESENTATION. 3632 02:40:36,280 --> 02:40:41,960 YOU SHOULD BE COMMENDED ON YOUR 3633 02:40:41,960 --> 02:40:44,280 ITERATIVE RIGOROUS PROCESS. 3634 02:40:44,280 --> 02:40:47,560 MY QUESTION IS FORWARD LOOKING, 3635 02:40:47,560 --> 02:40:52,480 IN ADDITION TO COMPENSATION AS 3636 02:40:52,480 --> 02:40:56,000 PART OF PROGRESSION, ASPECT OF 3637 02:40:56,000 --> 02:40:57,080 EFFORT OR FATIGUE, FATIGABILITY, 3638 02:40:57,080 --> 02:40:58,280 DO YOU ENVISION A WAY THAT 3639 02:40:58,280 --> 02:41:03,320 YOU'LL BE ABLE TO CAPTURE THAT 3640 02:41:03,320 --> 02:41:06,280 QUALITATIVELY, QUANTITATIVELY, 3641 02:41:06,280 --> 02:41:09,840 AS A COMPONENT OF THE PRESENCE 3642 02:41:09,840 --> 02:41:10,920 OF COMPENSATIONS. 3643 02:41:10,920 --> 02:41:13,400 >>THAT'S A REALLY INTERESTING 3644 02:41:13,400 --> 02:41:13,720 QUESTION. 3645 02:41:13,720 --> 02:41:14,760 IN DUCHENNE, WE'RE TRYING TO 3646 02:41:14,760 --> 02:41:18,560 KEEP IT AS MUCH OF A REFLECTION 3647 02:41:18,560 --> 02:41:21,480 OF EVERYDAY LIFE AS POSSIBLE. 3648 02:41:21,480 --> 02:41:24,320 AND SO IN DUCHENNE YOU WOULDN'T 3649 02:41:24,320 --> 02:41:27,200 GET OFF THE FLOOR THREE TIMES IN 3650 02:41:27,200 --> 02:41:27,480 SUCCESSION. 3651 02:41:27,480 --> 02:41:30,440 AS PARENTS I THINK WHAT WE'VE 3652 02:41:30,440 --> 02:41:33,760 NOTICED IS THAT THE BOYS DON'T 3653 02:41:33,760 --> 02:41:35,040 ADOPT NEW COMPENSATORY PATTERNS 3654 02:41:35,040 --> 02:41:36,240 UNTIL THEY NEED TO, IF THAT 3655 02:41:36,240 --> 02:41:37,040 MAKES SENSE. 3656 02:41:37,040 --> 02:41:40,000 YOU DON'T SEE A LOT OF INFLUENCE 3657 02:41:40,000 --> 02:41:41,280 BUT THAT'S ANECDOTAL. 3658 02:41:41,280 --> 02:41:49,200 WE DO HAVE A TEST-RETEST AS LACE 3659 02:41:49,200 --> 02:41:53,560 -- BASELINE. 3660 02:41:53,560 --> 02:41:54,920 THAT'S OUTSIDE. 3661 02:41:54,920 --> 02:41:56,560 WE'RE LOOKING AT DIFFICULTY, 3662 02:41:56,560 --> 02:41:57,880 EASE OF MOVEMENT, AS PATIENTS 3663 02:41:57,880 --> 02:42:01,160 MOVE ABOUT THEIR DAILY LIVES. 3664 02:42:01,160 --> 02:42:02,200 SO, FATIGUE ISN'T AS MUCH A 3665 02:42:02,200 --> 02:42:03,560 COMPONENT OF WHAT WE'RE TRYING 3666 02:42:03,560 --> 02:42:04,000 TO MEASURE. 3667 02:42:04,000 --> 02:42:06,000 HOW HARD IS IT FOR THEM TO DO 3668 02:42:06,000 --> 02:42:07,240 THOSE ACTIVITIES, THAT THEY ARE 3669 02:42:07,240 --> 02:42:08,400 REQUIRED TO DO THROUGHOUT THEIR 3670 02:42:08,400 --> 02:42:09,240 DAILY LIVES. 3671 02:42:09,240 --> 02:42:10,920 IT'S A GREAT QUESTION THOUGH AND 3672 02:42:10,920 --> 02:42:16,240 SOMETHING WE THOUGHT ABOUT A LOT 3673 02:42:16,240 --> 02:42:17,480 WITH CARDIOMYOPATHY, BASED ON 3674 02:42:17,480 --> 02:42:18,280 TALKING TO THOSE PATIENTS 3675 02:42:18,280 --> 02:42:21,960 THERE'S MUCH MORE OF AN 3676 02:42:21,960 --> 02:42:23,600 INFLUENCE ON FATIGABILITY AND 3677 02:42:23,600 --> 02:42:34,160 MYOPATHY IN TERMS OF THE NATURE 3678 02:42:34,560 --> 02:42:35,680 OF COMPENSATORY PATTERNS. 3679 02:42:35,680 --> 02:42:38,200 DOING A THREE-PEAT TO LOOK AT 3680 02:42:38,200 --> 02:42:38,640 THAT SPECIFICALLY. 3681 02:42:38,640 --> 02:42:39,440 IT WASN'T SOMETHING ARTICULATED 3682 02:42:39,440 --> 02:42:42,600 BY FAMILIES TOO MUCH INITIALLY 3683 02:42:42,600 --> 02:42:43,040 IN DUCHENNE. 3684 02:42:43,040 --> 02:42:45,080 >>I WAS THINKING TOO OF THE 3685 02:42:45,080 --> 02:42:48,640 MORE TIME IT TAKES TO EVEN DO 3686 02:42:48,640 --> 02:42:52,000 ONE ACTIVITY. 3687 02:42:52,000 --> 02:42:52,400 >>INTERESTING. 3688 02:42:52,400 --> 02:42:52,960 YEAH, THAT'S AN INTERESTING 3689 02:42:52,960 --> 02:42:55,720 THING TO TAKE A LOOK AT WITH 3690 02:42:55,720 --> 02:42:56,600 ARISE DATA. 3691 02:42:56,600 --> 02:42:59,320 WITH VIDEO RECORDING WE CAN 3692 02:42:59,320 --> 02:43:01,120 PRETTY ACCURATELY PINPOINT TIME 3693 02:43:01,120 --> 02:43:11,600 OF THOSE ASSESSMENTS, THAT'S A 3694 02:43:12,640 --> 02:43:13,600 GREAT ONE. 3695 02:43:13,600 --> 02:43:17,960 >>BRIEF QUESTION OR COMMENT, 3696 02:43:17,960 --> 02:43:23,640 DAN PEREZ. 3697 02:43:23,640 --> 02:43:34,000 >>JUST A COMMENT. 3698 02:43:39,240 --> 02:43:42,880 CONCEPTUALIZING THIS CLINICAL 3699 02:43:42,880 --> 02:43:51,320 TRIAL, COMPENSATORY MECHANISM, 3700 02:43:51,320 --> 02:43:52,200 SOMETHING VERY INTERESTING, 3701 02:43:52,200 --> 02:43:55,360 DIFFERENT PROBLEMS THROUGHOUT 3702 02:43:55,360 --> 02:43:57,240 THE LIFETIME, I WONDERED WHETHER 3703 02:43:57,240 --> 02:44:07,000 OR NOT THIS IS A TOOL THAT COULD 3704 02:44:07,000 --> 02:44:17,120 BE USED, PERCEPTION OF DISEASE 3705 02:44:17,120 --> 02:44:18,680 PROGRESSION, SOMEBODY SAYS I 3706 02:44:18,680 --> 02:44:29,200 HAVE A LOT OF DECLINE ALL OF A 3707 02:44:29,880 --> 02:44:35,200 SUDDEN, THE STEPS TO NO LONGER 3708 02:44:35,200 --> 02:44:39,280 COMPENSATE, A GUIDE WITH NATURAL 3709 02:44:39,280 --> 02:44:42,960 HISTORY STUDIES IN TERMS OF 3710 02:44:42,960 --> 02:44:49,680 DYNAMIC TOOL IN THE CLINICAL 3711 02:44:49,680 --> 02:44:54,400 TRIALS PROCESS, CLINICIAN AND 3712 02:44:54,400 --> 02:44:55,600 RESEARCHER TO A PARTICULAR 3713 02:44:55,600 --> 02:45:03,120 ACTION AS OPPOSED TO TRYING TO 3714 02:45:03,120 --> 02:45:13,640 QUANTIFY THE CHANGE IN A DRUG. 3715 02:45:22,960 --> 02:45:28,040 MRI-GUIDED BIOPSIES, SIGNAL WITH 3716 02:45:28,040 --> 02:45:36,760 DUX4, A TOOL TO GUIDE TO 3717 02:45:36,760 --> 02:45:37,440 UNDERSTANDING DISEASE 3718 02:45:37,440 --> 02:45:38,200 PROGRESSION. 3719 02:45:38,200 --> 02:45:39,400 >>YEAH, I APPRECIATE THAT. 3720 02:45:39,400 --> 02:45:41,520 I'M BREAKING WHAT YOU SAID INTO 3721 02:45:41,520 --> 02:45:43,480 A NUMBER OF DIFFERENT POINTS. 3722 02:45:43,480 --> 02:45:46,280 FIRST OF ALL, UTILIZING THIS IN 3723 02:45:46,280 --> 02:45:47,560 A CLINICAL TRIAL SETTING, IT IS 3724 02:45:47,560 --> 02:45:48,920 LIVE IN SEVERAL CLINICAL TRIALS 3725 02:45:48,920 --> 02:45:51,200 AS A SECONDARY AND EXPLORATORY 3726 02:45:51,200 --> 02:45:53,600 MEASURE, SO WHAT WE'RE TAKING A 3727 02:45:53,600 --> 02:45:56,120 LOOK AT IS WE'RE LOOKING AT THE 3728 02:45:56,120 --> 02:45:58,320 TRAJECTORY OF INDIVIDUAL 3729 02:45:58,320 --> 02:46:00,080 PATIENTS BY COMPARING CHANGE IN 3730 02:46:00,080 --> 02:46:03,440 ONE PATIENT OVER TIME, YOU KNOW, 3731 02:46:03,440 --> 02:46:05,120 COMPOSITE ACROSS THE GROUP TO A 3732 02:46:05,120 --> 02:46:06,640 TREATED ARM. 3733 02:46:06,640 --> 02:46:08,920 WE'RE NOT LOOKING AT A 3734 02:46:08,920 --> 02:46:09,680 CROSS-SECTIONAL COMPARISON 3735 02:46:09,680 --> 02:46:11,880 BETWEEN TREATED AND PLACEBO AND 3736 02:46:11,880 --> 02:46:12,760 THEN ANOTHER CROSS-SECTIONAL 3737 02:46:12,760 --> 02:46:13,080 COMPARISON. 3738 02:46:13,080 --> 02:46:15,280 WE'RE LOOKING AT CHANGE WITHIN 3739 02:46:15,280 --> 02:46:16,720 AN ARM OVER TIME BECAUSE WE WANT 3740 02:46:16,720 --> 02:46:21,560 TO SEE IF RATE OF CHANGE IN THIS 3741 02:46:21,560 --> 02:46:22,600 PROCESS IS SIGNIFICANTLY 3742 02:46:22,600 --> 02:46:24,320 DIFFERENT BETWEEN THE TREATED 3743 02:46:24,320 --> 02:46:25,800 ARM AND PLACEBO ARM. 3744 02:46:25,800 --> 02:46:27,560 SO THAT IS IN USE IN CLINICAL 3745 02:46:27,560 --> 02:46:30,200 TRIALS ALREADY BUT I THINK WHAT 3746 02:46:30,200 --> 02:46:32,920 YOU'RE TALKING ABOUT IS 3747 02:46:32,920 --> 02:46:36,320 POTENTIALLY ABILITY TO VALIDATE 3748 02:46:36,320 --> 02:46:37,720 THE RELIABILITY OF 3749 02:46:37,720 --> 02:46:38,240 PATIENT-REPORTED DISEASE 3750 02:46:38,240 --> 02:46:39,360 PROGRESSION AGAINST WHAT'S SEEN 3751 02:46:39,360 --> 02:46:43,000 ON VIDEO, IF I'M INTERPRETING 3752 02:46:43,000 --> 02:46:43,880 YOUR ANSWER CORRECTLY, WHICH 3753 02:46:43,880 --> 02:46:46,600 WOULD BE AN INTERESTING PROJECT 3754 02:46:46,600 --> 02:46:48,360 AND ADDRESSES CONCERNS I 3755 02:46:48,360 --> 02:46:52,960 ARTICULATED ABOUT RELIABILITY 3756 02:46:52,960 --> 02:46:54,160 OF PATIENT-REPORTED OUTCOMES. 3757 02:46:54,160 --> 02:46:54,680 A REALLY GREAT IDEA. 3758 02:46:54,680 --> 02:46:56,760 I APPRECIATE THAT VERY MUCH. 3759 02:46:56,760 --> 02:47:00,160 >>THANK YOU, EVERYONE, FOR YOUR 3760 02:47:00,160 --> 02:47:07,000 TREMENDOUS ENGAGEMENT FOLLOWING 3761 02:47:07,000 --> 02:47:08,480 THIS PRESENTATION. 3762 02:47:08,480 --> 02:47:12,080 FINAL PRESENTER, DR. DAMIANO 3763 02:47:12,080 --> 02:47:13,280 ZONOTTO, ASSOCIATE PROFESSOR IN 3764 02:47:13,280 --> 02:47:15,920 THE DEPARTMENT OF MECHANICAL 3765 02:47:15,920 --> 02:47:18,680 ENGINEERING, STEVENS INSTITUTE 3766 02:47:18,680 --> 02:47:20,920 OF TECHNOLOGY IN HOBOKEN, NEW 3767 02:47:20,920 --> 02:47:30,000 JERSEY, HAS BEEN COLLABORATING 3768 02:47:30,000 --> 02:47:34,880 WITH DR. JACKIE MONTES, 3769 02:47:34,880 --> 02:47:40,800 ASSOCIATE PROFESSOR AT COLUMBIA, 3770 02:47:40,800 --> 02:47:42,640 WEARABLE TECHNOLOGY FOR GAIT 3771 02:47:42,640 --> 02:47:44,840 ANALYSIS, A.I. ENABLED SHOE 3772 02:47:44,840 --> 02:47:47,560 INSOLES TO ASSESS WALKING 3773 02:47:47,560 --> 02:47:50,960 FUNCTION IN SMA AND DMD. 3774 02:47:50,960 --> 02:47:56,000 OVER TO YOU, DR. ZANOTTO AND 3775 02:47:56,000 --> 02:47:56,280 MONTES. 3776 02:47:56,280 --> 02:47:57,640 >>I'M GOING TO SUMMARIZE SOME 3777 02:47:57,640 --> 02:48:01,160 OF THE RECENT STUDIES THAT WE 3778 02:48:01,160 --> 02:48:03,880 CONDUCTED IN COLLABORATION WITH 3779 02:48:03,880 --> 02:48:06,760 JACKIE MONTES AND HER GROUP AT 3780 02:48:06,760 --> 02:48:08,480 COLUMBIA, HOW TO USE SMART 3781 02:48:08,480 --> 02:48:10,920 INSOLES TO ASSESS WALKING 3782 02:48:10,920 --> 02:48:13,840 FUNCTION IN SMA AND DMD IN 3783 02:48:13,840 --> 02:48:18,120 CONTROLLED ENVIRONMENTS AND IN 3784 02:48:18,120 --> 02:48:19,680 REAL LIFE. 3785 02:48:19,680 --> 02:48:22,280 DISEASE-MODIFYING TREATMENTS FOR 3786 02:48:22,280 --> 02:48:24,040 SEVERAL MONTHS, CLINICIANS, AND 3787 02:48:24,040 --> 02:48:25,640 OTHERS, OTHER TREATMENTS 3788 02:48:25,640 --> 02:48:28,400 CURRENTLY UNDER DEVELOPMENT, 3789 02:48:28,400 --> 02:48:33,760 THERE IS A GROWING NEED TO 3790 02:48:33,760 --> 02:48:37,600 INTRODUCE NEW WAYS, NEW TOOLS TO 3791 02:48:37,600 --> 02:48:39,480 ASSESS SAFETY AND EFFECTIVENESS 3792 02:48:39,480 --> 02:48:44,960 IN A MORE SUFFICIENT, SCALABLE, 3793 02:48:44,960 --> 02:48:47,760 AFFORDABLE WAY, MEASURING GAIT 3794 02:48:47,760 --> 02:48:49,440 AND BALANCE, CAPTURING CAPACITY, 3795 02:48:49,440 --> 02:48:55,200 IN CONTROLLED ENVIRONMENTS, 3796 02:48:55,200 --> 02:48:57,680 PROVIDE SNAPSHOT, MIGHT NOT BE 3797 02:48:57,680 --> 02:48:59,280 REPRESENTATIVE OF REAL LIFE. 3798 02:48:59,280 --> 02:49:06,040 ONE HAS POTENTIAL TO CAPTURE 3799 02:49:06,040 --> 02:49:09,000 DMOs, MATRIX THAT RELATE TO 3800 02:49:09,000 --> 02:49:10,400 REAL LIFE PERFORMANCE. 3801 02:49:10,400 --> 02:49:11,960 AS WAS MENTIONED AT THE 3802 02:49:11,960 --> 02:49:14,480 BEGINNING, THOSE WEARABLES CAN 3803 02:49:14,480 --> 02:49:16,000 ALSO REDUCE COST OF FUTURE 3804 02:49:16,000 --> 02:49:19,040 CLINICAL TRIALS, FOR EXAMPLE BY 3805 02:49:19,040 --> 02:49:23,440 REDUCING NUMBER OF IN-PERSON 3806 02:49:23,440 --> 02:49:25,880 VISITS REQUIRED BY STUDY 3807 02:49:25,880 --> 02:49:26,800 PARTICIPANT, POTENTIALLY 3808 02:49:26,800 --> 02:49:27,920 REDUCING TARGET ENROLLMENT AND 3809 02:49:27,920 --> 02:49:30,040 DURATION OF FUTURE TRIALS 3810 02:49:30,040 --> 02:49:31,760 THROUGH INTRODUCTION OF MORE 3811 02:49:31,760 --> 02:49:39,000 SENSITIVE AND STABLE OUTCOME 3812 02:49:39,000 --> 02:49:39,560 METRICS. 3813 02:49:39,560 --> 02:49:41,080 CONSUMER GRADE WEARABLES ARE 3814 02:49:41,080 --> 02:49:42,520 INCREASINGLY BEING USED IN 3815 02:49:42,520 --> 02:49:44,000 CLINICAL TRIALS, BUT THOSE 3816 02:49:44,000 --> 02:49:47,760 DEVICES IN TERMS OF MOBILITY 3817 02:49:47,760 --> 02:49:51,680 METRICS CAN PROVIDE A VERY SMALL 3818 02:49:51,680 --> 02:49:53,880 SET OF VOLUME METRICS, DISTANCE 3819 02:49:53,880 --> 02:49:56,600 WALKED PER DAY, STEP COUNT, 3820 02:49:56,600 --> 02:49:58,240 QUITE INACCURATE EVEN WHEN THEY 3821 02:49:58,240 --> 02:50:01,960 ARE USED WITH HEALTHY 3822 02:50:01,960 --> 02:50:02,440 INDIVIDUALS. 3823 02:50:02,440 --> 02:50:04,600 AND WE KNOW THAT ACCURACY IS A 3824 02:50:04,600 --> 02:50:07,880 MAJOR ISSUE FOR CLINICAL 3825 02:50:07,880 --> 02:50:13,680 RESEARCH, DOESN'T ALLOW US TO 3826 02:50:13,680 --> 02:50:14,640 MEASURE CLINICALLY MEANINGFUL 3827 02:50:14,640 --> 02:50:17,520 CHANGES IN MOTOR OUTCOME THAT 3828 02:50:17,520 --> 02:50:21,320 CAN REFLECT RESPONSIVENESS TO 3829 02:50:21,320 --> 02:50:22,160 TREATMENT. 3830 02:50:22,160 --> 02:50:23,120 THE RESEARCH-GRADE ACTIVITY 3831 02:50:23,120 --> 02:50:33,680 TRACKERS ARE MORE ACCURATE THAN 3832 02:50:41,560 --> 02:50:42,360 CONSUMER-GRADE. 3833 02:50:42,360 --> 02:50:44,440 ON THE OTHER HAND, MULTI-SENSOR 3834 02:50:44,440 --> 02:50:51,760 DEVICES CAN PROVIDE A LARGE 3835 02:50:51,760 --> 02:50:55,480 NUMBER OF PARAMETERS, THEY ARE 3836 02:50:55,480 --> 02:50:57,680 CUMBERSOME AND EXPENSIVE, NOT 3837 02:50:57,680 --> 02:50:59,000 REALLY DEPLOYABLE AND SCALABLE 3838 02:50:59,000 --> 02:51:09,480 AS A SOLUTION TO MEASURE REAL 3839 02:51:11,120 --> 02:51:13,320 LIFE GAIT. 3840 02:51:13,320 --> 02:51:20,400 THEY CAN NOT MEASURE PARAMETERS, 3841 02:51:20,400 --> 02:51:21,640 FOOT PRESSURE TRAJECTORY FOR 3842 02:51:21,640 --> 02:51:23,720 EXAMPLE, PARAMETERS ARE 3843 02:51:23,720 --> 02:51:26,120 IMPORTANT IN ASSESSING THE 3844 02:51:26,120 --> 02:51:28,080 NON-BALANCE. 3845 02:51:28,080 --> 02:51:31,360 WE BELIEVE AMONG TECHNOLOGIES 3846 02:51:31,360 --> 02:51:32,880 WELL POSITIONED TO MEASURE GAIT 3847 02:51:32,880 --> 02:51:35,080 IN REAL LIFE BECAUSE THEY SHOW 3848 02:51:35,080 --> 02:51:39,320 THE SAME ADVANTAGES OF FOOT AND 3849 02:51:39,320 --> 02:51:43,280 ANKLE MONITORS, IN ADDITION TO 3850 02:51:43,280 --> 02:51:44,400 MEASURE INTERLIMB PARAMETERS AND 3851 02:51:44,400 --> 02:51:45,560 KINETIC PARAMETERS. 3852 02:51:45,560 --> 02:51:47,800 IF WE LOOK AT ALGORITHMS THAT 3853 02:51:47,800 --> 02:51:51,840 ARE IMPLEMENTED IN MOST OF THE 3854 02:51:51,840 --> 02:51:52,400 COMMERCIALLY AVAILABLE 3855 02:51:52,400 --> 02:51:54,240 INSTRUMENT INSOLES ON THE 3856 02:51:54,240 --> 02:51:56,640 MARKET, THEY ONLY ALLOW FOR 3857 02:51:56,640 --> 02:51:57,080 MODERATE ACCURACY. 3858 02:51:57,080 --> 02:52:04,080 OUR WORK IN THE LAST FEW YEARS 3859 02:52:04,080 --> 02:52:09,320 HAS FOCUSED ALLOWED US TO HAVE 3860 02:52:09,320 --> 02:52:11,760 MORE ACCURATE METRICS. 3861 02:52:11,760 --> 02:52:14,280 THIS IS HOW OUR CURRENT DEVICE 3862 02:52:14,280 --> 02:52:21,720 LOOKS LIKE, A PAIR OF INSOLES 3863 02:52:21,720 --> 02:52:22,480 INSTRUMENTED WITH SENSORS, 3864 02:52:22,480 --> 02:52:28,280 SIMILAR TO REGULAR PAIR OF 3865 02:52:28,280 --> 02:52:30,400 INSOLES, LIGHT WEIGHT AND CAN 3866 02:52:30,400 --> 02:52:37,640 COLLECT DATA CONTINUOUSLY UP TO 3867 02:52:37,640 --> 02:52:38,400 FIVE HOURS. 3868 02:52:38,400 --> 02:52:43,800 APPROACH LIES IN MACHINE 3869 02:52:43,800 --> 02:52:46,320 LEARNING MODELS, TO LEVERAGE 3870 02:52:46,320 --> 02:52:48,840 WHAT WAS PREVIOUSLY COLLECTED, 3871 02:52:48,840 --> 02:52:54,000 ALLOWS US TO OBTAIN RELIABLE 3872 02:52:54,000 --> 02:52:56,880 SPATIAL, TEMPORAL METRICS, 3873 02:52:56,880 --> 02:52:59,680 VOLUME DMOs. 3874 02:52:59,680 --> 02:53:02,600 THIS IS THE PARAMETERS WITH 3875 02:53:02,600 --> 02:53:05,560 INSOLES, BOTH STRIDE BY STRIDE 3876 02:53:05,560 --> 02:53:07,560 DMOs THAT PERTAINS TO 3877 02:53:07,560 --> 02:53:11,400 DIFFERENT DOMAINS, AND ALSO MORE 3878 02:53:11,400 --> 02:53:12,720 STANDARD VOLUME DMOs, NUMBER 3879 02:53:12,720 --> 02:53:14,120 OF STEPS PER DAY, DISTANCE 3880 02:53:14,120 --> 02:53:16,880 COVERED PER HOUR, PER DAY, SO 3881 02:53:16,880 --> 02:53:18,640 FORTH. 3882 02:53:18,640 --> 02:53:21,880 WE'RE USING THIS TECHNOLOGY IN 3883 02:53:21,880 --> 02:53:22,880 COLLABORATIVE STUDIES WITH 3884 02:53:22,880 --> 02:53:26,600 SEVERAL RESEARCH HOSPITALS IN 3885 02:53:26,600 --> 02:53:26,920 THE NATION. 3886 02:53:26,920 --> 02:53:29,120 SO, I'M GOING TO TALK ABOUT SOME 3887 02:53:29,120 --> 02:53:34,560 RECENT STUDIES WE CARRY OUT TO 3888 02:53:34,560 --> 02:53:35,680 ASSESS VALIDITY OF MACHINE 3889 02:53:35,680 --> 02:53:36,280 LEARNING METHODS. 3890 02:53:36,280 --> 02:53:38,960 THIS WAS ONE OF THE AREAS STUDY, 3891 02:53:38,960 --> 02:53:40,520 WE RAN ON A GROUP OF HEALTHY 3892 02:53:40,520 --> 02:53:41,840 INDIVIDUALS, ASKED THEM TO WALK 3893 02:53:41,840 --> 02:53:44,360 AND RUN AT DIFFERENT SPEEDS ON 3894 02:53:44,360 --> 02:53:47,000 TREADMILL WHEEL THE GAIT -- 3895 02:53:47,000 --> 02:53:49,720 WHILE THE GAIT WAS MONITORED BY 3896 02:53:49,720 --> 02:53:51,920 A REFERENCE CAMERA SYSTEM TO 3897 02:53:51,920 --> 02:53:55,440 EXTRACT MEASUREMENT AND BY OUR 3898 02:53:55,440 --> 02:53:56,000 INSOLES. 3899 02:53:56,000 --> 02:54:01,000 WE WANTED TO VALIDATE TWO 3900 02:54:01,000 --> 02:54:02,480 MODELS, GENETIC MODEL 3901 02:54:02,480 --> 02:54:03,760 ONE-SIZE-FITS-ALL MODEL THAT 3902 02:54:03,760 --> 02:54:06,360 DOES NOT REQUIRE REFERENCE DATA 3903 02:54:06,360 --> 02:54:08,680 FROM THE INDIVIDUALS, AND STUDY 3904 02:54:08,680 --> 02:54:11,960 SPECIFIC MODEL THAT IS 3905 02:54:11,960 --> 02:54:13,920 ESSENTIALLY SPECIFICALLY FOR AN 3906 02:54:13,920 --> 02:54:16,120 INDIVIDUAL, USING REFERENCE DATA 3907 02:54:16,120 --> 02:54:18,520 COLLECTED WITH MOTION SYSTEM FOR 3908 02:54:18,520 --> 02:54:20,480 THAT PARTICULAR INDIVIDUAL. 3909 02:54:20,480 --> 02:54:22,640 IN ORDER TO ASSESS THE ACCURACY 3910 02:54:22,640 --> 02:54:33,160 OF OUR MODELS WE LOOK AT THREE 3911 02:54:34,480 --> 02:54:36,640 TARGET GAIT PARAMETERS, ASSESS, 3912 02:54:36,640 --> 02:54:38,920 MEASURE MEAN ABSOLUTE ERROR. 3913 02:54:38,920 --> 02:54:46,520 SUBJECT SPECIFIC MODELS WERE 3914 02:54:46,520 --> 02:54:49,000 MOST ACCURATE. 3915 02:54:49,000 --> 02:54:51,320 THE GENETIC MODELS WERE LIKELY 3916 02:54:51,320 --> 02:54:54,000 LESS ACCURACY, AROUND 2% FOR 3917 02:54:54,000 --> 02:54:58,080 WALKING, 5% FOR RUNNING. 3918 02:54:58,080 --> 02:55:02,040 BOTH MODELS PROVIDED EXCELLENT 3919 02:55:02,040 --> 02:55:10,480 TEST RELIABILITY. 3920 02:55:10,480 --> 02:55:15,320 THE TAKEHOME MESSAGE, WE KNOW IN 3921 02:55:15,320 --> 02:55:19,000 PRACTICE IT MIGHT BE HARD OR 3922 02:55:19,000 --> 02:55:20,360 IMPRACTICAL TO COLLECT REFERENCE 3923 02:55:20,360 --> 02:55:23,160 DATA FROM EACH STUDY PARTICIPANT 3924 02:55:23,160 --> 02:55:26,680 BECAUSE OF BUDGET OR TIME 3925 02:55:26,680 --> 02:55:27,440 CONSTRAINTS. 3926 02:55:27,440 --> 02:55:31,720 SO NEXT QUESTION WAS CAN WE 3927 02:55:31,720 --> 02:55:32,360 DETERMINE, DERIVE PERSONALIZED 3928 02:55:32,360 --> 02:55:38,280 MODELS WOULD THE -- WITHOUT NEED 3929 02:55:38,280 --> 02:55:39,720 FOR HAVING THE SUBJECT TEST IN A 3930 02:55:39,720 --> 02:55:41,120 GAIT LAB. 3931 02:55:41,120 --> 02:55:44,320 WE ASKED A GROUP OF 95 OLDER 3932 02:55:44,320 --> 02:55:45,880 ADULTS IN FIVE SENIOR CENTERS 3933 02:55:45,880 --> 02:55:49,360 AROUND THE NEW YORK CITY AREA TO 3934 02:55:49,360 --> 02:55:50,960 COMPLETE SIX-MINUTE WALK TEST 3935 02:55:50,960 --> 02:56:01,440 USING OUR WEARABLE SYSTEM. 3936 02:56:07,040 --> 02:56:09,360 WE SEE THE MEAN. 3937 02:56:09,360 --> 02:56:14,880 THOSE ARE ERRORS, THE LARGER THE 3938 02:56:14,880 --> 02:56:17,920 BARS, THE WORSE, SMALLER THE 3939 02:56:17,920 --> 02:56:18,520 BETTER. 3940 02:56:18,520 --> 02:56:20,120 INDIVIDUALS REQUIRE WALKING AID 3941 02:56:20,120 --> 02:56:22,520 TO PERFORM 6-MINUTE TEST VERSUS 3942 02:56:22,520 --> 02:56:31,720 THOSE THAT DID NOT REQUIRE ANY. 3943 02:56:31,720 --> 02:56:32,840 RED BOXES INDICATE THE 3944 02:56:32,840 --> 02:56:34,960 MEASUREMENT ERROR WE OBTAINED 3945 02:56:34,960 --> 02:56:41,080 WHEN APPLYING CONVENTIONAL 3946 02:56:41,080 --> 02:56:44,080 METHOD. 3947 02:56:44,080 --> 02:56:47,560 IT IS OFTEN IMPLEMENTED IN MANY 3948 02:56:47,560 --> 02:56:49,920 SMART INSULTS, FOOT TO ANKLE 3949 02:56:49,920 --> 02:56:52,040 TOUCH DEVICES IN THE MARKET. 3950 02:56:52,040 --> 02:56:57,120 THE ERRORS ARE QUITE 3951 02:56:57,120 --> 02:56:57,880 SUBSTANTIAL. 3952 02:56:57,880 --> 02:57:01,480 THE ERROR SUBSTANTIALLY REDUCED, 3953 02:57:01,480 --> 02:57:04,200 THIS METHOD IS NOT PRACTICAL. 3954 02:57:04,200 --> 02:57:09,240 WHAT WE INTRODUCED WAS TYPE OF 3955 02:57:09,240 --> 02:57:16,280 MODEL THAT IS BASED ON THE 3956 02:57:16,280 --> 02:57:20,560 TRANSDUCTIVE FRAMEWORK 3957 02:57:20,560 --> 02:57:21,440 EXTRACTING FROM A DATABASE, MOST 3958 02:57:21,440 --> 02:57:25,720 SIMILAR TO THE SUBJECT WE WANT 3959 02:57:25,720 --> 02:57:29,120 TO TRY TO TRAIN A NEW MODEL FOR. 3960 02:57:29,120 --> 02:57:35,000 WE'RE ABLE TO REDUCE MEASUREMENT 3961 02:57:35,000 --> 02:57:42,440 ERRORS BY 42% COMPARED TO 3962 02:57:42,440 --> 02:57:45,120 CONVENTIONAL, REDUCE ERRORS IN 3963 02:57:45,120 --> 02:57:48,360 SLEEP TIME BY 34%. 3964 02:57:48,360 --> 02:57:51,320 RESULTS WERE ENCOURAGING, BUT 3965 02:57:51,320 --> 02:57:53,680 OBTAINED FOR SIMPLE STRAIGHT 3966 02:57:53,680 --> 02:57:56,320 LINE WALKING TASK, DO NOT 3967 02:57:56,320 --> 02:57:59,760 NECESSARILY RESEMBLE REAL LIFE. 3968 02:57:59,760 --> 02:58:03,440 SO, TO EVALUATE THE VALIDITY OF 3969 02:58:03,440 --> 02:58:06,280 OUR METHODS IN TASKS THAT ARE 3970 02:58:06,280 --> 02:58:07,960 MORE CLOSE TO REAL LIFE WALKING, 3971 02:58:07,960 --> 02:58:10,440 WE'RE ASKED A GROUP OF 3972 02:58:10,440 --> 02:58:12,560 INDIVIDUALS TO PERFORM A SET OF 3973 02:58:12,560 --> 02:58:16,880 ACTIVITIES THAT ARE LISTED HERE. 3974 02:58:16,880 --> 02:58:18,320 FIRST, IN STRUCTURE CONDITION, 3975 02:58:18,320 --> 02:58:20,640 MEANING EACH ACTIVITY WAS 3976 02:58:20,640 --> 02:58:23,480 CARRIED OUT FOR SPECIFIC TIME 3977 02:58:23,480 --> 02:58:25,480 SLOT, SPECIFIC SEQUENCE. 3978 02:58:25,480 --> 02:58:28,520 AND AFTERWARDS THE SAME 3979 02:58:28,520 --> 02:58:29,840 ACTIVITIES BUT IN STRUCTURE 3980 02:58:29,840 --> 02:58:32,720 CONDITION MEANING WE LET THEM 3981 02:58:32,720 --> 02:58:35,000 NAVIGATE IN AN INDOOR 3982 02:58:35,000 --> 02:58:37,720 ENVIRONMENT WITH OBSTACLES, 3983 02:58:37,720 --> 02:58:43,000 CHAIRS, STAIRCASE, SO FORTH, AND 3984 02:58:43,000 --> 02:58:44,520 WE LET THEM PERFORM ALL THOSE 3985 02:58:44,520 --> 02:58:46,040 TASKS IN THE ORDER THEY WANT, 3986 02:58:46,040 --> 02:58:49,320 FOR THE TIME DURATION THEY WANT. 3987 02:58:49,320 --> 02:58:51,720 THIS DOES SHOW THE MEASUREMENT 3988 02:58:51,720 --> 02:58:54,480 ERRORS THAT WE FOUND, THE BLUE 3989 02:58:54,480 --> 02:58:55,680 BARS INDICATE COMMERCIAL 3990 02:58:55,680 --> 02:58:58,640 METHODS, THOSE THAT ARE 3991 02:58:58,640 --> 02:59:01,560 IMPLEMENTED IN MOST OFF THE 3992 02:59:01,560 --> 02:59:03,680 SHELF DEVICES VERSUS OUR MACHINE 3993 02:59:03,680 --> 02:59:05,520 LEARNING METHODS BASED ON 3994 02:59:05,520 --> 02:59:07,840 SUPPORT VECTOR ADRESS IN LIGHT 3995 02:59:07,840 --> 02:59:11,320 BLUE HERE AND FOUND THAT MACHINE 3996 02:59:11,320 --> 02:59:12,560 LEARNING METHOD REDUCED 3997 02:59:12,560 --> 02:59:15,160 MEASUREMENT ERRORS BOTH IN 3998 02:59:15,160 --> 02:59:15,880 STRUCTURE AROUND STRUCTURE 3999 02:59:15,880 --> 02:59:20,760 CONDITION REGARDLESS OF TYPE OF 4000 02:59:20,760 --> 02:59:21,480 WALKING TASKS. 4001 02:59:21,480 --> 02:59:26,320 WE LOOKED AT ACCURACY OF OUR 4002 02:59:26,320 --> 02:59:27,720 SYSTEM IN CLASSIFYING 4003 02:59:27,720 --> 02:59:29,840 ACTIVITIES, WE FOUND THE AVERAGE 4004 02:59:29,840 --> 02:59:34,200 ACCURACY FOR THE UNSTRUCTURED 4005 02:59:34,200 --> 02:59:37,920 CONDITION WAS 97%, AVERAGE 4006 02:59:37,920 --> 02:59:40,160 ACCURACY WE OBTAINED ASKING OUR 4007 02:59:40,160 --> 02:59:46,280 INDIVIDUALS TO USE THE DEVICE 4008 02:59:46,280 --> 02:59:48,040 FOR FOUR HOURS DOING THEIR 4009 02:59:48,040 --> 02:59:50,800 ACTIVITIES, IN THIS CASE 4010 02:59:50,800 --> 02:59:53,640 ACCURACY WAS 95%. 4011 02:59:53,640 --> 02:59:55,240 STILL PRETTY HIGH. 4012 02:59:55,240 --> 02:59:57,040 WE ALSO LOOKED AT HOW THE 4013 02:59:57,040 --> 02:59:58,880 LOCATION OF THE WEARABLE SENSOR 4014 02:59:58,880 --> 03:00:02,600 IN THE USER BODY MIGHT AFFECTS 4015 03:00:02,600 --> 03:00:04,240 ACCURACY OF THOSE ACTIVITY 4016 03:00:04,240 --> 03:00:05,320 CLASSIFICATION MODELS, AND SO TO 4017 03:00:05,320 --> 03:00:09,280 DO THAT WE TESTED THREE TYPES OF 4018 03:00:09,280 --> 03:00:10,760 SENSORS, WRIST WORN, SMARTPHONE 4019 03:00:10,760 --> 03:00:21,200 LOCATED IN THE SUBJECT'S 4020 03:00:23,240 --> 03:00:25,800 POCKETS, AND INSOLES. 4021 03:00:25,800 --> 03:00:28,560 THIS RESULTED IN SEVEN 4022 03:00:28,560 --> 03:00:29,840 INDEPENDENT ACTIVITY 4023 03:00:29,840 --> 03:00:30,160 CLASSIFIERS. 4024 03:00:30,160 --> 03:00:31,640 HERE YOU SEE THE AVERAGE 4025 03:00:31,640 --> 03:00:38,080 ACCURACY THAT WE OBTAINED FOR 4026 03:00:38,080 --> 03:00:44,120 THE UNSTRUCTURED CONDITION. 4027 03:00:44,120 --> 03:00:46,320 ALL THE CLASSIFIERS RESULTED IN 4028 03:00:46,320 --> 03:00:51,880 ACCURACY THAT WAS ABOVE 95%, 4029 03:00:51,880 --> 03:00:54,080 WHEREAS THE CLASSIFIER THAT 4030 03:00:54,080 --> 03:00:56,600 RELIED ON THE WRIST SENSOR ALONE 4031 03:00:56,600 --> 03:00:58,120 WAS CONSISTENTLY THE WORST 4032 03:00:58,120 --> 03:01:00,640 PERFORMING ONE IN THOSE 4033 03:01:00,640 --> 03:01:02,800 CONDITIONS, AND THIS IS 4034 03:01:02,800 --> 03:01:07,640 CONSISTENT WITH RESULTING IN THE 4035 03:01:07,640 --> 03:01:08,680 LITERATURE. 4036 03:01:08,680 --> 03:01:09,600 THIS IS ONGOING VALIDATION STUDY 4037 03:01:09,600 --> 03:01:14,680 IN A GROUP OF PATIENTS WITH SMA 4038 03:01:14,680 --> 03:01:16,920 AND DMD, AND ALTHOUGH THE 4039 03:01:16,920 --> 03:01:20,720 ENROLLMENT IS STILL ACTIVE WE 4040 03:01:20,720 --> 03:01:22,000 ARE ALMOST CLOSE TO COMPLETION. 4041 03:01:22,000 --> 03:01:24,280 WE'LL SHOW YOU THE PARTIAL 4042 03:01:24,280 --> 03:01:27,800 RESULT WE'VE OBTAINED SO FAR. 4043 03:01:27,800 --> 03:01:30,840 THIS STUDY IS CARRIED OUT 4044 03:01:30,840 --> 03:01:37,080 CLINICAL SITES, AND WE ASKED OUR 4045 03:01:37,080 --> 03:01:40,080 PARTICIPANTS TO COMPLETE THREE 4046 03:01:40,080 --> 03:01:40,360 ASSESSMENTS. 4047 03:01:40,360 --> 03:01:42,880 AFTER THAT WE ASKED THEM TO 4048 03:01:42,880 --> 03:01:46,080 COMPLETE A SET OF AD HOC WALKING 4049 03:01:46,080 --> 03:01:55,360 TASKS DESIGNED TO SIMULATE REAL 4050 03:01:55,360 --> 03:01:55,960 LIFE WALKING. 4051 03:01:55,960 --> 03:01:59,920 AS IN PREVIOUS STUDY I DESCRIBED 4052 03:01:59,920 --> 03:02:02,760 WE USE INSTRUMENTED WALKWAY TO 4053 03:02:02,760 --> 03:02:07,120 PROCEED REFERENCE SYSTEM TO 4054 03:02:07,120 --> 03:02:07,760 EXTRACT MEASUREMENT ERRORS. 4055 03:02:07,760 --> 03:02:11,200 SO THIS IS THE RESULT FROM 22 4056 03:02:11,200 --> 03:02:12,520 SUBJECTS, 13 CONTROLS WE'RE 4057 03:02:12,520 --> 03:02:14,680 SCREENING TO DATE. 4058 03:02:14,680 --> 03:02:17,640 TOP TABLE HERE SHOWS MEAN 4059 03:02:17,640 --> 03:02:25,520 OPPOSITE ERROR ON FIVE 4060 03:02:25,520 --> 03:02:26,160 REPRESENTING PARAMETERS, AND 4061 03:02:26,160 --> 03:02:29,120 CONTROLS AND PATIENT. 4062 03:02:29,120 --> 03:02:31,640 SO, WE FOUND WHEN INCLUDING ALL 4063 03:02:31,640 --> 03:02:33,200 DATA FROM WALKING TASKS, 4064 03:02:33,200 --> 03:02:35,920 STRAIGHT LINE WALKING AND CURBS 4065 03:02:35,920 --> 03:02:46,480 AND SO FORTH, THE ERROR FOR THE 4066 03:02:48,680 --> 03:02:50,480 CONTROLS WAS BELOW 5%, BOTTOM 4067 03:02:50,480 --> 03:02:59,640 SHOWS MEASUREMENT ERROR, FOR THE 4068 03:02:59,640 --> 03:03:00,960 CONTROLS AND PATIENT. 4069 03:03:00,960 --> 03:03:05,880 WE FOUND THAT WHEN COMBINING 4070 03:03:05,880 --> 03:03:07,520 DIRECTIONS COMBINED ERROR WAS 4071 03:03:07,520 --> 03:03:10,240 BELOW 3% FOR THE CONTROLS AND 4072 03:03:10,240 --> 03:03:12,560 BELOW 4% FOR THE PATIENT. 4073 03:03:12,560 --> 03:03:16,400 HERE YOU CAN SEE SOME 4074 03:03:16,400 --> 03:03:17,680 REPRESENTATIVE DATA IN TERMS OF 4075 03:03:17,680 --> 03:03:21,760 RIGHT FOOT THAT WE OBTAINED FROM 4076 03:03:21,760 --> 03:03:24,920 PARTICIPANT WITH DMD AND HEALTHY 4077 03:03:24,920 --> 03:03:31,160 CONTROL DURING THE 6-MINUTE WALK 4078 03:03:31,160 --> 03:03:31,600 TEST. 4079 03:03:31,600 --> 03:03:34,560 IN ORDER TO FURTHER VALIDATE OR 4080 03:03:34,560 --> 03:03:37,640 QUANTIFY, NOW WE'RE RUNNING 4081 03:03:37,640 --> 03:03:42,480 PILOT TESTS IN SMALL SAMPLE OF 4082 03:03:42,480 --> 03:03:45,000 PATIENTS WITH SMA AND DMD, FIVE 4083 03:03:45,000 --> 03:03:48,800 DAYS IN A ROW, FOUR HOURS PER 4084 03:03:48,800 --> 03:03:49,600 DAY. 4085 03:03:49,600 --> 03:03:51,800 I'M GOING TO SO YOU PRELIMINARY 4086 03:03:51,800 --> 03:03:52,600 DATA. 4087 03:03:52,600 --> 03:03:54,640 WE HAVE NOT COMPLETED DATA 4088 03:03:54,640 --> 03:03:57,600 COLLECTION BUT THIS IS DATA WE 4089 03:03:57,600 --> 03:04:05,040 COLLECTED FROM 16-YEAR-OLD BOY 4090 03:04:05,040 --> 03:04:10,680 WITH TYPE 3 SMA, TOP SHOWS 4091 03:04:10,680 --> 03:04:13,040 DISTRIBUTION OF DATA, 5 DAYS. 4092 03:04:13,040 --> 03:04:23,520 WE SPLIT DISTRIBUTION TO 3 4093 03:04:24,760 --> 03:04:32,720 COLORS, INDICATING DIFFERENT 4094 03:04:32,720 --> 03:04:33,000 DURATION. 4095 03:04:33,000 --> 03:04:33,920 WE SEPARATED. 4096 03:04:33,920 --> 03:04:37,720 THE PLOT SHOWS CORRESPONDING 4097 03:04:37,720 --> 03:04:39,720 RESULT OBTAINED FROM HEALTHY 4098 03:04:39,720 --> 03:04:40,800 CONTROLS. 4099 03:04:40,800 --> 03:04:42,560 PATIENTS TENDED TO WALK SLOWER 4100 03:04:42,560 --> 03:04:44,080 BY TAKING SHORTER STRIDES 4101 03:04:44,080 --> 03:04:47,560 COMPARED TO CONTROL, AND ALSO 4102 03:04:47,560 --> 03:04:49,560 OBVIOUSLY THE VOLUME OF THE 4103 03:04:49,560 --> 03:04:50,840 STRIDES WE RECORDED OVER FIVE 4104 03:04:50,840 --> 03:04:54,720 DAYS WAS MUCH SMALLER FOR THE 4105 03:04:54,720 --> 03:04:58,320 PATIENTS COMPARED TO CONTROL. 4106 03:04:58,320 --> 03:05:00,000 ANOTHER OBSERVATION HERE IS THAT 4107 03:05:00,000 --> 03:05:03,360 DISTRIBUTION IS MUCH MORE 4108 03:05:03,360 --> 03:05:07,560 OVERLAPPING, FOR THE SMA THAN 4109 03:05:07,560 --> 03:05:09,320 FOR THE HEALTHY INDIVIDUAL. 4110 03:05:09,320 --> 03:05:14,120 WE BELIEVE THIS MIGHT BE RELATED 4111 03:05:14,120 --> 03:05:16,800 TO VERSATILITY IN THE RANGE OF 4112 03:05:16,800 --> 03:05:19,160 REAL LIFE WALKING SPEED, THOSE 4113 03:05:19,160 --> 03:05:21,640 PATIENTS SHOW IN THEIR 4114 03:05:21,640 --> 03:05:23,600 DAY-TO-DAY LIFE, THIS MIGHT BE 4115 03:05:23,600 --> 03:05:26,200 RELATED TO MUSCLE WEAKNESS OR 4116 03:05:26,200 --> 03:05:27,120 MAYBE FEAR OF FALLING. 4117 03:05:27,120 --> 03:05:29,560 WE NEED TO COLLECT MORE DATA TO 4118 03:05:29,560 --> 03:05:31,840 CONFIRM WHETHER OR NOT THESE ARE 4119 03:05:31,840 --> 03:05:35,080 PLANS THAT WE'RE OBSERVING IN 4120 03:05:35,080 --> 03:05:36,760 OTHER PARTICIPANTS AS WELL. 4121 03:05:36,760 --> 03:05:38,320 TO CONCLUDE ONCE WE HAVE 4122 03:05:38,320 --> 03:05:41,720 FINISHED THIS PILOT TEST THE 4123 03:05:41,720 --> 03:05:46,320 NEXT IS TO EXTEND THE DURATION 4124 03:05:46,320 --> 03:05:51,160 OF THESE REAL LIFE GAIT MONITORS 4125 03:05:51,160 --> 03:05:54,600 TO FOLLOW THE MONITOR, MONITOR 4126 03:05:54,600 --> 03:05:55,760 STUDY PARTICIPANTS FOR 180 HOURS 4127 03:05:55,760 --> 03:05:58,160 OVER 30 DAYS, THIS HAS BEEN 4128 03:05:58,160 --> 03:06:01,120 REPORTED TO BE A GOOD TRADEOFF 4129 03:06:01,120 --> 03:06:04,640 BETWEEN NECESSITY OF REALLY 4130 03:06:04,640 --> 03:06:05,720 PROVIDING TRUTHFUL PICTURE OF 4131 03:06:05,720 --> 03:06:06,680 DAILY FUNCTION IN THOSE 4132 03:06:06,680 --> 03:06:11,320 INDIVIDUALS AND AT THE SAME TIME 4133 03:06:11,320 --> 03:06:14,120 PROVIDING OR GUARANTEEING GOOD 4134 03:06:14,120 --> 03:06:15,920 LEVEL OF PATIENT COMPLIANCE. 4135 03:06:15,920 --> 03:06:19,880 WE WANTED TO ADMINISTER IN 4136 03:06:19,880 --> 03:06:23,160 CLINICAL ASSESSMENT, TIME 4137 03:06:23,160 --> 03:06:24,880 POINTS, BASELINE, SIX MONTHS, 4138 03:06:24,880 --> 03:06:26,400 TWELVE MONTHS, AFTER EACH THE 4139 03:06:26,400 --> 03:06:30,960 IDEA IS TO FOLLOW, TO MONITOR 4140 03:06:30,960 --> 03:06:32,480 THE STUDY PARTICIPANT GAIT FOR 4141 03:06:32,480 --> 03:06:35,560 30 DAYS AND WE WILL USE THIS 4142 03:06:35,560 --> 03:06:39,840 DATA TO IDENTIFY THE SUBSET OF 4143 03:06:39,840 --> 03:06:43,320 DMOs THAT BEST DISCRIMINATE 4144 03:06:43,320 --> 03:06:46,160 ACROSS DIFFERENT TYPES OF 4145 03:06:46,160 --> 03:06:48,280 NEUROMUSCULAR CONDITIONS, AND 4146 03:06:48,280 --> 03:06:50,880 ALSO TO IDENTIFY THOSE DMOs 4147 03:06:50,880 --> 03:06:52,520 THAT BEST STRATIFY PATIENTS THAT 4148 03:06:52,520 --> 03:06:53,760 WITHIN THE SAME CONDITIONS IN 4149 03:06:53,760 --> 03:06:59,640 TERMS OF SEVERITY OF THEIR 4150 03:06:59,640 --> 03:07:01,000 FUNCTIONAL DEFICIT, USE 4151 03:07:01,000 --> 03:07:05,880 LONGITUDINAL DATA FOR EACH DMO 4152 03:07:05,880 --> 03:07:11,800 TO FIND THE MINIMAL DETECTABLE 4153 03:07:11,800 --> 03:07:13,240 CHANGE, AND SENSITIVITY TO 4154 03:07:13,240 --> 03:07:15,640 LONGITUDINAL CHANGES AT SIX 4155 03:07:15,640 --> 03:07:16,520 MONTHS AND TWELVE MONTHS. 4156 03:07:16,520 --> 03:07:18,120 WITH THAT I'D LIKE TO THANK YOU 4157 03:07:18,120 --> 03:07:21,960 FOR YOUR ATTENTION AND I'LL TAKE 4158 03:07:21,960 --> 03:07:23,720 ANY QUESTIONS. 4159 03:07:23,720 --> 03:07:32,160 >>THANK YOU VERY MUCH. 4160 03:07:32,160 --> 03:07:39,200 WE CAN TAKE QUESTIONS FROM BOTH 4161 03:07:39,200 --> 03:07:40,280 DR. ZONOTTO AND DR. MONTES. 4162 03:07:40,280 --> 03:07:41,880 THERE WAS ONE IN THE QUESTION 4163 03:07:41,880 --> 03:07:45,200 AND ANSWER BOX FROM DOUGLAS 4164 03:07:45,200 --> 03:07:47,560 ALBRECHT. 4165 03:07:47,560 --> 03:07:50,440 HAVE YOU ASSESSED YOUR SENSORS 4166 03:07:50,440 --> 03:07:51,280 MULTITUDEALLY AS COMPENSATIONS 4167 03:07:51,280 --> 03:07:53,520 CHANGE OVER TIME, RESULTS IN 4168 03:07:53,520 --> 03:07:54,800 EACH PERSON MAY CHANGE BUT NOT 4169 03:07:54,800 --> 03:07:57,560 NECESSARILY THE SAME WAY IN EACH 4170 03:07:57,560 --> 03:07:59,200 PERSON, THIS MAY COMPLICATE HOW 4171 03:07:59,200 --> 03:08:00,760 THESE MEASURES ARE USED TO 4172 03:08:00,760 --> 03:08:01,640 ASSESS TREATMENT EFFECT BECAUSE 4173 03:08:01,640 --> 03:08:05,160 IT COULD BE HARD TO DETERMINE 4174 03:08:05,160 --> 03:08:09,400 WHAT BENEFITS WILL LOOK LIKE. 4175 03:08:09,400 --> 03:08:13,120 >>AS OF NOW WE FOCUSED ON 4176 03:08:13,120 --> 03:08:14,760 FUNCTIONAL VALIDATION AND 4177 03:08:14,760 --> 03:08:16,440 ECOLOGICAL VALIDATION OF 4178 03:08:16,440 --> 03:08:20,480 TECHNOLOGY, LOOKING AT HOW WE 4179 03:08:20,480 --> 03:08:23,520 CAN REALLY LEVERAGE TECHNOLOGY 4180 03:08:23,520 --> 03:08:25,840 FOR THE DETECTING LONGITUDINAL 4181 03:08:25,840 --> 03:08:31,320 CHANGES, THE NEXT GOAL FOR US. 4182 03:08:31,320 --> 03:08:33,840 >>I THINK TO THE SECOND POINT, 4183 03:08:33,840 --> 03:08:35,920 A VERY GOOD SECOND QUESTION, 4184 03:08:35,920 --> 03:08:39,400 VERY GOOD POINT. 4185 03:08:39,400 --> 03:08:40,600 WHAT ABOUT THE INTERINDIVIDUAL 4186 03:08:40,600 --> 03:08:43,400 CHANGES OVER TIME AND HOW WOULD 4187 03:08:43,400 --> 03:08:48,000 THEY BE DIFFERENT, I MEAN, I 4188 03:08:48,000 --> 03:08:52,000 THINK IN DEVELOPING THESE 4189 03:08:52,000 --> 03:08:55,960 DMOs, COMPOSITE SIGNATURES, I 4190 03:08:55,960 --> 03:08:58,480 THINK BETTER ABLE TO UNDERSTAND 4191 03:08:58,480 --> 03:09:01,960 THE SORT OF INDIVIDUAL CHANGES 4192 03:09:01,960 --> 03:09:03,520 BUT IT'S DEFINITELY SOMETHING, 4193 03:09:03,520 --> 03:09:07,560 IT MIGHT BE DIFFERENT FOR EACH 4194 03:09:07,560 --> 03:09:10,400 CONDITION, SO I THINK NOT JUST 4195 03:09:10,400 --> 03:09:11,760 RELYING ON STRIDE LENGTH AND 4196 03:09:11,760 --> 03:09:16,880 USING COMPOSITE OF DMOs MIGHT 4197 03:09:16,880 --> 03:09:18,960 HELP UNDERSTAND THAT OR AT LEAST 4198 03:09:18,960 --> 03:09:25,160 BRING TO THE FRONT THOSE 4199 03:09:25,160 --> 03:09:26,520 NUANCES. 4200 03:09:26,520 --> 03:09:29,320 >>DAN HAS A QUESTION. 4201 03:09:29,320 --> 03:09:33,320 4202 03:09:33,320 --> 03:09:36,880 >>TWO QUESTIONS. 4203 03:09:36,880 --> 03:09:37,920 EXCELLENT TALK. 4204 03:09:37,920 --> 03:09:45,200 THE FIRST QUESTION HAS TO DO 4205 03:09:45,200 --> 03:09:51,880 WITH TECHNOLOGY WITH ANKLE 4206 03:09:51,880 --> 03:09:56,400 ORTHOTICS, AND THE SECOND 4207 03:09:56,400 --> 03:10:02,280 QUESTION, IN THE CLINICAL TRIALS 4208 03:10:02,280 --> 03:10:08,720 DO YOU THINK ABOUT INCLUSION 4209 03:10:08,720 --> 03:10:17,160 CRITERIA AND SO THE QUESTION IS 4210 03:10:17,160 --> 03:10:25,040 ONLY AMBULATING, NOT USING 4211 03:10:25,040 --> 03:10:26,920 ORTHOTICS OR USING ORTHOTICS, IN 4212 03:10:26,920 --> 03:10:32,840 A WHEELCHAIR OR NOT, AND THEN 4213 03:10:32,840 --> 03:10:43,520 ALSO WAS THE RESEARCH PEDIATRIC 4214 03:10:43,520 --> 03:10:44,760 VERSUS ADULT? 4215 03:10:44,760 --> 03:10:55,320 OR GERIATRIC SETTING, OR ADULTS 4216 03:10:57,680 --> 03:11:03,080 WITH MUSCULAR DYSTROPHY, THOSE 4217 03:11:03,080 --> 03:11:05,120 ARE THE TWO CONSIDERATIONS. 4218 03:11:05,120 --> 03:11:05,680 >>THANK YOU. 4219 03:11:05,680 --> 03:11:06,840 I APPRECIATE BOTH QUESTIONS. 4220 03:11:06,840 --> 03:11:10,280 I CAN TAKE THE ONE THAT MORE 4221 03:11:10,280 --> 03:11:15,160 RELATES TO TECHNOLOGY . 4222 03:11:15,160 --> 03:11:18,920 WE DID TEST THE TECHNOLOGY FOR 4223 03:11:18,920 --> 03:11:21,560 INDIVIDUALS THAT ALSO USE 4224 03:11:21,560 --> 03:11:26,800 ORTHOTICS, IN TERMS OF 4225 03:11:26,800 --> 03:11:27,600 SPATIOTEMPORAL PARAMETERS THERE 4226 03:11:27,600 --> 03:11:30,720 ARE NO PROBLEMS. 4227 03:11:30,720 --> 03:11:35,000 THE OTHER TECHNOLOGY QUESTION 4228 03:11:35,000 --> 03:11:39,400 WAS HOW THE -- WOULD TECHNOLOGY 4229 03:11:39,400 --> 03:11:40,800 WORK ACROSS DIFFERENT AGE 4230 03:11:40,800 --> 03:11:44,240 RANGES, THE ANSWER IS YES. 4231 03:11:44,240 --> 03:11:46,600 THE CURRENT PROTOTYPE WORKS, WE 4232 03:11:46,600 --> 03:11:49,920 HAVE VALIDATED THE CURRENT 4233 03:11:49,920 --> 03:11:53,760 TECHNOLOGY WITH OVER 500 4234 03:11:53,760 --> 03:11:55,080 INDIVIDUALS, AGE RANGE FROM 21 4235 03:11:55,080 --> 03:12:01,600 MONTHS TO 100 YEARS. 4236 03:12:01,600 --> 03:12:06,320 SO EIGHT DIFFERENT SIZES, OUR 4237 03:12:06,320 --> 03:12:10,600 MACHINE LEARNING MODELS WITH 4238 03:12:10,600 --> 03:12:12,760 CHANGES IN INSOLE SIZE. 4239 03:12:12,760 --> 03:12:18,080 >>I'LL ADD JUST WITH REGARDS TO 4240 03:12:18,080 --> 03:12:20,920 ORTHOTICS, THE INSOLES ARE SO 4241 03:12:20,920 --> 03:12:23,440 THIN THAT IT'S -- YOU KNOW, WE 4242 03:12:23,440 --> 03:12:24,720 CONSIDER THAT THEY ARE 4243 03:12:24,720 --> 03:12:26,520 DEFINITELY SOMETHING THAT YOU 4244 03:12:26,520 --> 03:12:30,960 COULD WEAR WITHIN A TYPICAL AFO 4245 03:12:30,960 --> 03:12:35,000 OR SMO, IN TERMS OUR NEXT -- WE 4246 03:12:35,000 --> 03:12:36,440 HAVEN'T STUDIED WITH ORTHOTICS 4247 03:12:36,440 --> 03:12:39,000 AT THE MOMENT BUT WE KNOW THAT 4248 03:12:39,000 --> 03:12:42,040 WE'RE SORT OF STRUGGLING WITH 4249 03:12:42,040 --> 03:12:44,640 UNDERSTANDING SORT OF THE 4250 03:12:44,640 --> 03:12:46,840 NATURAL HISTORY TRAJECTORY OF 4251 03:12:46,840 --> 03:12:51,800 GAIT IN THESE DISORDERS OVER 4252 03:12:51,800 --> 03:12:54,200 TIME, VERSUS HAVING IT ADAPTED, 4253 03:12:54,200 --> 03:12:56,520 BUT IT'S DEFINITELY SOMETHING 4254 03:12:56,520 --> 03:12:59,560 THAT IS FEASIBLE AND WILL 4255 03:12:59,560 --> 03:13:01,000 INCLUDE IF NOT SOONER RATHER 4256 03:13:01,000 --> 03:13:04,360 THAN LATER, AND IN TERMS OF SMA 4257 03:13:04,360 --> 03:13:08,440 AND DMD STUDIES THAT ARE ONGOING 4258 03:13:08,440 --> 03:13:11,480 AND PREDICT THAT THAT WE PREDICT 4259 03:13:11,480 --> 03:13:14,160 WHERE THE YOUNGEST CHILDREN 4260 03:13:14,160 --> 03:13:23,520 INCLUDING 4 OR 5 YEARS OLD, IT'S 4261 03:13:23,520 --> 03:13:25,280 BECAUSE WE'VE INCLUDED THE 4262 03:13:25,280 --> 03:13:26,800 SIX-MINUTE WALK TEST AS 4263 03:13:26,800 --> 03:13:27,560 REFERENCE CLINICAL ASSESSMENT 4264 03:13:27,560 --> 03:13:30,360 AND NEED SOME COOPERATION TO GET 4265 03:13:30,360 --> 03:13:31,920 THAT VALIDATED. 4266 03:13:31,920 --> 03:13:32,920 >>THANK YOU BOTH. 4267 03:13:32,920 --> 03:13:34,240 WE'LL TAKE QUESTIONS FROM GLEN 4268 03:13:34,240 --> 03:13:37,200 AND EMILY AND WE'LL MOVE ON. 4269 03:13:37,200 --> 03:13:39,280 >>YES, THANKS FOR A GREAT TALK. 4270 03:13:39,280 --> 03:13:41,440 WONDERING IF YOU'RE COLLECTING 4271 03:13:41,440 --> 03:13:43,760 DATA THAT WOULD LET YOU MONITOR 4272 03:13:43,760 --> 03:13:45,280 ANY FALLS THAT OCCUR IN 4273 03:13:45,280 --> 03:13:47,440 PATIENTS, SO THAT PERHAPS YOU 4274 03:13:47,440 --> 03:13:49,560 COULD USE THE DATA TO PREDICT 4275 03:13:49,560 --> 03:13:51,440 THE LIKELIHOOD OF FALLS AND 4276 03:13:51,440 --> 03:13:54,600 ACTUALLY TO HELP TRY AND PREVENT 4277 03:13:54,600 --> 03:13:55,600 FALLS IN INDIVIDUALS. 4278 03:13:55,600 --> 03:13:57,120 >>THAT'S A GREAT COMMENT. 4279 03:13:57,120 --> 03:13:59,720 WE HAVEN'T DONE IT YET BUT WE 4280 03:13:59,720 --> 03:14:02,040 ENVISION THAT THAT CAN BE A 4281 03:14:02,040 --> 03:14:06,360 POSSIBLE USE OF THIS TECHNOLOGY, 4282 03:14:06,360 --> 03:14:09,000 CERTAINLY COLLECT THE DATA, 4283 03:14:09,000 --> 03:14:11,400 THERE IS EVIDENCE THAT 4284 03:14:11,400 --> 03:14:15,680 COLLECTING DATA CAN BE USED TO 4285 03:14:15,680 --> 03:14:18,520 BUILD THOSE PREDICTION MODELS OF 4286 03:14:18,520 --> 03:14:23,120 FALLS OR DETECTION OF FALLS, 4287 03:14:23,120 --> 03:14:24,200 PREDICTION OF FALLS. 4288 03:14:24,200 --> 03:14:26,440 >>THANK YOU. 4289 03:14:26,440 --> 03:14:28,560 AND LAST IS EMILY. 4290 03:14:28,560 --> 03:14:29,120 >>HI. 4291 03:14:29,120 --> 03:14:29,560 THANKS. 4292 03:14:29,560 --> 03:14:33,160 THAT WAS A GREAT PRESENTATION. 4293 03:14:33,160 --> 03:14:35,680 DR. CAMPBELL HAD TO STEP AWAY, I 4294 03:14:35,680 --> 03:14:38,640 FEEL OBLIGATED TO KIND OF TIE IN 4295 03:14:38,640 --> 03:14:41,920 TO HER PRIOR QUESTIONS ABOUT 4296 03:14:41,920 --> 03:14:43,120 CLINICAL MEANINGFULNESS, JUST 4297 03:14:43,120 --> 03:14:45,080 WONDERING WITH ALL THIS KIND OF 4298 03:14:45,080 --> 03:14:47,280 FINE GRANULAR DATA THAT YOU'RE 4299 03:14:47,280 --> 03:14:50,440 ABLE TO COLLECT, HAVE YOU 4300 03:14:50,440 --> 03:14:53,400 THOUGHT ABOUT, LIKE GLEN SAID, 4301 03:14:53,400 --> 03:14:54,440 FALLS, WHAT CLINICAL MEANINGFUL 4302 03:14:54,440 --> 03:14:57,120 ENDPOINT DO YOU THINK WE COULD 4303 03:14:57,120 --> 03:14:58,680 LEVERAGE FROM A REGULATORY 4304 03:14:58,680 --> 03:14:59,480 PERSPECTIVE? 4305 03:14:59,480 --> 03:15:00,320 >>RIGHT. 4306 03:15:00,320 --> 03:15:10,800 SO AS I SAID, FOR NOW WE'RE 4307 03:15:19,120 --> 03:15:19,760 PRIMARILY FOCUSED APPROACH, 4308 03:15:19,760 --> 03:15:21,360 LOOKING AT LEVEL OF NOISE, THAT 4309 03:15:21,360 --> 03:15:22,760 IS IMPORTANT, RIGHT, BUT WE WANT 4310 03:15:22,760 --> 03:15:25,960 TO ASSESS WHAT IS MEANINGFUL FOR 4311 03:15:25,960 --> 03:15:28,640 THOSE PARTICIPANTS SO WE WANT TO 4312 03:15:28,640 --> 03:15:31,200 USE SURVEYS, QUESTIONNAIRES, 4313 03:15:31,200 --> 03:15:33,480 LIKERT SCALES AND TYPE OF DATA 4314 03:15:33,480 --> 03:15:36,000 TO REALLY LIKE FIGURE OUT WHAT 4315 03:15:36,000 --> 03:15:39,960 IS IMPORTANT, WHAT IS THE LEVEL 4316 03:15:39,960 --> 03:15:41,640 OF CLINICAL MEANINGFULNESS. 4317 03:15:41,640 --> 03:15:41,960 THOSE METRICS. 4318 03:15:41,960 --> 03:15:44,320 >>THANK YOU. 4319 03:15:44,320 --> 03:15:47,000 4320 03:15:47,000 --> 03:15:50,440 >>THANK YOU. 4321 03:15:50,440 --> 03:15:50,680 4322 03:15:50,680 --> 03:15:53,440 I THANK OUR PRESENTERS WHO 4323 03:15:53,440 --> 03:15:55,920 PRESENTED SOME REALLY AMAZING 4324 03:15:55,920 --> 03:15:56,800 TECHNOLOGY, INNOVATIVE OUTCOME 4325 03:15:56,800 --> 03:15:57,160 MEASURES. 4326 03:15:57,160 --> 03:16:00,320 WE LOOK FORWARD TO CONTINUED 4327 03:16:00,320 --> 03:16:02,320 PROGRESS ON THESE PROJECTS AND 4328 03:16:02,320 --> 03:16:04,920 FOR IMPLEMENTATION OF NOVEL 4329 03:16:04,920 --> 03:16:06,040 TECHNOLOGIES AND CLINICAL TRIALS 4330 03:16:06,040 --> 03:16:08,080 TO DELIVER HIGH QUALITY DATA AND 4331 03:16:08,080 --> 03:16:10,200 REDUCE BURDEN TO OUR STUDY 4332 03:16:10,200 --> 03:16:10,680 PARTICIPANTS. 4333 03:16:10,680 --> 03:16:13,480 NOW WE'RE GOING TO HAVE A COUPLE 4334 03:16:13,480 --> 03:16:17,920 BRIEF UPDATES FROM OUR NIH 4335 03:16:17,920 --> 03:16:20,720 STAFF, YOU'LL REALLY LAST JUNE 4336 03:16:20,720 --> 03:16:22,960 AT THE MDCC MEETING, JUNE LAST 4337 03:16:22,960 --> 03:16:27,040 YEAR, FOCUSED ON RESPIRATORY AND 4338 03:16:27,040 --> 03:16:28,600 SLEEP COMPLICATIONS. 4339 03:16:28,600 --> 03:16:31,440 WE ORGANIZED THAT WITH 4340 03:16:31,440 --> 03:16:34,200 COLLEAGUES FROM NHLBI AND DR. 4341 03:16:34,200 --> 03:16:36,600 NATARAJAN IS THE PROGRAM 4342 03:16:36,600 --> 03:16:37,800 DIRECTOR FOR PEDIATRIC AND 4343 03:16:37,800 --> 03:16:40,800 NEONATAL LUNG DISEASE AND 4344 03:16:40,800 --> 03:16:42,200 CRITICAL CARE AT NHLBI. 4345 03:16:42,200 --> 03:16:44,880 SHE'S HERE TO TELL US ABOUT THE 4346 03:16:44,880 --> 03:16:46,800 SPECIAL FUNDING OPPORTUNITY THAT 4347 03:16:46,800 --> 03:16:48,920 RESULTED FROM THAT MEETING. 4348 03:16:48,920 --> 03:16:50,600 SO, IN A WAY, THIS IS GETTING 4349 03:16:50,600 --> 03:16:53,120 BACK TO WHAT DAN ASKED ABOUT, 4350 03:16:53,120 --> 03:16:55,560 YOU KNOW, HOW DO WE MONITOR 4351 03:16:55,560 --> 03:16:57,080 OURSELVES TO SEE THAT WE DELIVER 4352 03:16:57,080 --> 03:17:01,800 ON THINGS THAT WE PROMISE. 4353 03:17:01,800 --> 03:17:03,440 SO, WELCOME, DR. NATARAJAN. 4354 03:17:03,440 --> 03:17:05,560 >>THANK YOU, DR. BIANCHI. 4355 03:17:05,560 --> 03:17:06,360 GOOD AFTERNOON. 4356 03:17:06,360 --> 03:17:08,360 THANK YOU FOR THIS OPPORTUNITY 4357 03:17:08,360 --> 03:17:11,640 TO PRESENT AN UPDATE ON A NOTICE 4358 03:17:11,640 --> 03:17:15,600 OF SPECIAL INTEREST, NOSI, TO 4359 03:17:15,600 --> 03:17:18,800 SPECIFICALLY ADDRESS RESPIRATORY 4360 03:17:18,800 --> 03:17:20,400 COMPLICATIONS OF MUSCULAR 4361 03:17:20,400 --> 03:17:20,720 DYSTROPHIES. 4362 03:17:20,720 --> 03:17:31,240 I THANK MY DIVISION DIRECTOR AND 4363 03:17:32,080 --> 03:17:40,000 OUR COLLEAGUES IN NINDS, NICHD, 4364 03:17:40,000 --> 03:17:41,640 AND NIAMS, HIGHLIGHTING RESEARCH 4365 03:17:41,640 --> 03:17:44,040 WE NEED IN THE MUSCULAR 4366 03:17:44,040 --> 03:17:46,840 DYSTROPHIES, RECOGNIZING MOST 4367 03:17:46,840 --> 03:17:48,720 PATIENTS OF NEUROMUSCULAR OR 4368 03:17:48,720 --> 03:17:52,920 CHEST WALL DISORDERS HAVE SOME 4369 03:17:52,920 --> 03:17:53,760 FORM OF RESPIRATORY 4370 03:17:53,760 --> 03:17:54,320 INSUFFICIENCY. 4371 03:17:54,320 --> 03:17:55,520 THE BURDEN ON PATIENTS AND 4372 03:17:55,520 --> 03:17:57,160 FAMILIES AS YOU'VE HEARD FROM 4373 03:17:57,160 --> 03:17:59,680 DR. DECKER AND OTHERS IS 4374 03:17:59,680 --> 03:18:02,400 SIGNIFICANT, CLINICIANS AND 4375 03:18:02,400 --> 03:18:03,960 FAMILIES SEEKING BETTER WAYS TO 4376 03:18:03,960 --> 03:18:08,480 BRING COMFORT TO THESE 4377 03:18:08,480 --> 03:18:10,520 INDIVIDUALS. 4378 03:18:10,520 --> 03:18:12,720 4379 03:18:12,720 --> 03:18:14,640 IT BRIEFLY RECAP WHAT LED US TO 4380 03:18:14,640 --> 03:18:18,480 THE NOTICE OF SPECIAL INTEREST, 4381 03:18:18,480 --> 03:18:20,480 AS YOU KNOW, RESPIRATORY 4382 03:18:20,480 --> 03:18:22,520 COMPLICATION LEADS TO MORTALITY 4383 03:18:22,520 --> 03:18:25,600 AND MORBIDITY, SIGNIFICANT 4384 03:18:25,600 --> 03:18:26,800 IMPACT ON HEALTH-RELATED QUALITY 4385 03:18:26,800 --> 03:18:27,560 OF LIFE. 4386 03:18:27,560 --> 03:18:29,440 SOME OF THESE DISEASES ARE 4387 03:18:29,440 --> 03:18:31,520 MENTIONED ON THE SLIDE, AS YOU 4388 03:18:31,520 --> 03:18:33,720 CAN SEE, BUT BY NO MEANS 4389 03:18:33,720 --> 03:18:37,960 EXHAUSTIVE, THERE ARE MANY 4390 03:18:37,960 --> 03:18:39,720 OTHERS. 4391 03:18:39,720 --> 03:18:46,080 WE FOUND ASSOCIATED PATHO 4392 03:18:46,080 --> 03:18:48,840 BIOLOGY INCLUDES DISORDERS THAT 4393 03:18:48,840 --> 03:18:50,040 AFFECT MUSCULOSKELETAL STRUCTURE 4394 03:18:50,040 --> 03:18:53,200 OF THE THORAX LEADING TO 4395 03:18:53,200 --> 03:18:55,440 PREDISPOSITION FOR RESTRICTIVE 4396 03:18:55,440 --> 03:18:56,800 LUNG DISEASE AND COLLAPSE. 4397 03:18:56,800 --> 03:19:04,280 THIS GRAPH ON THE LEFT OF YOUR 4398 03:19:04,280 --> 03:19:06,240 SLIDES SHOWS THE COMPLICATIONS 4399 03:19:06,240 --> 03:19:06,880 ACCOUNT FOR SIGNIFICANT 4400 03:19:06,880 --> 03:19:09,520 PERCENTAGE OF THE CAUSES OF 4401 03:19:09,520 --> 03:19:11,280 DEATH IN A VARIETY OF 4402 03:19:11,280 --> 03:19:16,640 CONDITIONS, AND THIS APPLIES TO 4403 03:19:16,640 --> 03:19:21,120 MANY MORE. 4404 03:19:21,120 --> 03:19:23,200 THIS LEADS TO SIGNIFICANT STRESS 4405 03:19:23,200 --> 03:19:26,360 AND BURDEN ON PATIENTS GIVEN A 4406 03:19:26,360 --> 03:19:30,720 PATIENT WITH ARDS WOULD BE AWARE 4407 03:19:30,720 --> 03:19:33,720 FOR MANY YEARS PRIOR TO THAT 4408 03:19:33,720 --> 03:19:35,920 EVENT OF THE DIFFICULTY IN 4409 03:19:35,920 --> 03:19:37,760 BREATHING AND LIMITATION OF 4410 03:19:37,760 --> 03:19:41,560 THEIR BREATHING AS MR. DECKER 4411 03:19:41,560 --> 03:19:44,480 ELOQUENTLY DESCRIBED EARLIER IN 4412 03:19:44,480 --> 03:19:45,400 THE SESSION. 4413 03:19:45,400 --> 03:19:48,040 THIS IS BROUGHT HOME BY 4414 03:19:48,040 --> 03:19:50,600 INTERVENTIONS THAT ESCALATE FROM 4415 03:19:50,600 --> 03:19:52,400 NON-INVASIVE VENTILATION TO 4416 03:19:52,400 --> 03:19:55,840 MECHANICAL VENTILATION, AND THE 4417 03:19:55,840 --> 03:19:59,640 USE OF COUGH ASSIST DEVICES AS 4418 03:19:59,640 --> 03:20:00,960 INDIVIDUALS GET WEAKER WITH 4419 03:20:00,960 --> 03:20:02,920 WORSE ABILITY TO CLEAR 4420 03:20:02,920 --> 03:20:04,000 SECRETIONS. 4421 03:20:04,000 --> 03:20:07,000 THIS IS RELATED TO UPPER AIRWAY, 4422 03:20:07,000 --> 03:20:09,560 REMAINING IN MOST OF THESE 4423 03:20:09,560 --> 03:20:12,120 CONDITIONS, ALSO AFFECT SLEEP. 4424 03:20:12,120 --> 03:20:14,760 AND COULD CONTRIBUTE 4425 03:20:14,760 --> 03:20:15,320 SIGNIFICANTLY TO OBSTRUCTIVE 4426 03:20:15,320 --> 03:20:16,360 SLEEP APNEA. 4427 03:20:16,360 --> 03:20:18,320 THE OTHER RESEARCH QUESTIONS 4428 03:20:18,320 --> 03:20:21,240 THAT WE WONDERED ABOUT WERE 4429 03:20:21,240 --> 03:20:22,560 DIFFERENCES IN TRAJECTORIES OF 4430 03:20:22,560 --> 03:20:24,600 DIFFERENT CONDITIONS AS WELL AS 4431 03:20:24,600 --> 03:20:27,400 DIFFERENCES IN THE TRAJECTORY OF 4432 03:20:27,400 --> 03:20:29,040 PROGRESSION OF DISEASE IN THE 4433 03:20:29,040 --> 03:20:33,720 SAME CONDITION, THIS SLIDE 4434 03:20:33,720 --> 03:20:36,480 DEMONSTRATES HOW ONE PATIENT IS 4435 03:20:36,480 --> 03:20:39,640 DIFFERENT WITH ANOTHER WITH THE 4436 03:20:39,640 --> 03:20:41,280 SAME DIAGNOSIS OF DUCHENNE 4437 03:20:41,280 --> 03:20:41,720 MESHED. 4438 03:20:41,720 --> 03:20:43,120 SEVERAL PRESSING KNOWLEDGE GAPS 4439 03:20:43,120 --> 03:20:46,080 WERE IDENTIFIED, AND I'LL ONLY 4440 03:20:46,080 --> 03:20:47,600 HIGHLIGHT A FEW, WHICH INCLUDED 4441 03:20:47,600 --> 03:20:51,480 IMPACT OF GENE THERAPY ON 4442 03:20:51,480 --> 03:20:52,400 RESPIRATORY FUNCTION ON ANIMAL 4443 03:20:52,400 --> 03:20:55,720 MODELS AND IN VITRO MODELS, 4444 03:20:55,720 --> 03:20:57,800 UPPER AIRWAYS OBSTRUCTION AND 4445 03:20:57,800 --> 03:21:00,480 IMPORTANCE OF SLEEP APNEA IN 4446 03:21:00,480 --> 03:21:01,520 DYSTROPHIES, OPTIMAL PRIMARY 4447 03:21:01,520 --> 03:21:06,240 FUNCTION TESTING IN SETTING OF 4448 03:21:06,240 --> 03:21:07,360 WEAKNESS, AND THERAPIES, 4449 03:21:07,360 --> 03:21:08,440 INCLUDING ASSESSMENT OF OUTCOMES 4450 03:21:08,440 --> 03:21:10,920 WHICH HAS ALSO BEEN DISCUSSED IN 4451 03:21:10,920 --> 03:21:13,480 THE SESSION TODAY, AND LASTLY 4452 03:21:13,480 --> 03:21:15,200 THE STUDY OF LONGITUDINAL 4453 03:21:15,200 --> 03:21:18,280 COHORTS TO UNDERSTAND THE 4454 03:21:18,280 --> 03:21:21,320 REASONS FOR AND VARIATIONS IN 4455 03:21:21,320 --> 03:21:25,400 THE TRAJECTORY OF PULMONARY 4456 03:21:25,400 --> 03:21:26,480 DECLINE. 4457 03:21:26,480 --> 03:21:28,800 SO, FOLKS AT THE NIH TOOK ALL 4458 03:21:28,800 --> 03:21:30,800 THE RECOMMENDATIONS OF THE 4459 03:21:30,800 --> 03:21:32,080 COMMITTEE VERY SERIOUSLY AND WE 4460 03:21:32,080 --> 03:21:38,080 PUT THEM TOGETHER TO BRING OUT A 4461 03:21:38,080 --> 03:21:42,160 NOTICE OF SPECIAL INTEREST, 4462 03:21:42,160 --> 03:21:46,520 ISSUED ON JULY 18, 2022, ON 4463 03:21:46,520 --> 03:21:52,000 RESPIRATORY COMPLICATIONS, 4464 03:21:52,000 --> 03:21:54,240 ISSUED BY THE NHLBI, EUNICE 4465 03:21:54,240 --> 03:21:55,280 KENNEDY SHRIVER INSTITUTE, 4466 03:21:55,280 --> 03:21:56,840 NATIONAL INSTITUTE OF NEUROLOGIC 4467 03:21:56,840 --> 03:21:58,680 DISEASE AND STROKE. 4468 03:21:58,680 --> 03:22:01,600 AND HERE IS THE LINK THAT I CAN 4469 03:22:01,600 --> 03:22:03,400 ALSO PROVIDE TO THE ORGANIZERS 4470 03:22:03,400 --> 03:22:05,680 TO SHARE WITH YOU. 4471 03:22:05,680 --> 03:22:09,720 THE FIRST AVAILABLE DUE DATE FOR 4472 03:22:09,720 --> 03:22:12,040 APPLICATION IS SEPTEMBER 25, 4473 03:22:12,040 --> 03:22:15,720 2022, AND NOSI WILL GO ON UNTIL 4474 03:22:15,720 --> 03:22:19,360 JANUARY 8, 2025, GIVING 4475 03:22:19,360 --> 03:22:20,520 INVESTIGATORS ABOUT A LITTLE 4476 03:22:20,520 --> 03:22:23,200 LESS THAN 3 YEARS TO BE ABLE TO 4477 03:22:23,200 --> 03:22:26,000 SUBMIT APPLICATIONS TO THIS 4478 03:22:26,000 --> 03:22:26,240 NOTICE. 4479 03:22:26,240 --> 03:22:28,000 THE MECHANISM UNDER WHICH 4480 03:22:28,000 --> 03:22:30,960 INVESTIGATORS CAN APPLY IS THE 4481 03:22:30,960 --> 03:22:33,720 CLASSIC NIH RESEARCH PROJECT 4482 03:22:33,720 --> 03:22:35,000 GRANT, PARENT R01, CLINICAL 4483 03:22:35,000 --> 03:22:36,400 TRIALS ARE NOT ALLOWED UNDER 4484 03:22:36,400 --> 03:22:39,040 THIS NOTICE OF SPECIAL INTEREST, 4485 03:22:39,040 --> 03:22:40,440 HOWEVER HUMAN SUBJECT RESEARCH 4486 03:22:40,440 --> 03:22:43,440 THAT IS LIMITED TO OBSERVATIONAL 4487 03:22:43,440 --> 03:22:47,240 STUDIES AND ALSO TO ANSWER 4488 03:22:47,240 --> 03:22:49,200 MECHANISTIC QUESTIONS IN HUMANS 4489 03:22:49,200 --> 03:22:51,160 ARE PERMITTED. 4490 03:22:51,160 --> 03:22:54,120 SOME RESEARCH EXAMPLES ARE SHOWN 4491 03:22:54,120 --> 03:22:56,120 ON THE SLIDE, AND MANY ISSUES 4492 03:22:56,120 --> 03:22:59,840 ARE ALREADY TALKED ABOUT. 4493 03:22:59,840 --> 03:23:03,480 BUT TO SORT OF SUMMARIZE THE 4494 03:23:03,480 --> 03:23:05,520 THEME OF THESE, THEY ARE REALLY 4495 03:23:05,520 --> 03:23:08,200 TO DRAW ATTENTION OF SCIENTISTS 4496 03:23:08,200 --> 03:23:10,440 AND FUNDED CENTERS, UNIVERSITY 4497 03:23:10,440 --> 03:23:13,920 CENTERS, TO INCREASE PRIMARY 4498 03:23:13,920 --> 03:23:16,400 RESEARCH EXPERTISE, TO INCREASE 4499 03:23:16,400 --> 03:23:17,520 COLLABORATION FOR HOLISTIC 4500 03:23:17,520 --> 03:23:19,200 ANIMAL MODEL RESEARCH BY THOSE 4501 03:23:19,200 --> 03:23:21,040 INVOLVED IN TESTING THERAPIES, 4502 03:23:21,040 --> 03:23:22,800 TO INCLUDE IMPACT ON RESPIRATORY 4503 03:23:22,800 --> 03:23:24,520 FUNCTION ON MUSCLES SUCH AS 4504 03:23:24,520 --> 03:23:26,520 DIAPHRAGM THAT HAS ALREADY BEEN 4505 03:23:26,520 --> 03:23:34,760 ALLUDED TO BEFORE, AS ONE OF THE 4506 03:23:34,760 --> 03:23:36,920 LARGEST SKELETAL MUSCLES, MOST 4507 03:23:36,920 --> 03:23:38,680 IMPORTANT EFFECT ON BREATHING 4508 03:23:38,680 --> 03:23:40,400 MITIGATION INTERVENTIONS IN 4509 03:23:40,400 --> 03:23:42,480 THESE POPULATIONS, SUCH THAT 4510 03:23:42,480 --> 03:23:43,920 WHEN THERE ARE NEW THERAPIES 4511 03:23:43,920 --> 03:23:46,360 AVAILABLES THE LUNGS ARE IN 4512 03:23:46,360 --> 03:23:47,880 THEIR BEST POSSIBLE CONDITION TO 4513 03:23:47,880 --> 03:23:49,960 BE ABLE TO BENEFIT FROM THEM. 4514 03:23:49,960 --> 03:23:52,400 IT IS OUR HOPE THIS WILL MAKE A 4515 03:23:52,400 --> 03:23:55,680 TANGIBLE DIFFERENCE TO THE LIVES 4516 03:23:55,680 --> 03:23:59,400 OF THOSE AFFECTED WITH THESE 4517 03:23:59,400 --> 03:24:00,480 CHALLENGING CONDITIONS. 4518 03:24:00,480 --> 03:24:02,760 OUR HOPE FOR THIS NOSI SPEAKING 4519 03:24:02,760 --> 03:24:04,440 MORE PRACTICALLY AND FOR THE 4520 03:24:04,440 --> 03:24:06,600 FIELD IS WE HOPE THIS WILL 4521 03:24:06,600 --> 03:24:08,560 ENCOURAGE SCIENTISTS IN THE 4522 03:24:08,560 --> 03:24:10,880 SPACE OF THE MUSCULAR 4523 03:24:10,880 --> 03:24:20,280 DYSTROPHIES TO INVESTIGATE 4524 03:24:20,280 --> 03:24:21,600 RESPIRATORY BREATHING, AND 4525 03:24:21,600 --> 03:24:22,840 SCIENTISTS ATTRACTED TO 4526 03:24:22,840 --> 03:24:23,680 INVESTIGATE NEUROMUSCULAR 4527 03:24:23,680 --> 03:24:24,960 DISEASES. 4528 03:24:24,960 --> 03:24:27,280 WE BELIEVE THE 4529 03:24:27,280 --> 03:24:28,920 CROSS-COLLABORATION BETWEEN 4530 03:24:28,920 --> 03:24:30,800 DISCIPLINES WILL BRING SYNERGY, 4531 03:24:30,800 --> 03:24:32,200 WHERE THE WHOLE IS GREATER THAN 4532 03:24:32,200 --> 03:24:35,520 THE SUM OF THE PARTS PROVIDING 4533 03:24:35,520 --> 03:24:36,800 GREATER BENEFIT TO OUR PATIENTS. 4534 03:24:36,800 --> 03:24:40,960 IT IS OUR HOPE THIS RESEARCH 4535 03:24:40,960 --> 03:24:44,800 THAT HELPS PATIENTS REMAIN 4536 03:24:44,800 --> 03:24:45,560 OPTIMAL PRIMARY HEALTH, 4537 03:24:45,560 --> 03:24:46,880 ULTIMATELY INCREASING POTENTIAL 4538 03:24:46,880 --> 03:24:52,600 FOR THEM TO BENEFIT FROM FUTURE 4539 03:24:52,600 --> 03:24:53,440 INNOVATIVE THERAPIES WHEN THEY 4540 03:24:53,440 --> 03:24:53,640 ARISE. 4541 03:24:53,640 --> 03:24:56,560 WITH THAT I'D BE HAPPY TO TAKE 4542 03:24:56,560 --> 03:24:57,040 QUESTIONS. 4543 03:24:57,040 --> 03:25:01,000 THANK YOU. 4544 03:25:01,000 --> 03:25:02,040 >>THANK YOU, ARUNA. 4545 03:25:02,040 --> 03:25:04,480 QUESTIONS? 4546 03:25:04,480 --> 03:25:06,480 4547 03:25:06,480 --> 03:25:11,320 I BELIEVE THAT GLEN PUT THE NOSI 4548 03:25:11,320 --> 03:25:20,160 LINK IN THE CHAT BOX. 4549 03:25:20,160 --> 03:25:30,640 >>I'VE GOT A QUESTION. 4550 03:25:43,960 --> 03:25:46,280 >>GO AHEAD, DAN. 4551 03:25:46,280 --> 03:25:56,800 >>LET ME TRY TO MOVE IT, IT 4552 03:26:00,000 --> 03:26:09,280 SEEMS TO BE STUCK. 4553 03:26:09,280 --> 03:26:19,720 >>IN TERMS OF CARDIAC, 4554 03:26:21,320 --> 03:26:21,600 (INDISCERNIBLE). 4555 03:26:21,600 --> 03:26:24,600 >>THIS PARTICULAR CHART, THE 4556 03:26:24,600 --> 03:26:30,520 NUMBER THEY STUDIED, WHICH WAS 4557 03:26:30,520 --> 03:26:32,480 21, AND 164 DUCHENNE PATIENTS, I 4558 03:26:32,480 --> 03:26:37,200 THINK IN THE NUMBER THAT THEY 4559 03:26:37,200 --> 03:26:38,400 STUDIED. 4560 03:26:38,400 --> 03:26:38,720 >>OKAY. 4561 03:26:38,720 --> 03:26:41,560 THAT SEEMS LIKE A LOT FOR 4562 03:26:41,560 --> 03:26:42,800 DUCHENNE, A LOT OF CANCER, WHICH 4563 03:26:42,800 --> 03:26:45,360 IS INTERESTING. 4564 03:26:45,360 --> 03:26:48,760 4565 03:26:48,760 --> 03:26:50,880 4566 03:26:50,880 --> 03:26:55,760 THIS IS IN ORDER OF -- OKAY. 4567 03:26:55,760 --> 03:27:01,120 I'M TRYING TO UNDERSTAND. 4568 03:27:01,120 --> 03:27:03,920 >>SURE. 4569 03:27:03,920 --> 03:27:04,240 >>OKAY. 4570 03:27:04,240 --> 03:27:06,040 THANK YOU AGAIN. 4571 03:27:06,040 --> 03:27:09,240 NEXT UP IS DR. EMILY CARIFI, 4572 03:27:09,240 --> 03:27:11,320 PROGRAM DIRECTOR FOR MUSCLE 4573 03:27:11,320 --> 03:27:14,360 DISORDERS AND THERAPIES AT 4574 03:27:14,360 --> 03:27:14,920 NIAMS. 4575 03:27:14,920 --> 03:27:15,720 SHE'S HERE TO GIVE A BRIEF 4576 03:27:15,720 --> 03:27:20,080 UPDATE ON THE NIH CENTERS OF 4577 03:27:20,080 --> 03:27:23,960 EXCELLENCE PROGRAM IN MUSCULAR 4578 03:27:23,960 --> 03:27:25,040 DYSTROPHIES, THE WELLSTONE 4579 03:27:25,040 --> 03:27:25,360 CENTERS. 4580 03:27:25,360 --> 03:27:25,600 WELCOME. 4581 03:27:25,600 --> 03:27:26,880 >>I DO KNOW I STAND BETWEEN YOU 4582 03:27:26,880 --> 03:27:32,240 AND THE END OF THE MEETING SO 4583 03:27:32,240 --> 03:27:33,360 I'LL GO QUICKLY. 4584 03:27:33,360 --> 03:27:38,160 I WANTED TO GIVE A BRIEF UPDATE 4585 03:27:38,160 --> 03:27:40,400 ON THE SPECIALIZED RESEARCH 4586 03:27:40,400 --> 03:27:41,240 CENTERS. 4587 03:27:41,240 --> 03:27:42,640 THIS IS PARTICULARLY TIMELY 4588 03:27:42,640 --> 03:27:45,480 BECAUSE TODAY IS THE DUE DATE 4589 03:27:45,480 --> 03:27:47,040 FOR THE MOST RECENT RFA, I 4590 03:27:47,040 --> 03:27:48,680 SUPPOSE IF YOU HAVEN'T WRITTEN 4591 03:27:48,680 --> 03:27:53,040 YOUR APPLICATION YOU'RE A BIT 4592 03:27:53,040 --> 03:27:56,920 LATE. 4593 03:27:56,920 --> 03:27:58,440 SO, PRETTY MUCH EVERYONE IN THE 4594 03:27:58,440 --> 03:28:00,840 ROOM, THE ZOOM HERE, KNOWS THE 4595 03:28:00,840 --> 03:28:02,480 HISTORY OF THE WELLSTONE 4596 03:28:02,480 --> 03:28:04,720 CENTERS, CREATED BY THE NIH IN 4597 03:28:04,720 --> 03:28:09,000 RESPONSE TO THE M.D. CARE ACT OF 4598 03:28:09,000 --> 03:28:10,800 2001, IN 2003, THEY REACHED A 4599 03:28:10,800 --> 03:28:17,880 STEADY STATE OF SIX CENTERS IN 4600 03:28:17,880 --> 03:28:22,360 2005, FUNDED BY NIAMS, NINDS, 4601 03:28:22,360 --> 03:28:25,360 NICHD, CO-FOUNDED BY NHLBI. 4602 03:28:25,360 --> 03:28:31,080 THE SIX CENTERS ARE STUDYING ALL 4603 03:28:31,080 --> 03:28:35,640 OF THE DISEASES LISTED AND HAVE 4604 03:28:35,640 --> 03:28:37,840 A SPREAD FROM BASIC PRE-CLINICAL 4605 03:28:37,840 --> 03:28:42,640 AND CLINICAL RESEARCH, BECAUSE 4606 03:28:42,640 --> 03:28:46,240 MANY OF THE PILLARS OF THE MDCC 4607 03:28:46,240 --> 03:28:50,760 IN 2005 AND 2015 ACTION PLANS, 4608 03:28:50,760 --> 03:28:51,640 DISEASE MECHANISM, BIOMARKERS, 4609 03:28:51,640 --> 03:28:54,920 ALL THE WAY TO THE CLINIC. 4610 03:28:54,920 --> 03:28:57,560 THIS PROGRAM WAS EVALUATED IN 4611 03:28:57,560 --> 03:28:59,640 2018 AND THE RESULTS OF THAT 4612 03:28:59,640 --> 03:29:02,360 EVALUATION WERE PRESENTED TO 4613 03:29:02,360 --> 03:29:06,760 THIS COMMITTEE IN 2019, IT WAS 4614 03:29:06,760 --> 03:29:08,520 AN OVERWHELMINGLY POSITIVE 4615 03:29:08,520 --> 03:29:10,480 EVALUATION, AND SUPPORTIVE OF 4616 03:29:10,480 --> 03:29:14,040 THE CENTERS PROGRAM AND 4617 03:29:14,040 --> 03:29:16,320 DIRECTION OF THE INVESTIGATORS. 4618 03:29:16,320 --> 03:29:19,240 THE CURRENT SIX CENTERS AS I 4619 03:29:19,240 --> 03:29:23,160 MENTIONED, ALL THE DISEASE AREAS 4620 03:29:23,160 --> 03:29:25,120 FROM REALLY BASIC ASPECTS OF 4621 03:29:25,120 --> 03:29:35,680 DISEASE MECHANISM TO THE CLINIC, 4622 03:29:36,320 --> 03:29:37,240 CAMPBELL, EMERSON, CHAMBERLAIN, 4623 03:29:37,240 --> 03:29:41,360 SWEENEY, WE REALLY DO HAVE A 4624 03:29:41,360 --> 03:29:43,560 WIDE RANGE GEOGRAPHICALLY AND 4625 03:29:43,560 --> 03:29:47,360 ALSO THE ENTIRE DISEASE, THEY 4626 03:29:47,360 --> 03:29:50,200 ARE VERY HAPPY WITH THE PROGRAM. 4627 03:29:50,200 --> 03:29:52,520 THIS TOOK ME A LONG TIME, IT 4628 03:29:52,520 --> 03:29:55,160 KEPT GETTING TOO BIG, I GOT IT 4629 03:29:55,160 --> 03:29:55,720 ON ONE SLIDE. 4630 03:29:55,720 --> 03:29:59,200 SOME OF THESE ALREADY MENTIONED 4631 03:29:59,200 --> 03:29:59,800 TODAY. 4632 03:29:59,800 --> 03:30:01,840 RESEARCH COMING OUT OF THESE 4633 03:30:01,840 --> 03:30:04,360 GROUPS IS JUST ENTIRELY TOP 4634 03:30:04,360 --> 03:30:08,280 NOTCH, BREAKING THE GROUND FOR 4635 03:30:08,280 --> 03:30:10,360 THERAPEUTICS, FOR UNDERSTANDING 4636 03:30:10,360 --> 03:30:11,680 DISEASE, FOR OUTCOME MEASURES, 4637 03:30:11,680 --> 03:30:14,960 BIOMARKERS, ANYTHING TO MOVE THE 4638 03:30:14,960 --> 03:30:19,520 FIELD FORWARD TO FUTURE 4639 03:30:19,520 --> 03:30:21,960 THERAPEUTIC PATIENT CARE. 4640 03:30:21,960 --> 03:30:23,600 THE OTHER ASPECTS OF THE 4641 03:30:23,600 --> 03:30:25,800 WELLSTONE CENTERS, IT'S THE FACT 4642 03:30:25,800 --> 03:30:28,560 THAT THEY HAVE RESEARCHERS THAT 4643 03:30:28,560 --> 03:30:30,200 THEY HAVE A MANDATE TO SHARE 4644 03:30:30,200 --> 03:30:32,840 WITH THE COMMUNITY AND WITH 4645 03:30:32,840 --> 03:30:34,400 THEIR OWN INVESTIGATORS. 4646 03:30:34,400 --> 03:30:37,360 AND THESE RESOURCES, AS I 4647 03:30:37,360 --> 03:30:40,920 MENTIONED, ARE AVAILABLE TO THE 4648 03:30:40,920 --> 03:30:41,560 ENTIRE MUSCULAR DYSTROPHY 4649 03:30:41,560 --> 03:30:44,960 COMMUNITY, SHARED VERY WELL BY 4650 03:30:44,960 --> 03:30:45,360 THE CENTERS. 4651 03:30:45,360 --> 03:30:49,360 SO, A LOT OF THESE THINGS MIGHT 4652 03:30:49,360 --> 03:30:50,680 BE HARDER FOR ANY ONE 4653 03:30:50,680 --> 03:30:58,840 INVESTIGATOR TO BE ABLE TO HAVE 4654 03:30:58,840 --> 03:31:01,280 OR TO FIND, AND ANIMAL MODELS 4655 03:31:01,280 --> 03:31:03,800 AND THE EXPERTISE TO DO EDITING, 4656 03:31:03,800 --> 03:31:06,960 TO DO TESTING ON EXISTING MODELS 4657 03:31:06,960 --> 03:31:08,840 TO CREATE NEW MODELS SO, AGAIN, 4658 03:31:08,840 --> 03:31:14,640 THE IDEA HERE IS TO CREATE A 4659 03:31:14,640 --> 03:31:17,000 WELL CURATED AND EXCELLENT 4660 03:31:17,000 --> 03:31:22,360 REPOSITORY FOR THE COMMUNITY AND 4661 03:31:22,360 --> 03:31:23,920 LEVEL OF EXPERTISE FOR THE 4662 03:31:23,920 --> 03:31:25,240 ENTIRE COMMUNITY. 4663 03:31:25,240 --> 03:31:27,760 THEY ALSO HAVE A MANDATE TO 4664 03:31:27,760 --> 03:31:30,280 CLEAN UP THE NEXT GENERATION OF 4665 03:31:30,280 --> 03:31:31,560 MUSCULAR DYSTROPHY RESEARCHERS, 4666 03:31:31,560 --> 03:31:36,240 SO THEY DO THIS IN TWO MAJOR 4667 03:31:36,240 --> 03:31:38,440 WAYS, ONE IN THE SPREADING OF 4668 03:31:38,440 --> 03:31:40,840 KNOWLEDGE, SO A LOT OF TIMES 4669 03:31:40,840 --> 03:31:45,440 THIS HAPPENS IN THE SEMINARS AND 4670 03:31:45,440 --> 03:31:47,480 INTERACTIVE COURSES, THEY DO A 4671 03:31:47,480 --> 03:31:51,320 LOT OF CROSS TRAINING, HAVING 4672 03:31:51,320 --> 03:31:53,400 PhDs IN THE CLINIC, M.D.s 4673 03:31:53,400 --> 03:31:59,640 IN THE LAB, LEADSSHIP TRAINING, 4674 03:31:59,640 --> 03:32:01,080 DEDICATED FUNDS FOR TRAINEES AND 4675 03:32:01,080 --> 03:32:04,120 MANY OF THESE TRAINEES HAVE GONE 4676 03:32:04,120 --> 03:32:07,960 ON TO SUCCESSFUL CAREERS, 4677 03:32:07,960 --> 03:32:11,560 SEVERAL OF THEM AGAIN I CUT OUT 4678 03:32:11,560 --> 03:32:14,760 THE SLIDES, I WAS GIVEN TEN 4679 03:32:14,760 --> 03:32:16,680 MINUTES, I HAVE PAGES OF NAMES, 4680 03:32:16,680 --> 03:32:20,760 THOSE WHO MOVED TO FACULTY, WHO 4681 03:32:20,760 --> 03:32:24,080 HAVE R01s, IT'S BEEN A GOOD 4682 03:32:24,080 --> 03:32:25,520 PIPELINE INTO THE DYSTROPHY 4683 03:32:25,520 --> 03:32:25,920 COMMUNITY. 4684 03:32:25,920 --> 03:32:28,760 THE FUTURE DIRECTIONS FOR THE 4685 03:32:28,760 --> 03:32:31,320 CENTER AS I MENTIONED THIS IS A 4686 03:32:31,320 --> 03:32:33,120 CENTER THAT'S BEEN AROUND 20 4687 03:32:33,120 --> 03:32:35,000 YEARS NOW. 4688 03:32:35,000 --> 03:32:36,320 WELL RESPECTED PROGRAM. 4689 03:32:36,320 --> 03:32:39,920 LIKE I SAID, THE EVALUATION IN 4690 03:32:39,920 --> 03:32:43,640 2018 WAS SUPPORTIVE BUT WE HAVE 4691 03:32:43,640 --> 03:32:46,720 IN PARTICULAR IN THE MOST RECENT 4692 03:32:46,720 --> 03:32:48,360 RFA STARTED TO MAKE SOME MINOR 4693 03:32:48,360 --> 03:32:51,520 CHANGES TO THE PROGRAM, JUST TO 4694 03:32:51,520 --> 03:32:54,720 HIGHLIGHT SOME PRIORITIES OF THE 4695 03:32:54,720 --> 03:33:02,920 NIH AND BASED ON THE COMMENTS IN 4696 03:33:02,920 --> 03:33:04,160 THE EVALUATION. 4697 03:33:04,160 --> 03:33:07,520 SO, IN THE RFA THERE IS AN 4698 03:33:07,520 --> 03:33:09,920 ENCOURAGEMENT OF SOME NIH 4699 03:33:09,920 --> 03:33:13,120 PRIORITIES INCLUDING RESEARCH 4700 03:33:13,120 --> 03:33:16,480 AREAS, INCLUDING PULMONARY 4701 03:33:16,480 --> 03:33:17,040 ASPECTS. 4702 03:33:17,040 --> 03:33:21,080 WE'RE ALSO TRYING TO CONTINUE TO 4703 03:33:21,080 --> 03:33:22,080 GROW THESE LEADERS OF THE 4704 03:33:22,080 --> 03:33:23,080 MUSCULAR DYSTROPHY COMMUNITY 4705 03:33:23,080 --> 03:33:30,080 HERE SO WE'RE ENCOURAGING THAT 4706 03:33:30,080 --> 03:33:31,520 NEW INVESTIGATORS LEAD 4707 03:33:31,520 --> 03:33:33,760 SCIENTIFIC PROJECTS AND GET 4708 03:33:33,760 --> 03:33:36,760 THEIR SKILLS TO THEIR OWN LABS 4709 03:33:36,760 --> 03:33:37,600 IN THE FUTURE. 4710 03:33:37,600 --> 03:33:39,360 THE GOAL HERE LIKE I SAID IS TO 4711 03:33:39,360 --> 03:33:41,680 REALLY INCREASE THE NUMBER OF 4712 03:33:41,680 --> 03:33:45,040 LEADERS THAT WE HAVE, AND ALSO 4713 03:33:45,040 --> 03:33:49,640 THE NIH COMMITMENT TO INCLUSION, 4714 03:33:49,640 --> 03:33:50,560 DIVERSITY, EQUITY. 4715 03:33:50,560 --> 03:33:54,480 BUT OUR GOAL HERE IS TO FOCUS ON 4716 03:33:54,480 --> 03:33:56,320 THE PATIENT COMMUNITY, MAKING 4717 03:33:56,320 --> 03:34:00,080 SURE THAT THESE CENTERS ARE 4718 03:34:00,080 --> 03:34:01,360 ABLE, USING RESOURCES WE'RE 4719 03:34:01,360 --> 03:34:05,120 GIVEN THEM TO REALLY REACH OUT 4720 03:34:05,120 --> 03:34:14,160 TO THOSE WHO ARE HISTORICALLY 4721 03:34:14,160 --> 03:34:15,000 UNDERREPRESENTED IN BIOMEDICAL. 4722 03:34:15,000 --> 03:34:17,920 THERE HAS TO BE STRONG AND 4723 03:34:17,920 --> 03:34:19,960 INCLUSIVE PLANS FOR ALL CLINICAL 4724 03:34:19,960 --> 03:34:26,240 PARTICIPANTS, AND ALSO AS I 4725 03:34:26,240 --> 03:34:27,960 MENTIONED TRAINEES, WHILE WE 4726 03:34:27,960 --> 03:34:33,240 DON'T -- THIS PROGRAM ISN'T -- 4727 03:34:33,240 --> 03:34:35,840 THE GOAL IS TO ENHANCE 4728 03:34:35,840 --> 03:34:36,960 RECRUITMENT OF TRAINEES SO 4729 03:34:36,960 --> 03:34:38,720 PEOPLE WHO STUDY PATIENTS LOOK 4730 03:34:38,720 --> 03:34:40,680 LIKE THE PATIENTS. 4731 03:34:40,680 --> 03:34:44,560 SO, AGAIN, I MENTIONED THE RFA 4732 03:34:44,560 --> 03:34:47,760 WAS DONE TODAY, THE PROGRAM IS 4733 03:34:47,760 --> 03:34:50,080 MOVING FORWARD, HOPEFULLY IN A 4734 03:34:50,080 --> 03:34:50,520 WONDERFUL DIRECTION. 4735 03:34:50,520 --> 03:34:51,080 THANK YOU. 4736 03:34:51,080 --> 03:34:53,360 I CAN TAKE ANY QUESTIONS IF 4737 03:34:53,360 --> 03:34:55,680 NEEDED. 4738 03:34:55,680 --> 03:34:59,640 4739 03:34:59,640 --> 03:35:04,680 >>DAN PEREZ AGAIN. 4740 03:35:04,680 --> 03:35:14,120 >>GO AHEAD, DAN. 4741 03:35:14,120 --> 03:35:19,240 >>THE QUESTION I HAVE, THE 4742 03:35:19,240 --> 03:35:25,400 CENTERS HAVE BEEN FUNDED, 4743 03:35:25,400 --> 03:35:30,640 WELLSTONE CENTERS, SINCE 2003, 4744 03:35:30,640 --> 03:35:33,440 FUNDING STATE CONSISTENT, I 4745 03:35:33,440 --> 03:35:35,960 REALIZE NOW REQUIRING TWO 4746 03:35:35,960 --> 03:35:37,480 SUBJECTS INSTEAD OF THREE, THERE 4747 03:35:37,480 --> 03:35:44,280 ARE SOME CHANGES IN TERMS OF NOT 4748 03:35:44,280 --> 03:35:46,920 HAVING THE FUNDS FOR THE 4749 03:35:46,920 --> 03:35:48,760 POSTDOCTORAL TRAIN, IS THERE A 4750 03:35:48,760 --> 03:35:52,720 WAY TO LOOK AT INCREASING THE 4751 03:35:52,720 --> 03:35:54,760 AMOUNT OF FUNDING PER CENTER? 4752 03:35:54,760 --> 03:36:05,320 BECAUSE THEY ARE LOSING -- WE'RE 4753 03:36:20,440 --> 03:36:22,560 LOSING AGAINST INFLATION, AND A 4754 03:36:22,560 --> 03:36:29,800 PROJECT WAS PUT TOGETHER, ALL 4755 03:36:29,800 --> 03:36:31,560 THE REQUIREMENTS, IS THERE ANY 4756 03:36:31,560 --> 03:36:41,400 FLEXIBILITY THERE, IS THERE 4757 03:36:41,400 --> 03:36:43,680 ANY -- DO YOU CHOOSE ANOTHER 4758 03:36:43,680 --> 03:36:54,240 MECHANISM, DO YOU CREATE ANOTHER 4759 03:36:57,760 --> 03:36:59,200 CLASS OF USES? 4760 03:36:59,200 --> 03:37:09,680 THE QUESTION IS HOW CAN WE 4761 03:37:17,920 --> 03:37:21,320 INCREASE THAT. 4762 03:37:21,320 --> 03:37:23,400 >>YEAH, SO, DAN, THANK YOU FOR 4763 03:37:23,400 --> 03:37:25,600 POSING THAT QUESTION. 4764 03:37:25,600 --> 03:37:27,040 YEAH, THERE'S A LOT WRAPPED IN 4765 03:37:27,040 --> 03:37:27,320 THERE. 4766 03:37:27,320 --> 03:37:31,080 I'M NOT SURE I FOLLOWED 4767 03:37:31,080 --> 03:37:31,960 EVERYTHING, BUT PART OF -- ONE 4768 03:37:31,960 --> 03:37:33,720 OF THE POINTS YOU RAISE IS 4769 03:37:33,720 --> 03:37:35,520 SOMETHING THAT WE'RE CHALLENGED 4770 03:37:35,520 --> 03:37:36,880 WITH GENERALLY, WHICH IS THAT 4771 03:37:36,880 --> 03:37:39,720 THE COST OF DOING RESEARCH IS 4772 03:37:39,720 --> 03:37:41,000 INCREASING FASTER THAN THE 4773 03:37:41,000 --> 03:37:43,640 SUPPORT THAT WE RECEIVED TO DO 4774 03:37:43,640 --> 03:37:46,200 RESEARCH, THAT'S A GENERAL 4775 03:37:46,200 --> 03:37:47,400 CHALLENGE, INVESTIGATORS FACE 4776 03:37:47,400 --> 03:37:49,680 THAT CHALLENGE, INSTITUTES AND 4777 03:37:49,680 --> 03:37:51,640 CENTERS FACE THAT CHALLENGE. 4778 03:37:51,640 --> 03:37:53,640 YOU KNOW, ONE OF THE WAYS WE 4779 03:37:53,640 --> 03:37:55,800 LOOK TO, YOU KNOW, ENSURE THAT 4780 03:37:55,800 --> 03:37:59,640 THE DOLLARS GO FURTHER IS BY 4781 03:37:59,640 --> 03:38:02,040 TRYING TO LEVERAGE OTHER 4782 03:38:02,040 --> 03:38:02,880 RESOURCES FUNDING IN-KIND 4783 03:38:02,880 --> 03:38:05,000 RESOURCES, SO I THINK SOMETIMES 4784 03:38:05,000 --> 03:38:07,400 THAT'S WHERE CENTERS CAN BE MORE 4785 03:38:07,400 --> 03:38:08,720 EFFICIENT IF THERE ARE 4786 03:38:08,720 --> 03:38:12,200 INSTITUTIONAL OR OTHER LEVERAGES 4787 03:38:12,200 --> 03:38:16,160 THAT CAN BE LEVERAGED, TO 4788 03:38:16,160 --> 03:38:18,240 SUPPORT THE RESEARCH, AND OF 4789 03:38:18,240 --> 03:38:22,600 COURSE ONE OF THE BENEFITS OF 4790 03:38:22,600 --> 03:38:23,600 FOCUSING ON TRAINING AND 4791 03:38:23,600 --> 03:38:24,800 EARLY-STAGE INVESTIGATORS IS SO 4792 03:38:24,800 --> 03:38:26,200 THAT THEY WILL DEVELOP INTEREST 4793 03:38:26,200 --> 03:38:27,520 AND COMMITMENT IN THESE AREAS 4794 03:38:27,520 --> 03:38:29,600 AND PROPOSE THEIR OWN STUDIES 4795 03:38:29,600 --> 03:38:35,600 THROUGH THE R01 OR OTHER 4796 03:38:35,600 --> 03:38:35,920 MECHANISMS. 4797 03:38:35,920 --> 03:38:38,960 IN TERMS OF RECEIVING MORE MONEY 4798 03:38:38,960 --> 03:38:41,520 FROM -- IN THE NIH BUDGETS FOR 4799 03:38:41,520 --> 03:38:42,160 THE WELLSTONE CENTER OR MUSCULAR 4800 03:38:42,160 --> 03:38:44,680 DYSTROPHY I DON'T KNOW THAT 4801 03:38:44,680 --> 03:38:48,520 THAT'S A QUESTION WE CAN TACKLE 4802 03:38:48,520 --> 03:38:50,840 IN THIS MEETING BUT I THINK OF 4803 03:38:50,840 --> 03:38:52,880 COURSE THE ADVOCACY THAT YOU AND 4804 03:38:52,880 --> 03:38:56,720 EVERYBODY ELSE PARTICIPATING IN 4805 03:38:56,720 --> 03:38:58,600 THIS MEETING PROVIDES IS ONE OF 4806 03:38:58,600 --> 03:39:02,880 THE MOST EFFECTIVE WAYS TO TRY 4807 03:39:02,880 --> 03:39:04,600 TO ENSURE THERE'S ONGOING 4808 03:39:04,600 --> 03:39:05,720 ATTENTION TO THESE CHALLENGES, 4809 03:39:05,720 --> 03:39:08,600 YOU KNOW, THE GAPS AS WELL AS 4810 03:39:08,600 --> 03:39:09,320 THE OPPORTUNITIES. 4811 03:39:09,320 --> 03:39:12,360 LET ME JUST PAUSE THERE AND SEE 4812 03:39:12,360 --> 03:39:14,480 IF OTHER -- IF DIANA OR WALTER 4813 03:39:14,480 --> 03:39:18,960 WANT TO ADD TO WHAT I HAD TO SAY 4814 03:39:18,960 --> 03:39:21,200 FROM THEIR PERSPECTIVES. 4815 03:39:21,200 --> 03:39:22,640 WALTER CAME OFF MUTE. 4816 03:39:22,640 --> 03:39:24,000 >>WELL, DAN, THE ANSWER IS 4817 03:39:24,000 --> 03:39:24,880 ALWAYS YES. 4818 03:39:24,880 --> 03:39:29,240 THEY COULD USE MORE MONEY. 4819 03:39:29,240 --> 03:39:35,320 THE ISSUE IS, AS LINDSEY PUT IT, 4820 03:39:35,320 --> 03:39:37,480 THIS APPLIES TO ALMOST EVERY 4821 03:39:37,480 --> 03:39:40,280 GRANT MECHANISM WE HAVE. 4822 03:39:40,280 --> 03:39:45,360 FOR INSTANCE, THE MODULAR R01, 4823 03:39:45,360 --> 03:39:48,040 HAS BEEN $250,000 FOR 20 YEARS, 4824 03:39:48,040 --> 03:39:49,240 IT HASN'T CHANGED, IT'S A BAD 4825 03:39:49,240 --> 03:39:51,400 THING TO KEEP UP WITH INFLATION, 4826 03:39:51,400 --> 03:39:57,440 BUT THE ONLY THING WE CAN DO, 4827 03:39:57,440 --> 03:39:58,560 WE'RE BALANCING ADDING MONEY TO, 4828 03:39:58,560 --> 03:40:01,640 YOU KNOW, X AND THAT MEANS WE 4829 03:40:01,640 --> 03:40:08,720 FUND FEWER GRANTS. 4830 03:40:08,720 --> 03:40:09,920 THE GENERAL CENSUS, IT'S 4831 03:40:09,920 --> 03:40:19,880 PROBABLY HEALTHY TO KEEP MORE 4832 03:40:19,880 --> 03:40:21,400 PEOPLE IN THE GAME, THE WORST 4833 03:40:21,400 --> 03:40:22,720 THING IS TO LOSE SMART PEOPLE, 4834 03:40:22,720 --> 03:40:26,080 BUT THIS IS SOMETHING WE 4835 03:40:26,080 --> 03:40:28,120 STRUGGLE WITH ALL THE TIME. 4836 03:40:28,120 --> 03:40:30,200 AND THE ONLY THING WE CAN -- 4837 03:40:30,200 --> 03:40:33,800 UNLESS WE GET MORE FUNDS, WE RUN 4838 03:40:33,800 --> 03:40:37,000 INTO THIS PROBLEM. 4839 03:40:37,000 --> 03:40:39,520 OUR PERCENTILE NOW FOR GETTING A 4840 03:40:39,520 --> 03:40:41,600 GRANT IS 14 PERCENTILE, 86% OF 4841 03:40:41,600 --> 03:40:46,160 PEOPLE DON'T GET THEIR GRANTS 4842 03:40:46,160 --> 03:40:46,400 FUNDED. 4843 03:40:46,400 --> 03:40:51,440 >>I'M TALKING ABOUT THE 4844 03:40:51,440 --> 03:41:01,280 WELLSTONE CENTERS, PREMIER 4845 03:41:01,280 --> 03:41:02,840 CENTERS OF NIH. 4846 03:41:02,840 --> 03:41:07,000 TOP DOCTORS IN THE FIELD. 4847 03:41:07,000 --> 03:41:09,160 WE'RE IN THE GOING TO GO IN 4848 03:41:09,160 --> 03:41:14,760 BECAUSE BY THE TIME WE END UP 4849 03:41:14,760 --> 03:41:21,000 NINE DIFFERENT WAYS, WE CAN'T 4850 03:41:21,000 --> 03:41:31,320 GET ANYTHING DONE. 4851 03:41:37,400 --> 03:41:40,920 ARE YOU NO LONGER INTERESTED 4852 03:41:40,920 --> 03:41:43,240 BECAUSE IT'S JUST NOT WORTH YOUR 4853 03:41:43,240 --> 03:41:43,720 WHILE? 4854 03:41:43,720 --> 03:41:44,640 >>THAT'S A GOOD POINT. 4855 03:41:44,640 --> 03:41:46,280 >>IT'S NOT A GOOD PLACE TO BE. 4856 03:41:46,280 --> 03:41:48,920 >>I'D LIKE TO ALSO THROW A 4857 03:41:48,920 --> 03:41:50,560 PUBLIC/PRIVATE PARTNERSHIP INTO 4858 03:41:50,560 --> 03:41:54,080 THE MIX, SOME SITUATIONS THE 4859 03:41:54,080 --> 03:41:56,560 ONLY, YOU KNOW, I MEAN IT'S A 4860 03:41:56,560 --> 03:41:58,120 ZERO SUM GAIN FOR EVERY 4861 03:41:58,120 --> 03:41:59,320 INSTITUTE AND WE'RE TRYING TO DO 4862 03:41:59,320 --> 03:42:03,160 AS BEST AS WE CAN TO BALANCE 4863 03:42:03,160 --> 03:42:04,720 MULTIPLE PRIORITIES. 4864 03:42:04,720 --> 03:42:06,440 SO THAT'S WHERE ORGANIZATIONS 4865 03:42:06,440 --> 03:42:08,640 LIKE THE FOUNDATION FOR NIH COME 4866 03:42:08,640 --> 03:42:14,520 IN, WHO CAN ARC STRAIGHT THESE 4867 03:42:14,520 --> 03:42:15,080 PUBLIC/PRIVATE PARTNERSHIPS. 4868 03:42:15,080 --> 03:42:17,480 AND THINK THAT THE NIH FUNDING, 4869 03:42:17,480 --> 03:42:20,320 IT'S SO PRESTIGIOUS AND IT'S 4870 03:42:20,320 --> 03:42:23,400 GONE THROUGH MULTIPLE LAYERS OF 4871 03:42:23,400 --> 03:42:26,120 REVIEW, THAT THAT CAN SOMETIMES 4872 03:42:26,120 --> 03:42:29,840 HELP TO ATTRACT ADDITIONAL 4873 03:42:29,840 --> 03:42:30,840 PRIVATE FUNDING. 4874 03:42:30,840 --> 03:42:32,280 IT'S NOT OPTIMAL BUT AT LEAST 4875 03:42:32,280 --> 03:42:33,160 IT'S SOMETHING. 4876 03:42:33,160 --> 03:42:37,000 YOU NOTICE THAT IN MANY 4877 03:42:37,000 --> 03:42:39,920 PRESENTATIONS WE SAW THAT GROUPS 4878 03:42:39,920 --> 03:42:44,280 ARE BEING FUNDED INCREASINGLY BY 4879 03:42:44,280 --> 03:42:46,520 COALITIONS OF FOUNDATION, 4880 03:42:46,520 --> 03:42:47,920 INDUSTRY, AND NIH FUNDING. 4881 03:42:47,920 --> 03:42:50,040 >>I WOULD ADD A GREAT EXAMPLE 4882 03:42:50,040 --> 03:42:52,720 ALONG THOSE LINES IS THE SUCCESS 4883 03:42:52,720 --> 03:42:54,160 OF THE ACCELERATING MEDICINES 4884 03:42:54,160 --> 03:42:55,280 PARTNERSHIP PROGRAMS. 4885 03:42:55,280 --> 03:42:56,680 I DON'T RECALL OFFHAND THE 4886 03:42:56,680 --> 03:42:59,840 PROPORTION OF FUNDING THAT COMES 4887 03:42:59,840 --> 03:43:02,520 FROM NIH INSTITUTES VERSUS 4888 03:43:02,520 --> 03:43:03,720 INDUSTRY AND NON-PROFIT 4889 03:43:03,720 --> 03:43:04,680 ORGANIZATIONS, BUT THE NIH 4890 03:43:04,680 --> 03:43:08,920 DOLLARS GO A VERY LONG WAY IN 4891 03:43:08,920 --> 03:43:10,280 THAT ENVIRONMENT, WHERE, YOU 4892 03:43:10,280 --> 03:43:13,120 KNOW, THERE ARE PARTNERSHIPS AND 4893 03:43:13,120 --> 03:43:22,800 IN THAT, THAT'S ANOTHER EXAMPLE 4894 03:43:22,800 --> 03:43:24,360 WHERE FRO SERVES A CRITICAL 4895 03:43:24,360 --> 03:43:26,160 LIAISING FUNCTION. 4896 03:43:26,160 --> 03:43:27,680 >>THANK YOU, LINDSEY. 4897 03:43:27,680 --> 03:43:30,040 SO WITH THAT, I THINK WE ARE 4898 03:43:30,040 --> 03:43:32,480 GOING TO MOVE TOWARDS CLOSING. 4899 03:43:32,480 --> 03:43:34,400 IT'S ALMOST 5:00. 4900 03:43:34,400 --> 03:43:36,360 I WOULD LIKE TO THANK OUR 4901 03:43:36,360 --> 03:43:38,920 SPEAKERS WHO ARE REALLY 4902 03:43:38,920 --> 03:43:39,880 OUTSTANDING TODAY. 4903 03:43:39,880 --> 03:43:42,520 I WAS REALLY IMPRESSED WITH SOME 4904 03:43:42,520 --> 03:43:45,800 OF THE NOVEL TECHNOLOGIES THAT 4905 03:43:45,800 --> 03:43:49,440 HAVE BEEN DEVELOPED, AS A 4906 03:43:49,440 --> 03:43:51,400 NEONATOLOGIST I REALLY WAS 4907 03:43:51,400 --> 03:43:51,840 IMPRESSED. 4908 03:43:51,840 --> 03:43:57,240 I WAS IMPRESSED BY ALL OF THEM 4909 03:43:57,240 --> 03:43:58,880 BUT DR. LOWES PRESENTATION WAS 4910 03:43:58,880 --> 03:44:00,480 NEWBORNS, AS A CLINICIAN I WAS 4911 03:44:00,480 --> 03:44:02,680 TRYING TO GUESS WHICH WAS THE 4912 03:44:02,680 --> 03:44:04,000 AFFECTED INFANT, I REALLY 4913 03:44:04,000 --> 03:44:04,520 COULDN'T. 4914 03:44:04,520 --> 03:44:06,720 BUT IT WAS PRETTY OBVIOUS WITH 4915 03:44:06,720 --> 03:44:08,320 THE PATTERNS THAT WERE GENERATED 4916 03:44:08,320 --> 03:44:10,680 BY THE NEW TECHNOLOGY. 4917 03:44:10,680 --> 03:44:12,440 SO THAT'S ONE OF MANY OF THE 4918 03:44:12,440 --> 03:44:16,240 EXCITING THINGS WE HEARD ABOUT 4919 03:44:16,240 --> 03:44:16,720 TODAY. 4920 03:44:16,720 --> 03:44:18,720 I'D LIKE TO THANK DR. NUCKOLLS 4921 03:44:18,720 --> 03:44:22,600 AND HIS STAFF FOR HELPING US TO 4922 03:44:22,600 --> 03:44:24,560 PUT TOGETHER THIS PROGRAM, WE'RE 4923 03:44:24,560 --> 03:44:26,760 WORKING ON BOTH THE TIMING AND 4924 03:44:26,760 --> 03:44:28,760 THE AGENDA FOR THE SPRING 4925 03:44:28,760 --> 03:44:29,280 MEETING. 4926 03:44:29,280 --> 03:44:32,040 I THINK WE'RE GOING TO OFFER A 4927 03:44:32,040 --> 03:44:34,440 HYBRID MEETING BUT STAY TUNED ON 4928 03:44:34,440 --> 03:44:34,760 THIS. 4929 03:44:34,760 --> 03:44:37,600 COULD YOU HAVE THE OPTION BE 4930 03:44:37,600 --> 03:44:42,200 BEING IN PERSON OR BEING ON 4931 03:44:42,200 --> 03:44:42,760 VIDEO. 4932 03:44:42,760 --> 03:44:46,280 PROBABLY MORE OR LESS THE SAME 4933 03:44:46,280 --> 03:44:49,440 TIMING, FOUR-HOUR MEETING ON A 4934 03:44:49,440 --> 03:44:50,560 SPRING AFTERNOON LIKELY. 4935 03:44:50,560 --> 03:44:52,000 GLEN, DID YOU HAVE ANYTHING ELSE 4936 03:44:52,000 --> 03:44:53,120 YOU WANTED TO ADD? 4937 03:44:53,120 --> 03:44:54,680 >>I'D LIKE TO ALSO SAY THANKS 4938 03:44:54,680 --> 03:45:00,280 TO ALL THE PRESENTERS WE HAD 4939 03:45:00,280 --> 03:45:00,480 TODAY. 4940 03:45:00,480 --> 03:45:01,120 GREAT PRESENTATIONS. 4941 03:45:01,120 --> 03:45:06,520 THANKS TO COMMITTEE MEMBERS AND 4942 03:45:06,520 --> 03:45:09,720 THANKS TO YOU, DR. BIANCHI, FOR 4943 03:45:09,720 --> 03:45:13,200 KEEPING US ON TIME AND A GREAT 4944 03:45:13,200 --> 03:45:13,680 MEETING. 4945 03:45:13,680 --> 03:45:17,040 WE'RE LOOKING FOR MARCH, APRIL, 4946 03:45:17,040 --> 03:45:22,000 OR MAY FOR FOUR-HOUR AFTERNOON 4947 03:45:22,000 --> 03:45:22,680 MEETING, I'LL BE CONTACTING 4948 03:45:22,680 --> 03:45:25,120 COMMITTEE MEMBERS TO IDENTIFY 4949 03:45:25,120 --> 03:45:28,520 THAT SPECIFIC DATE. 4950 03:45:28,520 --> 03:45:36,640 >>YEAH. 4951 03:45:36,640 --> 03:45:41,000 I THANK IT'S COMMITTEE MEMBERS. 4952 03:45:41,000 --> 03:45:43,320 ONE MORE POP QUIZ BEFORE WE SIGN 4953 03:45:43,320 --> 03:45:45,560 OFF, TOTAL NUMBER OF NOBEL 4954 03:45:45,560 --> 03:45:47,880 PRIZES, WHAT PERCENT HAVE BEEN 4955 03:45:47,880 --> 03:45:49,080 GIVEN TO WOMEN? 4956 03:45:49,080 --> 03:45:53,680 THESE ARE THE ONES IN SCIENCE. 4957 03:45:53,680 --> 03:45:55,640 ANYBODY WANT TO TAKE A GUESS? 4958 03:45:55,640 --> 03:45:59,240 TOO BAD WE DON'T HAVE POLLS. 4959 03:45:59,240 --> 03:45:59,640 >>FIVE. 4960 03:45:59,640 --> 03:46:02,800 >>SEVEN PERCENT. 4961 03:46:02,800 --> 03:46:03,440 >>CLOSE. 4962 03:46:03,440 --> 03:46:06,280 IT'S ACTUALLY UP TO 10% NOW, SO 4963 03:46:06,280 --> 03:46:07,760 IT'S A LITTLE BETTER, BUT WE 4964 03:46:07,760 --> 03:46:17,880 COULD DO BETTER IN THE FUTURE.. 4965 03:46:17,880 --> 03:46:20,720 ON THAT NOTE I SEE FLOATING 4966 03:46:20,720 --> 03:46:23,040 APPLAUSE HERE. 4967 03:46:23,040 --> 03:46:24,560 THANK YOU ALL. 4968 03:46:24,560 --> 03:46:26,360 HAVE A GREAT HOLIDAY SEASON. 4969 03:46:26,360 --> 03:46:32,360 THE END OF THE SUMMER, GOES BY 4970 03:46:32,360 --> 03:46:33,600 SO FAST, SUDDENLY THANKSGIVING. 4971 03:46:33,600 --> 03:46:35,320 HAPPY HOLIDAYS. 4972 03:46:35,320 --> 03:46:36,280 STAY SAFE. 4973 03:46:36,280 --> 03:46:39,040 GET YOUR BOOSTERS IF YOU HAVEN'T 4974 03:46:39,040 --> 03:46:40,560 GOTTEN THEM ALREADY, FLU SHOT, 4975 03:46:40,560 --> 00:00:00,000 WE'LL SEE YOU IN THE SPRING.