1 00:00:05,960 --> 00:00:09,600 HI, EVERYBODY, SO I'M GLEN 2 00:00:09,600 --> 00:00:10,880 NUCKOLIS AND I'M THE DESIGNATED 3 00:00:10,880 --> 00:00:12,600 FEDERAL OFFICIALS FOR THE 4 00:00:12,600 --> 00:00:13,720 MUSCULAR DYSTROPHY COORDINATING 5 00:00:13,720 --> 00:00:16,680 COMMCOORDINATECOMMITTEE. 6 00:00:16,680 --> 00:00:18,240 THIS IS OUR 28th MEETING OF 7 00:00:18,240 --> 00:00:19,640 THIS COMMITTEE. 8 00:00:19,640 --> 00:00:20,920 LET'S START BY DOING 9 00:00:20,920 --> 00:00:22,520 INTRODUCTIONS OF THE COMMITTEE 10 00:00:22,520 --> 00:00:26,000 MEMBERS AND I'LL DO A ROLL CALL 11 00:00:26,000 --> 00:00:30,160 STARTING WITH DIANA BIANCHI. 12 00:00:30,160 --> 00:00:30,760 >>Diana Bianchi: HELLO, 13 00:00:30,760 --> 00:00:32,320 EVERYBODY, I'M SIGNING IN 14 00:00:32,320 --> 00:00:33,040 REMOTELY FROM MASSACHUSETTS. 15 00:00:33,040 --> 00:00:34,520 I AM THE DIRECTOR OF THE 16 00:00:34,520 --> 00:00:35,840 NATIONAL INSTITUTE OF CHILD 17 00:00:35,840 --> 00:00:40,560 HEALTH AND HUMAN DEVELOPMENT AND 18 00:00:40,560 --> 00:00:42,560 HAPPY TO REPRESENT OUR IC ON 19 00:00:42,560 --> 00:00:45,800 THIS COMMITTEE. 20 00:00:45,800 --> 00:00:46,840 >>Glen Nuckolis: THANK YOU. 21 00:00:46,840 --> 00:00:52,240 NEXT IS IT THERESA AN AUGUSTINE. 22 00:00:52,240 --> 00:00:55,440 >>CAN YOU HEAR ME? 23 00:00:55,440 --> 00:00:55,960 >>Glen Nuckolis: YES. 24 00:00:55,960 --> 00:00:58,880 >>I'M HEARING IMPAIRED. 25 00:00:58,880 --> 00:01:02,280 I'M THERESA SAN AUGUSTINE FROM 26 00:01:02,280 --> 00:01:04,200 THE INSTITUTE OF DISABILITY, 27 00:01:04,200 --> 00:01:06,240 INDEPENDENT LIVING AND WE HAVE A 28 00:01:06,240 --> 00:01:06,520 RESEARCH. 29 00:01:06,520 --> 00:01:08,720 I WANTED TO SAY A LITTLE THING 30 00:01:08,720 --> 00:01:11,680 THAT I ADMIRE ALL THE WORK YOU 31 00:01:11,680 --> 00:01:13,400 HAVE DONE VERY WELL AND IT MAY 32 00:01:13,400 --> 00:01:18,240 BE MY LAST MEETING SO I WOULD 33 00:01:18,240 --> 00:01:21,680 HAVE INSTITUTE TO REPRESENT THIS 34 00:01:21,680 --> 00:01:22,840 COMMITTEE. 35 00:01:22,840 --> 00:01:25,680 AND OF COURSE, OUR MOTHER 36 00:01:25,680 --> 00:01:28,360 INSTITUTE IS THE ADMINISTRATION 37 00:01:28,360 --> 00:01:30,400 FOR COMMUNITY LIVING. 38 00:01:30,400 --> 00:01:33,680 SO, THANK YOU EVERYONE FOR THE 39 00:01:33,680 --> 00:01:34,800 RESEARCH THAT YOU HAVE DONE IN 40 00:01:34,800 --> 00:01:37,480 THE PAST. 41 00:01:37,480 --> 00:01:38,680 >>Glen Nuckolis: THANK YOU, 42 00:01:38,680 --> 00:01:39,600 THERESA AND THANK YOU FOR ALL 43 00:01:39,600 --> 00:01:41,360 THE WORK THAT YOU'VE DONE ON THE 44 00:01:41,360 --> 00:01:42,720 COMMITTEE OVER QUITE A NUMBER OF 45 00:01:42,720 --> 00:01:43,320 YEARS. 46 00:01:43,320 --> 00:01:45,320 WE REALLY APPRECIATE YOUR 47 00:01:45,320 --> 00:01:48,200 VALUABLE CONTRIBUTIONS. 48 00:01:48,200 --> 00:01:54,960 NEXT ON THE COMMITTEE IS CHRIS 49 00:01:54,960 --> 00:01:55,200 ANSELMO. 50 00:01:55,200 --> 00:01:57,320 >>HI, EVERYONE, I'M A MARKET 51 00:01:57,320 --> 00:01:59,240 INTELLIGENCE DIRECTOR AT THE 52 00:01:59,240 --> 00:01:59,840 MUSCULAR DYSTROPHY ASSOCIATION 53 00:01:59,840 --> 00:02:03,640 AND I'M ALSO A MEMBER OF THE MDA 54 00:02:03,640 --> 00:02:03,920 COMMUNITY. 55 00:02:03,920 --> 00:02:06,800 AND IT'S WONDERFUL TO BE WITH 56 00:02:06,800 --> 00:02:10,280 YOU TODAY. 57 00:02:10,280 --> 00:02:12,800 >>Glen Nuckolis: THANK YOU. 58 00:02:12,800 --> 00:02:15,000 NEXT OUR MEMBER LINDSEY CRISWELL 59 00:02:15,000 --> 00:02:17,320 IS NOT ABLE TO JOIN TODAY BUT 60 00:02:17,320 --> 00:02:18,480 REPRESENTING THE INSTITUTE IS 61 00:02:18,480 --> 00:02:21,560 BOB CARTER. 62 00:02:21,560 --> 00:02:23,680 >>GOOD AFTERNOON, GLAD TO BE 63 00:02:23,680 --> 00:02:25,640 JOINING YOU AND LOOK FORWARD TO 64 00:02:25,640 --> 00:02:28,240 HEARING THE PROGRESS. 65 00:02:28,240 --> 00:02:28,960 THANK YOU. 66 00:02:28,960 --> 00:02:30,600 >>Glen Nuckolis: THANK YOU, 67 00:02:30,600 --> 00:02:31,920 BOB. 68 00:02:31,920 --> 00:02:36,080 GUSTAVO DZIEWCZAPOLSKI. 69 00:02:36,080 --> 00:02:40,040 >>HELLO EVERYONE. 70 00:02:40,040 --> 00:02:47,480 SCIENTIFIC DIRECTOR AT THE 71 00:02:47,480 --> 00:02:48,480 ORGANIZATION QCMD FOR MUSCULAR 72 00:02:48,480 --> 00:02:48,800 DYSTROPHIES. 73 00:02:48,800 --> 00:02:54,800 >>Glen Nuckolis: THANK YOU. 74 00:02:54,800 --> 00:02:58,120 EMILY CARILLI. 75 00:02:58,120 --> 00:03:00,720 >>I'M A PEDIATRIC NUR OLE LIST 76 00:03:00,720 --> 00:03:02,760 OF THE DIVISION OF NEWER OLELY 77 00:03:02,760 --> 00:03:06,240 AT THE FDA. 78 00:03:06,240 --> 00:03:10,000 >>Glen Nuckolis: THANK YOU. 79 00:03:10,000 --> 00:03:10,600 JEAN. 80 00:03:10,600 --> 00:03:11,520 >>MEDICAL OFFICER WITH THE 81 00:03:11,520 --> 00:03:12,840 CENTERS FOR MEDICARE AND 82 00:03:12,840 --> 00:03:16,360 MEDICAID SERVICES. 83 00:03:16,360 --> 00:03:19,720 >>Glen Nuckolis: THANK YOU. 84 00:03:19,720 --> 00:03:25,520 MICHAEL GOLDSTEIN. 85 00:03:25,520 --> 00:03:25,800 >>MICHAEL. 86 00:03:25,800 --> 00:03:28,480 >>HEY, EVERYBODY, THIS IS 87 00:03:28,480 --> 00:03:29,160 MICHAEL GOLDSTEIN A APOLOGIZE 88 00:03:29,160 --> 00:03:30,840 I'LL PLAYING WITH MY CAL RA AND 89 00:03:30,840 --> 00:03:33,240 I CAN'T GET IT ON BUT I'M FROM 90 00:03:33,240 --> 00:03:34,200 THE OBSERVE SECURITY 91 00:03:34,200 --> 00:03:35,320 ADMINISTRATION AND THE DIRECTOR 92 00:03:35,320 --> 00:03:47,240 -->>IF I HAVE TROUBLE WITH MY 93 00:04:04,520 --> 00:04:07,280 CAMERA I'LL JOIN MY BY MY PHONE. 94 00:04:07,280 --> 00:04:09,520 AND I CAN PUT ON MY CAMERA. 95 00:04:09,520 --> 00:04:14,480 >>Glen Nuckolls: THANK YOU. 96 00:04:14,480 --> 00:04:14,800 ALICIA KEEN. 97 00:04:14,800 --> 00:04:15,320 >>GOOD AFTERNOON. 98 00:04:15,320 --> 00:04:19,880 I'M THE GENETIC SERVICES BRANCH 99 00:04:19,880 --> 00:04:22,680 CHIEF RESOURCE AND SERVICES 100 00:04:22,680 --> 00:04:24,320 ADMINISTRATION. 101 00:04:24,320 --> 00:04:28,200 >>Glen Nuckolls: THANK YOU. 102 00:04:28,200 --> 00:04:28,480 JIM KYLIE. 103 00:04:28,480 --> 00:04:29,480 >>GOOD AFTERNOON, EVERYONE. 104 00:04:29,480 --> 00:04:31,280 GOOD TO SEE YOU. 105 00:04:31,280 --> 00:04:33,040 JIM KYLIE THE DIRECTOR OF THE 106 00:04:33,040 --> 00:04:34,600 DIVISION OF LUNG DISEASES AT THE 107 00:04:34,600 --> 00:04:38,200 NATIONAL HEART LUNG AND BLOOD 108 00:04:38,200 --> 00:04:38,720 INSTITUTE. 109 00:04:38,720 --> 00:04:45,160 >>Glen Nuckolls: THANK YOU. 110 00:04:45,160 --> 00:04:46,760 WALTER KOROSHETZ. 111 00:04:46,760 --> 00:04:47,440 >>GOOD AFTERNOON. 112 00:04:47,440 --> 00:04:49,840 I'M THE DIRECTOR OF THE NINDS. 113 00:04:49,840 --> 00:04:51,680 >>Glen Nuckolls: THANK YOU. 114 00:04:51,680 --> 00:04:54,680 HELLO, I'M MARIE' LENA MCGWIRE 115 00:04:54,680 --> 00:04:57,400 THE PROGRAM MANAGER FOR THE DUE 116 00:04:57,400 --> 00:04:58,600 SCHENN MUSCULAR DYSTROPHIES 117 00:04:58,600 --> 00:04:59,720 RESEARCH PROGRAM WHICH IS UNDER 118 00:04:59,720 --> 00:05:04,960 THE DEPARTMENT OF DEFENSE. 119 00:05:04,960 --> 00:05:06,120 >>Glen Nuckolls: DENISE MILLER. 120 00:05:06,120 --> 00:05:11,040 >>HI, DIN SEES WITH THE 121 00:05:11,040 --> 00:05:14,080 CAREGIVERS ALLIANCE AND I HELP 122 00:05:14,080 --> 00:05:15,520 IN IDAHO WITH OUR ALZHEIMER'S 123 00:05:15,520 --> 00:05:17,400 DISEASE AND RELATED DEMENTIA AND 124 00:05:17,400 --> 00:05:21,080 I'M A DISABILITY ADVOCATE AND 125 00:05:21,080 --> 00:05:24,120 WHO KNOWS WHAT ELSE I CAN GET 126 00:05:24,120 --> 00:05:24,800 INTO. 127 00:05:24,800 --> 00:05:26,000 >>Glen Nuckolls: THANK YOU, 128 00:05:26,000 --> 00:05:26,720 DENISE. 129 00:05:26,720 --> 00:05:30,160 DAN PEREZ. 130 00:05:30,160 --> 00:05:40,280 >>GOOD EVENING, GOOD DAY, 131 00:05:40,280 --> 00:05:41,000 EVERYONE. 132 00:05:41,000 --> 00:05:43,000 DAN PEREZ. 133 00:05:43,000 --> 00:05:51,760 I'M CO-FOUNDER AND BOARD 134 00:05:51,760 --> 00:05:53,240 DIRECTOR. 135 00:05:53,240 --> 00:05:58,960 AND OBVIOUSLY I'M A PATIENT 136 00:05:58,960 --> 00:06:01,240 JUSTICE AND LIFELONG ADVOCATE 137 00:06:01,240 --> 00:06:03,520 FOR RESEARCH IN MUSCULAR 138 00:06:03,520 --> 00:06:05,160 DYSTROPHY. 139 00:06:05,160 --> 00:06:06,680 THANK YOU. 140 00:06:06,680 --> 00:06:09,920 >>Glen Nuckolls: THANK YOU. 141 00:06:09,920 --> 00:06:10,960 NATALIE STREET. 142 00:06:10,960 --> 00:06:11,960 >>HI. 143 00:06:11,960 --> 00:06:14,600 MY NAME IS NATALIE STREET I'M A 144 00:06:14,600 --> 00:06:16,840 HEALTH SCIENTIST ON THE RARE 145 00:06:16,840 --> 00:06:18,000 DISORDERS AND HEALTH OUTCOMES 146 00:06:18,000 --> 00:06:20,320 TEAM WHICH HOUSES THE MUSCULAR 147 00:06:20,320 --> 00:06:20,880 DYSTROPHY ACTIVITIES AT THE 148 00:06:20,880 --> 00:06:23,600 CENTERS FOR DISEASE CONTROL IN 149 00:06:23,600 --> 00:06:24,840 PREVENTION. 150 00:06:24,840 --> 00:06:28,560 >>Glen Nuckolls: THANK YOU. 151 00:06:28,560 --> 00:06:28,840 ERIC WANG. 152 00:06:28,840 --> 00:06:30,840 >>HI, EVERYBODY, I'M ASSOCIATE 153 00:06:30,840 --> 00:06:32,320 PROFESSOR AT UNIVERSITY OF 154 00:06:32,320 --> 00:06:33,640 FLORIDA. 155 00:06:33,640 --> 00:06:39,240 I'M ALSO A COMMUNITY ADVOCATE 156 00:06:39,240 --> 00:06:39,720 FOR MUSCULAR DYSTROPHY. 157 00:06:39,720 --> 00:06:40,400 >>Glen Nuckolls: THANK YOU. 158 00:06:40,400 --> 00:06:41,520 THOSE ARE OUR COMMITTEE MEMBERS. 159 00:06:41,520 --> 00:06:42,880 WE HAVE A COUPLE OF OTHER PEOPLE 160 00:06:42,880 --> 00:06:44,200 HERE IN THE ROOM AND I WANT 161 00:06:44,200 --> 00:06:46,360 TODAY GO AROUND AND HAVE THEM 162 00:06:46,360 --> 00:06:47,400 INTRODUCE THEMSELVES. 163 00:06:47,400 --> 00:06:50,760 WE'LL START WITH EMILY CARIFI. 164 00:06:50,760 --> 00:06:52,240 >>I'M THE PROGRAM OFFICER FOR 165 00:06:52,240 --> 00:06:54,280 THE MUSCLE DISEASES AND 166 00:06:54,280 --> 00:06:58,760 THERAPIES PROGRAM AT NIAMS. 167 00:06:58,760 --> 00:07:01,000 >>HELLO EVERYBODY, I'M PAUL, 168 00:07:01,000 --> 00:07:02,440 VICE PRESIDENT OF PUBLIC POLICY 169 00:07:02,440 --> 00:07:04,760 AND ADVOCACY AT THE MUSCULAR 170 00:07:04,760 --> 00:07:06,040 DYSTROPHY ASSOCIATION. 171 00:07:06,040 --> 00:07:07,280 GRATEFUL TO BE HERE. 172 00:07:07,280 --> 00:07:10,480 >>HI, EVERYBODY, I'M MARIE 173 00:07:10,480 --> 00:07:12,640 PIERRE AND I'M A HEALTH PROGRAM 174 00:07:12,640 --> 00:07:17,120 SPECIALIST AT NINDS. 175 00:07:17,120 --> 00:07:19,600 >>HI, EVERYONE, MY NAME IS 176 00:07:19,600 --> 00:07:24,400 JUSTIN WILSON AND I'M A SCIENCE 177 00:07:24,400 --> 00:07:27,400 ANNALIST HERE IN NIAMS. 178 00:07:27,400 --> 00:07:30,280 >>I'LL GALE LESTER THE DIRECTOR 179 00:07:30,280 --> 00:07:34,080 OF EXTRAMURAL RESEARCH AT NIAMS. 180 00:07:34,080 --> 00:07:38,000 >>I'M KATE SENIOR DIRECTOR OF 181 00:07:38,000 --> 00:07:38,640 POLICY. 182 00:07:38,640 --> 00:07:39,440 >>Glen Nuckolls: THANK YOU, 183 00:07:39,440 --> 00:07:40,080 EVERYBODY. 184 00:07:40,080 --> 00:07:42,000 SO, YOU KNOW, AGAIN, WELCOME TO 185 00:07:42,000 --> 00:07:43,480 THE 28th MEETING OF THE 186 00:07:43,480 --> 00:07:44,280 MUSCULAR DYSTROPHY COORDINATING 187 00:07:44,280 --> 00:07:44,840 COMMITTEE. 188 00:07:44,840 --> 00:07:46,520 THIS MEETING IS BEING CONDUCTED 189 00:07:46,520 --> 00:07:56,960 IN COMPLIANCE WITH THE -- TO 190 00:07:57,560 --> 00:07:58,280 INFORM DECISION-MAKING BY 191 00:07:58,280 --> 00:07:59,760 OFFICIALS AND AGENCIES IN THE 192 00:07:59,760 --> 00:08:01,520 EXECUTIVE BRANCH IN THE MEDICAL 193 00:08:01,520 --> 00:08:02,800 GOVERNMENT IN A MANNER 194 00:08:02,800 --> 00:08:03,920 TRANSPARENT AND FREE OF 195 00:08:03,920 --> 00:08:04,560 CONFLICTS. 196 00:08:04,560 --> 00:08:06,440 THERE'S NO CLOSED SESSION 197 00:08:06,440 --> 00:08:07,840 PLANNED FOR TODAY'S MEETING. 198 00:08:07,840 --> 00:08:09,400 WE DON'T ANTICIPATE DISCUSSION 199 00:08:09,400 --> 00:08:12,240 OF ACTIONS SPECIFIC TO THE 200 00:08:12,240 --> 00:08:13,080 ORGANIZATIONS OR FINANCIAL 201 00:08:13,080 --> 00:08:14,400 INTEREST OF OUR MEMBERS. 202 00:08:14,400 --> 00:08:17,920 SO THERE'S NO NEED FOR ANY 203 00:08:17,920 --> 00:08:18,680 RECUSEALS. 204 00:08:18,680 --> 00:08:20,520 WE DID POST A NOTICE IN THE 205 00:08:20,520 --> 00:08:21,520 FEDERAL REGISTER ABOUT THIS 206 00:08:21,520 --> 00:08:26,520 MEETING AND WE INVITED PUBLIC 207 00:08:26,520 --> 00:08:28,240 COMMENTS WE DID NOT RECEIVE A 208 00:08:28,240 --> 00:08:29,920 REQUEST SO WE DID NOT SCHEDULE A 209 00:08:29,920 --> 00:08:30,640 PUBLIC COMMENT PERIOD DURING 210 00:08:30,640 --> 00:08:31,360 THIS MEETING. 211 00:08:31,360 --> 00:08:33,720 THE MEETING IS BEING STREAMED 212 00:08:33,720 --> 00:08:36,440 LIVE VIA THE NIH VIDEO CAST THAT 213 00:08:36,440 --> 00:08:39,440 IS VIDEO CAST.NIH.GOV AND IT'S 214 00:08:39,440 --> 00:08:43,600 ALSO RECORDED AND ARCHIVED AND 215 00:08:43,600 --> 00:08:50,920 WE PROVIDE LINCOL PROVIDE LINK . 216 00:08:50,920 --> 00:08:53,040 FOR THOSE WHO ARE CONNECTED 217 00:08:53,040 --> 00:08:54,800 THROUGH THE ZOOM, IF YOU ARE 218 00:08:54,800 --> 00:08:55,920 COMMITTEE MEMBER OR OTHER 219 00:08:55,920 --> 00:08:58,360 DESIGNATED PANELIST, YOU CAN 220 00:08:58,360 --> 00:09:01,080 JUST RAISE YOUR VIRTUAL HAND OR 221 00:09:01,080 --> 00:09:05,320 JUST UNMUTE AND ASK QUESTIONS 222 00:09:05,320 --> 00:09:06,280 AND CONTRIBUTE TO THE 223 00:09:06,280 --> 00:09:06,600 CONVERSATION. 224 00:09:06,600 --> 00:09:07,560 IF YOU ARE NOT A COMMITTEE 225 00:09:07,560 --> 00:09:09,480 MEMBER OR DESIGNATED PANELIST, 226 00:09:09,480 --> 00:09:11,760 THEN YOU CAN SUBMIT QUESTIONS 227 00:09:11,760 --> 00:09:14,240 VIA THE CHAT FEATURE AND WE'LL 228 00:09:14,240 --> 00:09:15,440 READ THOSE QUESTIONS OUT FOR 229 00:09:15,440 --> 00:09:15,800 YOU. 230 00:09:15,800 --> 00:09:17,800 FOR THOSE OF YOU HERE ON THE 231 00:09:17,800 --> 00:09:19,560 ROOM, AS YOU DID WHEN 232 00:09:19,560 --> 00:09:21,600 INTRODUCING YOURSELVES REMEMBER 233 00:09:21,600 --> 00:09:23,600 TO CLICK YOUR TABLE MICROPHONE 234 00:09:23,600 --> 00:09:25,400 TO SPEAK AND CLICK IT AGAIN TO 235 00:09:25,400 --> 00:09:26,240 TURN IT OFF. 236 00:09:26,240 --> 00:09:29,440 SO WE'RE CURRENTLY 17thING 237 00:09:29,440 --> 00:09:30,400 NOMINATIONS FOR PUBLIC MEMBERS 238 00:09:30,400 --> 00:09:35,680 TO THE MDCC FOR THE TERMS OF 239 00:09:35,680 --> 00:09:37,520 2024 THROUGH 2026 AND WE'LL POST 240 00:09:37,520 --> 00:09:39,560 IN THE CHAT A LINK TO THE 241 00:09:39,560 --> 00:09:41,120 FEDERAL REGISTER NOTICE THAT IS 242 00:09:41,120 --> 00:09:42,520 DESCRIBED THE NOMINATION 243 00:09:42,520 --> 00:09:42,960 PROCESS. 244 00:09:42,960 --> 00:09:50,920 AND NOMINATIONS MUST BE RECEIVED 245 00:09:50,920 --> 00:09:51,280 BY APRIL 28th. 246 00:09:51,280 --> 00:09:52,920 THANK YOU FOR THOSE WHO HELPED 247 00:09:52,920 --> 00:09:55,280 PREPARE FOR THIS MEETING. 248 00:09:55,280 --> 00:09:57,720 INCLUDING MARIE PEE I REMEMBER, 249 00:09:57,720 --> 00:10:01,200 ELLA BLUE, DERIC SMITH, EMILY, 250 00:10:01,200 --> 00:10:05,320 MELISSA PEREASY I'LL TURN IT 251 00:10:05,320 --> 00:10:10,040 OVER TO Dr. BIANCHI FOR HER 252 00:10:10,040 --> 00:10:10,280 REPORT. 253 00:10:10,280 --> 00:10:10,960 >>Diana Bianchi: THANK YOU SO 254 00:10:10,960 --> 00:10:13,720 MUCH, GLEN, AND THEN WELCOME TO 255 00:10:13,720 --> 00:10:14,560 EVERYONE. 256 00:10:14,560 --> 00:10:18,280 DERIC, MAY HAVE THE SLIDES, 257 00:10:18,280 --> 00:10:20,600 PLEASE. 258 00:10:20,600 --> 00:10:22,120 I'D LIKE TO ACKNOWLEDGE AT THE 259 00:10:22,120 --> 00:10:23,800 BEGINNING OF MY SLIDE 260 00:10:23,800 --> 00:10:25,880 PRESENTATION THE VERY HELPFUL 261 00:10:25,880 --> 00:10:29,520 INPUT OF BOTH GLEN AND ELIZABETH 262 00:10:29,520 --> 00:10:38,080 AND MELISSA PARECE. 263 00:10:38,080 --> 00:10:39,800 SO TODAY I'M GOING TO DISCUSS 264 00:10:39,800 --> 00:10:42,160 THE APPROPRIATION AND THE FISCAL 265 00:10:42,160 --> 00:10:44,880 YEAR 24 PRESIDENT'S BUDGET 266 00:10:44,880 --> 00:10:45,200 REQUEST. 267 00:10:45,200 --> 00:10:46,440 I'M GOING TO GIVE A LITTLE BIT 268 00:10:46,440 --> 00:10:52,200 OF AN OVER OVERVIEW OVER NIH PY 269 00:10:52,200 --> 00:10:54,200 UPDATES AND RESEARCH AND THEN 270 00:10:54,200 --> 00:10:57,760 JUST HIGHLIGHT THE NIH-WIDE 271 00:10:57,760 --> 00:10:59,080 STRATEGIC PLAN FOR DIVERSITY, 272 00:10:59,080 --> 00:11:00,560 EQUITY, INCLUSION AND 273 00:11:00,560 --> 00:11:03,480 IMPORTANTLY ACCESSIBILITY. 274 00:11:03,480 --> 00:11:04,920 AND THEN A FEW THINGS THAT YOU 275 00:11:04,920 --> 00:11:06,200 MIGHT BE INTERESTED IN WITH 276 00:11:06,200 --> 00:11:08,120 REGARDS TO THE ADVISORY 277 00:11:08,120 --> 00:11:10,520 COMMITTEE TO THE NIH DIRECTORS 278 00:11:10,520 --> 00:11:13,600 WORKING GROUP RECOMMENDATIONS. 279 00:11:13,600 --> 00:11:17,440 JUST A VERY BRIEF HIGHLIGHT ON 280 00:11:17,440 --> 00:11:17,840 ARPA-H. 281 00:11:17,840 --> 00:11:21,400 I KNOW I MENTIONED IT LAST TIME. 282 00:11:21,400 --> 00:11:22,800 AND WE'LL GET INTO THE 283 00:11:22,800 --> 00:11:24,840 HIGHLIGHTS OF THE ADVANCES OF 284 00:11:24,840 --> 00:11:25,440 MUSCULAR DYSTROPHY RESEARCH. 285 00:11:25,440 --> 00:11:26,280 NEXT SLIDE, PLEASE. 286 00:11:26,280 --> 00:11:29,440 SO MONEY. 287 00:11:29,440 --> 00:11:31,480 NEXT SLIDE. 288 00:11:31,480 --> 00:11:36,880 SO, WE ARE NOW WELL INTO FISCAL 289 00:11:36,880 --> 00:11:39,080 YEAR '23 AND THIS CURRENT YEAR 290 00:11:39,080 --> 00:11:44,240 THE TOTAL NIH BUDGET IS 291 00:11:44,240 --> 00:11:45,440 $47.5 BILLION WHICH WAS AN 292 00:11:45,440 --> 00:11:48,160 INCREASE OF $2.5 BILLION OVER 293 00:11:48,160 --> 00:11:50,640 FISCAL YEAR '22. 294 00:11:50,640 --> 00:11:53,720 THE FISCAL YEAR '24 PRESIDENT'S 295 00:11:53,720 --> 00:11:55,680 BUDGET REQUEST WAS RELEASED 296 00:11:55,680 --> 00:11:58,240 PUBLICLY ON MARCH 13th. 297 00:11:58,240 --> 00:12:02,920 THE REQUEST FOR NIH WAS 48.6 298 00:12:02,920 --> 00:12:06,200 WHICH WAS AN INCREASE OF O 299 00:12:06,200 --> 00:12:08,080 $0.9 BILLION AND FOR MOST 300 00:12:08,080 --> 00:12:12,880 INSTITUTES IN PROGRAMS, IT WAS 301 00:12:12,880 --> 00:12:16,760 ESSENTIALLY A GNAT BUDGET. 302 00:12:16,760 --> 00:12:18,160 NORMALLY, AROUND THIS TIME, WE 303 00:12:18,160 --> 00:12:19,240 WOULD PREPARE FOR HOUSE AND 304 00:12:19,240 --> 00:12:21,720 SENATE APPROPRIATIONS HEARINGS. 305 00:12:21,720 --> 00:12:24,160 THERE IS A NEW CHAIR OF THE 306 00:12:24,160 --> 00:12:30,720 HOUSE OF APPROPRIATIONS 307 00:12:30,720 --> 00:12:33,000 COMMITTEE AND THEY ARE TAKING A 308 00:12:33,000 --> 00:12:34,040 DIFFERENT APPROACH. 309 00:12:34,040 --> 00:12:35,600 WHEREAS THE DIRECTOR ATTENDED 310 00:12:35,600 --> 00:12:38,920 THE HOUSE APPROPRIATIONS 311 00:12:38,920 --> 00:12:41,680 HEARINGS FLANKED BY SEVERAL 312 00:12:41,680 --> 00:12:42,840 INSTITUTES DIRECTORS, THIS YEAR 313 00:12:42,840 --> 00:12:44,880 DR. TABAK IS GOING TO BE GOING 314 00:12:44,880 --> 00:12:52,120 IT ALONE BY REQUEST FROM THE 315 00:12:52,120 --> 00:12:53,400 HOUSE, THE SENATE MEETINGS ARE 316 00:12:53,400 --> 00:12:55,360 LIKE THEY'VE BEEN WITH ACTING 317 00:12:55,360 --> 00:12:59,560 NIH DIRECTOR DR. TABAK AND 318 00:12:59,560 --> 00:13:02,000 SEVERAL OF THE LARGER INSTITUTES 319 00:13:02,000 --> 00:13:03,080 DIRECTORS. 320 00:13:03,080 --> 00:13:10,040 LARGER INSTITUTES. 321 00:13:10,040 --> 00:13:10,760 NEXT SLIDE. 322 00:13:10,760 --> 00:13:12,440 SO JUST A FEW THINGS THAT I 323 00:13:12,440 --> 00:13:15,480 THINK WOULD BE HELPFUL TO BE 324 00:13:15,480 --> 00:13:18,440 AWARE OF AND THERE'S CERTAINLY A 325 00:13:18,440 --> 00:13:21,720 PUSH TO MAKE THE RESULTS OF NIH 326 00:13:21,720 --> 00:13:26,040 FUNDED RESEARCH MORE ACCESSIBLE 327 00:13:26,040 --> 00:13:28,440 TO THE PUBLIC SO IN 2022 THE 328 00:13:28,440 --> 00:13:29,720 WHITE HOUSE OFFICE OF SCIENCE 329 00:13:29,720 --> 00:13:33,040 AND TECHNOLOGY POLICY RELEASED A 330 00:13:33,040 --> 00:13:35,840 MEMORANDUM ESTABLISHING NEW 331 00:13:35,840 --> 00:13:37,520 GUIDANCE FOR IMPROVING PUBLIC 332 00:13:37,520 --> 00:13:39,760 ACCESS TO SCHOLARLY PUBLICATIONS 333 00:13:39,760 --> 00:13:41,320 AND DATA RESULTING FROM 334 00:13:41,320 --> 00:13:44,680 FEDERALLY SUPPORTED RESEARCH. 335 00:13:44,680 --> 00:13:45,840 SINCE WE'RE JUST ABOUT AT THE 336 00:13:45,840 --> 00:13:46,800 TIME WHEN YOU ARE GOING TO BE 337 00:13:46,800 --> 00:13:48,080 PAYING YOUR TAXES, YOU WOULD 338 00:13:48,080 --> 00:13:50,000 LIKE TO SEE THE RETURN ON YOUR 339 00:13:50,000 --> 00:13:51,200 INVESTMENTS SO WE WANT TO MAKE 340 00:13:51,200 --> 00:13:54,120 IT POSSIBLE FOR YOU. 341 00:13:54,120 --> 00:13:56,600 THE NIH PUBLIC ACCESS PLAN 342 00:13:56,600 --> 00:13:58,320 OUTLINES NIH'S APPROACH TO 343 00:13:58,320 --> 00:14:00,720 IMPLEMENTING THE NEW GUIDANCE 344 00:14:00,720 --> 00:14:02,960 AND IT WILL INSTITUTE A ZERO 345 00:14:02,960 --> 00:14:05,120 EMBARGO PERIOD ON PUBLICATIONS 346 00:14:05,120 --> 00:14:08,720 SO THAT THERE'S A VERY TIMELY 347 00:14:08,720 --> 00:14:11,640 WAY TO RECEIVE THE RESULTS OF 348 00:14:11,640 --> 00:14:13,320 NIH FUNDED RESEARCH WITH NO 349 00:14:13,320 --> 00:14:16,520 DELAY. 350 00:14:16,520 --> 00:14:17,240 CURRENTLY, FOR EXAMPLE, IF 351 00:14:17,240 --> 00:14:18,520 APPROXIMATE I HAVE A PUB WILL 352 00:14:18,520 --> 00:14:20,320 INDICATION AND I AM MAKING IT 353 00:14:20,320 --> 00:14:21,880 PUBLICLY AVAILABLE, I HAVE TO 354 00:14:21,880 --> 00:14:24,240 ADHERE TO THE PUBLISHER 355 00:14:24,240 --> 00:14:26,880 STANDARDS AND I HAVE TO LOOK UP 356 00:14:26,880 --> 00:14:28,880 EACH TIME DOES THAT PUBLISHER 357 00:14:28,880 --> 00:14:32,160 ALLOW ONE MONTH OR 12 MONTHS, 12 358 00:14:32,160 --> 00:14:38,320 MONTHS BEING THE MAJORITY SO 359 00:14:38,320 --> 00:14:39,040 PUBLISHERS DON'T WANT THE PUBLIC 360 00:14:39,040 --> 00:14:40,240 TO HAVE ACCESS TO THIS 361 00:14:40,240 --> 00:14:42,360 INFORMATION BECAUSE IT AFFECT 362 00:14:42,360 --> 00:14:44,720 THE BUSINESS MODEL BUT THE WHITE 363 00:14:44,720 --> 00:14:46,000 HOUSE IS RECOMMENDING THAT 364 00:14:46,000 --> 00:14:51,080 THERE'S A ZERO EMBARGO PERIOD SO 365 00:14:51,080 --> 00:14:53,480 THERE IS A REQUEST FOR 366 00:14:53,480 --> 00:14:54,280 INFORMATION. 367 00:14:54,280 --> 00:14:56,000 YOU CAN STILL RESPOND. 368 00:14:56,000 --> 00:14:57,520 YOU CAN RESPOND UP TO 369 00:14:57,520 --> 00:14:59,080 APRIL 24th IF YOU HAVE 370 00:14:59,080 --> 00:15:04,120 OPINIONS ON THIS AND HERE IS THE 371 00:15:04,120 --> 00:15:09,360 LINK AND FEEL FREE TO WEIGH IN 372 00:15:09,360 --> 00:15:12,480 ON THE SIDE OF MORE INFORMATION 373 00:15:12,480 --> 00:15:14,000 OR THE PUBLISHER BIS MONTHSEL. 374 00:15:14,000 --> 00:15:23,000 NEXT SLIDE, PLEASE. 375 00:15:23,000 --> 00:15:25,720 THE NEW PROPOSED FRAMEWORK FOR 376 00:15:25,720 --> 00:15:26,800 SIMPLIFIED REVIEW OF GRANT 377 00:15:26,800 --> 00:15:31,200 APPLICATIONS SO THE NIH PROPOSES 378 00:15:31,200 --> 00:15:39,360 TO REORGANIZE THERE ARE FIVE AND 379 00:15:39,360 --> 00:15:40,760 THEY'RE RECOMMENDING WE GO TO 380 00:15:40,760 --> 00:15:43,360 THREE WITH FACTOR ONE BEING THE 381 00:15:43,360 --> 00:15:44,480 IMPORTANCE OF THE RESEARCH. 382 00:15:44,480 --> 00:15:47,280 THIS IS A CONSOLIDATION OF THE 383 00:15:47,280 --> 00:15:49,600 CURRENT CRITERIA SIGNIFICANCE 384 00:15:49,600 --> 00:15:51,840 AND INNOVATION. 385 00:15:51,840 --> 00:15:56,080 FACTOR TWO, IS FEASIBILITY AND 386 00:15:56,080 --> 00:16:01,160 RIG OR BASED ON THE CRITERION 387 00:16:01,160 --> 00:16:03,160 APPROACH AND FACTOR THREE IS 388 00:16:03,160 --> 00:16:10,200 EXPERTISE AND CREE SOU RESOURCE. 389 00:16:10,200 --> 00:16:11,680 THIS IS THE CURRENT CRITERIA 390 00:16:11,680 --> 00:16:14,920 INVESTIGATOR AND THE 391 00:16:14,920 --> 00:16:21,000 ENVIRONMENT. 392 00:16:21,000 --> 00:16:23,960 THE IMPORTANCE OF THE RESEARCH, 393 00:16:23,960 --> 00:16:26,360 THE RIGGER OF THE RESEARCH, THE 394 00:16:26,360 --> 00:16:28,120 FEASIBILITY OF THE RESEARCH AND 395 00:16:28,120 --> 00:16:29,880 THE CONTRIBUTIONS OF THE 396 00:16:29,880 --> 00:16:31,880 INVESTIGATOR AND INSTITUTION ARE 397 00:16:31,880 --> 00:16:35,680 FRAMED IN THE CONTEXT OF THE 398 00:16:35,680 --> 00:16:38,040 PROPOSED RESEARCH. 399 00:16:38,040 --> 00:16:40,680 IF YOU WOULD LIKE TO LEARN MORE, 400 00:16:40,680 --> 00:16:41,920 HERE IS THE LINK A LOT THE 401 00:16:41,920 --> 00:16:43,960 BOTTOM OF THE SLIDE. 402 00:16:43,960 --> 00:16:46,560 I KNOW THAT THIS PARTICULAR 403 00:16:46,560 --> 00:16:48,160 AUDIENCE IS VERY INTERESTED IN 404 00:16:48,160 --> 00:16:50,720 THE FUTURE OF THE NIH'S 405 00:16:50,720 --> 00:16:52,600 UNDIAGNOSED DISEASE NETWORK FOR 406 00:16:52,600 --> 00:16:54,680 THE UDN. 407 00:16:54,680 --> 00:16:56,800 THE GOAL OF THE UDN IS TO 408 00:16:56,800 --> 00:16:58,520 ESTABLISH AND TEST A NEW MODEL 409 00:16:58,520 --> 00:17:00,400 OF COLLABORATION ACROSS CLINICAL 410 00:17:00,400 --> 00:17:03,920 SITES TO EXTEND THE NIH UDP 411 00:17:03,920 --> 00:17:07,000 PROGRAM THE UNDIAGNOSED DISEASES 412 00:17:07,000 --> 00:17:10,680 PROGRAM TO EXTRAMURAL CENTERS. 413 00:17:10,680 --> 00:17:12,000 THE CURRENT PROGRAM WILL EXPIRE 414 00:17:12,000 --> 00:17:16,440 THIS SUMMER IN JULY. 415 00:17:16,440 --> 00:17:19,080 MULTIPLE INSTITUTES, INCLUDING 416 00:17:19,080 --> 00:17:20,520 NICHD ARE FUNDING A DATA 417 00:17:20,520 --> 00:17:22,040 MANAGEMENT AND COORDINATING 418 00:17:22,040 --> 00:17:23,240 CENTER THAT WILL START THIS 419 00:17:23,240 --> 00:17:25,000 SPRING AND SUMMER TO PROVIDE 420 00:17:25,000 --> 00:17:25,960 INFRASTRUCTURE AND RESEARCH 421 00:17:25,960 --> 00:17:29,160 SUPPORT FOR A NEW NETWORK OF 422 00:17:29,160 --> 00:17:30,080 CLINICAL SITES. 423 00:17:30,080 --> 00:17:32,400 CLINICAL SITES CAN APPLY FOR 424 00:17:32,400 --> 00:17:34,280 DESIGNATION AS A DIAGNOSTIC 425 00:17:34,280 --> 00:17:37,000 CENTER OF EXCELLENCE TO ACCESS 426 00:17:37,000 --> 00:17:39,080 COORDINATING CENTER RESOURCES SO 427 00:17:39,080 --> 00:17:42,840 THIS IS THE WAY THAT WE WILL 428 00:17:42,840 --> 00:17:44,200 CONTINUE THE UNDIAGNOSED 429 00:17:44,200 --> 00:17:47,320 DISEASES NETWORK. 430 00:17:47,320 --> 00:17:50,120 NOW WE MOVE TO THE NIH WIDE 431 00:17:50,120 --> 00:17:52,360 STRATEGIC PLAN FOR DIVERSITY, 432 00:17:52,360 --> 00:17:54,240 EQUITY, INCLUSION AND 433 00:17:54,240 --> 00:17:58,920 ACCESSIBILITY WHICH IS JUST 434 00:17:58,920 --> 00:18:01,560 RECENTLY RELEASED IN MARCH. 435 00:18:01,560 --> 00:18:04,040 THE MISSION OF THIS PLAN IS TO 436 00:18:04,040 --> 00:18:05,120 EMBRACE -- THE MISSION FOR NIH 437 00:18:05,120 --> 00:18:07,480 IS TO EMBRACE, STRENGTHEN AND 438 00:18:07,480 --> 00:18:09,520 INTEGRATE DIVERSITY, EQUITY, 439 00:18:09,520 --> 00:18:14,200 INCLUSION AND ACCESSIBILITY FOR 440 00:18:14,200 --> 00:18:15,680 DEIA ACROSS ALL NIH ACTIVITIES 441 00:18:15,680 --> 00:18:18,720 AND SERVICE OF THE NIH MISSION. 442 00:18:18,720 --> 00:18:23,560 THE VISION IS THAT NIH WILL BE A 443 00:18:23,560 --> 00:18:24,120 PEOPLE-CENTERED ORGANIZATION 444 00:18:24,120 --> 00:18:26,640 WHERE THE WORKFORCE AND RESEARCH 445 00:18:26,640 --> 00:18:29,200 PRIORITIES REFLECT OUR NATION'S 446 00:18:29,200 --> 00:18:31,000 DIVERSITY AND ALL PEOPLE FEEL A 447 00:18:31,000 --> 00:18:34,400 SENSE OF BELONGING AS THEY 448 00:18:34,400 --> 00:18:36,800 ADVANCE THE NIH MISSION. 449 00:18:36,800 --> 00:18:39,000 THE STRATEGIC PLAN IS PUBLICLY 450 00:18:39,000 --> 00:18:39,360 AVAILABLE. 451 00:18:39,360 --> 00:18:42,720 WE DID WANT TO MENTION, TO THIS 452 00:18:42,720 --> 00:18:44,760 AUDIENCE, THAT ON ONE OF THE 453 00:18:44,760 --> 00:18:47,240 PAGES, WE HAVE HIGHLIGHTED THE 454 00:18:47,240 --> 00:18:50,280 NATIONAL CENTER FOR MEDICAL 455 00:18:50,280 --> 00:18:52,840 REHABILITATION RESEARCH SO TO 456 00:18:52,840 --> 00:18:54,840 EMPHASIZE THE FACT THAT 457 00:18:54,840 --> 00:18:56,240 ACCESSIBILITY IS AN IMPORTANT 458 00:18:56,240 --> 00:19:03,280 PART OF THIS PLAN GOING FORWARD. 459 00:19:03,280 --> 00:19:05,000 SO THE OBJECTIVES OF THE PLAN 460 00:19:05,000 --> 00:19:08,200 ARE TO GROW AND SUSTAIN DIEA SO 461 00:19:08,200 --> 00:19:11,160 THIS GRAPHIC ON THE RIGHT 462 00:19:11,160 --> 00:19:12,320 "SQUARE OFF" PART OF -- THERE'S 463 00:19:12,320 --> 00:19:14,440 A TWO-PAGE EXECUTIVE 464 00:19:14,440 --> 00:19:15,840 HIGHLIGHTER. 465 00:19:15,840 --> 00:19:17,280 EXECUTIVE SUMMARY OF THE PLAN, 466 00:19:17,280 --> 00:19:19,280 WHICH WAS ALSO AVAILABLE 467 00:19:19,280 --> 00:19:19,800 PUBLICLY. 468 00:19:19,800 --> 00:19:21,920 THE OBJECTIVES ARE TO GROW AND 469 00:19:21,920 --> 00:19:25,760 SUSTAIN DEIA TO STRUCTURAL AND 470 00:19:25,760 --> 00:19:28,720 CULTURAL CHANGE AND TO IMPLEMENT 471 00:19:28,720 --> 00:19:29,840 ORGANIZATIONAL PRACTICES TO 472 00:19:29,840 --> 00:19:32,360 CENTER AND PRIORITIZE DEIA IN 473 00:19:32,360 --> 00:19:35,760 THE WORKSHOP IN THE WORKFORCE. 474 00:19:35,760 --> 00:19:39,440 AND THEN TO ADVANCE DEIA THROUGH 475 00:19:39,440 --> 00:19:39,840 RESEARCH. 476 00:19:39,840 --> 00:19:42,400 THE CROSS CUTTING THEMES INCLUDE 477 00:19:42,400 --> 00:19:45,600 PROMOTION OF TRANSPARENCY, 478 00:19:45,600 --> 00:19:47,320 COMMUNICATION, AND ENGAGEMENT, 479 00:19:47,320 --> 00:19:51,800 AND TO FOSTER SUSTAINABLE CHANGE 480 00:19:51,800 --> 00:19:57,480 AND HARNESS DATA. 481 00:19:57,480 --> 00:19:59,360 WE'RE MOVING TO THE ADVISORY 482 00:19:59,360 --> 00:20:01,320 COMMITTEE TO THE DIRECTOR. 483 00:20:01,320 --> 00:20:03,720 THAT'S TO DR. TABAK. 484 00:20:03,720 --> 00:20:06,560 THERE'S A WORKING GROUP ON 485 00:20:06,560 --> 00:20:10,520 DIVERSITY WHICH IS A SUBGROUP ON 486 00:20:10,520 --> 00:20:16,000 INDIVIDUALS WITH DISABILITIES. 487 00:20:16,000 --> 00:20:17,760 PEOPLE WITH DISABILITIES ARE THE 488 00:20:17,760 --> 00:20:19,200 SINGLE LARGEST POPULATION AND 489 00:20:19,200 --> 00:20:21,240 THE FASTEST GROWING MINORITY 490 00:20:21,240 --> 00:20:22,240 POPULATION IN THE UNITED STATES. 491 00:20:22,240 --> 00:20:25,120 PEOPLE WITH DIS A LOTS REPRESENT 492 00:20:25,120 --> 00:20:28,080 ABOUT 9% OF THE SCIENTIFIC 493 00:20:28,080 --> 00:20:29,040 WORKFORCE. 494 00:20:29,040 --> 00:20:30,200 RESEARCHERS WITH DISABILITIES 495 00:20:30,200 --> 00:20:35,280 ARE UNDER REPRESENTED IN NIH 496 00:20:35,280 --> 00:20:36,080 FUNDING. 497 00:20:36,080 --> 00:20:37,560 DATA ARE PUBLICLY AVAILABLE 498 00:20:37,560 --> 00:20:39,160 ABOUT DISABILITY PREVALENCE IN 499 00:20:39,160 --> 00:20:41,520 THE NIH AND THE BROADER RESEARCH 500 00:20:41,520 --> 00:20:43,560 WORKFORCE OR THE PARTICIPATION 501 00:20:43,560 --> 00:20:45,600 OF PEOPLE WITH DISABILITIES IN 502 00:20:45,600 --> 00:20:50,240 NIH FUNDED STUDIES. 503 00:20:50,240 --> 00:20:51,720 CURRENT NIH EFFORTS TO COLLECT 504 00:20:51,720 --> 00:20:54,520 DISABILITY DATA ON THE 505 00:20:54,520 --> 00:20:55,080 INADEQUATE. 506 00:20:55,080 --> 00:20:59,680 NEXT SLIDE, PLEASE. 507 00:20:59,680 --> 00:21:03,080 AND SO THE GROUP CONVICTION WITH 508 00:21:03,080 --> 00:21:04,280 DISABILITIES MADE SOME 509 00:21:04,280 --> 00:21:07,360 SUGGESTIONS IN THE WORKING GROUP 510 00:21:07,360 --> 00:21:08,000 REPORT. 511 00:21:08,000 --> 00:21:10,920 THEY SUGGESTED TO O UPDATE THE 512 00:21:10,920 --> 00:21:12,840 NIH MISSION STATEMENT CURRENTLY 513 00:21:12,840 --> 00:21:15,160 IT IS TO SEEK FUNDAMENTAL 514 00:21:15,160 --> 00:21:21,040 KNOWLEDGE ABOUT THE NATURE AND 515 00:21:21,040 --> 00:21:22,120 BEHAVIOR AND THE APPLICATION OF 516 00:21:22,120 --> 00:21:23,760 THAT KNOWLEDGE TO ENHANCE 517 00:21:23,760 --> 00:21:26,440 HEALTH, LENGTH LIFE AND REDUCE 518 00:21:26,440 --> 00:21:36,600 ILLNESS AND DIS A LOT. 519 00:21:36,600 --> 00:21:40,480 WERE ANY STRUCTURE ABLEISM THAT 520 00:21:40,480 --> 00:21:42,120 MAY EXIST AND DISABILITY 521 00:21:42,120 --> 00:21:43,440 INCLUSION INTERNALLY AND FOR THE 522 00:21:43,440 --> 00:21:46,760 NIH FUNDED WORKFORCE 523 00:21:46,760 --> 00:21:47,760 EXTRAMURALLY. 524 00:21:47,760 --> 00:21:49,280 THEY ALSO RECOMMENDED EXPANDING 525 00:21:49,280 --> 00:21:51,600 EFFORTS TO INCLUDE DISABILITY 526 00:21:51,600 --> 00:21:53,400 COMMUNITIES AND THE PERSPECTIVES 527 00:21:53,400 --> 00:21:56,400 OF PEOPLE WITH DISABILITIES AND 528 00:21:56,400 --> 00:21:59,000 TO CONDUCT RESEARCH ON 529 00:21:59,000 --> 00:22:01,800 DISABILITY HEALTH AND HEALTHCARE 530 00:22:01,800 --> 00:22:04,200 DISPARITIES AND EQUITY. 531 00:22:04,200 --> 00:22:06,240 THEY RECOMMENDED THAT WE ENSURE 532 00:22:06,240 --> 00:22:08,840 THAT DISABILITY INCLUSION AND 533 00:22:08,840 --> 00:22:13,160 ANTI ABLEISM ARE CORE COMPONENTS 534 00:22:13,160 --> 00:22:17,120 OF ALL NIH, DEIA EFFORTS AND TO 535 00:22:17,120 --> 00:22:22,840 MAINTAIN ACCOUNTABILITY FOR 536 00:22:22,840 --> 00:22:25,480 DISABILITY INCLUSION EFFORTS. 537 00:22:25,480 --> 00:22:28,040 SO AGAIN, I WOULD JUST LIKE TO 538 00:22:28,040 --> 00:22:31,880 BRIEFLY MENTION SOMETHING ABOUT 539 00:22:31,880 --> 00:22:33,560 ARPA-H. 540 00:22:33,560 --> 00:22:35,840 SO, IN THE FISCAL YEAR 541 00:22:35,840 --> 00:22:38,440 '23 BUDGET, CONGRESS PROVIDED 542 00:22:38,440 --> 00:22:42,960 ARPA-H WITH $2.5 BILLION THAT IS 543 00:22:42,960 --> 00:22:44,640 INDEPENDENT OF NIH. 544 00:22:44,640 --> 00:22:47,040 THE ARPA-H DIRECTOR REPORTS 545 00:22:47,040 --> 00:22:51,400 DIRECTLY TO THE HHS SECRETARY, 546 00:22:51,400 --> 00:22:52,960 ALTHOUGH NIH PROVIDES 547 00:22:52,960 --> 00:22:54,760 INFRASTRUCTURE TO ARPA-H. 548 00:22:54,760 --> 00:22:58,120 FOR EXAMPLE, SUCH AS HUMAN 549 00:22:58,120 --> 00:22:59,760 RESOURCES. 550 00:22:59,760 --> 00:23:01,200 ARPA-H, YOU MAY HAVE BEEN 551 00:23:01,200 --> 00:23:03,480 READING IN THE PRESS ABOUT ALL 552 00:23:03,480 --> 00:23:08,320 THIS COMPETITION FOR ARPA-H 553 00:23:08,320 --> 00:23:09,360 LABORATORIES OR SITES FOR 554 00:23:09,360 --> 00:23:10,000 ARPA-H. 555 00:23:10,000 --> 00:23:13,680 THERE ARE NO INTERNAL RESEARCH 556 00:23:13,680 --> 00:23:14,440 LABORATORIES. 557 00:23:14,440 --> 00:23:16,720 AND IMPORTANTLY FOR THIS 558 00:23:16,720 --> 00:23:19,480 AUDIENCE, ARPA-H IS DISEASE 559 00:23:19,480 --> 00:23:25,920 ACKNOWDIS AGNOSTICSO I'VE HEARD. 560 00:23:25,920 --> 00:23:29,360 WE REALLY WANT SOME PEDIATRIC 561 00:23:29,360 --> 00:23:31,360 PROJECTS IN ARPA-H. 562 00:23:31,360 --> 00:23:33,000 ANYONE CAN PROPOSE AN IMPORTANT 563 00:23:33,000 --> 00:23:35,280 PROBLEM THAT NEEDS TO BE SOLVED 564 00:23:35,280 --> 00:23:39,680 AND IT'S DISEASE AND DEMOGRAPHIC 565 00:23:39,680 --> 00:23:40,640 AGNOSTIC. 566 00:23:40,640 --> 00:23:45,920 SO, WHAT ARPA-H IS LOOKING FOR, 567 00:23:45,920 --> 00:23:48,440 ARE PROGRAMS MANAGERS WHO HAVE 568 00:23:48,440 --> 00:23:50,600 INNOVATIVE IDEAS AND THEY WILL 569 00:23:50,600 --> 00:23:54,520 ACT AS THE CATALYST FOR THAT 570 00:23:54,520 --> 00:23:58,520 PROGRAM AND THEY ARE GIVEN A LOT 571 00:23:58,520 --> 00:24:00,400 OF AUTONOMY TO MAKE DECISIONS SO 572 00:24:00,400 --> 00:24:03,520 ARPA-H IS SEEKING THESE PROGRAM 573 00:24:03,520 --> 00:24:07,840 MANAGERS WHO EY IDEALLY WHO ARE 574 00:24:07,840 --> 00:24:10,600 AGGRESSIVE AND WILLING TO FAIL 575 00:24:10,600 --> 00:24:12,000 QUICKLY BUT THEY NEED TO 576 00:24:12,000 --> 00:24:13,280 IDENTIFY DIFFICULT 577 00:24:13,280 --> 00:24:14,400 HEALTH-RELATED CHALLENGES AND 578 00:24:14,400 --> 00:24:16,040 THEY NEED TO ASSEMBLE, THEY NEED 579 00:24:16,040 --> 00:24:18,960 TO HIRE AND ASSEMBLE THEIR TEAMS 580 00:24:18,960 --> 00:24:22,080 FROM INDUSTRY, ACADEMIA, AND 581 00:24:22,080 --> 00:24:26,800 GOVERNMENT TO COLLECTIVELY SOLVE 582 00:24:26,800 --> 00:24:28,160 THESE PROBLEMS. 583 00:24:28,160 --> 00:24:30,360 THERE IS AN OPEN OPPORTUNITY 584 00:24:30,360 --> 00:24:32,640 CALLED DASH, WHICH IS MODELED 585 00:24:32,640 --> 00:24:34,920 AFTER MARCH MADNESS, TO PITCH 586 00:24:34,920 --> 00:24:36,960 HIGH-RISK, HIGH-IMPACT RESEARCH 587 00:24:36,960 --> 00:24:41,440 THAT AFFECTS ANY OR MULTIPLE 588 00:24:41,440 --> 00:24:42,040 POPULATIONS. 589 00:24:42,040 --> 00:24:44,560 SO WE SUGGEST THAT IF YOU ARE 590 00:24:44,560 --> 00:24:45,640 INTERESTED IN THOSE PROGRAMS, GO 591 00:24:45,640 --> 00:24:51,440 TO THE ARPA-H WEBSITE. 592 00:24:51,440 --> 00:24:54,440 NOW WE'LL GET TO THE ADVANCES 593 00:24:54,440 --> 00:24:55,240 AND MUSCULAR DYSTROPHY RESEARCH. 594 00:24:55,240 --> 00:24:59,760 SO THE FIRST PAPER WAS PUBLISHED 595 00:24:59,760 --> 00:25:04,920 IN E NEURO AND SUPPORTED BY 596 00:25:04,920 --> 00:25:12,480 NINDS HE WAS ENTITLED 597 00:25:12,480 --> 00:25:14,040 IMPLICATIONS FOR CENTRAL NERVOUS 598 00:25:14,040 --> 00:25:16,920 SYSTEMS SYMPTOMS IN MIO TONIC 599 00:25:16,920 --> 00:25:19,760 DYSTROPHY SO PATIENTS WHO HAVE 600 00:25:19,760 --> 00:25:23,040 MIO TONIC DYSTROPHY EXPERIENCE 601 00:25:23,040 --> 00:25:24,560 CENTRAL NERVOUS SYSTEM THAT 602 00:25:24,560 --> 00:25:27,680 INCLUDES HYPER SOMNIA AND 603 00:25:27,680 --> 00:25:34,120 INCREASE SENSITIVITY TO BAR BIT 604 00:25:34,120 --> 00:25:35,720 TO YOU ITS AND. 605 00:25:35,720 --> 00:25:40,280 PROTEIN 2 OR MBNL2 FUNCTION IS A 606 00:25:40,280 --> 00:25:42,280 MAJOR DRIVER OF CENTRAL NERVOUS 607 00:25:42,280 --> 00:25:52,680 SYSTEM SYMPTOMS IN DM1. 608 00:25:54,960 --> 00:25:58,560 IT WOULD EFFECT THE GAB A A AND 609 00:25:58,560 --> 00:26:02,080 MODULATION AND MAY SUGGEST THAT 610 00:26:02,080 --> 00:26:04,240 STRATEGIES TO TREAT THE SYMPTOMS 611 00:26:04,240 --> 00:26:06,720 EXPERIENCED BY PEOPLE WITH DM1 612 00:26:06,720 --> 00:26:11,120 MIGHT INCLUDE TARGETING THE GABA 613 00:26:11,120 --> 00:26:11,720 ACCESS. 614 00:26:11,720 --> 00:26:16,160 NEXT SLIDE, PLEASE. 615 00:26:16,160 --> 00:26:19,920 THE THE NEXT STUDY WAS CO 616 00:26:19,920 --> 00:26:24,080 SUPPORTED BY NIAGARA FALLS ICHD 617 00:26:24,080 --> 00:26:27,440 AND IT WAS CRISPR CAS 9 618 00:26:27,440 --> 00:26:28,560 CORRECTION OF MUSCULAR DYSTROPHY 619 00:26:28,560 --> 00:26:30,920 IN MICE BY A SELF-COMPLIMENTARY 620 00:26:30,920 --> 00:26:34,080 AAV DELIVERY SYSTEM. 621 00:26:34,080 --> 00:26:35,280 SO IMPLYING IT THAT A REFINED 622 00:26:35,280 --> 00:26:37,760 GENE EDITING APPROACH COULD 623 00:26:37,760 --> 00:26:39,920 IMPROVE CORRECTION OF DMD 624 00:26:39,920 --> 00:26:45,360 MUTATIONS IN ANIMAL MODELS OF 625 00:26:45,360 --> 00:26:45,640 DUE SCHENN. 626 00:26:45,640 --> 00:26:47,800 THEY MEDIATED GENE EDITING HAS 627 00:26:47,800 --> 00:26:49,480 BEEN USED SUCCESSFULLY IN MOUSE 628 00:26:49,480 --> 00:26:59,640 MODEL OF DUE SCHENN USING A 629 00:26:59,640 --> 00:27:01,040 SINGLE-STRANDED AAV WAS A RATE 630 00:27:01,040 --> 00:27:02,640 LIMITING FACTOR FOR 24 APPROACH 631 00:27:02,640 --> 00:27:05,200 N THIS PUBLICATION, THE RESEARCH 632 00:27:05,200 --> 00:27:08,680 DESIGNED A NEW METHOD USING 633 00:27:08,680 --> 00:27:11,280 SELF-COMPLIMENTARY AAV TO 634 00:27:11,280 --> 00:27:14,160 DELIVER CRISPR RNA FOR IN VIVO 635 00:27:14,160 --> 00:27:15,520 GENE EDITING. 636 00:27:15,520 --> 00:27:18,360 THIS NEW METHOD RESULTED IN 637 00:27:18,360 --> 00:27:20,520 HIGHER GENE EDITING AND 638 00:27:20,520 --> 00:27:22,160 RESTORATION COMPARED TO OTHER 639 00:27:22,160 --> 00:27:24,760 METHODS IN ANIMAL MODELS. 640 00:27:24,760 --> 00:27:28,240 AND THE METHOD TO QUANTIFY DIS 641 00:27:28,240 --> 00:27:31,280 OPEN RESTORATION AFTER THE 642 00:27:31,280 --> 00:27:34,000 CRISPR CAS 9 CORRECTION IS ALSO 643 00:27:34,000 --> 00:27:35,600 DESCRIBED AND THIS STUDY 644 00:27:35,600 --> 00:27:40,440 APPEARED AS PART OF METHODS IN 645 00:27:40,440 --> 00:27:43,400 MOLECULAR BIOLOGY THAT WAS 646 00:27:43,400 --> 00:27:44,680 PUBLISHED. 647 00:27:44,680 --> 00:27:45,520 NEXT SLIDE. 648 00:27:45,520 --> 00:27:46,480 THANK YOU. 649 00:27:46,480 --> 00:27:50,720 AND THE NEXT HIGHLIGHT WAS 650 00:27:50,720 --> 00:27:55,800 CO-SUPPORTED BY NICHD, NIAMS AND 651 00:27:55,800 --> 00:27:57,800 NHLBI PUBLISHED IN THE JOURNAL 652 00:27:57,800 --> 00:28:00,080 OF CLINICAL INVESTIGATION. 653 00:28:00,080 --> 00:28:02,200 TITLED LOSS OF FUNCTION OF THE 654 00:28:02,200 --> 00:28:05,880 NUCLEAR ENVELOPE PROTEIN LEMD2 655 00:28:05,880 --> 00:28:14,680 CAUSES DNA DAMAGE-DEPENDENT 656 00:28:14,680 --> 00:28:25,760 CARDIO MCARDMYOPHAHY SO THEY CAT 657 00:28:27,000 --> 00:28:28,520 PREVENT HEART AND SKELETAL 658 00:28:28,520 --> 00:28:29,200 MUSCLE. 659 00:28:29,200 --> 00:28:33,160 A MUTATION IN THE GENE LEMD2 660 00:28:33,160 --> 00:28:36,520 RESULTS IN LOSS OF FUNCTION OF 661 00:28:36,520 --> 00:28:38,840 THE NEP AND THAT CAN CAUSE 662 00:28:38,840 --> 00:28:41,280 SEVERE CARDIO MYOPATHY IN HUMANS 663 00:28:41,280 --> 00:28:42,640 BUT THE UNDERLINE DISEASE 664 00:28:42,640 --> 00:28:45,440 MECHANISM IS UNKNOWN AT PRESENT. 665 00:28:45,440 --> 00:28:47,680 IN THIS STUDY, THE INVESTIGATORS 666 00:28:47,680 --> 00:28:50,160 GENERATED MICE WITH THE 667 00:28:50,160 --> 00:28:53,920 MUTATION, AND THEY SERVED AS A 668 00:28:53,920 --> 00:28:56,640 PRECLINICAL DISEASE MODEL. 669 00:28:56,640 --> 00:29:00,960 CARDIO MYOCYTE-SPECIFIC LEMD2 670 00:29:00,960 --> 00:29:04,160 GENE THERAPY VIA AAV RESCUED 671 00:29:04,160 --> 00:29:07,320 CARDIAC FUNCTION IN THE MICE. 672 00:29:07,320 --> 00:29:08,840 SO, THE IMPORTANCE OF THIS IS 673 00:29:08,840 --> 00:29:11,440 THAT UNDERSTANDING THE MECHANISM 674 00:29:11,440 --> 00:29:13,440 OF ACTION CAN HELP UNDERSTAND 675 00:29:13,440 --> 00:29:21,960 OTHER RELATED SUCH AS 676 00:29:21,960 --> 00:29:22,480 'EMMER'-DREIFUSS MUSCULAR 677 00:29:22,480 --> 00:29:23,840 DYSTROPHY AND WHICH IS 678 00:29:23,840 --> 00:29:25,600 CHARACTERIZED BY SKELETAL MUSCLE 679 00:29:25,600 --> 00:29:29,480 WASTING AND CARDIAC PATHOLOGY. 680 00:29:29,480 --> 00:29:31,120 I THINK THIS IS THE LAST ONE. 681 00:29:31,120 --> 00:29:33,040 BUT IN THIS PUBLICATION, THE 682 00:29:33,040 --> 00:29:36,560 STUDY WAS SUPPORTED BY NIAMS, 683 00:29:36,560 --> 00:29:42,200 THE LGMD1DDNA FOUNDATION AND AN 684 00:29:42,200 --> 00:29:46,840 INTERNATIONAL REGISTRY AND AS 685 00:29:46,840 --> 00:29:48,040 WELL AS OTHER SUPPORT PUBLISHED 686 00:29:48,040 --> 00:29:49,840 IN THE CLINICAL AND 687 00:29:49,840 --> 00:29:52,280 TRANSLATIONAL NEUROLOGY. 688 00:29:52,280 --> 00:29:56,280 IN FEBRUARY OF 2023. 689 00:29:56,280 --> 00:29:58,280 SO, THIS PAPER REALLY DISCUSSED 690 00:29:58,280 --> 00:30:02,920 INCREASING UNDERSTANDING OF 691 00:30:02,920 --> 00:30:04,920 MUSCULAR DYSTROPHY TYPE D1 AND 692 00:30:04,920 --> 00:30:09,400 OTHERWISE KNOWN AS LGMDDD1 AND 693 00:30:09,400 --> 00:30:10,880 THAT WAS ONLY IDENTIFIED 694 00:30:10,880 --> 00:30:12,400 MUTATION INVOLVED WAS ONLY 695 00:30:12,400 --> 00:30:13,960 IDENTIFIED IN 2012 BUT IT'S ONE 696 00:30:13,960 --> 00:30:19,040 OF THE MORE COMMONLY DIAGNOSED 697 00:30:19,040 --> 00:30:20,080 MUSCULAR DYSTROPHIES. 698 00:30:20,080 --> 00:30:23,240 IT'S NATURAL HISTORY STUDY 699 00:30:23,240 --> 00:30:27,240 FOLLOWED 122 PATIENTS WITH 700 00:30:27,240 --> 00:30:27,920 LGMDD1 FROM 14 DIFFERENT 701 00:30:27,920 --> 00:30:31,120 COUNTRIES OVER A PERIOD OF 2.5 702 00:30:31,120 --> 00:30:31,800 YEARS. 703 00:30:31,800 --> 00:30:34,760 THEY IDENTIFIED GENOTYPE AS THE 704 00:30:34,760 --> 00:30:40,640 LIKELY SOURCE FOR FI PHENOTYPIC 705 00:30:40,640 --> 00:30:41,960 VARIABILITIES AND THE GENOTYPE 706 00:30:41,960 --> 00:30:43,560 APPEARS TO IMPACT SEVERAL 707 00:30:43,560 --> 00:30:46,200 ASPECTS WITH IMPLICATIONS FOR 708 00:30:46,200 --> 00:30:47,000 CLINICAL TRIALS. 709 00:30:47,000 --> 00:30:49,320 SO, DEPENDING ON THE MUTATION, 710 00:30:49,320 --> 00:30:51,680 THERE WERE DIFFERENT WEAKNESS 711 00:30:51,680 --> 00:30:53,040 PATTERNS, A DIFFERENT PATTERN OF 712 00:30:53,040 --> 00:30:55,280 AGE OF ONSET AND POSSIBLY RATE 713 00:30:55,280 --> 00:30:58,040 OF DISEASE PROGRESSION TO LOSS 714 00:30:58,040 --> 00:31:01,240 OF AMBULATION. 715 00:31:01,240 --> 00:31:03,640 SO, IT WAS RECOMMENDED THAT AN 716 00:31:03,640 --> 00:31:06,360 IN-DEPTH STUDY OF GENOTYPE AND 717 00:31:06,360 --> 00:31:07,840 PHENOTYPE LINKAGE IS NEEDED AS 718 00:31:07,840 --> 00:31:09,520 THE FIELD MOVES FORWARD TOWARDS 719 00:31:09,520 --> 00:31:09,920 CLINICAL TRIALS. 720 00:31:09,920 --> 00:31:11,680 IN OTHER WORDS, YOU CAN'T PUT 721 00:31:11,680 --> 00:31:15,280 ALL OF THE EFFECTED PATIENTS 722 00:31:15,280 --> 00:31:16,240 INTO ONE GROUP. 723 00:31:16,240 --> 00:31:18,480 PROBABLY THEY WILL NEED TO BE 724 00:31:18,480 --> 00:31:24,720 SORTED ACCORDING TO MUTATION. 725 00:31:24,720 --> 00:31:25,200 NEXT SLIDE. 726 00:31:25,200 --> 00:31:26,160 THERE'S ONE MORE. 727 00:31:26,160 --> 00:31:27,240 SORRY ABOUT THAT. 728 00:31:27,240 --> 00:31:31,080 THE LAST STUDY HAS BEEN 729 00:31:31,080 --> 00:31:33,840 SUPPORTED BY NINVS PUBLISHED IN 730 00:31:33,840 --> 00:31:37,560 THE LANCE I HAD LAST MONTH 731 00:31:37,560 --> 00:31:46,680 ENTITLED ANTISENSE OLE OLE 732 00:31:46,680 --> 00:31:49,760 OLIGONUCLEOTID TARGETING DMPK IN 733 00:31:49,760 --> 00:31:51,960 PATIENTS WITH MIO TONIC 734 00:31:51,960 --> 00:31:53,520 DYSTROPHY TYPE 1. 735 00:31:53,520 --> 00:31:55,880 SO THEY PROVIDE A PROMISING 736 00:31:55,880 --> 00:31:58,960 APPROACH TO TREATMENT OF MD1 737 00:31:58,960 --> 00:32:02,160 BECAUSE THEY REDUCE TOXIC RNA 738 00:32:02,160 --> 00:32:03,240 LEVELS. 739 00:32:03,240 --> 00:32:05,080 IN THIS PARTICULAR STUDY THE 740 00:32:05,080 --> 00:32:06,360 INVESTIGATORS LOOKED AT THE 741 00:32:06,360 --> 00:32:12,640 SAFETY OF BALIFORSEN AND 742 00:32:12,640 --> 00:32:14,080 ANTISENSE NUCLEOTIDE THAT 743 00:32:14,080 --> 00:32:17,960 TARGETS THE DMT PROTEIN KINASE 744 00:32:17,960 --> 00:32:18,720 mRNA. 745 00:32:18,720 --> 00:32:20,360 THE TREATMENT WAS WELL TOLERATE 746 00:32:20,360 --> 00:32:23,120 BY THE STUDY PARTICIPANTS WITH 747 00:32:23,120 --> 00:32:26,400 ONLY MILD ADVERSE EVENTS. 748 00:32:26,400 --> 00:32:28,400 HOWEVER, SKELETAL MUSCLE DRUG 749 00:32:28,400 --> 00:32:29,920 CONCENTRATIONS WERE BELOW THE 750 00:32:29,920 --> 00:32:31,640 LEVELS THAT WERE PREDICT TODAY 751 00:32:31,640 --> 00:32:34,320 ACHIEVE A SUBSTANTIAL TARGET 752 00:32:34,320 --> 00:32:35,360 REDUCTION. 753 00:32:35,360 --> 00:32:37,960 THE RESULTS SUPPORT FURTHER 754 00:32:37,960 --> 00:32:45,080 INVESTIGATION OF ASO AS A 755 00:32:45,080 --> 00:32:45,920 THERAPEUTIC APPROACH BUT 756 00:32:45,920 --> 00:32:47,520 IMPROVED DELIVERY TO MUSCLE IS 757 00:32:47,520 --> 00:32:48,000 NEEDED. 758 00:32:48,000 --> 00:32:53,160 I THINK THIS IS THE LAST ONE. 759 00:32:53,160 --> 00:32:57,080 SO, THOSE ARE MY UPDATES FOR 760 00:32:57,080 --> 00:32:57,440 NOW. 761 00:32:57,440 --> 00:32:59,720 WE WANTED TO GIVE YOU A HEADS-UP 762 00:32:59,720 --> 00:33:02,560 ABOUT A TOPIC THAT WE INTEND TO 763 00:33:02,560 --> 00:33:05,800 DISCUSS AT THE FALL MDCC MEETING 764 00:33:05,800 --> 00:33:08,640 AND THAT WILL INVOLVE NEWBORN 765 00:33:08,640 --> 00:33:11,880 SCREENING FOR DUCHENNE MUSCULAR 766 00:33:11,880 --> 00:33:12,160 DYSTROPHY. 767 00:33:12,160 --> 00:33:14,280 WE HAD A SESSION ON THIS TOPIC 768 00:33:14,280 --> 00:33:19,560 IN DECISION OF 2020, BUT THERE'S 769 00:33:19,560 --> 00:33:24,520 BEEN SOME RESENT ACTIVITY IN 770 00:33:24,520 --> 00:33:27,240 TERMS OF DECISIONS THAT WERE 771 00:33:27,240 --> 00:33:29,760 MADE WHETHER OR NOT TO RECOMMEND 772 00:33:29,760 --> 00:33:32,240 SCREENING ROUTINELY FOR THIS 773 00:33:32,240 --> 00:33:34,640 CONDITION. 774 00:33:34,640 --> 00:33:38,360 O, WE ARE IN THE PRODUCTS OF 775 00:33:38,360 --> 00:33:39,320 IDENTIFYING SPEAKER THAT CAN 776 00:33:39,320 --> 00:33:40,360 SPEAK ON BOTH SIDES OF THE 777 00:33:40,360 --> 00:33:40,840 TOPIC. 778 00:33:40,840 --> 00:33:43,040 WE WANTED TO GIVE YOU A HEADS-UP 779 00:33:43,040 --> 00:33:47,280 WE WEREN'T QUITE READY TO 780 00:33:47,280 --> 00:33:49,400 DISCUSS THIS YET. 781 00:33:49,400 --> 00:33:50,360 WE INTEND TO HAVE A FULL 782 00:33:50,360 --> 00:33:51,080 DISCUSSION IN THE FALL. 783 00:33:51,080 --> 00:33:53,400 SO I THINK THAT'S THE END OF MY 784 00:33:53,400 --> 00:33:55,080 FORMAL PRESENTATION. 785 00:33:55,080 --> 00:33:57,240 AND WE ARE DOING PRETTY WELL ON 786 00:33:57,240 --> 00:33:57,720 TIME. 787 00:33:57,720 --> 00:33:59,040 SO I HAVE ABOUT FIVE MINUTES, 788 00:33:59,040 --> 00:34:00,680 FIVE OR SIX MINUTES FOR 789 00:34:00,680 --> 00:34:01,160 QUESTIONS. 790 00:34:01,160 --> 00:34:11,560 AND I WELCOME THEM. 791 00:34:11,560 --> 00:34:12,600 >>Glen Nuckolls: ERIC WANG WAS 792 00:34:12,600 --> 00:34:15,800 A SENIOR AUTHOR ON THE RECEPTORS 793 00:34:15,800 --> 00:34:20,280 GIDSTUDY SO I WONDER IF HE WILL 794 00:34:20,280 --> 00:34:23,000 PROVIDE COMMENTS ON THAT STUDY? 795 00:34:23,000 --> 00:34:25,440 >>THIS STUDY WAS PERFORMED IN 796 00:34:25,440 --> 00:34:27,680 CORRELATION WITH GARY AND 797 00:34:27,680 --> 00:34:28,840 UNIVERSITY AND I DON'T HAVE TOO 798 00:34:28,840 --> 00:34:34,360 MUCH TO ADD OTHER THAN WE THANK 799 00:34:34,360 --> 00:34:35,960 NINDS FOR SUPPORTING THIS. 800 00:34:35,960 --> 00:34:39,320 IT'S IN THE AREA OF MIO TONIC 801 00:34:39,320 --> 00:34:42,040 THAT HASN'T GOT AS MUCH 802 00:34:42,040 --> 00:34:48,680 ATTENTION AS PERIPHERAL AND SO 803 00:34:48,680 --> 00:34:50,720 THE STUDY SEEKS TO LAY SOME 804 00:34:50,720 --> 00:34:52,280 GROUNDWORK TO IT TO INVESTIGATE 805 00:34:52,280 --> 00:34:52,800 THAT AREA. 806 00:34:52,800 --> 00:34:55,080 THANK YOU, AGAIN, FOR THE 807 00:34:55,080 --> 00:34:55,320 SUPPORT. 808 00:34:55,320 --> 00:34:57,080 >>Diana Bianchi: WHAT ARE YOUR 809 00:34:57,080 --> 00:34:57,360 NEXT STEPS? 810 00:34:57,360 --> 00:35:00,400 >>YEAH, ONE OF THE 811 00:35:00,400 --> 00:35:02,120 PHARMACOLOGICAL APPROACHES IN 812 00:35:02,120 --> 00:35:07,360 THE STUDY WAS TO TEST A MOLECULE 813 00:35:07,360 --> 00:35:14,760 THAT IS FDA APPROVED FOR 814 00:35:14,760 --> 00:35:15,640 BENZODIAZEPINES OVERDOSE AND IT 815 00:35:15,640 --> 00:35:17,000 WAS APPROVED IN THE SAME YEAR 816 00:35:17,000 --> 00:35:19,600 THE GENE FOR MIO TONIC DYSTROPHY 817 00:35:19,600 --> 00:35:22,960 WAS DISCOVERED IN 1992 AND WE 818 00:35:22,960 --> 00:35:25,280 ARE FURTHER PURSUING MECHANISTIC 819 00:35:25,280 --> 00:35:27,320 STUDIES HOW THIS MOLECULE IS 820 00:35:27,320 --> 00:35:28,880 HAVING THE AFFECTS THAT WE 821 00:35:28,880 --> 00:35:30,800 OPENED IN THE MOUSE MODEL. 822 00:35:30,800 --> 00:35:34,000 AND OUR HOPE IS TO GAIN 823 00:35:34,000 --> 00:35:34,480 SUFFICIENT MECHANISTIC 824 00:35:34,480 --> 00:35:37,760 UNDERSTANDING TO SUPPORT FUTURE 825 00:35:37,760 --> 00:35:43,640 CLINICAL STUDIES, FOR EXAMPLE, 826 00:35:43,640 --> 00:35:47,520 INFORMING APPROPRIATE PHARMA CO 827 00:35:47,520 --> 00:35:48,800 KINETIC RELATIONSHIPS AND THE 828 00:35:48,800 --> 00:35:50,040 APPROPRIATE TIMES AND ENDPOINTS 829 00:35:50,040 --> 00:35:52,400 TO LOOK AT IN CLINICAL TRIALS. 830 00:35:52,400 --> 00:35:54,280 SO, THOSE ARE ON GOING NEXT 831 00:35:54,280 --> 00:35:56,840 STEPS FOR THIS PARTICULAR 832 00:35:56,840 --> 00:35:57,840 INVESTIGATION. 833 00:35:57,840 --> 00:35:58,400 >>Diana Bianchi: GREAT. 834 00:35:58,400 --> 00:35:59,520 IT'S REALLY A TREAT TO HAVE YOU 835 00:35:59,520 --> 00:36:01,480 HERE AND TO SHARE THAT WITH US. 836 00:36:01,480 --> 00:36:02,120 >>THANK YOU. 837 00:36:02,120 --> 00:36:03,240 >>Glen Nuckolls: THANK YOU, I 838 00:36:03,240 --> 00:36:06,880 ALSO WANT TO POINT OUT, THE 839 00:36:06,880 --> 00:36:11,120 FIRST AUTHOR WAS RECIPIENT OF A 840 00:36:11,120 --> 00:36:12,800 DIVERSITY SUPPLEMENT TO ERIC AND 841 00:36:12,800 --> 00:36:13,560 GARY'S GRANTS. 842 00:36:13,560 --> 00:36:15,440 SO, DIVERSITY SUPPLEMENT IS A 843 00:36:15,440 --> 00:36:18,840 PROGRAM THAT NIH HAS TO PROVIDE 844 00:36:18,840 --> 00:36:20,280 ADDITIONAL FUNDS TO EXISTING 845 00:36:20,280 --> 00:36:24,680 GRANTS TO SUPPORT STUDENTS, 846 00:36:24,680 --> 00:36:27,000 POSTDOCS, EVEN JUNIOR FACULTY 847 00:36:27,000 --> 00:36:29,360 THAT SPRING DIVERSITY INTO THE 848 00:36:29,360 --> 00:36:34,880 RESEARCH WORKFORCE. 849 00:36:34,880 --> 00:36:39,080 >>Diana Bianchi: GREAT. 850 00:36:39,080 --> 00:36:49,600 ANY OTHER QUESTIONS OR COMMENTS? 851 00:36:51,200 --> 00:36:52,640 >>IS THERE A QUESTION IN THE 852 00:36:52,640 --> 00:36:53,680 CHAT? 853 00:36:53,680 --> 00:36:55,120 >> 854 00:36:55,120 --> 00:36:55,960 >>Glen Nuckolls: YEAH. 855 00:36:55,960 --> 00:37:01,360 HOW WILL BIO RIVET -- BIO 856 00:37:01,360 --> 00:37:04,040 ARCHIVE MODIFY NIH RULES FOR 857 00:37:04,040 --> 00:37:07,480 PUBLICATION AND THAT'S FROM 858 00:37:07,480 --> 00:37:08,400 KEVIN CAMPBELL. 859 00:37:08,400 --> 00:37:09,080 >>Diana Bianchi: THAT'S AN 860 00:37:09,080 --> 00:37:09,560 INTERESTING QUESTION. 861 00:37:09,560 --> 00:37:11,680 WELL, I THINK THE INTENT, I 862 00:37:11,680 --> 00:37:13,120 REALLY DON'T KNOW THE ACTUAL 863 00:37:13,120 --> 00:37:17,520 ANSWER BUT THE INTENT IS MAYBE 864 00:37:17,520 --> 00:37:18,680 TO SCHOLARSHIP OVER BIO ARCHIVE 865 00:37:18,680 --> 00:37:26,400 AND GO DIRECTLY TO MAKING THE 866 00:37:26,400 --> 00:37:27,400 PUBLISHER VERSION AVAILABLE. 867 00:37:27,400 --> 00:37:29,440 BIO ARCHIVE IS NOT THE 868 00:37:29,440 --> 00:37:32,960 PEER-REVIEWED VERSION OF THE 869 00:37:32,960 --> 00:37:34,240 PUBLICATION SO WE CERTAINLY WANT 870 00:37:34,240 --> 00:37:39,680 THE PEER-REVIEWED VERSION TO BE 871 00:37:39,680 --> 00:37:47,920 MADE PUBLIC. 872 00:37:47,920 --> 00:37:48,800 >>Glen Nuckolls: ANY OTHER 873 00:37:48,800 --> 00:37:53,760 QUESTIONS FOR Dr. BIANCHI? 874 00:37:53,760 --> 00:37:55,600 >>Diana Bianchi: I'M GOING TO 875 00:37:55,600 --> 00:37:57,880 YIELD BACK THE TWO MINUTES. 876 00:37:57,880 --> 00:38:00,200 NEXT ON AT AGAIN DA ASK A RECAP 877 00:38:00,200 --> 00:38:03,000 OF THE 2020 MUSCULAR DYSTROPHY 878 00:38:03,000 --> 00:38:04,320 ASSOCIATION CLINICAL AND 879 00:38:04,320 --> 00:38:05,880 SCIENTIFIC CONFERENCE AND THAT 880 00:38:05,880 --> 00:38:11,640 IS GOING TO BE PROVIDED BY 881 00:38:11,640 --> 00:38:12,720 SHARON HESTERLEE. 882 00:38:12,720 --> 00:38:14,520 SO WELCOME, SHARON. 883 00:38:14,520 --> 00:38:16,480 >>Glen Nuckolls: WE HAD A LAST 884 00:38:16,480 --> 00:38:16,960 MINUTE SUBSTITUTION, THE 885 00:38:16,960 --> 00:38:18,760 PRESENTATION IS ACTUALLY GOING 886 00:38:18,760 --> 00:38:20,920 TO BE FROM PAUL MILLMEYER HERE 887 00:38:20,920 --> 00:38:21,880 HERE AT THE TABLE. 888 00:38:21,880 --> 00:38:24,560 >>Diana Bianchi: I'M SORRY, 889 00:38:24,560 --> 00:38:26,280 THAT WAS NOT ON THE RUN OF SHOW 890 00:38:26,280 --> 00:38:27,120 BUT THAT'S OKAY. 891 00:38:27,120 --> 00:38:27,760 >>Glen Nuckolls: WE JUST FOUND 892 00:38:27,760 --> 00:38:29,560 OUT VERY RECENTLY. 893 00:38:29,560 --> 00:38:30,240 >>Diana Bianchi: WELCOME, PAUL. 894 00:38:30,240 --> 00:38:33,360 >>THANK YOU. 895 00:38:33,360 --> 00:38:35,600 APOLOGIES FOR THOSE WHO ARE 896 00:38:35,600 --> 00:38:37,880 EXPECTING Dr. HESTERLEE. 897 00:38:37,880 --> 00:38:41,080 SHE IS NOT DOUBLE BOOKED BUT 898 00:38:41,080 --> 00:38:43,960 TRIPLE BOOKED SO SHE ASKED ME TO 899 00:38:43,960 --> 00:38:45,040 TAKE HER PLACE. 900 00:38:45,040 --> 00:38:46,840 I'M GRATEFUL TO BE TELLING YOU 901 00:38:46,840 --> 00:38:48,880 ABOUT OUR SCIENTIFIC AND 902 00:38:48,880 --> 00:38:51,880 CLINICAL CONFERENCE THAT THE MDA 903 00:38:51,880 --> 00:38:53,800 CONVENED JUST A FEW WEEKS BACK. 904 00:38:53,800 --> 00:38:57,120 SO, I DO KNOW WE HAVE A FEW 905 00:38:57,120 --> 00:38:57,440 SLIDES. 906 00:38:57,440 --> 00:39:07,880 AM I RUNNING THOSE MYSELF? 907 00:39:22,800 --> 00:39:29,680 I DON'T SEE THEM A SPEAR ON MY 908 00:39:29,680 --> 00:39:33,480 SCREEN. 909 00:39:33,480 --> 00:39:33,880 ALL RIGHT. 910 00:39:33,880 --> 00:39:36,640 >>YOU ARE SEEING A SLIDES BUT 911 00:39:36,640 --> 00:39:39,640 THEY'RE NOT IN SCREEN FORMAT. 912 00:39:39,640 --> 00:39:43,200 THE FULL SCREEN. 913 00:39:43,200 --> 00:39:43,400 OKAY. 914 00:39:43,400 --> 00:39:50,600 LET ME DRY THIS AGAIN. 915 00:39:50,600 --> 00:39:51,360 THERE WE GO. 916 00:39:51,360 --> 00:39:52,240 ALL RIGHT. 917 00:39:52,240 --> 00:39:54,480 SO YES WE WANT TO SPEND A FEW 918 00:39:54,480 --> 00:39:55,080 MINUTES TALKING ABOUT THE 919 00:39:55,080 --> 00:39:56,280 TAKEAWAYS FROM THE CONFERENCE 920 00:39:56,280 --> 00:39:58,000 THAT WE CONVENED JUST A FEW 921 00:39:58,000 --> 00:40:00,360 WEEKS AGO BECAUSE WE REALLY ARE 922 00:40:00,360 --> 00:40:01,760 HAPPY WITH HOW IT WENT AND I 923 00:40:01,760 --> 00:40:03,160 THINK THERE'S ACTUALLY A NUMBER 924 00:40:03,160 --> 00:40:04,800 OF THINGS THAT WE'RE DISCUSSED 925 00:40:04,800 --> 00:40:06,280 TURNS THAT CONFERENCE THAT COULD 926 00:40:06,280 --> 00:40:08,360 BE RELEVANT TO THE GENERAL 927 00:40:08,360 --> 00:40:09,960 MISSION OF THIS COORDINATING 928 00:40:09,960 --> 00:40:11,160 COMMITTEE BUT EVEN MORE 929 00:40:11,160 --> 00:40:12,720 SPECIFICALLY THE ACTION PLAN 930 00:40:12,720 --> 00:40:14,480 WE'RE GOING TO DISCUSS LATER 931 00:40:14,480 --> 00:40:18,680 THIS AGENDA. 932 00:40:18,680 --> 00:40:21,240 AS I TRY TO MOVE FORWARD. 933 00:40:21,240 --> 00:40:25,320 LET'S SEE HERE. 934 00:40:25,320 --> 00:40:35,640 USE THE COMPUTER. 935 00:40:36,240 --> 00:40:36,640 COMPUTER. 936 00:40:36,640 --> 00:40:39,120 WE HAD 1500 PEOPLE IN ATTENDANCE 937 00:40:39,120 --> 00:40:40,360 IN-PERSON AT OUR CONFERENCE 938 00:40:40,360 --> 00:40:42,480 WHICH IS ACTUALLY THE BIGGEST 939 00:40:42,480 --> 00:40:43,120 CONFERENCE WE'VE HELD WHICH WE 940 00:40:43,120 --> 00:40:45,560 ARE VERY EXCITED ABOUT. 941 00:40:45,560 --> 00:40:48,040 OF COURSE, DUE TO THE EVOLUTION 942 00:40:48,040 --> 00:40:49,600 THAT THE PANDEMIC HAS BROUGHT 943 00:40:49,600 --> 00:40:51,600 ABOUT, WE ALSO HAVE A VIRTUAL 944 00:40:51,600 --> 00:40:57,760 OPTIONS FOR ATTENDEES AND HAD 945 00:40:57,760 --> 00:40:58,960 550 AND COUNTING BECAUSE OF 946 00:40:58,960 --> 00:41:00,520 COURSE THAT'S THE CONFERENCE 947 00:41:00,520 --> 00:41:02,080 SESSIONS ARE AVAILABLE ON DEMAND 948 00:41:02,080 --> 00:41:06,240 AND ANYBODY WHO WAS UNABLE TO 949 00:41:06,240 --> 00:41:08,560 JOIN US. 950 00:41:08,560 --> 00:41:11,280 WE HAD A NUMBER OF SESSIONS ON A 951 00:41:11,280 --> 00:41:13,440 VARIETY OF TOPICS WHICH WE'LL 952 00:41:13,440 --> 00:41:15,200 GET TO HERE MOMENT TAR TEE AND 953 00:41:15,200 --> 00:41:17,560 YOU CAN SEE THE ADDITIONAL 954 00:41:17,560 --> 00:41:19,040 STATISTICS OF WHO IS THERE WITH 955 00:41:19,040 --> 00:41:21,040 US AND NUMBER OF SPEAKERS, 956 00:41:21,040 --> 00:41:23,680 POSTERS, EXHIBITERS, GRATEFUL TO 957 00:41:23,680 --> 00:41:26,000 HAVE 14 PATIENT ADVOCACY EXHIBIT 958 00:41:26,000 --> 00:41:27,920 NORTH OUR PAVILION AND A NUMBER 959 00:41:27,920 --> 00:41:30,040 OF INDUSTRY FORUMS AND MANY MORE 960 00:41:30,040 --> 00:41:34,400 VIRTUAL POSTERS FROM THERE AND 961 00:41:34,400 --> 00:41:35,960 SO JUST TO TELL YOU ABOUT WHAT 962 00:41:35,960 --> 00:41:37,480 WE COVERED WITHIN THE CONFERENCE 963 00:41:37,480 --> 00:41:39,400 SO THE CONFERENCE ITSELF WAS 964 00:41:39,400 --> 00:41:40,560 MARCH 20th THROUGH 22nd. 965 00:41:40,560 --> 00:41:42,920 AS MOST OF THESE CONFERENCES GO, 966 00:41:42,920 --> 00:41:44,400 WE HAD A VARIETY OF PRE MEETINGS 967 00:41:44,400 --> 00:41:46,040 BECAUSE AS YOU GATHER EVERYBODY 968 00:41:46,040 --> 00:41:47,160 TOGETHER FOR A CONFERENCE, YOU 969 00:41:47,160 --> 00:41:48,440 WANT TO TAKE THE OPPORTUNITY TO 970 00:41:48,440 --> 00:41:50,360 TALK ABOUT SOME OTHER STUFF AT 971 00:41:50,360 --> 00:41:50,960 THE SAME TIME. 972 00:41:50,960 --> 00:41:53,400 SO THE DAY BEFORE OUR 973 00:41:53,400 --> 00:41:54,600 MARCH 19th WE CONVENED OUR 974 00:41:54,600 --> 00:41:56,120 CARE CENTER DIRECTORS AND WE 975 00:41:56,120 --> 00:41:58,400 CONVENED THE PATIENT ADVOCACY 976 00:41:58,400 --> 00:41:59,440 ORGANIZATIONS IN ATTENDANCE AND 977 00:41:59,440 --> 00:42:03,640 HAD A TRAINING NETWORKING 978 00:42:03,640 --> 00:42:06,880 SESSION AND THEN, THE CONFERENCE 979 00:42:06,880 --> 00:42:09,200 WITH Dr. PETER MARKS FOR 980 00:42:09,200 --> 00:42:12,080 EVALUATION AND RESEARCH AT FDA 981 00:42:12,080 --> 00:42:14,200 KICKING THINGS OFF WITH 982 00:42:14,200 --> 00:42:15,000 PRESENTATION WHICH I'LL GET INTO 983 00:42:15,000 --> 00:42:16,920 A LITTLE BIT MORE DETAIL 984 00:42:16,920 --> 00:42:18,120 MOMENTARILY AND A RECOGNITION OF 985 00:42:18,120 --> 00:42:23,840 THE GREAT WORK THAT Dr. 986 00:42:23,840 --> 00:42:26,600 CUDKOWICZ HAS DONE AND I'LL SEE 987 00:42:26,600 --> 00:42:28,000 A FEW EXTRA WORDS MOM TARLY. 988 00:42:28,000 --> 00:42:31,480 THE REMAINDER WAS FOCUSED WAS 989 00:42:31,480 --> 00:42:36,800 TRACKS WITHIN ALS, ULTRA--RARE 990 00:42:36,800 --> 00:42:38,080 MYOPATHY AND BIG DATA AND 991 00:42:38,080 --> 00:42:39,400 PRACTICAL CONSIDERATIONS AND 992 00:42:39,400 --> 00:42:43,880 GENE THERAPY AND SO FOURTH. 993 00:42:43,880 --> 00:42:47,160 SO, WE STARTED OUR EFFORTS FOR 994 00:42:47,160 --> 00:42:48,720 THOSE FOUR DAYS WITH OUR 995 00:42:48,720 --> 00:42:49,880 DIRECTOR MEETING AND FOR THOSE 996 00:42:49,880 --> 00:42:51,800 OF YOU THAT DON'T KNOW ABOUT OUR 997 00:42:51,800 --> 00:42:54,360 CARE CENTER NETWORK, WE HAVE 998 00:42:54,360 --> 00:42:55,560 OVER 150 CARE CENTERS ACROSS THE 999 00:42:55,560 --> 00:42:57,520 UNITED STATES AND YOU SEE THOSE 1000 00:42:57,520 --> 00:42:59,200 HIGHLIGHTED IN THE MAP ON THE 1001 00:42:59,200 --> 00:43:00,760 LEFT SIDE OF THE SCREEN. 1002 00:43:00,760 --> 00:43:03,600 AND THESE OF COURSE ARE THE 1003 00:43:03,600 --> 00:43:05,440 NEURO MUSCULAR DISEASE CLINICS 1004 00:43:05,440 --> 00:43:07,240 ON THE FRONTLINES OF TREATING 1005 00:43:07,240 --> 00:43:08,720 THOSE WITH MUIR OWE MUSCULAR 1006 00:43:08,720 --> 00:43:11,320 DISEASE AND WE PARTNER WITH 1007 00:43:11,320 --> 00:43:13,040 THESE CLINICS TO NOT ONLY OF 1008 00:43:13,040 --> 00:43:14,640 COURSE ASSIST THEM IN THE PRE 1009 00:43:14,640 --> 00:43:16,280 VISION OF CARE AND ALSO TO 1010 00:43:16,280 --> 00:43:18,080 COORDINATE RESOURCES GOING TO 1011 00:43:18,080 --> 00:43:19,120 COMMUNITY MEMBERS WHO ATTEND 1012 00:43:19,120 --> 00:43:21,240 THESE CLINICS FOR THEIR CARE AND 1013 00:43:21,240 --> 00:43:22,880 IT'S AT OUR ANNUAL CARE CENTER 1014 00:43:22,880 --> 00:43:24,680 DIRECTOR MEETING WE TALK ABOUT 1015 00:43:24,680 --> 00:43:28,320 SOME OF THE BIGGEST ISSUES AND 1016 00:43:28,320 --> 00:43:33,000 FACING CLINICAL CARE NEURO MUSS 1017 00:43:33,000 --> 00:43:34,560 YOU LAR DISEASE AND WE TOOK A 1018 00:43:34,560 --> 00:43:35,960 FOCUS, ESPECIALLY THIS YEAR, ON 1019 00:43:35,960 --> 00:43:39,800 GENE THERAPY AND IT'S GOING TO 1020 00:43:39,800 --> 00:43:42,760 BE A TREND YOU WILL HEAR ABOUT 1021 00:43:42,760 --> 00:43:43,800 FROM MY HUGH MINUTES HERE. 1022 00:43:43,800 --> 00:43:45,240 THE INTENSE INTEREST? 1023 00:43:45,240 --> 00:43:49,680 GENE THERAPY ACROSS THE ENTIRE 1024 00:43:49,680 --> 00:43:55,920 FIELD AND THERE ARE GENE THERAPY 1025 00:43:55,920 --> 00:43:57,720 THAT ARE PROMISING AND EXCITING 1026 00:43:57,720 --> 00:43:59,080 AND ALSO RATHER CHALLENGING. 1027 00:43:59,080 --> 00:44:01,920 SO IN THE AFTERNOON, WE HAD A 1028 00:44:01,920 --> 00:44:02,960 SESSION CHAIRED BY Dr. BARRIE 1029 00:44:02,960 --> 00:44:04,960 BURN AND Dr. JOHN DAY TWO OF 1030 00:44:04,960 --> 00:44:07,400 THE MEDICAL ADVISERS ON 1031 00:44:07,400 --> 00:44:08,520 ESSENTIALLY CLINICAL READINESS 1032 00:44:08,520 --> 00:44:12,160 FOR THE APPROVAL OF MUSCULAR 1033 00:44:12,160 --> 00:44:13,600 DYSTROPHY GENE THERAPY AND AT 1034 00:44:13,600 --> 00:44:15,480 THE END OF MAY SO JUST AROUND 1035 00:44:15,480 --> 00:44:17,040 THE CORNER HERE. 1036 00:44:17,040 --> 00:44:18,720 AND I BELIEVE IT WAS Dr. BURN 1037 00:44:18,720 --> 00:44:20,760 WHO ASKED THE 100 OR SO CARE 1038 00:44:20,760 --> 00:44:22,080 CENTERS DIRECTORS IN THE 1039 00:44:22,080 --> 00:44:24,960 AUDIENCE TO RAISE THEIR HAND IF 1040 00:44:24,960 --> 00:44:26,360 THEY THOUGHT WE WERE 1041 00:44:26,360 --> 00:44:27,680 SYSTEMICALLY PREPARED FOR THE 1042 00:44:27,680 --> 00:44:31,520 APPROVAL OF THIS THERAPY AND THE 1043 00:44:31,520 --> 00:44:32,920 PROVISION FROM THERE AND NOT ONE 1044 00:44:32,920 --> 00:44:36,760 HAND WAS RAISED BECAUSE WE SEE 1045 00:44:36,760 --> 00:44:38,040 THE POSSIBILITY OF HUNDREDS IF 1046 00:44:38,040 --> 00:44:40,440 NOT THOUSANDS DEPENDING ON THE 1047 00:44:40,440 --> 00:44:43,080 LABEL WITH BOYS DUCHENNE OR 1048 00:44:43,080 --> 00:44:45,720 YOUNG MEN WITH DUCHENNE WHO WILL 1049 00:44:45,720 --> 00:44:49,040 SEEK THIS GENE THERAPY UPON 1050 00:44:49,040 --> 00:44:50,640 APPROVAL AND HOW STRAINING THAT 1051 00:44:50,640 --> 00:44:57,520 MAY BE TO THIS SYSTEM. 1052 00:44:57,520 --> 00:44:58,480 THERE'S CONCERN THERE ARE 1053 00:44:58,480 --> 00:45:00,800 CLINICS VERSED AND PREPARED FOR 1054 00:45:00,800 --> 00:45:02,000 THE PROVISION OF GENE THERAPY, 1055 00:45:02,000 --> 00:45:05,640 IN PART DUE TO THEIR PROVISION 1056 00:45:05,640 --> 00:45:07,960 OF FOR SMA AND ALSO JUST IN 1057 00:45:07,960 --> 00:45:09,720 GENERAL DUE TO THEIR RESEARCH 1058 00:45:09,720 --> 00:45:11,560 AND CLINICAL EXPERTISE IN AGAIN 1059 00:45:11,560 --> 00:45:12,320 THERAPY AND THEY'RE NOT GOING TO 1060 00:45:12,320 --> 00:45:14,480 BE ABLE TO ACCOMMODATE HUNDREDS 1061 00:45:14,480 --> 00:45:19,840 OR OVER A THOUSAND BOYS AND 1062 00:45:19,840 --> 00:45:21,360 YOUNG MEN AVAILABLE LATER THIS 1063 00:45:21,360 --> 00:45:23,280 SPRING SO IT'S SOMETHING THAT WE 1064 00:45:23,280 --> 00:45:24,560 ARE CERTAINLY VERY FOCUSED ON 1065 00:45:24,560 --> 00:45:26,320 OVER THE NEXT COUPLE OF MONTHS 1066 00:45:26,320 --> 00:45:28,480 AND CERTAINLY OTHERS IN THE 1067 00:45:28,480 --> 00:45:29,280 DUCHENE FIELD ARE AS WELL. 1068 00:45:29,280 --> 00:45:31,800 SO MOVING ON FROM THERE, WE ALSO 1069 00:45:31,800 --> 00:45:35,320 CONVENE THE PATIENT ADVOCACY 1070 00:45:35,320 --> 00:45:36,920 ORGANIZATIONS IN ATTENDANCE AND 1071 00:45:36,920 --> 00:45:38,680 WE'RE GRATEFUL TO HAVE 24 OF OUR 1072 00:45:38,680 --> 00:45:40,960 PARTNERING NON-PROFIT 1073 00:45:40,960 --> 00:45:42,200 ORGANIZATIONS WITH US AND THIS 1074 00:45:42,200 --> 00:45:43,880 IS ACTUALLY BUILDING UPON AN 1075 00:45:43,880 --> 00:45:46,760 EFFORT THAT WE'VE HAD ON GOING 1076 00:45:46,760 --> 00:45:48,000 FOR SEVERAL YEARS NOW AND THIS 1077 00:45:48,000 --> 00:45:51,600 BEING WITHIN OUR NEURO MUSCULAR 1078 00:45:51,600 --> 00:45:52,800 ADVOCACY COLLABORATIVE. 1079 00:45:52,800 --> 00:45:55,280 WITHIN THIS COLLABORATIVE, WE EX 1080 00:45:55,280 --> 00:45:56,840 TOGETHER TO DISCUSSION WHAT ARE 1081 00:45:56,840 --> 00:45:59,200 THE PRESSING POLICY ISSUES AT 1082 00:45:59,200 --> 00:46:01,480 THE TIME AT THE MOMENT FOR EACH 1083 00:46:01,480 --> 00:46:03,280 OF THE INDIVIDUAL PATIENT 1084 00:46:03,280 --> 00:46:05,080 POPULATIONS PERHAPS THAT WE 1085 00:46:05,080 --> 00:46:06,280 SERVE AND WHAT WE FOUND THROUGH 1086 00:46:06,280 --> 00:46:07,920 OUR WORK OVER THE COURSE OF THE 1087 00:46:07,920 --> 00:46:10,160 LAST YEAR OR SO, IS THAT 1088 00:46:10,160 --> 00:46:11,280 COLLABORATION REALLY ON THESE 1089 00:46:11,280 --> 00:46:13,200 FOUR TOPICS YOU SEE IN FRONT OF 1090 00:46:13,200 --> 00:46:15,480 YOU, IS OF GREAT INTEREST FOR 1091 00:46:15,480 --> 00:46:17,160 THE ORGANIZATIONS THAT WE WORK 1092 00:46:17,160 --> 00:46:18,040 WITH. 1093 00:46:18,040 --> 00:46:28,360 FIRST BEING DIAGN DIAGNOSTIC 1094 00:46:28,360 --> 00:46:28,960 SEQUENCING AND MORE SO WHAT 1095 00:46:28,960 --> 00:46:30,040 HAPPENS FROM THERE. 1096 00:46:30,040 --> 00:46:31,200 ACTUALLY HAVING THIS SPECIALISTS 1097 00:46:31,200 --> 00:46:33,120 HAVING THE CLINICIANS AND HAVING 1098 00:46:33,120 --> 00:46:35,680 THE SYSTEMS TO SET UP 1099 00:46:35,680 --> 00:46:37,080 INDIVIDUALS DIAGNOSED WITH THE 1100 00:46:37,080 --> 00:46:39,200 NEURO MUSCULAR DISEASE WITH 1101 00:46:39,200 --> 00:46:40,480 OBTAINING THE APPROPRIATE CARE 1102 00:46:40,480 --> 00:46:40,920 FROM THERE. 1103 00:46:40,920 --> 00:46:42,480 WE OF COURSE SIMILAR TO OUR 1104 00:46:42,480 --> 00:46:46,120 FOCUS ELSEWHERE WITHIN THE 1105 00:46:46,120 --> 00:46:46,760 CONFERENCE, ALSO TALKED ABOUT 1106 00:46:46,760 --> 00:46:47,680 GENE THERAPY AS THIS IS ON THE 1107 00:46:47,680 --> 00:46:50,160 MIND OF MOST INDIVIDUALS IN THE 1108 00:46:50,160 --> 00:46:50,680 PATIENT ADVOCACY COMMUNITY 1109 00:46:50,680 --> 00:46:52,640 PERHAPS THEY ALREADY HAVE EYE 1110 00:46:52,640 --> 00:46:56,560 GENE THERAPY APPROVED SUCH AS 1111 00:46:56,560 --> 00:46:57,960 THOSE WITHIN DUCHENNE BUT REALLY 1112 00:46:57,960 --> 00:46:59,480 THE MAJORITY OF OTHER 1113 00:46:59,480 --> 00:47:00,600 ORGANIZATIONS IN THE SPACE ARE 1114 00:47:00,600 --> 00:47:02,480 HOPING FOR A GENE THERAPY 1115 00:47:02,480 --> 00:47:03,680 APPROVAL AND ONLY THE NEXT 1116 00:47:03,680 --> 00:47:08,280 COUPLE OF YEARS WITH LGMD BEING 1117 00:47:08,280 --> 00:47:12,280 IN THE NEXT HANDFUL YEARS AS 1118 00:47:12,280 --> 00:47:12,880 WELL AS OTHERS. 1119 00:47:12,880 --> 00:47:15,160 WE TALKED ABOUT THE FINANCIAL 1120 00:47:15,160 --> 00:47:16,440 BURDENS OF LIVING WITH NEURO 1121 00:47:16,440 --> 00:47:18,320 MUSCULAR DISEASE AND TRYING TO 1122 00:47:18,320 --> 00:47:19,600 LESSEN THOSE BURDENS AND HOW WE 1123 00:47:19,600 --> 00:47:22,760 CAN REALLY COLLECTIVELY DEVELOP 1124 00:47:22,760 --> 00:47:24,160 ADVOCACY AND INITIATIVES TO 1125 00:47:24,160 --> 00:47:25,000 TACKLE THESE TOGETHER BECAUSE I 1126 00:47:25,000 --> 00:47:27,400 THINK IT'S COMMONSENSE THAT WHEN 1127 00:47:27,400 --> 00:47:28,400 ONE ORGANIZATIONS IS WORKING ON 1128 00:47:28,400 --> 00:47:31,480 THIS WE CAN BE EFFECTIVE AND 1129 00:47:31,480 --> 00:47:32,680 WHEN 24, AND WE REALLY CAN BE 1130 00:47:32,680 --> 00:47:36,240 EVEN MORE EFFECTIVE THAN JUST 1131 00:47:36,240 --> 00:47:36,720 INDIVIDUALLY. 1132 00:47:36,720 --> 00:47:39,040 SO ALSO ON SUNDAY, WE HAD A 1133 00:47:39,040 --> 00:47:40,080 TRAINING NETWORKING SESSION AND 1134 00:47:40,080 --> 00:47:41,280 WE BROUGHT TOGETHER THE NEXT 1135 00:47:41,280 --> 00:47:42,320 GENERATION OF YOUNG 1136 00:47:42,320 --> 00:47:43,160 INVESTIGATORS AND I'LL THANK 1137 00:47:43,160 --> 00:47:46,960 Dr. MICHAELS NOR BEING THERE 1138 00:47:46,960 --> 00:47:48,720 HIMSELF FOR THIS SESSION AS WELL 1139 00:47:48,720 --> 00:47:51,320 AS Dr. GUBITZ WHO WORKED 1140 00:47:51,320 --> 00:47:52,920 TOGETHER ALONG WITH OTHERS IN 1141 00:47:52,920 --> 00:47:55,120 THE ROOM ON JUST TRYING TO LOOK 1142 00:47:55,120 --> 00:47:58,560 AT WHAT THE NEXT GENERATION OF 1143 00:47:58,560 --> 00:48:00,760 YOUNG INVESTIGATORS TRYING TO 1144 00:48:00,760 --> 00:48:02,680 GET THEM TO THE NEXT STEPS 1145 00:48:02,680 --> 00:48:05,320 ESSENTIALLY IN THIS CAREER AND 1146 00:48:05,320 --> 00:48:07,440 HOW MDA CAN SERVE THEM AND IN 1147 00:48:07,440 --> 00:48:09,200 GENERAL THE COMMUNITY CAN MOVE 1148 00:48:09,200 --> 00:48:09,840 FORWARD. 1149 00:48:09,840 --> 00:48:12,680 SO MOVING ON TO THE CONFERENCE 1150 00:48:12,680 --> 00:48:14,680 ITSELF, CONTINUING ON FOCUSING 1151 00:48:14,680 --> 00:48:18,160 ON GENE THERAPY IT'S SAID WE HAD 1152 00:48:18,160 --> 00:48:19,680 PETER MARKS OFFERING OUR KEYNOTE 1153 00:48:19,680 --> 00:48:21,320 AND HE WAS REALIZING THE PROMISE 1154 00:48:21,320 --> 00:48:21,960 OF GONE THERAPY. 1155 00:48:21,960 --> 00:48:24,000 IT WAS REALLY GREAT TO HEAR 1156 00:48:24,000 --> 00:48:25,600 THINGS Dr. MARKS HAD TO SAY 1157 00:48:25,600 --> 00:48:27,760 ABOUT FDA'S APPROACH TO GENE 1158 00:48:27,760 --> 00:48:28,240 THERAPY. 1159 00:48:28,240 --> 00:48:30,000 FOR ONE, OF COURSE, THERE'S A 1160 00:48:30,000 --> 00:48:31,920 GREATER FOCUS WITHIN THE CENTER 1161 00:48:31,920 --> 00:48:34,360 FOR BUY LOGICS ON GENE THERAPY 1162 00:48:34,360 --> 00:48:35,160 GOING FORWARD. 1163 00:48:35,160 --> 00:48:36,440 THEY HAVE A SUPER OFFICE. 1164 00:48:36,440 --> 00:48:39,920 THE OFFICE OF THERAPEUTIC 1165 00:48:39,920 --> 00:48:42,040 PRODUCTS AND THEY'RE HIRING 150 1166 00:48:42,040 --> 00:48:42,920 PEOPLE IN THIS OFFICE AS WELL AS 1167 00:48:42,920 --> 00:48:44,520 A NEW DIRECTOR OF THIS OFFICE 1168 00:48:44,520 --> 00:48:46,680 AND THEY'RE AS Dr. MARKS 1169 00:48:46,680 --> 00:48:49,880 DISCUSSED, POTENTIALLY LAUNCHING 1170 00:48:49,880 --> 00:48:51,080 AN OPERATION WARP SPEED FOR RARE 1171 00:48:51,080 --> 00:48:52,120 DISEASE WITHIN GENE THERAPIES 1172 00:48:52,120 --> 00:48:58,640 FOR RARE DISEASES AND THIS IS 1173 00:48:58,640 --> 00:49:00,440 TAKING THE LESSON BETWEEN THE 1174 00:49:00,440 --> 00:49:02,360 BIOTECHNOLOGY INDUSTRY AND FDA 1175 00:49:02,360 --> 00:49:04,000 FROM COVID AND DEVELOPMENT OF 1176 00:49:04,000 --> 00:49:05,280 VACCINES AND TREATMENTS FOR 1177 00:49:05,280 --> 00:49:07,400 COVID AND SEEING WHAT COULD BE 1178 00:49:07,400 --> 00:49:09,400 APPLIED TO GENE THERAPY FOR RARE 1179 00:49:09,400 --> 00:49:10,560 DISEASES, SOMETHING WE THINK IS 1180 00:49:10,560 --> 00:49:12,000 WARRANTED AND WE'RE EXCITED 1181 00:49:12,000 --> 00:49:14,240 COULD HAVE A NUMBER OF POSITIVE 1182 00:49:14,240 --> 00:49:17,760 IMPLICATIONS FOR DEVELOPMENT IN 1183 00:49:17,760 --> 00:49:19,080 NEURO MUSCULAR DISEASES. 1184 00:49:19,080 --> 00:49:20,680 Dr. MARKS TALKED ABOUT THE 1185 00:49:20,680 --> 00:49:23,840 ACCELERATED APPROVAL PATHWAY, 1186 00:49:23,840 --> 00:49:25,000 FLEXIBILITY, AND PERTAINS TO 1187 00:49:25,000 --> 00:49:26,920 GENE THERAPY DEVELOPMENTS AND 1188 00:49:26,920 --> 00:49:30,200 REEL TRYING TO ACCELERATE EVEN 1189 00:49:30,200 --> 00:49:31,960 FURTHER OF NEW GENE THERAPIES 1190 00:49:31,960 --> 00:49:33,160 BEING DEVELOPED AND BEING 1191 00:49:33,160 --> 00:49:34,560 CONSIDERED AND HOPEFULLY 1192 00:49:34,560 --> 00:49:37,840 APPROVED BY THE FDA AND BEING 1193 00:49:37,840 --> 00:49:38,720 ACCESSIBLE FOR THE COMMUNITY 1194 00:49:38,720 --> 00:49:39,400 FROM THERE. 1195 00:49:39,400 --> 00:49:47,720 WE CELEBRATED Dr. THE 1196 00:49:47,720 --> 00:49:48,920 PRESENTATION TODAY OF COURSE 1197 00:49:48,920 --> 00:49:50,920 BECAUSE ALS IS OUTSIDE THE SCOPE 1198 00:49:50,920 --> 00:49:52,680 OF OUR CONVERSATION TODAY. 1199 00:49:52,680 --> 00:49:56,360 ONE THING THOUGH THAT IS GOOD TO 1200 00:49:56,360 --> 00:49:59,880 MENTION, ONE OF THE MOST 1201 00:49:59,880 --> 00:50:01,840 IMPACTFUL ITEMS OF PROGRESS 1202 00:50:01,840 --> 00:50:06,440 PERHAPS IS Dr. CUKKOWICZ IS 1203 00:50:06,440 --> 00:50:08,000 THE PLATFORM TRIALS OF COURSE 1204 00:50:08,000 --> 00:50:09,320 OFFERING THE OPPORTUNITY TO 1205 00:50:09,320 --> 00:50:12,520 MINIMIZE EXPOSURE TO PLACEBOS 1206 00:50:12,520 --> 00:50:14,760 FOR THOSE WITHIN OR COMMUNITY 1207 00:50:14,760 --> 00:50:16,560 AND FIND SIGNALS TEST SAFETY AND 1208 00:50:16,560 --> 00:50:18,680 SIGNALS OF EFFICACY EVEN QUICKER 1209 00:50:18,680 --> 00:50:21,680 THAN TRADITIONALLY CONSTRUCTED 1210 00:50:21,680 --> 00:50:22,240 CLINICAL TRIALS. 1211 00:50:22,240 --> 00:50:24,880 THERE'S A LOT TO BE LEARNED FROM 1212 00:50:24,880 --> 00:50:26,600 THERE AND APPLIED IN OTHER 1213 00:50:26,600 --> 00:50:27,600 MUSCULAR DYSTROPHY. 1214 00:50:27,600 --> 00:50:28,880 CONVERSATIONS WITH DUCHENNE HAVE 1215 00:50:28,880 --> 00:50:30,880 HAD ON PLATFORMS TRIALS WE SEE 1216 00:50:30,880 --> 00:50:32,480 IMPLICATIONS POTENTIALLY EVEN 1217 00:50:32,480 --> 00:50:36,320 BEYOND DUCHENNE IN THE USE OF 1218 00:50:36,320 --> 00:50:37,520 PLATFORMS TRIALS SO WE WERE 1219 00:50:37,520 --> 00:50:39,720 GRATEFUL TO BE ABLE TO CELEBRATE 1220 00:50:39,720 --> 00:50:40,800 HER IN MUCH OF THE WORK SHE 1221 00:50:40,800 --> 00:50:43,520 CONTINUES TO DO. 1222 00:50:43,520 --> 00:50:45,160 WITHIN THE CONFERENCE SESSIONS 1223 00:50:45,160 --> 00:50:47,760 ITSELF, WE HELD A PANEL ON 1224 00:50:47,760 --> 00:50:50,360 RESEARCH INVESTING AND IT WAS A 1225 00:50:50,360 --> 00:50:51,800 SUMMIT THAT WAS INCLUDED WITHIN 1226 00:50:51,800 --> 00:50:52,880 OUR CONFERENCE. 1227 00:50:52,880 --> 00:50:54,520 AND GRATEFUL TO BE TALKING ABOUT 1228 00:50:54,520 --> 00:50:56,920 THE INVESTING LANDSCAPE WITHIN 1229 00:50:56,920 --> 00:50:59,040 NEURO MUSCULAR DISEASES 1230 00:50:59,040 --> 00:51:00,680 PRESENTATIONS FROM 12 EARLY 1231 00:51:00,680 --> 00:51:03,600 STAGE BIO TECHS ON SOME OF THE 1232 00:51:03,600 --> 00:51:05,560 PROJECTS THAT THEY'RE ON GOING 1233 00:51:05,560 --> 00:51:06,200 AND OPPORTUNITIES FOR INVESTORS 1234 00:51:06,200 --> 00:51:08,400 IN THE ROOM TO CONSIDER 1235 00:51:08,400 --> 00:51:09,720 INVESTING IN THOSE PROJECTS. 1236 00:51:09,720 --> 00:51:10,560 IT'S SOMETHING WE HAVE CONVENED 1237 00:51:10,560 --> 00:51:12,320 IN THE PAST AND WE'RE GRATEFUL 1238 00:51:12,320 --> 00:51:18,360 TO CONVENE AGAIN. 1239 00:51:18,360 --> 00:51:19,720 AGAIN, THE GENE THERAPY WAS 1240 00:51:19,720 --> 00:51:22,400 QUITE THE FOCUS AND IT WAS 1241 00:51:22,400 --> 00:51:23,840 EVIDENT IT WAS THE FOCUS OF 1242 00:51:23,840 --> 00:51:28,920 THOSE WHO ARE ATTENDING BECAUSE, 1243 00:51:28,920 --> 00:51:30,640 WE HAD FOUR TRACKS ON GOING AT 1244 00:51:30,640 --> 00:51:32,440 ANY ONE TIME AND THE GENE 1245 00:51:32,440 --> 00:51:34,680 THERAPY TRACK WAS ACTUALLY, WE 1246 00:51:34,680 --> 00:51:36,880 HAD TO CON INFRASTRUCTURE A VERY 1247 00:51:36,880 --> 00:51:38,040 RAPIDLY OVERFLOW ROOM BECAUSE WE 1248 00:51:38,040 --> 00:51:38,880 RAN OUT OF SPACE. 1249 00:51:38,880 --> 00:51:41,560 WE NEEDED AN OVERFLOW ROOM FOR 1250 00:51:41,560 --> 00:51:42,480 THE OVERFLOW ROOM. 1251 00:51:42,480 --> 00:51:43,880 WHICH SHOWS JUST HOW SUBSTANTIAL 1252 00:51:43,880 --> 00:51:45,680 THE INTEREST IN GENE THERAPY IS 1253 00:51:45,680 --> 00:51:49,040 WITHIN BOTH OF CLINICAL AND THE 1254 00:51:49,040 --> 00:51:50,480 RESEARCH COMMUNITY, WHO WAS 1255 00:51:50,480 --> 00:51:51,400 ATTENDING OUR SUMMIT. 1256 00:51:51,400 --> 00:51:52,520 THERE WAS A NUMBER EVER THINGS 1257 00:51:52,520 --> 00:51:53,920 THAT WERE DISCUSSED THROUGHOUT 1258 00:51:53,920 --> 00:51:55,720 THIS SESSION INCLUDING POTENTIAL 1259 00:51:55,720 --> 00:52:00,000 SAFETY ISSUES AND ADMINISTRATION 1260 00:52:00,000 --> 00:52:01,320 AND PRESSING QUESTIONS FOR THE 1261 00:52:01,320 --> 00:52:03,160 GENE THERAPY AND INCLUDING ON 1262 00:52:03,160 --> 00:52:05,680 DOSING AND ANTIBODY POSITIVE 1263 00:52:05,680 --> 00:52:07,680 PATIENTS AND REDOSING STRATEGIES 1264 00:52:07,680 --> 00:52:09,800 AND THOSE KINDS OF TOPICS AND 1265 00:52:09,800 --> 00:52:11,520 ALSO WITHIN IMPLICATIONS OF 1266 00:52:11,520 --> 00:52:12,960 NEWBORN SCREENING AND CLINICAL 1267 00:52:12,960 --> 00:52:14,400 TRIAL DESIGN AND THE GENE 1268 00:52:14,400 --> 00:52:16,880 THERAPY MIGHT BE HAVING AND 1269 00:52:16,880 --> 00:52:18,160 INCLUDING LESSON LEARNED FROM 1270 00:52:18,160 --> 00:52:19,920 NEWBORN SCREENING IN SMA AND HOW 1271 00:52:19,920 --> 00:52:21,840 IT IMPACTS DUCHENE AND SCREENS 1272 00:52:21,840 --> 00:52:24,320 THAT COULD COME DOWN THE ROAD IF 1273 00:52:24,320 --> 00:52:27,280 OR WHEN GENE THERAPIES ARE 1274 00:52:27,280 --> 00:52:28,600 APPROVED FOR CONDITIONS. 1275 00:52:28,600 --> 00:52:30,600 AND THEN FINALLY ON TRIAL 1276 00:52:30,600 --> 00:52:32,240 DESIGN, HOW THE DEVELOPED 1277 00:52:32,240 --> 00:52:34,040 PROTOCOLS, HOW TO DEVELOP TRIALS 1278 00:52:34,040 --> 00:52:35,760 WITH INCREASING NUMBER OF 1279 00:52:35,760 --> 00:52:37,440 PATIENTS ALREADY BEING TREATED 1280 00:52:37,440 --> 00:52:42,320 BY APPROVED THERAPIES AND TO YOU 1281 00:52:42,320 --> 00:52:45,280 TO APPROACH WHETHER A PLACEBO 1282 00:52:45,280 --> 00:52:45,880 ARM SHOULD BE WIRED. 1283 00:52:45,880 --> 00:52:47,440 DISCUSSING THE PRACTICAL 1284 00:52:47,440 --> 00:52:49,320 MANAGEMENT OF GENE THERAPY AND 1285 00:52:49,320 --> 00:52:50,520 PROCUREMENT ADMINISTRATION AND 1286 00:52:50,520 --> 00:52:52,080 IT'S COMPLEX AND THINGS OF THOSE 1287 00:52:52,080 --> 00:52:53,600 WHO HAVE ACTUALLY NOT 1288 00:52:53,600 --> 00:52:55,120 ADMINISTERED GENE THERAPIES MAY 1289 00:52:55,120 --> 00:53:04,200 NOT BE AWARE OF AND SO 1290 00:53:04,200 --> 00:53:05,640 DISCUSSING AND TRYING TO EDUCATE 1291 00:53:05,640 --> 00:53:07,360 OTHER CLINICS WHO MAY BE GOING 1292 00:53:07,360 --> 00:53:11,200 THROUGH THIS PERHAPS THIS 1293 00:53:11,200 --> 00:53:13,080 SUMMER, FOR EXAMPLE, IF THIS 1294 00:53:13,080 --> 00:53:14,680 APPROVED AND OTHER CLINICAL 1295 00:53:14,680 --> 00:53:16,240 CHALLENGES WITHIN THE GENE 1296 00:53:16,240 --> 00:53:17,520 THERAPY AREA INCLUDING OTHER 1297 00:53:17,520 --> 00:53:19,640 REAL WORLD CHALLENGES ON SAFETY 1298 00:53:19,640 --> 00:53:21,720 AND PRACTICAL AND ETHICAL 1299 00:53:21,720 --> 00:53:22,800 CONSIDERATIONS AND HOW 1300 00:53:22,800 --> 00:53:24,240 CLINICIANS ARE RESPONDING 1301 00:53:24,240 --> 00:53:27,760 REAL-TIME FRANKLY TO THOSE 1302 00:53:27,760 --> 00:53:28,440 PROBLEMS. 1303 00:53:28,440 --> 00:53:30,080 ONE THING WE ANNOUNCED WAS OUR 1304 00:53:30,080 --> 00:53:30,520 KICK-START PROGRAM. 1305 00:53:30,520 --> 00:53:32,200 THIS IS AN EXCITING INITIATIVE 1306 00:53:32,200 --> 00:53:35,640 IN WHICH WE'RE PARTNERING WITH 1307 00:53:35,640 --> 00:53:38,480 THOSE AT U.S. DAVIS TO BRING A 1308 00:53:38,480 --> 00:53:39,600 POTENTIAL GENE THERAPY THROUGH 1309 00:53:39,600 --> 00:53:42,680 THE PRE IND STAGE TO THE CLINIC 1310 00:53:42,680 --> 00:53:45,040 AND WE SEE MANY ISSUES STILL 1311 00:53:45,040 --> 00:53:46,840 WITHIN THIS PROCESS FOR SOME 1312 00:53:46,840 --> 00:53:50,800 GENE THERAPY AND ULTRARARE NEURO 1313 00:53:50,800 --> 00:53:53,080 MUSCULAR DISEASES SO WE'LL 1314 00:53:53,080 --> 00:53:54,320 PILOTING A PROGRAM NOT LIKE 1315 00:53:54,320 --> 00:53:57,040 THOSE ON GOING WITHIN NIH BUT 1316 00:53:57,040 --> 00:53:58,920 STILL BEING A NEEDED FOR GREATER 1317 00:53:58,920 --> 00:54:01,360 STUDY AND GREATER RESEARCH AND 1318 00:54:01,360 --> 00:54:03,240 GREATER TESTING AS SUCH EFFORTS 1319 00:54:03,240 --> 00:54:08,000 AND WE'RE PARTNER WHAT IS Dr. 1320 00:54:08,000 --> 00:54:13,760 HERANO AND OTHERS AT U.S. DAVIS 1321 00:54:13,760 --> 00:54:14,440 ON KICK-START. 1322 00:54:14,440 --> 00:54:24,880 SO WHAT WE'RE FOCUSING ON. 1323 00:54:28,760 --> 00:54:30,480 IT'S SOMETHING THAT MDA HAS BEEN 1324 00:54:30,480 --> 00:54:31,800 WORKING CLOSELY WITH OUR 1325 00:54:31,800 --> 00:54:34,360 PARTNERS AT PPMD AND I'M SURE IF 1326 00:54:34,360 --> 00:54:36,840 FOLKS FROM PPMD ARE ON TODAY BUT 1327 00:54:36,840 --> 00:54:38,160 NIKKI ARM STRONG IS LEADING THE 1328 00:54:38,160 --> 00:54:42,920 CHARGE AND WE'VE BEEN CO 1329 00:54:42,920 --> 00:54:45,200 NOMINATE THE NOMINATION AND 1330 00:54:45,200 --> 00:54:47,760 WE'LL BE HAVING A LOT TO SAY 1331 00:54:47,760 --> 00:54:48,840 CERTAINLY COME FALL TIME. 1332 00:54:48,840 --> 00:54:50,480 Dr. BIANCHI, THANK YOU FOR 1333 00:54:50,480 --> 00:54:52,400 PUTTING THAT ON THE AGENDA AND 1334 00:54:52,400 --> 00:54:53,800 WE'RE EXCITED TO TALK ABOUT 1335 00:54:53,800 --> 00:54:54,120 THAT. 1336 00:54:54,120 --> 00:54:55,160 I'LL TURN IT BACK. 1337 00:54:55,160 --> 00:54:55,680 >>Diana Bianchi: GREAT. 1338 00:54:55,680 --> 00:54:56,880 THANK YOU SO MUCH, PAUL, FOR 1339 00:54:56,880 --> 00:54:58,960 PINCH HITTING AND 1340 00:54:58,960 --> 00:55:01,320 CONGRATULATIONS TO YOU, SHARON 1341 00:55:01,320 --> 00:55:03,680 AND YOUR COLLEAGUES FOR HAVING 1342 00:55:03,680 --> 00:55:06,040 SUCH AN INFORMATIVE AND SUPER 1343 00:55:06,040 --> 00:55:08,160 WELL ATTENDED MEETING. 1344 00:55:08,160 --> 00:55:11,560 THAT'S AMAZING THAT WE'RE NOW 1345 00:55:11,560 --> 00:55:13,360 BACK IN-PERSON. 1346 00:55:13,360 --> 00:55:17,400 ARE THERE ANY QUESTIONS FOR 1347 00:55:17,400 --> 00:55:22,880 PAUL? 1348 00:55:22,880 --> 00:55:30,800 I DON'T SEE ANY IN THE CHATBOX. 1349 00:55:30,800 --> 00:55:34,160 GLEN OR PAUL CAN YOU VAN THE 1350 00:55:34,160 --> 00:55:34,360 ROOM? 1351 00:55:34,360 --> 00:55:35,000 >>Glen Nuckolls: NO QUESTIONS 1352 00:55:35,000 --> 00:55:35,320 IN THE ROOM. 1353 00:55:35,320 --> 00:55:38,800 I WAS ASKING TO ASK IF YOU 1354 00:55:38,800 --> 00:55:40,840 PICKED THE PLACE FOR NEXT YEAR? 1355 00:55:40,840 --> 00:55:45,240 >>ORLANDO, FLORIDA, MARCH 3-6. 1356 00:55:45,240 --> 00:55:46,560 HOPE TO SEE EVERYBODY THERE. 1357 00:55:46,560 --> 00:55:47,160 >>Diana Bianchi: EXCELLENT. 1358 00:55:47,160 --> 00:55:47,760 >>Glen Nuckolls: THANK YOU, 1359 00:55:47,760 --> 00:55:50,840 PAUL. 1360 00:55:50,840 --> 00:55:51,400 >>Diana Bianchi: AGAIN, 1361 00:55:51,400 --> 00:55:53,960 QUESTIONS? 1362 00:55:53,960 --> 00:55:54,520 OKAY. 1363 00:55:54,520 --> 00:55:55,720 WELL, WITHOUT FURTHER ADIEU, 1364 00:55:55,720 --> 00:55:57,600 WE'RE GOING TO GO TO OUR NEXT 1365 00:55:57,600 --> 00:55:59,960 TOPIC WHICH IS AN IMPORTANT ONE 1366 00:55:59,960 --> 00:56:02,560 AND WE'RE GOING TO DISCUSS THE 1367 00:56:02,560 --> 00:56:05,440 MDCC ACTION PLAN FOR THE 1368 00:56:05,440 --> 00:56:05,880 MUSCULAR DYSTROPHIES. 1369 00:56:05,880 --> 00:56:10,440 THIS IS A DOCUMENT THAT IS 1370 00:56:10,440 --> 00:56:12,120 PRODUCED BY OR COMMITTEE WITH 1371 00:56:12,120 --> 00:56:13,160 CONTRIBUTIONS WITH A WIDE RANGE 1372 00:56:13,160 --> 00:56:14,960 OF STAKEHOLDERS INCLUDING 1373 00:56:14,960 --> 00:56:16,520 PATIENTS AND ADVOCATES, 1374 00:56:16,520 --> 00:56:20,120 RESEARCHERS AND FEDERAL 1375 00:56:20,120 --> 00:56:20,600 AGENCIES. 1376 00:56:20,600 --> 00:56:22,240 WHEN IT WAS LAST RENEWED A 1377 00:56:22,240 --> 00:56:23,800 DECADE AGO IN 2015, IT 1378 00:56:23,800 --> 00:56:24,680 REPRESENTED THE CURRENT VIEWS AT 1379 00:56:24,680 --> 00:56:30,280 THE TIME FOR ADVANCING RESEARCH 1380 00:56:30,280 --> 00:56:31,920 TOWARDS A BETTER UNDERSTANDING 1381 00:56:31,920 --> 00:56:34,440 OF MUSCULAR DYSTROPHY. 1382 00:56:34,440 --> 00:56:35,960 AK SET RAILING PRODUCTS AND 1383 00:56:35,960 --> 00:56:37,720 STRATEGIES TO IMPROVE QUALITY OF 1384 00:56:37,720 --> 00:56:40,120 LIFE AND ACCESS TO CARR AND 1385 00:56:40,120 --> 00:56:42,040 SERVICES. 1386 00:56:42,040 --> 00:56:43,320 NOW, I THINK EVERYBODY 1387 00:56:43,320 --> 00:56:44,560 RECOGNIZES A LOT HAS HAPPENED IN 1388 00:56:44,560 --> 00:56:47,360 THE LAST DECADE. 1389 00:56:47,360 --> 00:56:49,160 SO GLEN WOULD LIKE TO PRESENT A 1390 00:56:49,160 --> 00:56:51,640 PLAN FOR RENEWING THE ACTION 1391 00:56:51,640 --> 00:56:54,280 PLAN FOR 2025 AND BEYOND. 1392 00:56:54,280 --> 00:56:55,800 SO I'M GOING TO HAND IT OVER TO 1393 00:56:55,800 --> 00:56:57,800 YOU. 1394 00:56:57,800 --> 00:56:58,240 GLEN. 1395 00:56:58,240 --> 00:56:58,880 >>Glen Nuckolls: THANK YOU, 1396 00:56:58,880 --> 00:57:00,280 Dr. BIANCHI. 1397 00:57:00,280 --> 00:57:02,040 YES, SO I'LL GO THROUGH A 1398 00:57:02,040 --> 00:57:04,640 PROPOSAL THAT WE HAVE FOR 1399 00:57:04,640 --> 00:57:06,920 RENEWAL OF THE ACTION PLAN FOR 1400 00:57:06,920 --> 00:57:07,840 2025 AND BEYOND. 1401 00:57:07,840 --> 00:57:10,040 AND THEN WE HAVE TIME SCHEDULED 1402 00:57:10,040 --> 00:57:11,320 AFTER THIS WHERE WE CAN DISCUSS 1403 00:57:11,320 --> 00:57:13,000 THIS PLAN AND GET IT A LITTLE 1404 00:57:13,000 --> 00:57:23,240 MORE DETAILED. 1405 00:57:24,080 --> 00:57:34,440 MY CLICKER IS NOT WORKING. 1406 00:57:37,000 --> 00:57:38,280 DO YOU WANT TO JUST ADVANCE THE 1407 00:57:38,280 --> 00:57:48,520 SLIDE FOR ME. 1408 00:57:52,800 --> 00:57:54,880 DO YOU DO YOU WANT TO ADVANCE 1409 00:57:54,880 --> 00:57:55,440 THE SLIDE. 1410 00:57:55,440 --> 00:57:57,720 THE ACTION PLAN IS ONE OF THE 1411 00:57:57,720 --> 00:58:01,040 MAIN PRODUCTS OF THIS COMMITTEE 1412 00:58:01,040 --> 00:58:02,480 AND IT WAS -- THE ACTION PLAN 1413 00:58:02,480 --> 00:58:05,640 WAS FIRST DEVELOPED IN 2005 IN 1414 00:58:05,640 --> 00:58:07,800 RESPONSE TO GUIDANCE FROM THE 1415 00:58:07,800 --> 00:58:09,880 LEGISLATION, THE MD CARE ACT AND 1416 00:58:09,880 --> 00:58:13,320 THEN WE RENEWED THE ACTION PLAN 1417 00:58:13,320 --> 00:58:14,480 IN 2025 AND THAT IS THE LAST 1418 00:58:14,480 --> 00:58:16,880 TIME IT WAS RENEWED. 1419 00:58:16,880 --> 00:58:18,680 SO, IT DOES REPRESENT WHAT WAS 1420 00:58:18,680 --> 00:58:23,480 AT THE TIME CURRENT VIEWS OF 1421 00:58:23,480 --> 00:58:24,800 RESEARCHERS AND OTHER 1422 00:58:24,800 --> 00:58:27,120 STAKEHOLDERS ON NOT ONLY KIND OF 1423 00:58:27,120 --> 00:58:29,600 RESEARCH ASPECT OZ RESEARCH AND 1424 00:58:29,600 --> 00:58:30,320 TREATMENT, RESEARCH AND 1425 00:58:30,320 --> 00:58:34,720 UNDERSTANDING DISEASES AND IT 1426 00:58:34,720 --> 00:58:36,200 HAS A NUMBER OF OBJECTIVES THAT 1427 00:58:36,200 --> 00:58:42,440 ARE ORIENTED TOWARDS ENHANCING 1428 00:58:42,440 --> 00:58:43,640 QUALITIES OF LIFE AND ACCESS TO 1429 00:58:43,640 --> 00:58:46,040 CARE AND SERVICES SO, YOU KNOW, 1430 00:58:46,040 --> 00:58:49,000 WE AS FUNDING ORGANIZATIONS AND 1431 00:58:49,000 --> 00:58:51,600 POLICYMAKERS, WE USE THE ACTION 1432 00:58:51,600 --> 00:58:55,920 PLAN TO HELP GUIDE SOME OF OUR 1433 00:58:55,920 --> 00:58:58,720 DECISION-MAKING IT GUIDES 1434 00:58:58,720 --> 00:58:59,960 RESEARCHERS IN PLANNING STUDIES 1435 00:58:59,960 --> 00:59:02,480 AND PARTICULARLY RESEARCHERS WHO 1436 00:59:02,480 --> 00:59:02,960 ARE NEW TO THE MUSCULAR 1437 00:59:02,960 --> 00:59:03,920 DYSTROPHY FIELD AND IT'S NOT 1438 00:59:03,920 --> 00:59:07,640 UNUSUAL THAT WE GET GRANT 1439 00:59:07,640 --> 00:59:10,120 APPLICATIONS AND IN THE 1440 00:59:10,120 --> 00:59:10,680 SIGNIFICANT SECTION THEY'LL 1441 00:59:10,680 --> 00:59:12,720 REFERENCE THE MDCC ACTION PLAN. 1442 00:59:12,720 --> 00:59:17,240 I THINK THE PLAN ALSO HELPS HAD 1443 00:59:17,240 --> 00:59:18,920 EDUCATE PATIENTS AND WHAT IS AT 1444 00:59:18,920 --> 00:59:21,000 THE TIME THE CURRENT LANDSCAPE 1445 00:59:21,000 --> 00:59:24,400 AND FUTURE DIRECTIONS OF 1446 00:59:24,400 --> 00:59:25,320 MUSCULAR DYSTROPHIES RESEARCH 1447 00:59:25,320 --> 00:59:26,400 AND THESE OTHER ACTIVITIES. 1448 00:59:26,400 --> 00:59:37,000 THE MD CARE ACT AS THAT THEY 1449 00:59:37,000 --> 00:59:38,560 WILL REVIEW AND REVISE THE PLAN. 1450 00:59:38,560 --> 00:59:39,880 GIVEN THE PRECEDENCE WITH THE 1451 00:59:39,880 --> 00:59:42,160 LAST RENEWAL WE'RE EXPECTING 1452 00:59:42,160 --> 00:59:43,320 THAT 2025 WOULD BE THE NEXT TIME 1453 00:59:43,320 --> 00:59:47,400 FOR RENEWAL TO BE DONE. 1454 00:59:47,400 --> 00:59:49,720 SO, THE CURRENT PLAN, 20915 1455 00:59:49,720 --> 00:59:52,160 ACTION PLAN AND HERE IS A LINK 1456 00:59:52,160 --> 00:59:54,680 TO THE FULL ACTION PLAN ON OUR 1457 00:59:54,680 --> 00:59:55,280 WEBSITE. 1458 00:59:55,280 --> 00:59:58,680 IT HAS SIX DIFFERENT SECTIONS 1459 00:59:58,680 --> 01:00:01,120 AND MECHANISMS OF MUSCULAR 1460 01:00:01,120 --> 01:00:03,720 DYSTROPHY, DIAGNOSTIC AND 1461 01:00:03,720 --> 01:00:06,160 PRECLINICAL THERAPY THERAPY 1462 01:00:06,160 --> 01:00:06,720 DEVELOPMENT AND LIVING WITH 1463 01:00:06,720 --> 01:00:09,000 MUSCULAR DYSTROPHY WHICH 1464 01:00:09,000 --> 01:00:09,960 INCLUDES NOT ONLY THINGS LIKE 1465 01:00:09,960 --> 01:00:13,240 QUALITY OF LIFE AND ACCESS TO 1466 01:00:13,240 --> 01:00:16,680 CARE BUT ALSO STRATEGIES FOR 1467 01:00:16,680 --> 01:00:17,600 FACILITATING CLINICAL TRIALS AND 1468 01:00:17,600 --> 01:00:18,720 A SECTION ON INFRASTRUCTURE AND 1469 01:00:18,720 --> 01:00:22,080 THAT'S BOTH RESEARCH AND 1470 01:00:22,080 --> 01:00:22,600 INFRASTRUCTURE AND 1471 01:00:22,600 --> 01:00:24,680 INFRASTRUCTURE FOR CARE AND SO 1472 01:00:24,680 --> 01:00:26,120 WITHIN THOSE SIX HEADINGS, THERE 1473 01:00:26,120 --> 01:00:30,720 ARE SUBHEADINGS THAT END UP WITH 1474 01:00:30,720 --> 01:00:34,320 A TOTAL OF 81 OBJECTIVES AND 1475 01:00:34,320 --> 01:00:34,960 GOALS AND IT WAS WRITTEN WITH 1476 01:00:34,960 --> 01:00:36,760 THE A TIME WHERE THERE WAS NO 1477 01:00:36,760 --> 01:00:41,200 APPROVED TREATMENTS FOR ANY FORM 1478 01:00:41,200 --> 01:00:41,880 OF MUSCULAR DYSTROPHY AND WE'VE 1479 01:00:41,880 --> 01:00:42,680 SEEN A LOT OF PROGRESS IN THE 1480 01:00:42,680 --> 01:00:44,400 FIELD SINCE THEN AND A LOT OF 1481 01:00:44,400 --> 01:00:47,360 INCREASE IN THE INVOLVEMENT OF 1482 01:00:47,360 --> 01:00:49,120 INDUSTRY GROUPS AND SOL THE 2015 1483 01:00:49,120 --> 01:00:50,880 PLAN WAS DEVELOPED WITH REALLY 1484 01:00:50,880 --> 01:00:54,840 FAIRLY LIMITED INVOLVEMENT OF 1485 01:00:54,840 --> 01:00:55,320 INDUSTRY RESEARCHERS. 1486 01:00:55,320 --> 01:00:57,200 ANOTHER THING WE'VE SEEN IN THE 1487 01:00:57,200 --> 01:00:59,200 FIELD IS THAT ADVOCACY GROUPS 1488 01:00:59,200 --> 01:01:02,280 AND OTHER ORGANIZATIONS HAVE 1489 01:01:02,280 --> 01:01:03,520 DEVELOPED THEIR OWN MUSCULAR 1490 01:01:03,520 --> 01:01:05,800 DYSTROPHY RELEVANCE STRATEGIC 1491 01:01:05,800 --> 01:01:08,920 PLANS SO THE CURRENT PLAN REALLY 1492 01:01:08,920 --> 01:01:10,080 DOESN'T REFERENCE THOSE OR TAKE 1493 01:01:10,080 --> 01:01:12,080 ADVANTAGE OF THE EFFORTS AND 1494 01:01:12,080 --> 01:01:14,600 BUILD ON THE STRATEGIC PLANNING 1495 01:01:14,600 --> 01:01:19,480 DONE BY OTHER ORGANIZATIONS. 1496 01:01:19,480 --> 01:01:20,680 SO THIS IS A LIST OF THE PEOPLE 1497 01:01:20,680 --> 01:01:22,720 WHO CONTRIBUTED TO THE 2015 1498 01:01:22,720 --> 01:01:25,120 ACTION PLAN SO WE HAD WORKING 1499 01:01:25,120 --> 01:01:26,920 GROUPS FOR EACH OF THE DIFFERENT 1500 01:01:26,920 --> 01:01:29,240 SIX SUBHEADINGS AND A NUMBER OF 1501 01:01:29,240 --> 01:01:33,240 THE NDCC MEMBERS AND CERTAINLY A 1502 01:01:33,240 --> 01:01:35,280 LOT OF EXPERTISE REPRESENTED 1503 01:01:35,280 --> 01:01:37,520 BOTH ACADEMIC AND INVESTIGATORS 1504 01:01:37,520 --> 01:01:44,720 AND PATIENT PATIENT ADVOCATES 1505 01:01:44,720 --> 01:01:45,920 AND FEDERAL AGENCY 1506 01:01:45,920 --> 01:01:48,840 REPRESENTATIVES AND SO FOURTH. 1507 01:01:48,840 --> 01:01:51,080 I WANT TODAY GIVE YOU AN IDEA OF 1508 01:01:51,080 --> 01:01:52,960 GUIDING PRINCIPLES FOR THE 1509 01:01:52,960 --> 01:01:54,280 ACTION PLAN RENEWAL AND WE WOULD 1510 01:01:54,280 --> 01:01:57,000 LIKE FOR IT TO CONTINUE TO 1511 01:01:57,000 --> 01:02:01,640 UPDATE AND REPRESENT THE NOW 1512 01:02:01,640 --> 01:02:04,080 CURRENT VIEWS AN STRATEGIZE FOR 1513 01:02:04,080 --> 01:02:06,680 RESEARCH AND ACCELERATING 1514 01:02:06,680 --> 01:02:10,520 DIAGNOSIS AND DEVELOPING 1515 01:02:10,520 --> 01:02:12,120 EFFECTIVE TREATMENTS AND WHEN 1516 01:02:12,120 --> 01:02:14,080 ENHANCING QUALITY OF LIFE FOR 1517 01:02:14,080 --> 01:02:14,800 INDIVIDUALS LIVING WITH MUD. 1518 01:02:14,800 --> 01:02:17,440 WE WANT TO INCORPORATE THE BROAD 1519 01:02:17,440 --> 01:02:24,840 PERSPECTIVES OF STAKEHOLDERS AND 1520 01:02:24,840 --> 01:02:27,560 PATIENTS FAMILY MEMBERS AND THEY 1521 01:02:27,560 --> 01:02:30,400 WILL SHAPE THE ACTION PLAN AND 1522 01:02:30,400 --> 01:02:31,680 WE'D LIKE TO HAVE REPRESENTATION 1523 01:02:31,680 --> 01:02:33,200 ON EACH OF THE WORKING GROUPS 1524 01:02:33,200 --> 01:02:35,120 FROM A PATIENT OR PATIENT 1525 01:02:35,120 --> 01:02:36,880 ADVOCATE AND WE WOULD LIKE TO 1526 01:02:36,880 --> 01:02:45,880 INCLUDE IN THE ACTION PLAN, 1527 01:02:45,880 --> 01:02:47,120 PATIENT STORIES OR STATEMENTS 1528 01:02:47,120 --> 01:02:48,480 THAT MIGHT BE RELEVANT TO THE 1529 01:02:48,480 --> 01:02:50,600 PLAN SO WE'D LIKE TO INCLUDE NOT 1530 01:02:50,600 --> 01:02:55,880 ONLY ACADEMIC BUT INDUSTRY 1531 01:02:55,880 --> 01:02:57,400 INVESTIGATORS AND IN 1532 01:02:57,400 --> 01:02:58,880 CONTRIBUTING THEIR VIEWS TO THE 1533 01:02:58,880 --> 01:02:59,960 RENEWED ACTION PLAN AND OF 1534 01:02:59,960 --> 01:03:05,440 COURSE, FEDERAL AGENCIES THAT 1535 01:03:05,440 --> 01:03:06,480 REPRESENT FUNDING ORGANIZATIONS 1536 01:03:06,480 --> 01:03:10,440 AS WELL AS AGENCIES THAT PROVIDE 1537 01:03:10,440 --> 01:03:14,560 SERVICES AND WE WOULD LIKE IT TO 1538 01:03:14,560 --> 01:03:16,160 BE USER FRIENDLY AND STREAMLINED 1539 01:03:16,160 --> 01:03:20,480 FROM THE 2015 ACTION PLAN AND 1540 01:03:20,480 --> 01:03:23,760 WE'LL ENCOURAGE EACH OF THE 1541 01:03:23,760 --> 01:03:24,720 WORKING GROUPS DEVELOP SMALLER 1542 01:03:24,720 --> 01:03:26,320 MORE GENERALIZED GOALS SO WE'RE 1543 01:03:26,320 --> 01:03:28,120 THINKING MAYBE THREE TO FOUR PER 1544 01:03:28,120 --> 01:03:29,760 WORKING GROUP AND SO THE TOTAL 1545 01:03:29,760 --> 01:03:32,680 NUMBER OF OBJECTIVES WOULD GO 1546 01:03:32,680 --> 01:03:34,800 FROM 81 DOWN TO 15 OR 20 BUT 1547 01:03:34,800 --> 01:03:37,680 AGAIN THEY WOULD BE BROADER 1548 01:03:37,680 --> 01:03:40,920 GOALS AND THEN WE'LL LINK OUT TO 1549 01:03:40,920 --> 01:03:45,480 INDIVIDUAL STRATEGIC PLANS 1550 01:03:45,480 --> 01:03:46,640 DEVELOPED DID I DISEASE SPECIFIC 1551 01:03:46,640 --> 01:03:48,280 ADVOCACY GROUPS TO PROVIDE MORE 1552 01:03:48,280 --> 01:03:50,760 OF THAT RESOLUTIONS INTO 1553 01:03:50,760 --> 01:03:54,520 INDIVIDUAL DISTRIBUTIONS AWAY 1554 01:03:54,520 --> 01:03:56,000 WANT TO ADDRESS DIVERSITY, 1555 01:03:56,000 --> 01:03:57,240 EQUITY INCLUSION AND 1556 01:03:57,240 --> 01:03:58,080 ACCESSIBILITY AND THE ACTION 1557 01:03:58,080 --> 01:04:01,360 PLAN AND THAT'S APPLYING TO 1558 01:04:01,360 --> 01:04:04,840 PATIENTS TO ACCESS TO CARE AND 1559 01:04:04,840 --> 01:04:06,960 ALSO DIVERSITY OF THE RESEARCH 1560 01:04:06,960 --> 01:04:10,960 STUDIES AND THE RESEARCH 1561 01:04:10,960 --> 01:04:11,920 WORKFORCE AND WOAD LIKE TO 1562 01:04:11,920 --> 01:04:14,440 PROPOSE THE SECTIONS OF THE 25 1563 01:04:14,440 --> 01:04:16,400 ACTION PLANS WOULD BE LISTED 1564 01:04:16,400 --> 01:04:18,760 HERE AND WE HAVE MECHANISMS OF 1565 01:04:18,760 --> 01:04:20,760 MUSCULAR DYSTROPHY INCLUDING 1566 01:04:20,760 --> 01:04:22,320 THOSE THAT ARE UNIQUE TO 1567 01:04:22,320 --> 01:04:23,880 SPECIFIC FORMS OF MUSCULAR 1568 01:04:23,880 --> 01:04:26,080 DYSTROPHY AND THOSE THAT ARE 1569 01:04:26,080 --> 01:04:30,160 COMMON AND IF WE TRY TO PICK 1570 01:04:30,160 --> 01:04:31,760 THREE OR FOUR AGREED GOALS WE 1571 01:04:31,760 --> 01:04:33,120 WON'T BE ABLE TO GO INTO THE 1572 01:04:33,120 --> 01:04:36,920 KIND OF RESOLUTION THAT THE 2015 1573 01:04:36,920 --> 01:04:39,200 PLAN DOES FOR INDIVIDUAL 1574 01:04:39,200 --> 01:04:40,600 DYSTROPHY AND IT WILL LINK TO 1575 01:04:40,600 --> 01:04:43,120 OTHER STRATEGIC PLANS THAT HAVE 1576 01:04:43,120 --> 01:04:44,320 THAT RESOLUTION. 1577 01:04:44,320 --> 01:04:47,280 WE'LL HAVE PRECLINICAL THERAPY 1578 01:04:47,280 --> 01:04:48,920 DEVELOPMENTS AND A SECTION ON 1579 01:04:48,920 --> 01:04:50,480 CLINICAL STUDIES THAT WOULD 1580 01:04:50,480 --> 01:04:54,680 INCLUDE NATURAL HISTORY STUDIES 1581 01:04:54,680 --> 01:04:56,280 AND TRIAL READINESS AND 1582 01:04:56,280 --> 01:05:00,280 BIOMARKERS AND CLINICAL OUTCOME 1583 01:05:00,280 --> 01:05:03,920 ASSESSMENT AND NEWBORN SCREENING 1584 01:05:03,920 --> 01:05:08,360 OR DIAGNOSTICS STRATEGIES AND 1585 01:05:08,360 --> 01:05:10,560 CLINICAL TRIALS AND WE'LL HAVE 1586 01:05:10,560 --> 01:05:11,520 ENHANCING QUALITY OF LIFE AND 1587 01:05:11,520 --> 01:05:13,200 ACCESS TO CARE WHICH WAS KIND OF 1588 01:05:13,200 --> 01:05:20,200 THE LIVING WITH SECTION FROM THE 1589 01:05:20,200 --> 01:05:26,960 2015 PLAN AND A SECTION THAT 1590 01:05:26,960 --> 01:05:29,960 ADDRESSES AND PARTNERSHIP AMONG 1591 01:05:29,960 --> 01:05:31,760 ADVOCACY GROUPS OR INDUSTRY 1592 01:05:31,760 --> 01:05:33,440 GROUPS, WHERE THEY ARE AND KIND 1593 01:05:33,440 --> 01:05:37,360 OF TEAMS SCIENCE AND DATA 1594 01:05:37,360 --> 01:05:47,320 SHARING AND CAREER DEVELOPMENT 1595 01:05:47,320 --> 01:05:49,000 AND RESEARCH INFRASTRUCTURE AND 1596 01:05:49,000 --> 01:05:50,640 INTO EACH OF THE RELEVANT 1597 01:05:50,640 --> 01:05:54,720 SECTIONS OF THE ACTION PLAN AND 1598 01:05:54,720 --> 01:05:57,000 AS MENTIONED WE WANT TO BUILD 1599 01:05:57,000 --> 01:05:59,360 UPON INNER GUYS WITH AND REALLY 1600 01:05:59,360 --> 01:06:00,920 INTEGRATE THIS STRATEGIC PLANS 1601 01:06:00,920 --> 01:06:03,480 THAT ARE DEVELOPED BY MDCC 1602 01:06:03,480 --> 01:06:07,920 MEMBER ORGANIZATION AND OTHER 1603 01:06:07,920 --> 01:06:10,120 ADVOCACY GROUPS. 1604 01:06:10,120 --> 01:06:12,440 OF COURSE, THE MUSCULAR 1605 01:06:12,440 --> 01:06:15,800 DYSTROPHY COORDINATING COMMITTEE 1606 01:06:15,800 --> 01:06:17,960 AS A WHOLE WOULD BE CONSIDERING 1607 01:06:17,960 --> 01:06:22,320 THE RENEWED PLAN FOR APPROVAL 1608 01:06:22,320 --> 01:06:23,000 AND WE'RE FIGURING THAT CAN 1609 01:06:23,000 --> 01:06:26,160 HAPPEN IN THE SPRING MEETING OF 1610 01:06:26,160 --> 01:06:27,360 2015 AND SO LEADERSHIP OF THE 1611 01:06:27,360 --> 01:06:31,440 OVER ALL PROCESS WOULD INCLUDE 1612 01:06:31,440 --> 01:06:34,240 Dr. BIANCHI AND Dr. CRISWELL 1613 01:06:34,240 --> 01:06:39,320 FROM NIAMS SHE'S THE CHAIR FOR 1614 01:06:39,320 --> 01:06:41,080 2024 AND 2025 AND WE'D LIKE TO 1615 01:06:41,080 --> 01:06:43,960 HAVE A MDCC ACTION PLAN STEERING 1616 01:06:43,960 --> 01:06:47,320 GROUP THAT WOULD BE COMPOSED OF 1617 01:06:47,320 --> 01:06:51,080 VOLUNTEERS AMONG THE MEMBERS OF 1618 01:06:51,080 --> 01:06:53,200 MDCC AND THE HOLES OF THIS 1619 01:06:53,200 --> 01:06:56,720 STEERING GROUP WOULD BE TO 1620 01:06:56,720 --> 01:06:58,160 IDENTIFY OR NOMINATE THE MEMBERS 1621 01:06:58,160 --> 01:06:59,360 OF EACH OF THE WORKING GROUPS 1622 01:06:59,360 --> 01:07:08,200 AND TO BE MORE INVOLVED WITH ALL 1623 01:07:08,200 --> 01:07:09,800 THE RENEWED ACTION PLAN 1624 01:07:09,800 --> 01:07:12,920 INCLUDES. 1625 01:07:12,920 --> 01:07:14,240 AND SO I WILL SEND OUT AFTER 1626 01:07:14,240 --> 01:07:15,880 THIS MEETING I'LL SEND OUT AN 1627 01:07:15,880 --> 01:07:17,880 E-MAIL TO MDCC MEMBERS INVITING 1628 01:07:17,880 --> 01:07:23,280 YOU IF YOU ARE INTERESTED, TO BE 1629 01:07:23,280 --> 01:07:24,040 PART OF THIS STEERING GROUP. 1630 01:07:24,040 --> 01:07:25,600 AND THEN WE HAVE THE TIME 1631 01:07:25,600 --> 01:07:27,400 WORKING GROUPS AND EACH WOULD BE 1632 01:07:27,400 --> 01:07:29,560 PROBABLY SIX TO EIGHT 1633 01:07:29,560 --> 01:07:30,160 PARTICIPANTS INCLUDING 1634 01:07:30,160 --> 01:07:31,800 RESEARCHERS AND PATIENT 1635 01:07:31,800 --> 01:07:36,600 ADVOCATES AND OTHER STAKEHOLDERS 1636 01:07:36,600 --> 01:07:38,440 AND WE NEED TO CONSIDER GENDER, 1637 01:07:38,440 --> 01:07:42,680 RACE, ETHNICITY AND GEOGRAPHIC 1638 01:07:42,680 --> 01:07:44,240 REPRESENTATION ON THESE WORKING 1639 01:07:44,240 --> 01:07:46,600 GROUPS BUT WE CONSIDER WHAT 1640 01:07:46,600 --> 01:07:47,440 TYPES OF MUSCULAR DYSTROPHY ARE 1641 01:07:47,440 --> 01:07:51,000 INCLUDED IN EACH GROW AND WE 1642 01:07:51,000 --> 01:07:58,520 HAVE NIH STAFF AS DELAY ONS FOR. 1643 01:07:58,520 --> 01:08:01,600 THE OVER ALL OUTLINE IS PROSED 1644 01:08:01,600 --> 01:08:02,400 AND INTRODUCTION TA TALKS ABOUT 1645 01:08:02,400 --> 01:08:06,520 THE DIFFERENT FORMS OF DYSTROPHY 1646 01:08:06,520 --> 01:08:09,800 AND LEGISLATION IN NDCC AND THE 1647 01:08:09,800 --> 01:08:11,080 PROCESS IN THE RENEWAL AND WE 1648 01:08:11,080 --> 01:08:14,960 WANTED TO HAVE A SECTION WHERE 1649 01:08:14,960 --> 01:08:16,880 IT DISCUSSES PROGRESS ON THE 1650 01:08:16,880 --> 01:08:18,160 2015 ACTION PLAN SO WHAT HAPPENS 1651 01:08:18,160 --> 01:08:19,960 SINCE THEN AND WE THOUGHT ABOUT 1652 01:08:19,960 --> 01:08:23,520 DIFFERENT WAYS TO DO THIS AND WE 1653 01:08:23,520 --> 01:08:26,240 CAN DO AN EXAUSTIVE PORTFOLIO 1654 01:08:26,240 --> 01:08:27,880 ANALYSIS AND DETERMINE YOU KNOW 1655 01:08:27,880 --> 01:08:29,760 WHAT GRANTS WERE AWARDED RELATED 1656 01:08:29,760 --> 01:08:31,560 TO THE DIFFERENT OBJECTS AND 1657 01:08:31,560 --> 01:08:33,000 WHAT CAME OUT AND I THINK WE'RE 1658 01:08:33,000 --> 01:08:35,000 TRYING TO MAKE THIS A USER 1659 01:08:35,000 --> 01:08:37,800 FRIENDLY DOCUMENT. 1660 01:08:37,800 --> 01:08:39,200 WE THOUGHT IT MIGHT BE HELPFUL 1661 01:08:39,200 --> 01:08:41,440 AND MORE USER FRIENDLY TO JUST 1662 01:08:41,440 --> 01:08:43,360 PROVIDE FOR EACH OF THE 81 1663 01:08:43,360 --> 01:08:45,160 OBJECTIVES, JUST A COUPLE OF SEN 1664 01:08:45,160 --> 01:08:46,600 ESSENTIALS THAT SUMMARIZES THE 1665 01:08:46,600 --> 01:08:48,240 PROGRESS THAT HAS BEEN MADE ON 1666 01:08:48,240 --> 01:08:51,080 THAT OBJECTIVE AND I THINK IT 1667 01:08:51,080 --> 01:08:54,080 WOULD TAKE A LOT OF NIH STAFF 1668 01:08:54,080 --> 01:08:55,520 AND ALSO MEMBERS OF THE WORKING 1669 01:08:55,520 --> 01:08:57,160 GROUP TO HELP FORMULATE WHAT 1670 01:08:57,160 --> 01:09:00,920 THOSE SUMMARIES ARE SO THAT WE 1671 01:09:00,920 --> 01:09:02,840 KNOW FOR EACH OBJECTIVE IS 1672 01:09:02,840 --> 01:09:05,200 THINGS HAVE PROGRESSED AND WE'D 1673 01:09:05,200 --> 01:09:07,480 HAVE THE FIVE SECTIONS THAT ARE 1674 01:09:07,480 --> 01:09:11,600 DESCRIBED EARLIER WITH THREE TO 1675 01:09:11,600 --> 01:09:12,560 FOUR PRIORITIES IDENTIFIED IN 1676 01:09:12,560 --> 01:09:17,640 EACH OF THOSE SECTIONS AND THEN 1677 01:09:17,640 --> 01:09:19,160 HAVE SECTION FOUR WOULD BE THE 1678 01:09:19,160 --> 01:09:21,280 STRATEGIC PLANS OF OTHER 1679 01:09:21,280 --> 01:09:24,400 ORGANIZATIONS RELATIVE TO THE 1680 01:09:24,400 --> 01:09:25,480 MESS KULAR DES TROPHY AND THIS 1681 01:09:25,480 --> 01:09:29,760 AFTERNOON WE'RE HEAL FROM THREE 1682 01:09:29,760 --> 01:09:32,320 DIFFERENT GROUPS FROM MDCC ABOUT 1683 01:09:32,320 --> 01:09:35,200 THEIR PLAN THAT GETS INTO MORE 1684 01:09:35,200 --> 01:09:40,000 OF A FINE DETAILS FOR VIN TYPES 1685 01:09:40,000 --> 01:09:41,440 OF MESS KULAR DYSTROPHY AND WE 1686 01:09:41,440 --> 01:09:45,640 HAVE ANNA PEN DICTION WITH 1687 01:09:45,640 --> 01:09:48,040 SUMMARY OF PUBLIC COMMENTS AND 1688 01:09:48,040 --> 01:09:49,400 LIZARRAGA LISTING OF CONTRIBUTE 1689 01:09:49,400 --> 01:09:51,920 TOURS TO THE PLAN. 1690 01:09:51,920 --> 01:09:54,280 SO, OVER ALL KIND OF PROPOSED 1691 01:09:54,280 --> 01:09:56,200 TIMELINE FOR THIS RENEWAL, WOULD 1692 01:09:56,200 --> 01:10:01,240 CONTINUE FROM NOW MUCH AND 1693 01:10:01,240 --> 01:10:02,720 THROUGH 2025 AND WE ARE 1694 01:10:02,720 --> 01:10:04,640 PRESENTING PLANS INFORM THE NDCC 1695 01:10:04,640 --> 01:10:09,680 AT OUR SPRING MEETING FOR 2023 1696 01:10:09,680 --> 01:10:11,720 AND WE'LL INVITE MEMBERS TO BE 1697 01:10:11,720 --> 01:10:13,160 PART OF THIS STEERING GROUP AND 1698 01:10:13,160 --> 01:10:17,360 THAT HELPS IDENTIFY THE WORKING 1699 01:10:17,360 --> 01:10:18,840 GROUP MEMBERS AND WEEP ESTABLISH 1700 01:10:18,840 --> 01:10:22,280 EX CHARGE THOSE WORKING GROUPS 1701 01:10:22,280 --> 01:10:23,640 AND WE'RE THINKING MAYBE TWO ON 1702 01:10:23,640 --> 01:10:26,080 LIBRARY MEETINGS WITH INDIVIDUAL 1703 01:10:26,080 --> 01:10:26,840 WORKING GROUPS AND CERTAINLY A 1704 01:10:26,840 --> 01:10:28,560 LOT OF E-MAIL EXCHANGE WITH THEM 1705 01:10:28,560 --> 01:10:31,600 AND OTHER TIMES TO GIVE THEM THE 1706 01:10:31,600 --> 01:10:33,760 IDEA OF WHAT WE'RE LOOKING FOR 1707 01:10:33,760 --> 01:10:35,440 AND LET THEM LOOK OVER THE PRIOR 1708 01:10:35,440 --> 01:10:39,320 PLAN AND THINKING ABOUT WHAT IS 1709 01:10:39,320 --> 01:10:42,000 GOING ON IN THE FIELD AND 1710 01:10:42,000 --> 01:10:42,640 FORMULATE THOSE PRIORITIES AND 1711 01:10:42,640 --> 01:10:44,760 AS I MENTIONED WE REALLY LIKE TO 1712 01:10:44,760 --> 01:10:47,560 INCLUDE INDUSTRY RESEARCHERS 1713 01:10:47,560 --> 01:10:51,200 MORE DIRECTLY IN THE RENEWAL AND 1714 01:10:51,200 --> 01:10:54,240 WE ARE A FACA COMMITTEE PART OF 1715 01:10:54,240 --> 01:10:56,840 THE EXECUTIVE BRANCH AND SO WE 1716 01:10:56,840 --> 01:11:00,520 HAVE TO DO THIS KIND OF NOT WITH 1717 01:11:00,520 --> 01:11:04,280 RESPECT TO CURRENT POLICIES AND 1718 01:11:04,280 --> 01:11:05,560 IT NEEDS TO BE TRANSPARENT WHAT 1719 01:11:05,560 --> 01:11:07,760 WE'RE DOING SO WE'D LIKE TO HAVE 1720 01:11:07,760 --> 01:11:10,120 A SEPARATE ON-LINE MEETING WITH 1721 01:11:10,120 --> 01:11:13,680 INDUSTRY RESEARCHERS AND WE CAN 1722 01:11:13,680 --> 01:11:14,960 MAKE SURE WE GET THE WORD OUT TO 1723 01:11:14,960 --> 01:11:17,520 AS MANY WHO ARE INTERESTED IN 1724 01:11:17,520 --> 01:11:21,400 PARTICIPATING AND HAVE THAT 1725 01:11:21,400 --> 01:11:22,720 EITHER BE LIVESTREAM SO PEOPLE 1726 01:11:22,720 --> 01:11:24,360 FROM THE PUBLIC CAN SEE WHAT IS 1727 01:11:24,360 --> 01:11:27,520 GOING ON OR HAVE DETAILED 1728 01:11:27,520 --> 01:11:29,880 SUMMARY OF THE DISCUSSIONS THERE 1729 01:11:29,880 --> 01:11:35,200 AND REALLY TO GET THE INPUT FROM 1730 01:11:35,200 --> 01:11:36,120 INDUSTRY HERE DIRECTLY INTO THE 1731 01:11:36,120 --> 01:11:38,200 RENEWAL OF THE ACTION PLAN AND 1732 01:11:38,200 --> 01:11:39,760 WE'D LIKE TO HAVE AN IN-PERSON 1733 01:11:39,760 --> 01:11:41,680 MEETING THAT WOULD INCLUDE NOT 1734 01:11:41,680 --> 01:11:44,640 ONLY ALL OF THE WORKING GROUPS 1735 01:11:44,640 --> 01:11:48,320 BUT ALSO THE MDCC ITSELF AND WE 1736 01:11:48,320 --> 01:11:50,360 CAN SCHEDULE THIS TO CORRESPOND 1737 01:11:50,360 --> 01:11:52,360 WITH THE FALL MEETING IN 2024 1738 01:11:52,360 --> 01:11:53,960 AND HAVE THE WORKING GROUPS ALL 1739 01:11:53,960 --> 01:11:56,200 MEETING THE DAY BEFORE AND THE 1740 01:11:56,200 --> 01:11:58,440 DAY AFTER AND THE MDCC MEETING 1741 01:11:58,440 --> 01:12:01,920 IS IF PEOPLE WANT TO PARTICIPATE 1742 01:12:01,920 --> 01:12:03,160 ATTEND BOTH THAT WOULD BE 1743 01:12:03,160 --> 01:12:04,600 AVAILABLE AND THEN WE'LL FINISH 1744 01:12:04,600 --> 01:12:06,960 UP SOME EDITING WITH HELP FROM 1745 01:12:06,960 --> 01:12:08,760 THE WORKING GROUPS AND STEERING 1746 01:12:08,760 --> 01:12:13,080 GROUP AND SOLICIT FOR PUBLIC 1747 01:12:13,080 --> 01:12:15,000 COMMENT AND AND ALSO AS I 1748 01:12:15,000 --> 01:12:16,360 MENTIONED BEFORE, IN THE SPRING 1749 01:12:16,360 --> 01:12:20,520 MEETING OF 2025, BRING THE 1750 01:12:20,520 --> 01:12:23,240 FINALIZED RENEWAL FOR 2025 TO 1751 01:12:23,240 --> 01:12:28,600 VOTE FOR NDCC AND WITH WOULD 1752 01:12:28,600 --> 01:12:30,880 PUBLISH IT IN OUR WEBSITE AND 1753 01:12:30,880 --> 01:12:36,200 REPORT OUT TO THE RELEVANT NIH 1754 01:12:36,200 --> 01:12:38,360 INSTITUTE COUNCIL BRIEFLY SO IN 1755 01:12:38,360 --> 01:12:42,120 20915 PLAN WE DEVELOPED A 1756 01:12:42,120 --> 01:12:49,120 PUBLICATION IN MUSCLE AND NERVE 1757 01:12:49,120 --> 01:12:51,720 THAT WE WERE LAST AUTHORS ON 1758 01:12:51,720 --> 01:12:54,200 TALKED ABOUT THE ACTION PLAN AND 1759 01:12:54,200 --> 01:12:54,920 THE MUSCULAR DYSTROPHY 1760 01:12:54,920 --> 01:12:56,920 COORDINATING COMMITTEE AND KIND 1761 01:12:56,920 --> 01:13:02,040 OF THE PROCESS THAT LED TO THE 1762 01:13:02,040 --> 01:13:04,280 ACTION PLAN AND IT INCREASED THE 1763 01:13:04,280 --> 01:13:06,320 VIS A LOT OF WHAT WE'VE DONE SO 1764 01:13:06,320 --> 01:13:10,440 WE MIGHT DO THAT AGAIN FOR THE 1765 01:13:10,440 --> 01:13:10,960 2025 PLAN. 1766 01:13:10,960 --> 01:13:12,840 THAT'S THE PROPOSAL AND I'D BE 1767 01:13:12,840 --> 01:13:21,080 HAPPY TO OPEN THE FLOOR FOR ANY 1768 01:13:21,080 --> 01:13:31,280 QUESTIONS. 1769 01:13:32,080 --> 01:13:40,200 I OPEN IT FOR ANY QUESTIONS. 1770 01:13:40,200 --> 01:13:44,560 EVERYBODY COOL WITH THAT? 1771 01:13:44,560 --> 01:13:45,360 >>Diana Bianchi: I THINK IT 1772 01:13:45,360 --> 01:13:46,080 LOOKS GREAT. 1773 01:13:46,080 --> 01:13:47,680 THERE'S ALWAYS QUITE A BIT WORK 1774 01:13:47,680 --> 01:13:51,280 DONE IN THIS DRAFT PLAN. 1775 01:13:51,280 --> 01:13:52,600 CONGRATULATIONS TO YOU AND YOUR 1776 01:13:52,600 --> 01:13:53,200 TEAM. 1777 01:13:53,200 --> 01:13:54,880 THE LINK THAT IS IN THE CHATBOX 1778 01:13:54,880 --> 01:13:57,600 IS THAT THE PROPOSED ACTION PLAN 1779 01:13:57,600 --> 01:13:59,920 OR THE 2015 ACTION PLAN? 1780 01:13:59,920 --> 01:14:00,720 I HAVEN'T CLICKED ON IT. 1781 01:14:00,720 --> 01:14:03,680 >>IT'S THE 2015 ACTION PLAN THE 1782 01:14:03,680 --> 01:14:08,000 ONE WE CURRENTLY HAVE ON OUR 1783 01:14:08,000 --> 01:14:12,240 WEBSITE. 1784 01:14:12,240 --> 01:14:14,000 FROM DONOVAN, HOW CAN 1785 01:14:14,000 --> 01:14:15,200 NON-MEMBERS BE INVOLVED? 1786 01:14:15,200 --> 01:14:17,920 WELL, YOU KNOW, MULTIPLE WAYS, I 1787 01:14:17,920 --> 01:14:24,400 THINK THAT WE'LL CERTAINLY BE 1788 01:14:24,400 --> 01:14:25,040 MEMBERS OF THE STEERING GROUP 1789 01:14:25,040 --> 01:14:28,240 WILL BE ABLE TO RECOMMEND WHO TO 1790 01:14:28,240 --> 01:14:32,520 INCLUDE ON THE DIFFERENT WORKING 1791 01:14:32,520 --> 01:14:34,480 GROUPS AND THAT IS ONE 1792 01:14:34,480 --> 01:14:36,520 OPPORTUNITY FOR NON MEMBERS TO 1793 01:14:36,520 --> 01:14:40,280 PARTICIPATE AND THEN OF COURSE 1794 01:14:40,280 --> 01:14:42,280 WE WILL BE POSTING THE DRAFT TO 1795 01:14:42,280 --> 01:14:49,360 THE VIA REQUEST SO ANYONE CAN 1796 01:14:49,360 --> 01:14:51,640 THE PUBLIC AND ON THE ACTION 1797 01:14:51,640 --> 01:14:55,400 PLAN SO THOSE ARE THE PLUS 1798 01:14:55,400 --> 01:14:57,680 WHATEVER WE HAVE NDCC MEETINGS 1799 01:14:57,680 --> 01:15:03,400 AND RELEVANT TO IT THEN OTHER 1800 01:15:03,400 --> 01:15:04,720 PLANTS CAN DEVELOP IF THE 1801 01:15:04,720 --> 01:15:14,000 DEVELOPMENT OF THE PLAN. 1802 01:15:14,000 --> 01:15:16,040 >>YEAH, HOW ARE THE 1803 01:15:16,040 --> 01:15:18,400 RECOMMENDATIONS IN THE DOCUMENT 1804 01:15:18,400 --> 01:15:20,400 INCORPORATED INTO FINAL 1805 01:15:20,400 --> 01:15:22,520 DECISION-MAKING AND HOW MUCH 1806 01:15:22,520 --> 01:15:24,760 AGENCY DO WE HAVE AS A COMMITTEE 1807 01:15:24,760 --> 01:15:32,200 OVER THESE DECISIONS? 1808 01:15:32,200 --> 01:15:36,960 >>I WOULD DEFER TO AN INSTITUTE 1809 01:15:36,960 --> 01:15:41,760 DIRECTOR AT LEAST IN TERMS OF 1810 01:15:41,760 --> 01:15:46,800 DESCRIBING MAYBE HOW NIH USES 1811 01:15:46,800 --> 01:15:47,160 THE ACTION PLAN. 1812 01:15:47,160 --> 01:15:51,120 >>I CAN FOLLOW WITH WALTER. 1813 01:15:51,120 --> 01:15:53,320 YOU HAVE THE HISTORY OF THE 1814 01:15:53,320 --> 01:15:56,280 PRIOR ACTION PLAN AS WELL. 1815 01:15:56,280 --> 01:15:59,880 >>THANK YOU, DIANA. 1816 01:15:59,880 --> 01:16:03,880 YOU KNOW, I THINK WE HAVE DONE 1817 01:16:03,880 --> 01:16:06,080 THIS VERY SIMILAR THINGS 1818 01:16:06,080 --> 01:16:09,320 MULTIPLE DIFFERENT TIMES FOR 1819 01:16:09,320 --> 01:16:11,120 DIFFERENT CONDITIONS AND IT'S 1820 01:16:11,120 --> 01:16:14,520 REALLY VERY IMPACTFUL IN TERMS 1821 01:16:14,520 --> 01:16:16,000 OF WHAT WE DO AS AN AGENCY GOING 1822 01:16:16,000 --> 01:16:19,280 FORWARD ON THE INSIDE AND ALSO 1823 01:16:19,280 --> 01:16:22,000 IT IS A GOOD GUIDE TO 1824 01:16:22,000 --> 01:16:26,560 INVESTIGATORS IN THE OUTSIDE 1825 01:16:26,560 --> 01:16:28,080 INTERESTED IN WHAT THE COMMUNITY 1826 01:16:28,080 --> 01:16:29,520 THINKS IS MOST IMPORTANT. 1827 01:16:29,520 --> 01:16:32,880 I THINK GLEN, FOR THIS ONE, YOU 1828 01:16:32,880 --> 01:16:37,680 KNOW, WHEN THEY ARE PURELY NIH 1829 01:16:37,680 --> 01:16:39,480 PLANS, WE'RE IN OUR COMFORT 1830 01:16:39,480 --> 01:16:39,960 ZONE. 1831 01:16:39,960 --> 01:16:42,480 THIS ONE IS GOING TO CUT ACROSS 1832 01:16:42,480 --> 01:16:43,640 MULTIPLE DIFFERENT AGENCIES SO 1833 01:16:43,640 --> 01:16:47,280 THAT'S WHERE I THINK WE'RE GOING 1834 01:16:47,280 --> 01:16:50,840 TO NEED A LITTLE BIT OF GUIDANCE 1835 01:16:50,840 --> 01:16:52,480 FROM EVERYBODY AROUND THE TABLE 1836 01:16:52,480 --> 01:16:56,400 ON HOW TO DO THIS, RIGHT. 1837 01:16:56,400 --> 01:17:01,480 WE DID SOMETHING LIKE THIS IN 1838 01:17:01,480 --> 01:17:05,000 THE INCOMPETENTER AGENCY PAIN 1839 01:17:05,000 --> 01:17:07,240 RESEARCH COORDINATING COMMITTEE 1840 01:17:07,240 --> 01:17:09,520 PUT TOGETHER A FEDERAL STRATEGY 1841 01:17:09,520 --> 01:17:14,240 FOR PAIN RESEARCH AND THAT WAS 1842 01:17:14,240 --> 01:17:15,320 NOT JUST NIH. 1843 01:17:15,320 --> 01:17:17,320 THAT MIGHT BE THE GROUP, LINDA 1844 01:17:17,320 --> 01:17:19,120 PORTER RAN THAT. 1845 01:17:19,120 --> 01:17:19,840 AT NIH. 1846 01:17:19,840 --> 01:17:21,960 BUT THERE WERE MULTIPLE AGENCIES 1847 01:17:21,960 --> 01:17:22,480 INVOLVED. 1848 01:17:22,480 --> 01:17:26,360 SO THAT MIGHT BE A GOOD ONE TO 1849 01:17:26,360 --> 01:17:30,400 TRY TO USE AS A PRECEDENT. 1850 01:17:30,400 --> 01:17:31,760 I'M TRYING TO THINK OF OTHER 1851 01:17:31,760 --> 01:17:34,200 ONES THAT WERE DONE WITH 1852 01:17:34,200 --> 01:17:34,960 MULTIPLE AGENCIES. 1853 01:17:34,960 --> 01:17:39,080 >>I WOULD SUGGEST THE RESEARCH 1854 01:17:39,080 --> 01:17:42,480 ON PREGNANT AND LACTATING WOMEN. 1855 01:17:42,480 --> 01:17:45,880 THE TASK FORCE THERE WAS 1856 01:17:45,880 --> 01:17:48,080 MANDATED THROUGH THE 21s 1857 01:17:48,080 --> 01:17:57,360 21st CURSE ACT IN 20716 AND 1858 01:17:57,360 --> 01:17:58,800 THEN HHS ASKED NIH TO LEAD THAT 1859 01:17:58,800 --> 01:18:00,360 TASK FORCE AND Dr. COLLINS 1860 01:18:00,360 --> 01:18:03,400 ASKED NICHD TO LEAD THE TASK 1861 01:18:03,400 --> 01:18:03,760 FORCE. 1862 01:18:03,760 --> 01:18:05,200 WE HAD REPRESENTATIVES FROM 1863 01:18:05,200 --> 01:18:06,880 MULTIPLE INSTITUTES AND CENTERS 1864 01:18:06,880 --> 01:18:08,440 AT NIH AND OTHER FEDERAL 1865 01:18:08,440 --> 01:18:11,320 AGENCIES SUCH AS THE CDC AND THE 1866 01:18:11,320 --> 01:18:12,040 NDA INVOLVED. 1867 01:18:12,040 --> 01:18:14,760 WE HAD ADVOCACY ORGANIZATIONS 1868 01:18:14,760 --> 01:18:18,400 AND WE HAD PHARMA INVOLVED SO 1869 01:18:18,400 --> 01:18:22,560 WITH A SERIES OF FOUR PUBLIC 1870 01:18:22,560 --> 01:18:24,120 MEETINGS, WE CAME UP WITH A 1871 01:18:24,120 --> 01:18:26,280 SERIES OF RECOMMENDATIONS AND 1872 01:18:26,280 --> 01:18:28,760 THEN WORKED AS A GROUP TO NARROW 1873 01:18:28,760 --> 01:18:36,000 THEM DOWN TOP 15 AND THEN IN AN 1874 01:18:36,000 --> 01:18:38,880 ANEXTENSION WE WORKED ON 1875 01:18:38,880 --> 01:18:39,960 RECOMMENDATIONS SO IT'S ANOTHER 1876 01:18:39,960 --> 01:18:41,720 EXAMPLE OF WORKING ACROSS MANY 1877 01:18:41,720 --> 01:18:43,520 GROUPS TO COME UP WITH A 1878 01:18:43,520 --> 01:18:45,160 CONSENSUS. 1879 01:18:45,160 --> 01:18:46,240 THERE WERE INDIVIDUAL 1880 01:18:46,240 --> 01:18:55,160 INVESTIGATORS INVOLVED AS WELL. 1881 01:18:55,160 --> 01:18:58,880 THERE'S OT QUESTION, GLEN. 1882 01:18:58,880 --> 01:19:02,960 >>Glen Nuckolls: WILL NBSTRN 1883 01:19:02,960 --> 01:19:13,360 HAVE A ROLE AND ALSO, A CM ACMG. 1884 01:19:13,360 --> 01:19:17,320 DO YOU RECOGNIZE THESE? 1885 01:19:17,320 --> 01:19:18,400 >>Diana Bianchi: YES, THE 1886 01:19:18,400 --> 01:19:19,920 AMERICAN COLLEGE OF MEDICAL 1887 01:19:19,920 --> 01:19:24,880 GENETICS AND GENOMICS SO THESE 1888 01:19:24,880 --> 01:19:34,400 ARE INVOLVED STAKEHOLDERS AND WN 1889 01:19:34,400 --> 01:19:35,240 WE HAD THE TASK FORCE FOR 1890 01:19:35,240 --> 01:19:36,560 EXAMPLE WE WOULD HAVE THE TASK 1891 01:19:36,560 --> 01:19:38,200 FORCE MEMBERS SITTING AROUND A 1892 01:19:38,200 --> 01:19:40,040 TABLE SUCH AS YOUR ALL SITTING 1893 01:19:40,040 --> 01:19:44,880 NOW BUT IN THE AUDIENCE AND WE 1894 01:19:44,880 --> 01:19:48,440 HAD MANY PROFESSIONAL SOCIETIES 1895 01:19:48,440 --> 01:19:49,600 PARTICIPATING AND THEY COULD GET 1896 01:19:49,600 --> 01:19:53,440 UP AND USE THE MICROPHONE. 1897 01:19:53,440 --> 01:19:55,560 IN A HYBRID ENVIRONMENT WE CAN 1898 01:19:55,560 --> 01:19:56,880 EXTEND THAT TO PEOPLE WHO DON'T 1899 01:19:56,880 --> 01:19:59,280 HAVE TO BE IN THE ROOM. 1900 01:19:59,280 --> 01:20:02,240 I AM ASSUMING THERE WILL BE AT 1901 01:20:02,240 --> 01:20:05,200 LEAST ONE OR MORE MULTIPLE 1902 01:20:05,200 --> 01:20:06,680 REQUESTS FOR INFORMATION SO THAT 1903 01:20:06,680 --> 01:20:07,960 IF PEOPLE ARE NOT IN THE ROOM OR 1904 01:20:07,960 --> 01:20:09,520 NOT AT THE MEETINGS, THEY WILL 1905 01:20:09,520 --> 01:20:11,880 DEFINITELY HAVE A CHANCE TO 1906 01:20:11,880 --> 01:20:16,040 WEIGH IN AND EXPRESS THEIR 1907 01:20:16,040 --> 01:20:26,240 OPINIONS. 1908 01:20:35,200 --> 01:20:37,400 >>BEFORE THIS MEETING, DAN, AID 1909 01:20:37,400 --> 01:20:42,040 MEETING ABOUT WHERE DOES 1910 01:20:42,040 --> 01:20:43,800 SCREENING AND DIAGNOSIS GOES 1911 01:20:43,800 --> 01:20:45,840 BECAUSE IT WAS ONE CHANGE FROM 1912 01:20:45,840 --> 01:20:50,760 THE 2015 PLAN INFORM THE 2025 1913 01:20:50,760 --> 01:20:58,240 PROPOSAL IS THAT IF IF IT'S 1914 01:20:58,240 --> 01:21:00,520 RESEARCH RELATED TO DEVELOPING 1915 01:21:00,520 --> 01:21:10,680 NEW DIAGNOSTIC STRATEGIES, OR 1916 01:21:10,680 --> 01:21:20,840 NEWBORN -- 1917 01:21:46,240 --> 01:21:56,320 >>THAT I'M MORE CONCERNED ABOUT 1918 01:21:56,320 --> 01:22:01,120 AS A DISEASE WE DON'T SEE ON THE 1919 01:22:01,120 --> 01:22:02,400 OUTSIDE AND THE CLINICIAN SEIZE 1920 01:22:02,400 --> 01:22:04,720 FROM THE OUTSIDE IN AND THE 1921 01:22:04,720 --> 01:22:08,680 DISEASE ALSO WORKS FROM THE 1922 01:22:08,680 --> 01:22:09,880 INSIDE OUT. 1923 01:22:09,880 --> 01:22:12,880 WE'RE NOT AT THE POINT WHERE WE 1924 01:22:12,880 --> 01:22:14,280 THINK ABOUT IT LIKE WE THINK 1925 01:22:14,280 --> 01:22:15,320 ABOUT CANCER. 1926 01:22:15,320 --> 01:22:19,160 AND THE DISEASE WHAT WAS STAGED 1927 01:22:19,160 --> 01:22:21,200 THE DISEASE. 1928 01:22:21,200 --> 01:22:27,760 WE TALK ABOUT MORE IN TERMS OF 1929 01:22:27,760 --> 01:22:32,080 SCREENING, AND SEQUENCING. 1930 01:22:32,080 --> 01:22:37,360 WE THINK ABOUT SINGLE GENE 1931 01:22:37,360 --> 01:22:37,640 DISEASES. 1932 01:22:37,640 --> 01:22:42,440 I DON'T THINK AND I WONDER AND 1933 01:22:42,440 --> 01:22:45,600 THINK ABOUT LIKE CANCER. 1934 01:22:45,600 --> 01:22:49,160 STAGE ONE, STAGE 2, STAGE 3 AND 1935 01:22:49,160 --> 01:22:51,800 STAGE 4 WHERE YOU MAY HAVE AN 1936 01:22:51,800 --> 01:22:55,200 ACTIVE DISEASE PROCESS AND IT'S 1937 01:22:55,200 --> 01:22:59,200 GOING ON INSIDE AND YOU THINK 1938 01:22:59,200 --> 01:23:05,840 ABOUT SCREENING, I'M WONDERING 1939 01:23:05,840 --> 01:23:07,960 WHETHER YOU ARE PUTTING THAT IN 1940 01:23:07,960 --> 01:23:14,160 THE CLINICAL END OF THINGS WE 1941 01:23:14,160 --> 01:23:16,040 WANT TO SEE THE DISEASE AND WE 1942 01:23:16,040 --> 01:23:20,040 HAVE SOME ENTER CLINICAL TRIALS 1943 01:23:20,040 --> 01:23:25,640 AND IT MAY LEAD TO DISEASES OR 1944 01:23:25,640 --> 01:23:31,520 THE GOVERNMENT ALF HOLL HAPPENED 1945 01:23:31,520 --> 01:23:36,640 THAT MILD DEVELOPMENT ASPECTS OF 1946 01:23:36,640 --> 01:23:40,200 LAST WEEK. 1947 01:23:40,200 --> 01:23:43,640 AND THAT WE NEED TO TREAT THESE 1948 01:23:43,640 --> 01:23:48,840 DISEASES REALLY EARLY ON IN THE 1949 01:23:48,840 --> 01:23:49,280 PROCESS. 1950 01:23:49,280 --> 01:23:54,280 WHEN THE DISEASE IS NOT 1951 01:23:54,280 --> 01:23:56,000 MANIFESTING IN A CLINICAL 1952 01:23:56,000 --> 01:24:00,080 STRUCTURE THAT WE NEED TO BE 1953 01:24:00,080 --> 01:24:06,760 LOOKING FOR DISEASE IS AK IT 1954 01:24:06,760 --> 01:24:09,160 TIVE AND VERY ACTIVE AND GOING 1955 01:24:09,160 --> 01:24:13,000 ON IN THE MUSCLE. 1956 01:24:13,000 --> 01:24:19,320 AND I WORRY THAT YOU NOT BE 1957 01:24:19,320 --> 01:24:23,440 ABOUT THIS HOLISTICALLY AND WE 1958 01:24:23,440 --> 01:24:25,520 WANT TO MOVE INTO A TRIAL AND WE 1959 01:24:25,520 --> 01:24:27,560 WANT TO HAVE GENE THERAPIES AND 1960 01:24:27,560 --> 01:24:31,120 WE'RE WORKING ON DELIVERING 1961 01:24:31,120 --> 01:24:32,320 TOXICITY AND WE KNOW SOMETHING 1962 01:24:32,320 --> 01:24:39,280 ABOUT HOW DO WE ACQUISITION, 1963 01:24:39,280 --> 01:24:41,560 LOCATE, FUND THESE AND CREATE 1964 01:24:41,560 --> 01:24:44,640 THIS AS A DISEASE WHEN THEY'RE 1965 01:24:44,640 --> 01:24:45,720 AT STAGE 1. 1966 01:24:45,720 --> 01:24:54,040 NOT STAGE 4. 1967 01:24:54,040 --> 01:24:57,560 AND HOW WE GAGING MUSCULAR 1968 01:24:57,560 --> 01:24:58,440 DYSTROPHY. 1969 01:24:58,440 --> 01:25:07,600 MY QUESTION TO YOU WAS, IN THE 1970 01:25:07,600 --> 01:25:11,520 SLIDE INVOLVED AND MECHANISMS 1971 01:25:11,520 --> 01:25:12,960 WITH TREATMENT AND GIVING AN 1972 01:25:12,960 --> 01:25:18,480 INFRASTRUCTURE AND SCREENING THE 1973 01:25:18,480 --> 01:25:22,680 NUMBER OF THINGS AND THIS IS AN 1974 01:25:22,680 --> 01:25:25,520 AREA THAT IS MORE THAN JUST 1975 01:25:25,520 --> 01:25:36,080 SENDING IT ACROSS THE AND OTHER 1976 01:25:38,760 --> 01:25:42,560 AND ARE ARE WE GOING TO LOCK 1977 01:25:42,560 --> 01:25:53,040 THESE PATTERNS OF DISEASE AND 1978 01:25:53,400 --> 01:25:56,920 AND I'M THINKING ABOUT IT AND 1979 01:25:56,920 --> 01:26:04,160 THE REASON I HAVE THE E-MAIL WAS 1980 01:26:04,160 --> 01:26:08,000 THERE'S CONCERNS AND THEY HAVE 1981 01:26:08,000 --> 01:26:10,320 IT FROM AND THANK YOU FOR YOUR 1982 01:26:10,320 --> 01:26:13,480 TIME. 1983 01:26:13,480 --> 01:26:15,360 I'M DONE. 1984 01:26:15,360 --> 01:26:17,080 >>Glen Nuckolls: I THINK THAT 1985 01:26:17,080 --> 01:26:18,960 THE GENERIC ISSUE IN ALL THE 1986 01:26:18,960 --> 01:26:27,120 GENE THERAPIES IS TRYING TO GET 1987 01:26:27,120 --> 01:26:28,440 IT AS EARLY AS POSSIBLE. 1988 01:26:28,440 --> 01:26:29,560 WE HAD A DISCUSSION ABOUT 1989 01:26:29,560 --> 01:26:31,600 SOMETHING SIMILAR WHERE SOMEONE 1990 01:26:31,600 --> 01:26:34,040 WANTED TO GO IN AND WITH NOVEL 1991 01:26:34,040 --> 01:26:35,960 THERAPY AND THEY'RE AFRAID TO GO 1992 01:26:35,960 --> 01:26:38,080 INTO KIDS AND THEY WANT TO START 1993 01:26:38,080 --> 01:26:39,280 IN ADULT AND SOME PEOPLE SAY 1994 01:26:39,280 --> 01:26:43,000 IT'S TOO LATE YOU HAVE TO GO 1995 01:26:43,000 --> 01:26:44,680 INTO KIDS AND THIS IS SOMETHING 1996 01:26:44,680 --> 01:26:46,760 WE'RE ALL WRESTLING WITH. 1997 01:26:46,760 --> 01:26:51,680 IIS REALLY A BENEFIT IS GREATER 1998 01:26:51,680 --> 01:26:53,560 THE EARLIER YOU GO. 1999 01:26:53,560 --> 01:26:56,400 THE ISSUE IS REALLY, YOU KNOW, 2000 01:26:56,400 --> 01:27:06,400 HOW SAFE DO YOU HAVE TO AND THIS 2001 01:27:06,400 --> 01:27:11,680 AND AND WE DECIDED TO GO INTO 2002 01:27:11,680 --> 01:27:13,080 INFANTS BECAUSE THEY WERE GOING 2003 01:27:13,080 --> 01:27:17,160 TO DIE BY YEAR ONE AND IT 2004 01:27:17,160 --> 01:27:23,760 WORKED. 2005 01:27:23,760 --> 01:27:27,920 THIS IS AN ISSUE FOR A LONG 2006 01:27:27,920 --> 01:27:29,360 TIME. 2007 01:27:29,360 --> 01:27:31,760 >>I SEE JENNIFER LEVEE WROTE 2008 01:27:31,760 --> 01:27:32,680 SOMETHING IN THE CHATBOX. 2009 01:27:32,680 --> 01:27:36,840 DO YOU WANT TO COME OFF MUTE? 2010 01:27:36,840 --> 01:27:41,040 >> 2011 01:27:41,040 --> 01:27:43,440 >>I CAN READ IT. 2012 01:27:43,440 --> 01:27:46,120 SO, IT IS IMPORTANT TO CONSIDER 2013 01:27:46,120 --> 01:27:48,040 HOW VARIOUS MUSCULAR DYSTROPHIES 2014 01:27:48,040 --> 01:27:49,920 ARE AT VARIOUS DIFFERENT PLACES 2015 01:27:49,920 --> 01:27:51,000 IN DRUG DEVELOPMENT. 2016 01:27:51,000 --> 01:27:53,280 SOME HAVE APPROVED THERAPIES BUT 2017 01:27:53,280 --> 01:27:55,400 OTHERS HAVE HAD VERY LITTLE 2018 01:27:55,400 --> 01:27:56,960 RESEARCH DONE AND A MINIMAL 2019 01:27:56,960 --> 01:27:59,200 UNDERSTANDING OF THE 2020 01:27:59,200 --> 01:27:59,720 PATHOLOGICAL MECHANISMS. 2021 01:27:59,720 --> 01:28:02,560 BE CAREFUL NOT TO OVER REPRESENT 2022 01:28:02,560 --> 01:28:03,640 WORKING GROUPS WITH STAKEHOLDERS 2023 01:28:03,640 --> 01:28:06,200 THAT REPRESENT THE DISEASES 2024 01:28:06,200 --> 01:28:07,880 FURTHEST ALONG IN DEVELOPMENT AS 2025 01:28:07,880 --> 01:28:12,000 THIS WILL INFORM THE ACTION 2026 01:28:12,000 --> 01:28:14,640 PLAN. 2027 01:28:14,640 --> 01:28:20,640 AND I AGREE, UNLIKE SOME OF THE 2028 01:28:20,640 --> 01:28:22,160 OTHER STRATEGIC PLANS THAT NIH 2029 01:28:22,160 --> 01:28:25,000 HAS DEVELOPED, THINKING ABOUT 2030 01:28:25,000 --> 01:28:32,040 ALS PLAN, ADRD, FOR THE MUSCULAR 2031 01:28:32,040 --> 01:28:33,720 DYSTROPHY WE'RE TALKING ABOUT 2032 01:28:33,720 --> 01:28:36,960 MANY, MANY DIFFERENT TYPES OF 2033 01:28:36,960 --> 01:28:38,240 MUSCULAR DYSTROPHY WITH PERHAPS 2034 01:28:38,240 --> 01:28:41,200 35 DIFFERENT GENETIC CAUSES THAT 2035 01:28:41,200 --> 01:28:42,640 ARE ALL KIND OF LUMPED TOGETHER 2036 01:28:42,640 --> 01:28:50,960 AND WE HAVE TO CONSIDER WHERE 2037 01:28:50,960 --> 01:28:53,480 THEY ARE ALONG THE SPECTRUM OF 2038 01:28:53,480 --> 01:28:55,000 DISEASE AND DEVELOPING THERAPIES 2039 01:28:55,000 --> 01:28:57,080 AND IMPLEMENTING THOSE THERAPIES 2040 01:28:57,080 --> 01:28:58,760 SO THAT'S WHY WE'LL START THE 2041 01:28:58,760 --> 01:29:02,400 ACTION PLAN WITH MECHANISMS OF 2042 01:29:02,400 --> 01:29:04,080 DISEASE WHICH EVEN I THINK 2043 01:29:04,080 --> 01:29:06,400 DISEASES LIKE DUCHENNE COULD 2044 01:29:06,400 --> 01:29:07,160 BENEFIT FROM BETTER 2045 01:29:07,160 --> 01:29:13,200 UNDERSTANDING OF MECHANISMS AND 2046 01:29:13,200 --> 01:29:15,440 THAT MAY BE THE PRIMARY FOCUS 2047 01:29:15,440 --> 01:29:19,920 FOR OTHER FORMS OF MUSCULAR 2048 01:29:19,920 --> 01:29:30,120 DYSTROPHY. 2049 01:29:48,800 --> 01:29:59,080 IS THIS AND AND AND -- 2050 01:30:48,480 --> 01:30:49,240 I CAN RESPONDS TO THAT. 2051 01:30:49,240 --> 01:30:53,320 THAT'S COME UP AT A NUMBER OF 2052 01:30:53,320 --> 01:30:54,240 DIFFERENT TIMES WHEN WE'VE 2053 01:30:54,240 --> 01:30:55,600 LOOKED AT OUR FUNDING IN THE 2054 01:30:55,600 --> 01:30:57,600 MUSCULAR DYSTROPHIES AND WE HAD 2055 01:30:57,600 --> 01:31:01,160 IN 2018, WE HAD AN OUTSIDE GROUP 2056 01:31:01,160 --> 01:31:03,520 THAT DID AN EVALUATION OF THE 2057 01:31:03,520 --> 01:31:05,080 WELL STONE MUSCULAR DYSTROPHY 2058 01:31:05,080 --> 01:31:08,040 RESEARCH PROGRAM AND ONE OF THE 2059 01:31:08,040 --> 01:31:11,720 SUGGESTIONS IS THAT IT -- THERE 2060 01:31:11,720 --> 01:31:15,200 ARE THE SAME GENES IN WHAT ARE 2061 01:31:15,200 --> 01:31:18,400 CALLED MYOPATHY SOME OF WHAT WE 2062 01:31:18,400 --> 01:31:19,160 CALL MUSCULAR DYSTROPHIES AND 2063 01:31:19,160 --> 01:31:23,640 YOU KNOW, THE BOUNDARY BETWEEN 2064 01:31:23,640 --> 01:31:26,400 DYSTROPHY AND OTHER DISEASES 2065 01:31:26,400 --> 01:31:27,360 EFFECTS SKELETAL MUSCLE AND 2066 01:31:27,360 --> 01:31:31,760 OTHER TYPES OF MUSCLE IS NOT ALL 2067 01:31:31,760 --> 01:31:32,560 THAT CLEAR. 2068 01:31:32,560 --> 01:31:39,000 IN THIS CASE, WHAT WE DO BEST IS 2069 01:31:39,000 --> 01:31:39,840 TO FOLLOW THE GUIDANCE FROM THE 2070 01:31:39,840 --> 01:31:42,640 LEGISLATION THE MD CARE ACT AND 2071 01:31:42,640 --> 01:31:45,200 IT IDENTIFIES A NUMBER OF TYPES 2072 01:31:45,200 --> 01:31:47,400 OF MUSCULAR DYSTROPHY AND THAT 2073 01:31:47,400 --> 01:31:50,200 WILL FOCUS ON BUT WE COULD END 2074 01:31:50,200 --> 01:31:54,120 INDICATE IN RENEWED ACTION PLAN 2075 01:31:54,120 --> 01:31:55,120 THAT A LOT OF THE SAME PROCESS 2076 01:31:55,120 --> 01:32:01,000 WILL CARRY OVER INTO CARE 2077 01:32:01,000 --> 01:32:05,280 DISEASES EFFECTING SKELETAL 2078 01:32:05,280 --> 01:32:09,040 MUSCLE. 2079 01:32:09,040 --> 01:32:10,560 >>WHAT STOPPING US FROM DEALING 2080 01:32:10,560 --> 01:32:15,480 WITH THE PRIMARY MUSCLE DISEASES 2081 01:32:15,480 --> 01:32:18,040 IN THIS? 2082 01:32:18,040 --> 01:32:22,840 STRATEGIC PLAN IF WE WANTED 2083 01:32:22,840 --> 01:32:23,120 ANYTHING? 2084 01:32:23,120 --> 01:32:25,200 >>SO I DON'T WANT TO PUT HER ON 2085 01:32:25,200 --> 01:32:27,200 THE SPOT BUT OUR PROGRAM 2086 01:32:27,200 --> 01:32:31,960 DIRECTOR FROM NIAMS HAS A LOT OF 2087 01:32:31,960 --> 01:32:34,200 DIFFERENT MUSCLE DISORDERS IN 2088 01:32:34,200 --> 01:32:37,840 HER PORTFOLIO AND SO HOW MUCH 2089 01:32:37,840 --> 01:32:40,040 WOULD IT EXPAND THE PLAN DO YOU 2090 01:32:40,040 --> 01:32:47,680 THINK IF WE INCLUDED CONGENITAL 2091 01:32:47,680 --> 01:32:50,920 HIMY ON AGENT' AND SO FOURTH. 2092 01:32:50,920 --> 01:32:52,440 >>THERE'S MANY MUSCLE DISORDERS 2093 01:32:52,440 --> 01:32:54,960 AND IT'S NOT A CLEAR BOUNDARY 2094 01:32:54,960 --> 01:32:56,400 BETWEEN SKELETAL MUSCLE AND WE 2095 01:32:56,400 --> 01:32:58,960 ALSO HAVE TO INCLUDE MOVING AWAY 2096 01:32:58,960 --> 01:33:01,320 FROM MUSCLE BECAUSE MUSCULAR 2097 01:33:01,320 --> 01:33:05,720 DYSTROPHIES HAVE COMPLICATIONS 2098 01:33:05,720 --> 01:33:12,240 SO MY PORTFOLIO HAS BOTH, ALL, 2099 01:33:12,240 --> 01:33:13,840 AND IT COULD POTENTIALLY DOUBLE 2100 01:33:13,840 --> 01:33:18,080 THE SIZE OF THE PROPOSED 2101 01:33:18,080 --> 01:33:19,120 RESEARCH AREAS. 2102 01:33:19,120 --> 01:33:22,560 MANY OF THEM ARE EARLIER LIKE A 2103 01:33:22,560 --> 01:33:24,600 LOT OF DYSTROPHY ARE LEADING THE 2104 01:33:24,600 --> 01:33:28,840 CHARGE SO IT MIGHT TILT THE PLAN 2105 01:33:28,840 --> 01:33:30,840 TOWARDS PERHAPS A MEMBERS THAT 2106 01:33:30,840 --> 01:33:32,280 FEEL COMFORTABLE BUT I AGREE 2107 01:33:32,280 --> 01:33:35,560 THAT A LOT OF THE PATHWAYS BEING 2108 01:33:35,560 --> 01:33:37,160 FORMED BY THE DYSTROPHIES THAT 2109 01:33:37,160 --> 01:33:39,320 ARE LEADING THE CHARGE ARE BEING 2110 01:33:39,320 --> 01:33:41,840 FOLLOWED VERY CLOSELY BY SOME OF 2111 01:33:41,840 --> 01:33:52,720 THE OTHER MY M MYOPATHY AND SOE 2112 01:33:55,080 --> 01:33:57,720 IN A VERY DIFFERENT PLACE. 2113 01:33:57,720 --> 01:34:00,280 >>IT MAY BE CHALLENGING TO 2114 01:34:00,280 --> 01:34:02,920 INCLUDE THE LIST OF WHAT WE 2115 01:34:02,920 --> 01:34:06,160 CURRENTLY CALL MUSCULAR 2116 01:34:06,160 --> 01:34:06,800 DYSTROPHIES IN OUR PLAN AND I 2117 01:34:06,800 --> 01:34:11,560 HAVE TO DRAW A BOUNDARY 2118 01:34:11,560 --> 01:34:14,360 SOMEWHERE. 2119 01:34:14,360 --> 01:34:20,440 ANY OTHER THOUGHTS ON THAT? 2120 01:34:20,440 --> 01:34:23,680 >>I GUESS I WOULD BE OPPOSED OT 2121 01:34:23,680 --> 01:34:34,160 BOUNDARIES OF THIS SEMANTIC. 2122 01:34:34,160 --> 01:34:38,360 I DON'T KNOW WHAT'S DO YOU 2123 01:34:38,360 --> 01:34:38,560 THINK. 2124 01:34:38,560 --> 01:34:41,320 >>IT'S SOMETHING THAT THE GROUP 2125 01:34:41,320 --> 01:34:44,280 SHOULD DISCUSS WHOEVER IS ON ON 2126 01:34:44,280 --> 01:34:45,240 THE INITIAL GROUP? 2127 01:34:45,240 --> 01:34:49,000 I MEAN, I WOULD FAVOR BEING 2128 01:34:49,000 --> 01:34:50,120 INCLUSIVE AND THEN LOOKING AT 2129 01:34:50,120 --> 01:34:51,680 THE PRACTICALITY AND THEN TRYING 2130 01:34:51,680 --> 01:34:52,760 TO NARROW IT DOWN. 2131 01:34:52,760 --> 01:34:55,920 I MEAN, WHEN WE DID THE NICHD 2132 01:34:55,920 --> 01:34:59,240 STRATEGIC PLAN WE INITIALLY HAD 2133 01:34:59,240 --> 01:35:09,720 270 SCIENTIFIC THEMES AND -- 2134 01:35:43,480 --> 01:35:47,360 >>MAYBE WE CAN JUST FRAME A 2135 01:35:47,360 --> 01:35:48,640 QUESTION, PROS AND CONS AND 2136 01:35:48,640 --> 01:35:50,200 OFFER A VOTE AND SEE WHAT THE 2137 01:35:50,200 --> 01:35:51,640 GROUP WANTS TO DO. 2138 01:35:51,640 --> 01:35:53,920 >>YEAH, WELL, AS Dr. BIANCHI 2139 01:35:53,920 --> 01:35:56,840 SUGGESTED THAT MAYBE THAT 2140 01:35:56,840 --> 01:35:58,160 STEERING GROUP THAT IS 2141 01:35:58,160 --> 01:36:00,920 VOLUNTEERS FROM THE MDCC CAN TRY 2142 01:36:00,920 --> 01:36:03,160 TO ADDRESS THAT. 2143 01:36:03,160 --> 01:36:05,320 AND CONSIDER WHAT WOULD IT LOOK 2144 01:36:05,320 --> 01:36:11,640 LIKE IF WE EXPAND TO PERHAPS 2145 01:36:11,640 --> 01:36:17,760 GENETICALLY SIMILAR OR OTHER MY 2146 01:36:17,760 --> 01:36:19,880 ON THEE THAT LEAD TO MUSCLE 2147 01:36:19,880 --> 01:36:30,400 WASTING AND THE MERCHANDISE AND 2148 01:36:31,760 --> 01:36:31,960 SCREENING. 2149 01:36:31,960 --> 01:36:33,680 >>OKAY, I HOPE WE GET GOOD 2150 01:36:33,680 --> 01:36:35,120 VOLUNTEERS AND A LOT OF 2151 01:36:35,120 --> 01:36:36,440 VOLUNTEERS FOR THAT COMMITTEE 2152 01:36:36,440 --> 01:36:38,000 BECAUSE IT'S GOING TO BE A 2153 01:36:38,000 --> 01:36:44,600 IMPORTANT WORK TO DO. 2154 01:36:44,600 --> 01:36:50,360 >>I GUESS, WHAT ARE YOUR 2155 01:36:50,360 --> 01:36:54,160 CONCERNS? 2156 01:36:54,160 --> 01:36:59,400 20 MENS AGO WE TALKED ABOUT 2157 01:36:59,400 --> 01:37:01,520 MUSCULAR DYSTROPHY ALS AND THEY 2158 01:37:01,520 --> 01:37:06,440 WERE SYNONYMOUS WITH THE 2159 01:37:06,440 --> 01:37:07,000 MUSCULAR DYSTROPHIES. 2160 01:37:07,000 --> 01:37:11,680 AND IT TOOK A LOT OF WORK TO 2161 01:37:11,680 --> 01:37:16,160 WHERE YOU GET NIH AND CDC AND 2162 01:37:16,160 --> 01:37:21,400 OTHER AGENCIES TO GET THE 2163 01:37:21,400 --> 01:37:24,960 MUSCULAR DYSTROPHIES PRIMARY 2164 01:37:24,960 --> 01:37:33,000 TALKING AND DISCUSSION BUT Dr. 2165 01:37:33,000 --> 01:37:36,880 BIANCHI SUCCESSES FOR 2166 01:37:36,880 --> 01:37:39,440 GENERATIONS AGO WHO WOULD GO TO 2167 01:37:39,440 --> 01:37:40,720 NIH WHAT ARE YOU DOING FOR 2168 01:37:40,720 --> 01:37:41,760 MUSCULAR DYSTROPHIES AND THEY 2169 01:37:41,760 --> 01:37:45,440 WOULD SAY WE'RE ACCEPTING THE 2170 01:37:45,440 --> 01:37:48,560 $80 MILLION TO MUSCULAR 2171 01:37:48,560 --> 01:37:51,160 DYSTROPHIES. 2172 01:37:51,160 --> 01:37:59,120 WHAT ARE YOU SPENDING ON ON THE 2173 01:37:59,120 --> 01:38:01,360 TENDENCY AND LOOKING THEIR WAY 2174 01:38:01,360 --> 01:38:07,720 AND BACK INTO THE LEARNED AND 2175 01:38:07,720 --> 01:38:12,520 MUSCULAR DYSTROPHIES AND 2176 01:38:12,520 --> 01:38:13,840 VARIATION AND I WOULD ENCOURAGE 2177 01:38:13,840 --> 01:38:19,080 THE COMMITTEE TO KEEP A SHARP 2178 01:38:19,080 --> 01:38:20,880 EYE OUT FOR MUSCULAR DYSTROPHY 2179 01:38:20,880 --> 01:38:24,960 AND DISEASES OF THE PRIMARY 2180 01:38:24,960 --> 01:38:29,240 MUSCLE NOT NEURONS AND THINGS 2181 01:38:29,240 --> 01:38:33,480 AND SECOND THING, I THINK THAT 2182 01:38:33,480 --> 01:38:37,360 THE ISSUE OF GETTING DRUGS INTO 2183 01:38:37,360 --> 01:38:41,200 MUSCLE DELIVERING AND TOXICITY 2184 01:38:41,200 --> 01:38:42,480 AND THEN THOSE DRIVERS THEY NEED 2185 01:38:42,480 --> 01:38:48,040 A LOT OF REINFORCEMENT NOT ON 2186 01:38:48,040 --> 01:38:55,520 THE BACK END AND WE ARE STILL 2187 01:38:55,520 --> 01:39:02,840 FLYING BLIND WITH GETTING THINGS 2188 01:39:02,840 --> 01:39:11,080 AND CRISPR AND SO I WOULD 2189 01:39:11,080 --> 01:39:14,600 ENCOURAGE YOU TO KEEP A VERY 2190 01:39:14,600 --> 01:39:18,240 SHARP FOCUS TO HOW TO GET DRUGS 2191 01:39:18,240 --> 01:39:22,120 INTO MUSCLE INTO MUSCLE 2192 01:39:22,120 --> 01:39:27,840 SPECIFICALLY AND THEN JUST GO TO 2193 01:39:27,840 --> 01:39:30,200 THE THRESHOLD. 2194 01:39:30,200 --> 01:39:33,480 THESE DISEASES, AND THE OTHER 2195 01:39:33,480 --> 01:39:38,360 ONE MIGHT NEED JUST A 2196 01:39:38,360 --> 01:39:40,480 INTERACTION AND SO WITH THE 2197 01:39:40,480 --> 01:39:45,760 TOXICITY JUST THE ISSUE HERE OF 2198 01:39:45,760 --> 01:39:46,600 INDUSTRIES AND THEY KEEP TURNING 2199 01:39:46,600 --> 01:39:48,840 THEIR HEADS AND TOXICITY AND 2200 01:39:48,840 --> 01:39:50,840 TOXICITY AND DELIVERY, HOW TO 2201 01:39:50,840 --> 01:39:57,480 GET IT IN AND HOW TO ACCESS AND 2202 01:39:57,480 --> 01:40:01,200 IT'S ENOUGH WORK JUST TO FOCUS 2203 01:40:01,200 --> 01:40:09,920 ON THE EXISTENCE AND WHAT THE 2204 01:40:09,920 --> 01:40:20,160 DEFINITION IS. 2205 01:40:22,000 --> 01:40:22,800 >>Diana Bianchi: THANK YOU, 2206 01:40:22,800 --> 01:40:26,560 DAN, FOR THOSE COMMENTS. 2207 01:40:26,560 --> 01:40:27,200 >>Glen Nuckolls: WE MIGHT ASK 2208 01:40:27,200 --> 01:40:30,440 PAUL IF HE WOULD LIKE TO COMMENT 2209 01:40:30,440 --> 01:40:33,880 BECAUSE FOR YOUR ORGANIZATION, 2210 01:40:33,880 --> 01:40:34,600 MUSCULAR DYSTROPHY ASSOCIATION, 2211 01:40:34,600 --> 01:40:39,320 YOU GUYS INCLUDE AL S AND QUITE 2212 01:40:39,320 --> 01:40:41,320 A NUMBER OF OTHER CONDITIONS SO 2213 01:40:41,320 --> 01:40:42,640 KIND OF OPERATIONALLY, DO YOU 2214 01:40:42,640 --> 01:40:45,440 HAVE A DEFINITION FOR 2215 01:40:45,440 --> 01:40:45,840 DYSTROPHIES? 2216 01:40:45,840 --> 01:40:49,120 >>MYOPATHY TOO. 2217 01:40:49,120 --> 01:40:49,680 >>Glen Nuckolls: UH-HUH. 2218 01:40:49,680 --> 01:40:54,600 >>THAT'S RIGHT. 2219 01:40:54,600 --> 01:40:58,680 I THINK THE PLAN TO BRING THIS 2220 01:40:58,680 --> 01:40:59,240 QUESTION TO THE STEERING 2221 01:40:59,240 --> 01:41:01,440 COMMITTEE PROBABLY IS THE MOST 2222 01:41:01,440 --> 01:41:02,240 PRO AT BECAUSE THAT IS THE 2223 01:41:02,240 --> 01:41:03,280 COMMITTEE THAT WILL LEAD THE 2224 01:41:03,280 --> 01:41:04,360 WORK. 2225 01:41:04,360 --> 01:41:07,440 I THINK WITHIN OUR VIEWPOINT, WE 2226 01:41:07,440 --> 01:41:09,200 CERTAINLY HAVE BEEN LOOKING AT 2227 01:41:09,200 --> 01:41:12,320 THE CONDITIONS UNDER OUR 2228 01:41:12,320 --> 01:41:15,240 UMBRELLA MORE AND MORE 2229 01:41:15,240 --> 01:41:16,920 GENETICALLY THAN HOW THEY'VE 2230 01:41:16,920 --> 01:41:18,400 BEEN CLINICALLY DEFINED IN THE 2231 01:41:18,400 --> 01:41:20,440 PAST. 2232 01:41:20,440 --> 01:41:22,400 THE DIFFERENCE BETWEEN MYOPATHY 2233 01:41:22,400 --> 01:41:23,320 AND MUSCULAR DYSTROPHY CAUSED BY 2234 01:41:23,320 --> 01:41:25,440 THE SAME GENETIC UNDERPINNINGS, 2235 01:41:25,440 --> 01:41:26,880 IS BECOMING IF YOU WILL, LESS 2236 01:41:26,880 --> 01:41:28,440 AND LESS IMPACTFUL. 2237 01:41:28,440 --> 01:41:29,840 AND CERTAINLY HOW WE LOOK AT IT 2238 01:41:29,840 --> 01:41:32,440 AND FRANKLY IN OUR VIEWPOINT 2239 01:41:32,440 --> 01:41:33,520 HEALTH THE FIELD IS LOOKING AT 2240 01:41:33,520 --> 01:41:34,440 IT AS WELL. 2241 01:41:34,440 --> 01:41:37,960 I WOULDN'T WANT US TO BE CAUGHT 2242 01:41:37,960 --> 01:41:42,000 UP IN, LET'S ONLY FOCUS THE PLAN 2243 01:41:42,000 --> 01:41:43,600 ON A NARROW DEFINITION OF 2244 01:41:43,600 --> 01:41:45,680 MUSCULAR DYSTROPHY WHEN THE 2245 01:41:45,680 --> 01:41:46,320 IMPLICATIONS OF THE PLAN COULD 2246 01:41:46,320 --> 01:41:49,720 HAVE SOME REALLY RELEVANT 2247 01:41:49,720 --> 01:41:51,640 BENEFITS TO THE MYOPATHY THAT 2248 01:41:51,640 --> 01:41:53,200 ARE GENETICALLY RELATED IF NOT 2249 01:41:53,200 --> 01:41:55,960 CAUSED BY THE SAME GENETIC 2250 01:41:55,960 --> 01:41:56,760 UNDERPINNING. 2251 01:41:56,760 --> 01:41:59,000 WHEREAS, WHEN WE START EXPANDING 2252 01:41:59,000 --> 01:42:01,880 INTO DISEASES SUCH AS ALS AND 2253 01:42:01,880 --> 01:42:03,560 OTHER PERHAPS CLASSICALLY 2254 01:42:03,560 --> 01:42:06,120 DEFINED DISEASES AS NEURO 2255 01:42:06,120 --> 01:42:07,920 MUSCULAR BUT PERHAPS STILL 2256 01:42:07,920 --> 01:42:10,640 GENETICALLY UNRELATED TO A 2257 01:42:10,640 --> 01:42:11,480 MUSCULAR DYSTROPHY, THERE MIGHT 2258 01:42:11,480 --> 01:42:13,840 BE MORE LOGIC TO DRAW A LINE 2259 01:42:13,840 --> 01:42:14,480 AROUND THE MUSCULAR DYSTROPHIES 2260 01:42:14,480 --> 01:42:15,400 IN THAT WAY. 2261 01:42:15,400 --> 01:42:16,840 CERTAINLY, IF THERE'S THAT 2262 01:42:16,840 --> 01:42:19,720 GENETIC UNDERPINNING AND LESSONS 2263 01:42:19,720 --> 01:42:20,480 WITHIN A MUSCULAR DYSTROPHY ARE 2264 01:42:20,480 --> 01:42:24,040 VERY APPLICABLE AND TO THE 2265 01:42:24,040 --> 01:42:26,520 RELATED CON TEN DAL MYOPATHY WE 2266 01:42:26,520 --> 01:42:28,160 WOULDN'T SEE BOTH OF THEM BEING 2267 01:42:28,160 --> 01:42:34,800 INCLUDED IN THE SAME EFFORT. 2268 01:42:34,800 --> 01:42:45,320 >>Glen Nuckolls: THANK YOU. 2269 01:42:47,840 --> 01:42:48,280 >>Diana Bianchi: OKAY. 2270 01:42:48,280 --> 01:42:51,920 WELL, PEOPLE MIGHT BE REED FOR A 2271 01:42:51,920 --> 01:42:52,520 BREAK. 2272 01:42:52,520 --> 01:42:53,800 WE'RE RUNNING JUST A LITTLE BIT 2273 01:42:53,800 --> 01:42:54,400 AHEAD. 2274 01:42:54,400 --> 01:42:57,440 SO HOW ABOUT IF WE GIVE EVERYONE 2275 01:42:57,440 --> 01:42:58,160 A 17-MINUTE BREAK. 2276 01:42:58,160 --> 01:43:01,600 I HAVE 2:43 ON MY COMPUTER IT'S 2277 01:43:01,600 --> 01:43:03,160 2:42 IN THE ROOM. 2278 01:43:03,160 --> 01:43:06,440 BUT PLEASE BE BACK PROMPTLY AT 2279 01:43:06,440 --> 01:43:07,640 3:00 AND WE WILL THEN HEAR FROM 2280 01:43:07,640 --> 01:43:13,120 THE STRATEGIC PLANS OF THE MDCC 2281 01:43:13,120 --> 01:43:14,240 ORGANIZATIONS. 2282 01:43:14,240 --> 01:43:16,520 THE MEMBER ORGANIZATIONS. 2283 01:43:16,520 --> 01:43:18,600 DOES THAT SEEM LIKE A PLAN? 2284 01:43:18,600 --> 01:43:22,680 EVERYONE CAN STRETCH THEIR LEGS. 2285 01:43:22,680 --> 01:43:24,680 THE FACILITIES ARE DOWN THE 2286 01:43:24,680 --> 01:43:25,200 HALL, BE BACK AT 3:00. 2287 01:43:25,200 --> 01:43:25,680 WELCOME EVERYBODY BACK. 2288 01:43:25,680 --> 01:43:27,840 IT'S 3:00. 2289 01:43:27,840 --> 01:43:30,040 NEXT ON THE AGENDA WE'LL HAVE 2290 01:43:30,040 --> 01:43:33,840 PRESENTATIONS FROM SEVERAL MDCC 2291 01:43:33,840 --> 01:43:37,120 MEMBER ORGANIZATIONS WITH THEIR 2292 01:43:37,120 --> 01:43:37,600 STRATEGIC PLANS. 2293 01:43:37,600 --> 01:43:40,320 AS WE WORK TO RENEW THE MDCC 2294 01:43:40,320 --> 01:43:41,560 ACTION PLAN YOU JUST HEARD 2295 01:43:41,560 --> 01:43:43,320 ABOUT, WE NEED TO KNOW ABOUT AND 2296 01:43:43,320 --> 01:43:44,720 BUILD UPON THE PLANS DEVELOPED 2297 01:43:44,720 --> 01:43:47,800 BY THE PATIENT ADVOCACY GROUPS 2298 01:43:47,800 --> 01:43:50,600 AND OTHER FUNDING ORGANIZATIONS. 2299 01:43:50,600 --> 01:43:53,080 THE MDCC ACTION PLAN WILL 2300 01:43:53,080 --> 01:43:55,440 DESCRIBE OVERARCHING GOALS AND 2301 01:43:55,440 --> 01:43:57,240 STRATEGIC PLANS OF OTHER 2302 01:43:57,240 --> 01:43:59,600 ORGANIZATIONS WILL PROVIDE MORE 2303 01:43:59,600 --> 01:44:02,480 DETAILED DISEASE SPECIFIC 2304 01:44:02,480 --> 01:44:02,960 OBJECTIVES. 2305 01:44:02,960 --> 01:44:04,280 TODAY WILL BE PART ONE OF THIS 2306 01:44:04,280 --> 01:44:05,920 SESSION FOR OUR NEXT MEETING IN 2307 01:44:05,920 --> 01:44:07,800 THE FALL, WE'RE GOING TO HAVE 2308 01:44:07,800 --> 01:44:10,520 PRESENTATIONS FROM ADDITIONAL 2309 01:44:10,520 --> 01:44:13,320 DYSTROPHY ADVOCACY GROUPS. 2310 01:44:13,320 --> 01:44:17,120 SO, FIRST UP IN THIS SESSION IS 2311 01:44:17,120 --> 01:44:20,920 JAMSHID ARJOMAND THE CHIEF 2312 01:44:20,920 --> 01:44:25,720 SCIENTIFIC OFFICER FOR THE FSHD 2313 01:44:25,720 --> 01:44:29,000 SOCIETY. 2314 01:44:29,000 --> 01:44:34,240 OVER TO YOU. 2315 01:44:34,240 --> 01:44:35,880 >>HI, THANK YOU, VERY MUCH. 2316 01:44:35,880 --> 01:44:38,600 THANK YOU TO THE NDCC COMMITTEE 2317 01:44:38,600 --> 01:44:40,520 FOR THE INVITATION AND I WANTED 2318 01:44:40,520 --> 01:44:42,640 TO EXTEND MY THANKS TO DIANNE, 2319 01:44:42,640 --> 01:44:45,360 THE CO FOUNDERS OF THE SOCIETY 2320 01:44:45,360 --> 01:44:48,040 FOR HIS TIRELESS AND ON GOING 2321 01:44:48,040 --> 01:44:51,000 EFFORTS WITH THE MDC AND AND 2322 01:44:51,000 --> 01:44:54,480 THANK YOU VERY MUCH SO, TO GET 2323 01:44:54,480 --> 01:44:56,120 STARTED I DIDN'T KNOW THIS IS MY 2324 01:44:56,120 --> 01:44:57,400 FIRST TIME ATTENDING THIS 2325 01:44:57,400 --> 01:45:00,840 MEETING I DIDN'T KNOW IF PEOPLE 2326 01:45:00,840 --> 01:45:03,320 WERE FAMILIAR WITH THE DISEASE 2327 01:45:03,320 --> 01:45:05,480 AND WHO WE ARE AT THE SOCIETY 2328 01:45:05,480 --> 01:45:11,760 AND THEN TALK ABOUT THE 2329 01:45:11,760 --> 01:45:13,880 STRATEGIC PLAN THE THERAPEUTIC 2330 01:45:13,880 --> 01:45:15,520 ACCESS RATER WHICH IS AIMED AT 2331 01:45:15,520 --> 01:45:17,480 ADDRESSING GAPS AND CLINICAL 2332 01:45:17,480 --> 01:45:19,320 TRIAL READINESS AND THE PLANS WE 2333 01:45:19,320 --> 01:45:22,800 HAVE GOING FORWARD WITH AN 2334 01:45:22,800 --> 01:45:25,160 ALLIANCE WITH INDUSTRY TO 2335 01:45:25,160 --> 01:45:29,200 ADDRESS THESE GAPS. 2336 01:45:29,200 --> 01:45:35,400 WHO MAY NOT BE FAMILIAR, FHSD IS 2337 01:45:35,400 --> 01:45:37,880 AS THE NAME IMPLIES THAT THE 2338 01:45:37,880 --> 01:45:41,160 DISEASE AFFECTS THE UPPER BODY 2339 01:45:41,160 --> 01:45:44,560 MUSCLES AND AS YOU SEE IN THE 2340 01:45:44,560 --> 01:45:49,360 MRI AND IT CAN EFFECT IN THE 2341 01:45:49,360 --> 01:45:52,320 MAUD' AND IT'S QUITE OFTEN VERY 2342 01:45:52,320 --> 01:45:55,320 A SEM ET IT TRICK AND THE LEFT 2343 01:45:55,320 --> 01:45:58,800 SIDE AND RIGHT SIDE MORE EFFECTS 2344 01:45:58,800 --> 01:46:00,840 AND IT'S PROGRESSIVE AND ABOUT 2345 01:46:00,840 --> 01:46:03,120 25% OF THE POPULATION AND DOES 2346 01:46:03,120 --> 01:46:07,680 END UP REQUIRING WHEELCHAIRS AND 2347 01:46:07,680 --> 01:46:13,880 THEY LOOSE MOBILITY AND THE IT'S 2348 01:46:13,880 --> 01:46:18,840 COMPLICATED AND THE PREVALENCE 2349 01:46:18,840 --> 01:46:22,160 AND IN 20,000 AND THE REASON WHY 2350 01:46:22,160 --> 01:46:23,720 WE DON'T KNOW THIS IT'S VERY 2351 01:46:23,720 --> 01:46:25,360 DIFFICULT TO DO THE GENETIC 2352 01:46:25,360 --> 01:46:27,880 TESTING IN THE POPULATION TO SEE 2353 01:46:27,880 --> 01:46:32,400 HOW MANY ASYMPTOMATIC GENE 2354 01:46:32,400 --> 01:46:33,920 CARRIERS ARE WALKING AMONG US. 2355 01:46:33,920 --> 01:46:38,840 THERE ARE TWO TYPES OF FSHD TYPE 2356 01:46:38,840 --> 01:46:39,560 ONE OR TYPE TWO. 2357 01:46:39,560 --> 01:46:42,120 BOTH OF THEM ARE CAUSED BY THE 2358 01:46:42,120 --> 01:46:47,080 MISS INVESTIGATION OF A TOXIC 2359 01:46:47,080 --> 01:46:53,480 GENE CALLED DUX4. 2360 01:46:53,480 --> 01:46:56,920 FSHD TYPE 2 ARE MUTATIONS IN 2361 01:46:56,920 --> 01:46:59,080 GENES THAT EPIGENETIC REGULATORY 2362 01:46:59,080 --> 01:47:05,600 GENES SO THEY BASICALLY AND THAT 2363 01:47:05,600 --> 01:47:07,600 IS WHEN YOU HAVE DUX4 EXPRESSION 2364 01:47:07,600 --> 01:47:10,440 AND IT'S ESTIMATED THAT MAYBE 2365 01:47:10,440 --> 01:47:13,360 ABOUT 20% OR SO OF THE 2366 01:47:13,360 --> 01:47:15,360 POPULATION ARE ACTUALLY 2367 01:47:15,360 --> 01:47:16,680 SPONTANEOUS AND CONVERSIONS 2368 01:47:16,680 --> 01:47:19,160 HOWEVER WHEN YOU SPEAK WITH 2369 01:47:19,160 --> 01:47:20,960 EXPERT CLINICIANS AND THEY SAY 2370 01:47:20,960 --> 01:47:23,200 THEY DIAGNOSED A PATIENT AND 2371 01:47:23,200 --> 01:47:25,560 THEY SEE THE ENTIRE FAMILY QUITE 2372 01:47:25,560 --> 01:47:28,440 OFTEN, FAMILY MEMBERS ARE ALSO 2373 01:47:28,440 --> 01:47:30,720 EFFECTED AND THIS IS BEEN 2374 01:47:30,720 --> 01:47:33,120 UNDIAGNOSED SO IT'S AN UNDER 2375 01:47:33,120 --> 01:47:33,560 DIAGNOSE INDICATION. 2376 01:47:33,560 --> 01:47:37,360 SO WHO WE ARE IS THE FSHD 2377 01:47:37,360 --> 01:47:39,720 SOCIETY AND RESEARCH FOCUSED 2378 01:47:39,720 --> 01:47:41,880 ADVOCACY NON-PROFIT GROUP AND 2379 01:47:41,880 --> 01:47:44,200 OUR MISSION IS TO HELP FAMILIES 2380 01:47:44,200 --> 01:47:54,720 LIFT BEST LIFE THAT THEY CAN WE 2381 01:48:01,920 --> 01:48:03,480 HAVE A RESEARCH GROUP ON ONE 2382 01:48:03,480 --> 01:48:06,560 SIDE AND THE ADVOCACY GROUP IS 2383 01:48:06,560 --> 01:48:09,880 TO BUILD COMMUNITY AWARENESS TO 2384 01:48:09,880 --> 01:48:14,400 DISSEMINATE IMPORTANT 2385 01:48:14,400 --> 01:48:17,800 INFORMATION ON AND AND SO WE 2386 01:48:17,800 --> 01:48:19,960 KNOW WHAT IS COMING UP. 2387 01:48:19,960 --> 01:48:22,080 WE'RE TRYING TO HAVE AN 2388 01:48:22,080 --> 01:48:23,840 INTERNATIONAL OUTREACH BECAUSE 2389 01:48:23,840 --> 01:48:25,840 THERE ARE OTHER ADVOCACY GROUPS 2390 01:48:25,840 --> 01:48:27,240 IN OTHER COUNTRIES AND I'LL 2391 01:48:27,240 --> 01:48:28,440 TOUCH ON THAT MOMENTARILY. 2392 01:48:28,440 --> 01:48:31,920 ON THE RESEARCH SIDE, LIKE MANY 2393 01:48:31,920 --> 01:48:33,320 OTHER ADVOCACY GROUPS WE RAISED 2394 01:48:33,320 --> 01:48:37,120 FUNDING AND WE SUPPORT RESEARCH 2395 01:48:37,120 --> 01:48:38,800 TO AND SOME OF THE FUNDING THAT 2396 01:48:38,800 --> 01:48:41,600 NIH PROVIDES AND I'LL GO INTO 2397 01:48:41,600 --> 01:48:43,520 MORE DETAIL ABOUT THE 2398 01:48:43,520 --> 01:48:45,160 THERAPEUTIC ACCELERATOR AND THIS 2399 01:48:45,160 --> 01:48:47,560 IS A FOCUS ON TRYING TO ADDRESS 2400 01:48:47,560 --> 01:48:49,120 THE GAPS THAT INDUSTRY MIGHT 2401 01:48:49,120 --> 01:48:50,800 HAVE AND BRINGING THEIR DRUGS 2402 01:48:50,800 --> 01:48:54,120 AND THEIR THERAPEUTICS FORWARD. 2403 01:48:54,120 --> 01:48:56,960 OF COURSE, I WANTED TO HIGHLIGHT 2404 01:48:56,960 --> 01:49:00,840 HERE IS THE ADVOCACY GROUP AND 2405 01:49:00,840 --> 01:49:02,600 OVERLAP WHEN THE RESEARCH 2406 01:49:02,600 --> 01:49:04,600 INVOLVES THE PATIENT COMMUNITY 2407 01:49:04,600 --> 01:49:06,440 SO WE'VE BEEN VERY SUCCESSFUL 2408 01:49:06,440 --> 01:49:08,400 AND I GIVE AN EXAMPLE OF HOW 2409 01:49:08,400 --> 01:49:12,160 WE'RE ABLE TO GET THE COMMUNITY 2410 01:49:12,160 --> 01:49:14,760 TO PARTICIPATE AND THE FDAs 2411 01:49:14,760 --> 01:49:16,560 EXTREMELY LED PATIENT FOCUSED 2412 01:49:16,560 --> 01:49:21,240 DRUG DEVELOPMENT AND AND AS WE 2413 01:49:21,240 --> 01:49:23,960 GO AND TRYING TO INCLUDE AN EX 2414 01:49:23,960 --> 01:49:26,880 PANNED OUR EFFORTS BY ENGAGING 2415 01:49:26,880 --> 01:49:30,400 WITH INDUSTRY AND ALSO WITH THE 2416 01:49:30,400 --> 01:49:30,840 REGULATORY. 2417 01:49:30,840 --> 01:49:32,360 SO JUST TO GIVE YOU AN EXAMPLE 2418 01:49:32,360 --> 01:49:34,400 OF WHAT OUR COMMUNITY ENGAGEMENT 2419 01:49:34,400 --> 01:49:36,760 DOES IS WE STARTED A CHAPTER 2420 01:49:36,760 --> 01:49:39,400 PROGRAM A COUPLE YEARS BEFORE I 2421 01:49:39,400 --> 01:49:43,200 JOINED AND THIS IS PROVIDING 2422 01:49:43,200 --> 01:49:46,760 PATIENTS AND CHAPTERS AND AND 2423 01:49:46,760 --> 01:49:48,160 METROPOLITAN AREAS AND THEY DO 2424 01:49:48,160 --> 01:49:50,200 SOME FUNDRAISING AND PROVIDE 2425 01:49:50,200 --> 01:49:52,560 SUPPORT AND INFORMATION TO THE 2426 01:49:52,560 --> 01:49:55,760 COMMUNITY IN THAT AND IN THAT 2427 01:49:55,760 --> 01:49:58,560 AREA GEOGRAPHICAL REGION SO WHEN 2428 01:49:58,560 --> 01:50:01,240 THERE'S A WE FIND AND IN THOSE 2429 01:50:01,240 --> 01:50:02,880 AREAS AND THESE CHAPTERS, WE 2430 01:50:02,880 --> 01:50:04,520 HAVE OVER 30 OF THEM IN THE U.S. 2431 01:50:04,520 --> 01:50:06,680 AND THEY'RE STARTING TO EXPAND 2432 01:50:06,680 --> 01:50:10,880 IN CANADA AND I BELIEVE THIS MAP 2433 01:50:10,880 --> 01:50:13,200 IS ALREADY AND IN CANADA AND ON 2434 01:50:13,200 --> 01:50:14,720 EDUCATIONAL FRONT, WE DO A LOT 2435 01:50:14,720 --> 01:50:16,760 OF DIFFERENT TYPES OF 2436 01:50:16,760 --> 01:50:18,160 EDUCATIONAL PROGRAMS AND ONE OF 2437 01:50:18,160 --> 01:50:22,440 THEM IS THE FSH D AND THESE ARE 2438 01:50:22,440 --> 01:50:24,320 PATIENT FOCUSED MEETINGS THAT 2439 01:50:24,320 --> 01:50:26,840 TAKE PLACE AT CLINICAL CARE 2440 01:50:26,840 --> 01:50:29,200 CENTER AND IT'S AN OPPORTUNITY 2441 01:50:29,200 --> 01:50:31,320 FOR THE COMMUNITY TO IMMEDIATE 2442 01:50:31,320 --> 01:50:34,920 AND COMMERCIALISTS AND 2443 01:50:34,920 --> 01:50:36,440 CLINICIANS AND AND KNOW WHAT IS 2444 01:50:36,440 --> 01:50:40,840 OFFERED IN THEIR REGION AND 2445 01:50:40,840 --> 01:50:41,680 EDUCATIONAL PROGRAMS AND THE 2446 01:50:41,680 --> 01:50:43,760 UNIVERSITY AND THESE ARE 2447 01:50:43,760 --> 01:50:44,840 WEBINARS AND WE JUST STARTED 2448 01:50:44,840 --> 01:50:46,120 THESE ON-LINE COMMUNITIES CALLED 2449 01:50:46,120 --> 01:50:50,240 THE GATHERING PLACE ON SPECIFIC 2450 01:50:50,240 --> 01:50:51,280 TOPICS SO FOR INSTANCE, WE HAVE 2451 01:50:51,280 --> 01:50:56,080 A GATHERING PLACE AND THOSE WHO 2452 01:50:56,080 --> 01:50:57,640 ARE EFFECTED WITH THE DISEASE. 2453 01:50:57,640 --> 01:50:59,680 AS I MENTIONED, OUR OUTREACH IS 2454 01:50:59,680 --> 01:51:02,360 TRYING TO BE GLOBAL AND WE 2455 01:51:02,360 --> 01:51:04,720 HELPED ORGANIZE THE FSHD WORLD 2456 01:51:04,720 --> 01:51:06,240 ALLIANCE AS YOU CAN IMAGINE. 2457 01:51:06,240 --> 01:51:08,840 THERE ARE MANY PATIENT ADVOCACY 2458 01:51:08,840 --> 01:51:09,920 GROUPS INTERNATIONALLY IN 2459 01:51:09,920 --> 01:51:11,600 DIFFERENT COUNTRIES AND SO WE 2460 01:51:11,600 --> 01:51:14,200 HELP ORGANIZE THEM INTO THE 2461 01:51:14,200 --> 01:51:16,200 WORLD ALLIANCE AND NOW WE WERE 2462 01:51:16,200 --> 01:51:21,040 AND ALLIANCE AND ADVANCE 2463 01:51:21,040 --> 01:51:25,920 PROGRAMS AND MAKE SURE THAT THE 2464 01:51:25,920 --> 01:51:30,920 PRIORITIES AND GOING FORWARD, WE 2465 01:51:30,920 --> 01:51:41,160 TAP INTO OUR WE'LL FOCUS ON 2466 01:51:41,160 --> 01:51:42,560 ACCESS FOR THE DRUGS. 2467 01:51:42,560 --> 01:51:44,200 SO ON THE RESEARCH SIDE, THIS IS 2468 01:51:44,200 --> 01:51:49,880 WHERE I'M GOING TO FOCUS MY 2469 01:51:49,880 --> 01:51:55,320 PRESENTATION IS ALL OF DRUG 2470 01:51:55,320 --> 01:51:56,760 DEVELOPMENT BEGINS WITH A BASIC 2471 01:51:56,760 --> 01:51:59,720 UNDERSTANDING OF THE PATH OF 2472 01:51:59,720 --> 01:52:01,360 PHYSIOLOGY OF THE DISEASE SO OUR 2473 01:52:01,360 --> 01:52:03,040 ORGANIZATION LIKE OTHER NON 2474 01:52:03,040 --> 01:52:04,360 PROFITS IN THE AREA SUCH AS 2475 01:52:04,360 --> 01:52:09,040 FRIENDS OF FSH RESEARCH AND 2476 01:52:09,040 --> 01:52:13,120 CHRIS CAREEN OWE FOUNDATION AND 2477 01:52:13,120 --> 01:52:14,680 IN AUSTRALIA AND GLOBAL AND 2478 01:52:14,680 --> 01:52:16,600 WE'VE BEEN FUNDING A LOT OF NIH 2479 01:52:16,600 --> 01:52:20,040 AND DA AND ALL THESE OTHER 2480 01:52:20,040 --> 01:52:23,880 ORGANIZATIONS AND SOME BASIC 2481 01:52:23,880 --> 01:52:26,120 RESEARCH AND HOPEFULLY IT'S AN 2482 01:52:26,120 --> 01:52:28,240 INCENTIVE FOR DRUG DEVELOPERS 2483 01:52:28,240 --> 01:52:30,400 THEN TO START LOOKING AT 2484 01:52:30,400 --> 01:52:34,520 DEVELOPING DRUGS AND TREATMENTS 2485 01:52:34,520 --> 01:52:42,400 AND SO THE IMD ENABLING OR 2486 01:52:42,400 --> 01:52:51,280 INITIAL WE HAVEN'T DONE A LOT IN 2487 01:52:51,280 --> 01:52:56,680 THAT SECTOR AND AND TO ENTER THE 2488 01:52:56,680 --> 01:52:58,560 CLINIC, THERE ARE SOME 2489 01:52:58,560 --> 01:52:59,640 FUNDAMENTAL THINGS THAT EVERY 2490 01:52:59,640 --> 01:53:01,320 DRUG COMPANY IS GOING TO NEED 2491 01:53:01,320 --> 01:53:06,360 AND SOME OF THESE INCLUDE ASSAY 2492 01:53:06,360 --> 01:53:07,920 OUTCOME MEASURES, MODELS AND SO 2493 01:53:07,920 --> 01:53:09,200 THESE ARE THE INSTRUMENTS AND 2494 01:53:09,200 --> 01:53:10,760 THE TOOLS THAT THEY'RE GOING 2495 01:53:10,760 --> 01:53:12,680 INFORM NEED AND IN THE CLINIC 2496 01:53:12,680 --> 01:53:16,560 AND THEY'RE GOING TO NEED AN 2497 01:53:16,560 --> 01:53:17,280 INFRASTRUCTURE AND THEY CAN 2498 01:53:17,280 --> 01:53:24,280 CARRY OUT THEIR CLINICAL TILES 2499 01:53:24,280 --> 01:53:34,800 AND AND IN 2019 COLLABORATIVE 2500 01:53:54,080 --> 01:53:56,640 MEETING AND THIS AND MODERATED 2501 01:53:56,640 --> 01:54:07,120 ORDER AND YOU ALL KNOW AND AND 2502 01:54:08,320 --> 01:54:10,600 LEGAL REPRESENTATION FROM THE 2503 01:54:10,600 --> 01:54:12,160 FDA AND AND NIH GROUPS AND AT 2504 01:54:12,160 --> 01:54:16,200 THIS MEETING AND WE WANTED MAKE 2505 01:54:16,200 --> 01:54:22,160 PRO PHOTO IT AS ATTRACTABLE TO 2506 01:54:22,160 --> 01:54:25,680 ADD TO THEIR PIPELINE SO AND 2507 01:54:25,680 --> 01:54:27,640 ATTENDED THAT MEETING FOUR OF 2508 01:54:27,640 --> 01:54:32,760 WHICH, AN FSHD PROGRAM AND THEIR 2509 01:54:32,760 --> 01:54:36,360 PIPELINE AND THE AND TO IDENTIFY 2510 01:54:36,360 --> 01:54:36,920 THE GAPS IN CLINICAL TRIALS 2511 01:54:36,920 --> 01:54:39,120 RIGHT NOW AND ONE ARE THE MAIN 2512 01:54:39,120 --> 01:54:40,320 HURDLES THAT COMPANIES ARE GOING 2513 01:54:40,320 --> 01:54:42,480 TO NEED IN ORDER TO ADVANCE 2514 01:54:42,480 --> 01:54:43,520 THEIR THERAPEUTICS. 2515 01:54:43,520 --> 01:54:47,880 SO AS YOU CAN IMAGINE, THESE ARE 2516 01:54:47,880 --> 01:54:50,360 COMMON TO A LOT OF DISEASES AND 2517 01:54:50,360 --> 01:54:52,000 WE HEARD THAT THERE'S A GREAT 2518 01:54:52,000 --> 01:54:55,320 NEED FOR BIOMARKERS AND BOTH 2519 01:54:55,320 --> 01:54:57,720 BLOOD BASED BIOMARKERS TO AVOID 2520 01:54:57,720 --> 01:55:08,280 THE MUSCLE BIOPSIES AND THERE'SA 2521 01:55:09,400 --> 01:55:10,480 BETTER NEED FOR OUTCOMES MEASURE 2522 01:55:10,480 --> 01:55:12,920 AND ASSESSMENT TOOLS. 2523 01:55:12,920 --> 01:55:15,400 THEY HAVE REALLY BEEN TOUCHDOWN 2524 01:55:15,400 --> 01:55:17,920 IN MAKING SURE THAT THE THERE 2525 01:55:17,920 --> 01:55:21,960 WAS A NETWORK FOR CLINICAL 2526 01:55:21,960 --> 01:55:32,400 TRIALS AND WE HAD HELPED. 2527 01:56:17,000 --> 01:56:17,920 >>THEY HAVE HISTORY AND IF 2528 01:56:17,920 --> 01:56:19,400 THERE'S A WAY OF INTEGRATING 2529 01:56:19,400 --> 01:56:23,480 THESE IT WOULD BE BENEFICIAL AND 2530 01:56:23,480 --> 01:56:26,040 THE GENETICS ARE COMPLICATED AND 2531 01:56:26,040 --> 01:56:36,560 IMPROVING THE GENETIC AND I'LL 2532 01:57:02,520 --> 01:57:03,880 EXPLAIN WHAT WE DID AND 2533 01:57:03,880 --> 01:57:06,200 REGARDING THE BLOOD BIOMARKERS 2534 01:57:06,200 --> 01:57:08,800 WE LAUNCHED TWO STUDIES THE LOW 2535 01:57:08,800 --> 01:57:13,240 HANGING FRUIT TYPE OF STUDIES 2536 01:57:13,240 --> 01:57:19,000 LOOKING FOR DUX4 IS A TRANCE 2537 01:57:19,000 --> 01:57:20,760 SCRIPPS FACTOR THAT TURNED ON A 2538 01:57:20,760 --> 01:57:21,840 COUPLE HUNDRED DIFFERENT GENES 2539 01:57:21,840 --> 01:57:23,880 AND WE WERE LOOKING FOR THE 2540 01:57:23,880 --> 01:57:25,160 DOWNSTREAM AGAINST THAT ARE 2541 01:57:25,160 --> 01:57:26,200 ACTIVATED BY TO SEE IF THEY 2542 01:57:26,200 --> 01:57:29,520 COULD BE DETECTED IN THE BLOOD 2543 01:57:29,520 --> 01:57:41,320 AND SO -- PATIENT GROUPS THAT 2544 01:57:41,320 --> 01:57:43,280 MIGHT BE A PROCESSING AT A 2545 01:57:43,280 --> 01:57:44,560 DIFFERENT RATE OR FASTER AND IF 2546 01:57:44,560 --> 01:57:47,800 WE CAN EYE FINE THOSE PATIENTS 2547 01:57:47,800 --> 01:57:49,600 THEN WE CAN RECRUIT THEM INTO 2548 01:57:49,600 --> 01:57:51,160 TRIALS MORE EFFECTIVELY. 2549 01:57:51,160 --> 01:57:52,560 AND THE OTHER COLLABORATION WE 2550 01:57:52,560 --> 01:57:55,040 SET UP WITH THE CRITICAL 2551 01:57:55,040 --> 01:57:56,280 INSTITUTE AND WITH THE 2552 01:57:56,280 --> 01:57:58,120 SPECIFICALLY WITH THE RARE 2553 01:57:58,120 --> 01:58:00,480 DISEASE CURES ACCELERATOR DATA 2554 01:58:00,480 --> 01:58:04,160 ANALYTICS PROGRAM AND THIS IS TO 2555 01:58:04,160 --> 01:58:06,840 HARNESS THE TRIAL RESULTS FROM 2556 01:58:06,840 --> 01:58:07,920 PREVIOUS CLINICAL TRIALS AND THE 2557 01:58:07,920 --> 01:58:11,160 FUTURE OF CLINICAL TRIALS AND 2558 01:58:11,160 --> 01:58:13,680 GET THEM PUT INTO A REPOSITORY 2559 01:58:13,680 --> 01:58:16,000 SO THAT WE CAN USE THEM MAYBE TO 2560 01:58:16,000 --> 01:58:21,200 MODEL BETTER CLINICAL DESIGN. 2561 01:58:21,200 --> 01:58:31,720 AS I MENTIONED, WE RAN WE HAVE A 2562 01:58:33,920 --> 01:58:35,160 HEALTH ECONOMIC STUDY THE COST 2563 01:58:35,160 --> 01:58:38,680 BURDEN OF LIVING WITH THE 2564 01:58:38,680 --> 01:58:43,680 DISEASES AND WE ALSO WE ARE 2565 01:58:43,680 --> 01:58:45,000 TAPPING INTO THE REIMBURSEMENT 2566 01:58:45,000 --> 01:58:49,000 OR CLAIMS DATA FROM USING THE 2567 01:58:49,000 --> 01:58:50,000 HEALTHCARE DATABASE. 2568 01:58:50,000 --> 01:58:53,240 ON THE JOINTLY FUNDED 2569 01:58:53,240 --> 01:58:55,120 INITIATIVES, WE HELPED EXPAND 2570 01:58:55,120 --> 01:58:56,520 THE CLINICAL TRIAL RESEARCH 2571 01:58:56,520 --> 01:59:01,560 NETWORK WITH FOUR MORE SITES AND 2572 01:59:01,560 --> 01:59:04,400 WE HELPED LAUNCH THE RESULT 2573 01:59:04,400 --> 01:59:07,600 STUDIES CALLED MOVE AND NOW 2574 01:59:07,600 --> 01:59:09,320 THERE'S SUPPORT FROM OTHER 2575 01:59:09,320 --> 01:59:12,520 GROUPS SUCH AS FSHD CANADA IS 2576 01:59:12,520 --> 01:59:14,600 SUPPORTING THE CONTINUED 2577 01:59:14,600 --> 01:59:15,800 EXPANSION OF SIDES INTO CANADA 2578 01:59:15,800 --> 01:59:19,560 AND U.S. AND EUROPEAN SITES. 2579 01:59:19,560 --> 01:59:21,480 I'LL TALK ABOUT THIS AND THIS IS 2580 01:59:21,480 --> 01:59:23,480 WHERE WE SET UP A SPONSORED 2581 01:59:23,480 --> 01:59:24,560 AGAIN AT THE TIME I CAN TESTING 2582 01:59:24,560 --> 01:59:25,680 PROGRAM FOR THE COMMUNITY. 2583 01:59:25,680 --> 01:59:26,720 THIS WAS A PILOT PROGRAM AND 2584 01:59:26,720 --> 01:59:35,680 I'LL GO INTO THAT. 2585 01:59:35,680 --> 01:59:39,680 AND TRIED TO CREATE AN ALLIANCE 2586 01:59:39,680 --> 01:59:44,040 WHERE THE FACT AND ALLIANCE AND 2587 01:59:44,040 --> 01:59:47,120 ASSESSMENT AND CLINICAL TOOLS 2588 01:59:47,120 --> 01:59:49,440 AND WHAT WE'RE REALLY LOOK TO GO 2589 01:59:49,440 --> 01:59:51,600 DO IS ONCE THE ALLIANCE IS 2590 01:59:51,600 --> 01:59:53,440 ESTABLISHED IT CREATES AN 2591 01:59:53,440 --> 01:59:55,400 OPPORTUNITY FOR PUBLIC-PRIVATE 2592 01:59:55,400 --> 01:59:57,960 PARTNERSHIP AND IT WAS GREAT TO 2593 01:59:57,960 --> 02:00:00,200 HEAR Dr. NUCKOLIS MENTIONED IT 2594 02:00:00,200 --> 02:00:02,200 WAS PART OF WHAT THE NDCC IS 2595 02:00:02,200 --> 02:00:12,680 LOOK TO GO DO IN THE FUTURE. 2596 02:00:14,040 --> 02:00:17,240 WOWEWERE THE THIRD VIRTUAL MEETG 2597 02:00:17,240 --> 02:00:18,200 THAT TOOK PLACE. 2598 02:00:18,200 --> 02:00:20,600 WE STILL MANAGED TO GET ABOUT 2599 02:00:20,600 --> 02:00:21,520 400 PARTICIPANTS AND PATIENTS TO 2600 02:00:21,520 --> 02:00:25,840 COME INTO THE MEETING AND WE 2601 02:00:25,840 --> 02:00:29,080 DRAFTED A REPORT THAT WAS THEN 2602 02:00:29,080 --> 02:00:30,720 SUBMITTED TO FDA AND I BELIEVE 2603 02:00:30,720 --> 02:00:32,640 THAT THERE ARE LINKS TO THIS AND 2604 02:00:32,640 --> 02:00:36,280 IT WILL GO ON OUR WEBSITE AND 2605 02:00:36,280 --> 02:00:37,040 GOING FORWARD, THERE ARE 2606 02:00:37,040 --> 02:00:39,280 OPPORTUNITIES TO ENGAGE WITH THE 2607 02:00:39,280 --> 02:00:41,200 FDA ON MORE SPECIFIC TOPICS AND 2608 02:00:41,200 --> 02:00:42,160 WE'LL LOOK INTO DOING THAT. 2609 02:00:42,160 --> 02:00:44,880 FOR INSTANCE, IMPORTANCE OF 2610 02:00:44,880 --> 02:00:45,720 UPPER MOBILITY IS SOMETHING THAT 2611 02:00:45,720 --> 02:00:47,040 WE'VE HEARD FROM THE COMMUNITY 2612 02:00:47,040 --> 02:00:48,880 AS BEING REALLY CRITICAL FOR 2613 02:00:48,880 --> 02:00:52,320 THEIR QUALITY OF LIFE AND 2614 02:00:52,320 --> 02:00:55,560 TYPICAL MEASUREMENTS ARE LOOKING 2615 02:00:55,560 --> 02:00:57,960 FOR MOVING AND WHEELCHAIR USE 2616 02:00:57,960 --> 02:00:59,280 HOWEVER WHEN PEOPLE CAN'T FEED 2617 02:00:59,280 --> 02:01:01,640 THEMSELVES OR CAN'T TAKE CARE OF 2618 02:01:01,640 --> 02:01:02,600 THEMSELVES BECAUSE THEIR ARMS 2619 02:01:02,600 --> 02:01:04,400 DON'T WORK, IT'S REALLY EFFECTS 2620 02:01:04,400 --> 02:01:05,720 THEIR QUALITY OF LIFE. 2621 02:01:05,720 --> 02:01:07,480 SO WE WANT TO MAYBE HAVE A 2622 02:01:07,480 --> 02:01:10,840 MEETING SPECIFICALLY ON THAT. 2623 02:01:10,840 --> 02:01:12,920 REGARDING GENETIC TESTING, WHERE 2624 02:01:12,920 --> 02:01:17,560 BORROWED A PAGE FROM THE JANE 2625 02:01:17,560 --> 02:01:20,400 FOUNDATION AND THE LEN GIRDLE 2626 02:01:20,400 --> 02:01:23,000 DISEASE GROUPS TRYING TO PROVIDE 2627 02:01:23,000 --> 02:01:24,920 FULLY RESPONSE ORDER GENETIC 2628 02:01:24,920 --> 02:01:26,440 TESTING FOR THE COMMUNITY AND 2629 02:01:26,440 --> 02:01:28,480 THE REASON WHY THIS IS REALLY 2630 02:01:28,480 --> 02:01:30,000 IMPORTANT IS BECAUSE GENETIC 2631 02:01:30,000 --> 02:01:32,760 TESTING IS A CRITICAL INCLUSION 2632 02:01:32,760 --> 02:01:34,640 CRITERIA FOR ENROLLMENT INTO A 2633 02:01:34,640 --> 02:01:36,000 CLINICAL TRIAL AND THAT'S THE 2634 02:01:36,000 --> 02:01:37,600 FIRST STEP AND WE'VE HEARD FROM 2635 02:01:37,600 --> 02:01:42,080 A LOT OF OUR PATIENTS THAT FIRST 2636 02:01:42,080 --> 02:01:44,280 OF ALL, MANY CLINICIANS WERE NOT 2637 02:01:44,280 --> 02:01:49,080 EXPERTS IN FSHD DON'T ORDER IN 2638 02:01:49,080 --> 02:01:52,000 THE PAST, GENETICS ARE 2639 02:01:52,000 --> 02:01:54,000 COMPLICATED SO QUITE OFTEN, THE 2640 02:01:54,000 --> 02:01:56,160 TESTS ARE VERY EXPENSIVE AND 2641 02:01:56,160 --> 02:01:57,320 INSURANCE COMPANIES NOT OFTEN 2642 02:01:57,320 --> 02:01:58,960 DON'T WANT TO REIMBURSE FOR THE 2643 02:01:58,960 --> 02:02:01,360 COST OF THE TEST WHICH CAN RANGE 2644 02:02:01,360 --> 02:02:03,960 BETWEEN $2,000 TO $3,000. 2645 02:02:03,960 --> 02:02:05,760 BUT ALSO, WHEN WITH WE'VE HEARD 2646 02:02:05,760 --> 02:02:07,360 FROM THOSE PATIENTS WHO HAVE 2647 02:02:07,360 --> 02:02:10,200 RECEIVED THE TEST, IT'S MUCH 2648 02:02:10,200 --> 02:02:11,800 THEY DON'T HAVE GENETIC 2649 02:02:11,800 --> 02:02:14,280 COUNSELING AND IF THIS GOES ON 2650 02:02:14,280 --> 02:02:16,160 THE RECORD IT CAN AFFECT THEIR 2651 02:02:16,160 --> 02:02:17,720 ABILITY TO GET LONG-TERM CARE 2652 02:02:17,720 --> 02:02:18,880 AND LIFE INSURANCE AND THOSE 2653 02:02:18,880 --> 02:02:21,120 THINGS SO WE WANTED TO SETTING 2654 02:02:21,120 --> 02:02:23,040 UP SOMETHING COMPREHENSIVE AND 2655 02:02:23,040 --> 02:02:24,680 RE REACHED OUT TO INDUSTRY WHO 2656 02:02:24,680 --> 02:02:31,000 WOULD SUPPORT TH THIS DEFER ANDE 2657 02:02:31,000 --> 02:02:33,120 SET UP A PILOT PROJECT WHERE WE 2658 02:02:33,120 --> 02:02:35,560 DID SEVERAL THINGS. 2659 02:02:35,560 --> 02:02:37,360 ALL OF THE PATIENTS, IT'S A 2660 02:02:37,360 --> 02:02:41,120 DIRECT TO PATIENT PROCESS, 2661 02:02:41,120 --> 02:02:41,960 PROGRAM, SO THEY CAN REACH OUT 2662 02:02:41,960 --> 02:02:44,000 TO A GENETIC COUNSELING GROUP 2663 02:02:44,000 --> 02:02:45,600 AND THEY RECEIVED THE GENETIC 2664 02:02:45,600 --> 02:02:47,280 COUNSELING IF THEY NEED THE 2665 02:02:47,280 --> 02:02:50,200 INCLUSION EXCLUSION CRITERIA FOR 2666 02:02:50,200 --> 02:02:51,760 MOST CLINICAL WHICH IS THEY HAVE 2667 02:02:51,760 --> 02:02:54,520 TO MANIFEST SYSTEMS AND HAVE A 2668 02:02:54,520 --> 02:02:57,680 CLINICAL DIAGNOSIS AND HAVE A 2669 02:02:57,680 --> 02:03:02,040 FAMILY HISTORY OF DISEASE AND 2670 02:03:02,040 --> 02:03:04,160 THE ADULTS WERE QUALIFIED WITH 2671 02:03:04,160 --> 02:03:05,880 THIS PROGRAM AND THEY HAVE A 2672 02:03:05,880 --> 02:03:08,560 TEST WITH FSHD TYPE 1 AND TYPE 2673 02:03:08,560 --> 02:03:09,120 2. 2674 02:03:09,120 --> 02:03:12,360 FOR THE 150 PEOPLE THAT FIRST 2675 02:03:12,360 --> 02:03:13,360 SIGNED UP, WE HAVE RESULTS NOW 2676 02:03:13,360 --> 02:03:18,360 ON ABOUT 90 OF THEM AND IT'S 2677 02:03:18,360 --> 02:03:19,560 GREAT TO KNOW THAT SOME PEOPLE 2678 02:03:19,560 --> 02:03:21,960 GO ON THEIR MEDICAL RECORD 2679 02:03:21,960 --> 02:03:23,240 DECLINED A TEST AND OVER ALL, 2680 02:03:23,240 --> 02:03:25,240 WE'VE GOT VERY GOOD RESPONSE AND 2681 02:03:25,240 --> 02:03:27,400 WE'RE STILL RECEIVING THE 2682 02:03:27,400 --> 02:03:29,440 RESULTS FROM THIS TEST SO WHAT 2683 02:03:29,440 --> 02:03:31,320 WE PLAN TO DO GOING FORWARD IS 2684 02:03:31,320 --> 02:03:33,920 SEE IF WE CAN EXTEND THE 2685 02:03:33,920 --> 02:03:35,040 SPONSORED PROGRAM IN THE U.S. 2686 02:03:35,040 --> 02:03:38,280 AND HOPEFULLY HAVE IT BE OPEN 2687 02:03:38,280 --> 02:03:38,880 INDEFINITELY AND ALSO WE'RE 2688 02:03:38,880 --> 02:03:41,840 LOOKING NOW TO EXPAND THIS 2689 02:03:41,840 --> 02:03:43,920 TESTING PROCESS IN OTHER 2690 02:03:43,920 --> 02:03:47,360 COUNTRIES WHERE THEY HAVE A VERY 2691 02:03:47,360 --> 02:03:48,400 LIMITED CAPACITY. 2692 02:03:48,400 --> 02:03:50,640 SO, AS I MENTIONED, GOING 2693 02:03:50,640 --> 02:03:51,600 FORWARD, WHAT WE'RE LOOKING TO 2694 02:03:51,600 --> 02:03:54,880 DO IS SET UP THIS INDUSTRY 2695 02:03:54,880 --> 02:03:56,840 ALLIANCE CALLED FACT ALLIANCE 2696 02:03:56,840 --> 02:03:58,840 AND THE BENEFITS TO INDUSTRY 2697 02:03:58,840 --> 02:04:00,600 REALLY IS THAT BY JOINING FORCE 2698 02:04:00,600 --> 02:04:02,640 AND JOINING THE RESOURCES IT 2699 02:04:02,640 --> 02:04:05,240 REALLY DERISKS ALL OF THESE 2700 02:04:05,240 --> 02:04:06,480 ACTIVITIES AND COLLECTIVELY THEY 2701 02:04:06,480 --> 02:04:09,040 CAN PROBABLY ADDRESS MORE GAPS 2702 02:04:09,040 --> 02:04:10,840 THAN THEY WOULD BE ABLE TO DO ON 2703 02:04:10,840 --> 02:04:12,120 THEIR OWN. 2704 02:04:12,120 --> 02:04:14,320 IT ALSO IMPORTANTLY AVOIDS 2705 02:04:14,320 --> 02:04:17,840 WASTING RESOURCES AND AVOIDS 2706 02:04:17,840 --> 02:04:18,920 REDUNDANCIES AND FOR THIS 2707 02:04:18,920 --> 02:04:20,120 PROGRAM TO BE SUCCESSFUL, 2708 02:04:20,120 --> 02:04:23,240 OBVIOUSLY, IT HAS TO BE TIMELY 2709 02:04:23,240 --> 02:04:25,040 AND IT HAS TO BE QUICK SO IT 2710 02:04:25,040 --> 02:04:27,600 MATCHES THEIR DRUG DEVELOPMENT 2711 02:04:27,600 --> 02:04:29,480 TIME LINES AND IT NEEDS TO HAVE 2712 02:04:29,480 --> 02:04:31,240 OPEN DATA SHARING SO I WAS 2713 02:04:31,240 --> 02:04:33,960 REALLY GREAT TO HEAR Dr. 2714 02:04:33,960 --> 02:04:37,720 BIANCHI MENTIONING THAT RESEARCH 2715 02:04:37,720 --> 02:04:42,960 NOW WILL HOPEFULLY BE NO MORE 2716 02:04:42,960 --> 02:04:45,680 MORATORIUM ON DATA ACCESS GOING 2717 02:04:45,680 --> 02:04:46,880 FORWARD AND THAT WOULD BE GREAT. 2718 02:04:46,880 --> 02:04:48,280 OBVIOUSLY ANOTHER THING THAT 2719 02:04:48,280 --> 02:04:48,920 WOULD BE HELP [SPEAKING FOREIGN 2720 02:04:48,920 --> 02:04:55,800 TOHAVE REGULATORY INPUT. 2721 02:04:55,800 --> 02:04:57,080 THESE STUDIOS WOULD BE THROUGH 2722 02:04:57,080 --> 02:04:59,080 SUBJECT MATTER EXPERTS AND WITH 2723 02:04:59,080 --> 02:05:01,600 INPUT FROM INDUSTRY AND 2724 02:05:01,600 --> 02:05:02,640 HOPEFULLY REGULATORY BODIES AND 2725 02:05:02,640 --> 02:05:04,080 THIS IS REALLY THE AREA WHERE 2726 02:05:04,080 --> 02:05:06,880 THERE'S AN OPPORTUNITY FOR THE 2727 02:05:06,880 --> 02:05:07,840 PUBLIC-PRIVATE PARTNERSHIP. 2728 02:05:07,840 --> 02:05:10,960 AND AS YOU CAN SEE THAT THE 2729 02:05:10,960 --> 02:05:12,480 TOPICS HAVEN'T CHANGED MUCH FROM 2730 02:05:12,480 --> 02:05:17,840 WHAT I SHOWED IN 2019. 2731 02:05:17,840 --> 02:05:19,520 THERE'S GREAT INTEREST IN 2732 02:05:19,520 --> 02:05:24,480 BIOMARKERS, IMAGING BIOMARKERS 2733 02:05:24,480 --> 02:05:27,040 AND SEEING IF CHANGES IN FATTY 2734 02:05:27,040 --> 02:05:29,520 AND MUSCLE CAN BE SURROGATE END 2735 02:05:29,520 --> 02:05:32,440 POINT FOR DISEASE PROCESSION AND 2736 02:05:32,440 --> 02:05:35,800 THERE'S AN INTEREST IN PEDIATRIC 2737 02:05:35,800 --> 02:05:36,800 NATURAL HISTORY AND THERE'S 2738 02:05:36,800 --> 02:05:39,040 STUDIES THAT HAVE BEEN PROPOSED 2739 02:05:39,040 --> 02:05:41,040 TO DO SOMETHING VERY SIMILAR TO 2740 02:05:41,040 --> 02:05:44,040 WHAT THE ADULT MOVEMENT ARE 2741 02:05:44,040 --> 02:05:46,680 TAKING PLACE AND THERE'S ALSO A 2742 02:05:46,680 --> 02:05:48,360 GREAT DEAL IN REMOTE AND REAL 2743 02:05:48,360 --> 02:05:55,320 WORLD ASSESSMENTS AND HERE, AFTR 2744 02:05:55,320 --> 02:05:57,000 THE PANDEMIC, CLINICAL TRIALS 2745 02:05:57,000 --> 02:06:02,000 STARTED AGAIN AND THE TYPICAL 2746 02:06:02,000 --> 02:06:03,960 TRIAL SITES ARE INUNDATED WITH 2747 02:06:03,960 --> 02:06:04,920 DEMAND. 2748 02:06:04,920 --> 02:06:06,520 HAVING PATIENTS GO IN-PERSON TO 2749 02:06:06,520 --> 02:06:08,480 THE CLIN SICK A BURDEN TO THE 2750 02:06:08,480 --> 02:06:10,760 PATIENT AND IT'S ALSO VERY 2751 02:06:10,760 --> 02:06:12,440 DIFFICULT FOR THE SIDES TO 2752 02:06:12,440 --> 02:06:13,840 ACCOMMODATE SO ANY TOOLS MAKING 2753 02:06:13,840 --> 02:06:18,080 IT EASIER TO PARTICIPATE IN THE 2754 02:06:18,080 --> 02:06:19,880 TRIAL IS QUITE A BENEFIT SOLVE 2755 02:06:19,880 --> 02:06:21,840 GO ALONG WITH THAT IS INNOVATIVE 2756 02:06:21,840 --> 02:06:23,480 TRIAL DESIGNS BECAUSE WE ARE 2757 02:06:23,480 --> 02:06:25,880 DEALING WITH A RARE DISEASE AND 2758 02:06:25,880 --> 02:06:27,320 THERE'S A LIMITED NUMBER OF 2759 02:06:27,320 --> 02:06:29,840 PATIENTS THAT WOULD QUALIFY FOR 2760 02:06:29,840 --> 02:06:32,560 ANY SUCH TRIAL AND JUST AS TO GO 2761 02:06:32,560 --> 02:06:34,720 ALONG WITH THAT ALSO IS THAT IT 2762 02:06:34,720 --> 02:06:37,000 WOULD BE NICE TO EXPAND ON THE 2763 02:06:37,000 --> 02:06:40,160 CLINICAL TRIAL NETWORK AND 2764 02:06:40,160 --> 02:06:41,600 CURRENTLY THERE'S ABOUT 18 SITES 2765 02:06:41,600 --> 02:06:46,440 IN THE U.S. THAT HAVE A 2766 02:06:46,440 --> 02:06:48,120 FAMILIARITY WITH FSHD BUT 2767 02:06:48,120 --> 02:06:51,080 THERE'S MORE AND WESTBOUND WORK 2768 02:06:51,080 --> 02:06:51,960 WITH MUSCULAR DYSTROPHY 2769 02:06:51,960 --> 02:06:56,600 ASSOCIATION AND 150 CLINICAL 2770 02:06:56,600 --> 02:06:58,720 SITES THAT QUALIFY FOR RUNNING 2771 02:06:58,720 --> 02:07:00,480 TRIALS. 2772 02:07:00,480 --> 02:07:01,960 AND I JUST WANTED TO FINISH OFF 2773 02:07:01,960 --> 02:07:07,680 HERE WITH THE CURRENT LANDSCAPE 2774 02:07:07,680 --> 02:07:08,880 IN FSHD. 2775 02:07:08,880 --> 02:07:10,960 WHEN I JOINED IN 2019, THERE 2776 02:07:10,960 --> 02:07:14,240 WERE EIGHT COMPANIES THAT 2777 02:07:14,240 --> 02:07:18,440 ATTENDED THE INDUSTRY 2778 02:07:18,440 --> 02:07:19,640 COLLABORATIVE MEETINGS AND 2779 02:07:19,640 --> 02:07:22,960 WITHIN THE THREE THREE DROPPED 2780 02:07:22,960 --> 02:07:24,120 OUT. 2781 02:07:24,120 --> 02:07:25,720 NOWER WE'RE COUNTING 20 2782 02:07:25,720 --> 02:07:26,880 COMPANIES IN THE SPACE 2783 02:07:26,880 --> 02:07:27,560 DEVELOPING DRUGS. 2784 02:07:27,560 --> 02:07:28,760 AND IT'S FANTASTIC. 2785 02:07:28,760 --> 02:07:37,520 A LOT OF THEM THEM ARE STALL AE 2786 02:07:37,520 --> 02:07:42,320 STILL ON STEALTH MODE BUT THIS 2787 02:07:42,320 --> 02:07:49,000 IS FROM THE WEBSITE AND THREE 2788 02:07:49,000 --> 02:07:50,400 ACTIVE TRIALS AND WE HAVE TWO OR 2789 02:07:50,400 --> 02:07:51,560 THREE NEXT YEAR AND THERE'S A 2790 02:07:51,560 --> 02:07:53,560 LOT OF INTEREST IN THIS KIND OF 2791 02:07:53,560 --> 02:07:55,040 INDUSTRY COLLABORATIVE APPROACH 2792 02:07:55,040 --> 02:07:56,800 TO ADDRESSING THE GAPS AND 2793 02:07:56,800 --> 02:08:07,320 CLINICAL TRIALS READINESS AND 2794 02:08:25,440 --> 02:08:28,560 WHERE WE'RE GOING TO LAUNCH THIS 2795 02:08:28,560 --> 02:08:29,880 INDUSTRY ALLIANCE AND THE 2796 02:08:29,880 --> 02:08:31,520 MEETING WILL THEN FINISH WITH 2797 02:08:31,520 --> 02:08:33,280 WORLD ALLIANCE MEETING OF ALL 2798 02:08:33,280 --> 02:08:37,480 THE VOLUNTEERS GROUPS FROM 2799 02:08:37,480 --> 02:08:37,960 INTERNATIONAL COUNTRIES. 2800 02:08:37,960 --> 02:08:41,000 THANK YOU, VERY MUCH. 2801 02:08:41,000 --> 02:08:42,520 I'LL TAKE SOME QUESTIONS. 2802 02:08:42,520 --> 02:08:46,480 >>Diana Bianchi: THANK YOU FOR 2803 02:08:46,480 --> 02:08:51,800 THAT VVERY CLEAR AND EXCITING 2804 02:08:51,800 --> 02:08:52,160 PRESENTATION. 2805 02:08:52,160 --> 02:08:54,040 THIS IS WHY WE NEED A NEW 2806 02:08:54,040 --> 02:08:54,520 STRATEGIC PLAN. 2807 02:08:54,520 --> 02:08:58,040 THE OLD STRATEGIC PLAN FOR THE 2808 02:08:58,040 --> 02:09:00,280 MDCC IS CLEARLY OUTDATED AND SO 2809 02:09:00,280 --> 02:09:02,760 WE LOOK FORWARD TO INCORPORATING 2810 02:09:02,760 --> 02:09:05,600 SOME OF THESE GREAT ADVANCES. 2811 02:09:05,600 --> 02:09:09,480 SO, THE FLOOR IS OPEN FOR 2812 02:09:09,480 --> 02:09:19,680 QUESTIONS. 2813 02:09:19,960 --> 02:09:21,240 GUSTAVO, YOU CAME ON VIDEO. 2814 02:09:21,240 --> 02:09:24,240 DO YOU HAVE A QUESTION? 2815 02:09:24,240 --> 02:09:24,760 >>Glen Nuckolls: NO. 2816 02:09:24,760 --> 02:09:35,840 THE ROOMIS VERY QUIET TODAY. 2817 02:09:38,480 --> 02:09:39,080 >>Glen Nuckolls: IF THERE ARE 2818 02:09:39,080 --> 02:09:41,320 QUESTIONS, PUT IT IN THE CHAT 2819 02:09:41,320 --> 02:09:46,200 AND I CAN ANSWER ON-LINE. 2820 02:09:46,200 --> 02:09:46,800 >>Diana Bianchi: ON-LINE. 2821 02:09:46,800 --> 02:09:47,480 >>Diana Bianchi: LET'S MOVE ON 2822 02:09:47,480 --> 02:09:52,320 TO OUR NEXT PRESENTER, Dr. 2823 02:09:52,320 --> 02:09:54,800 TANYA STEVENSON THE MIO TONIC 2824 02:09:54,800 --> 02:09:57,120 DYSTROPHY FOUNDATION AND TONIA 2825 02:09:57,120 --> 02:10:07,600 WILL BE PRESENTING REMOTELY. 2826 02:10:08,400 --> 02:10:09,720 >>THANK YOU, I'M GOING TO 2827 02:10:09,720 --> 02:10:19,400 PRESENT MY SCREEN HERE. 2828 02:10:19,400 --> 02:10:29,560 AT THIS. 2829 02:10:33,520 --> 02:10:34,640 >>THANK YOU FOR IN INVITING ME 2830 02:10:34,640 --> 02:10:35,000 TODAY. 2831 02:10:35,000 --> 02:10:37,280 I'M EXCITE TODAY TALK ABOUT THE 2832 02:10:37,280 --> 02:10:39,400 STRATEGIC WORK THE MYOTONIC DIS 2833 02:10:39,400 --> 02:10:41,520 TOE FEE FOUNDATION HAS DONE OVER 2834 02:10:41,520 --> 02:10:44,080 THE YEARS. 2835 02:10:44,080 --> 02:10:45,280 SO, THE MYOTONIC FOUNDATION IS 2836 02:10:45,280 --> 02:10:47,400 THE LEADING GLOBAL ADVOCATE 2837 02:10:47,400 --> 02:10:50,120 HELPING PATIENTS AND FAMILIES 2838 02:10:50,120 --> 02:10:51,400 NAVIGATE THE MYOTONIC DYSTROPHY 2839 02:10:51,400 --> 02:10:53,640 PROCESS AND THE VERY FIRST 2840 02:10:53,640 --> 02:10:55,120 RESEARCH CONTACTED BY NEWLY 2841 02:10:55,120 --> 02:10:56,560 DIAGNOSED PATIENTS, FAMILIES, 2842 02:10:56,560 --> 02:10:58,480 SOCIAL WORKERS, PHYSICIANS, AND 2843 02:10:58,480 --> 02:10:59,800 ALL OVER THE WORLD. 2844 02:10:59,800 --> 02:11:01,320 THERE ARE OVER 25,000 PEOPLE 2845 02:11:01,320 --> 02:11:04,760 RIGHT NOW IN OUR MDF COMMUNITY. 2846 02:11:04,760 --> 02:11:08,160 WE'RE SORT OF CONSIDERED THE 2847 02:11:08,160 --> 02:11:11,720 NECK SUN OF COMMUNITY CARE AND 2848 02:11:11,720 --> 02:11:18,760 CURE. 2849 02:11:18,760 --> 02:11:20,440 WE SUPPORT AND CONNECT THE 2850 02:11:20,440 --> 02:11:21,720 MYOTONIC DISTRICT COMMUNITY. 2851 02:11:21,720 --> 02:11:23,800 WE PROVIDE RESOURCE AND ADVOCATE 2852 02:11:23,800 --> 02:11:25,320 FOR CARE. 2853 02:11:25,320 --> 02:11:27,960 WE ACCELERATE RESEARCH TOWARDS A 2854 02:11:27,960 --> 02:11:30,560 CURE AND RESEARCH. 2855 02:11:30,560 --> 02:11:32,680 OUR WORK AND OUR DECISIONS ARE 2856 02:11:32,680 --> 02:11:34,560 GUIDED BY SIX CORE VALUES. 2857 02:11:34,560 --> 02:11:37,000 COMMUNITY, COL APP RACE, I AM 2858 02:11:37,000 --> 02:11:40,840 PATHY, HOPE, URGENCY, AND 2859 02:11:40,840 --> 02:11:41,480 KNOWLEDGE. 2860 02:11:41,480 --> 02:11:43,880 AND FOR THOSE WHO ARE A LITTLE 2861 02:11:43,880 --> 02:11:44,840 LESS FAMILIAR WITH MYOTONIC 2862 02:11:44,840 --> 02:11:47,400 DYSTROPHY IT'S A GENETIC RARE 2863 02:11:47,400 --> 02:11:49,240 PROGRESSIVE REPEAT EXPANSION 2864 02:11:49,240 --> 02:11:49,960 DISEASE. 2865 02:11:49,960 --> 02:11:52,080 IT'S OFTEN CONSIDERED THE MOST 2866 02:11:52,080 --> 02:11:53,880 VARIABLE OF ALL KNOWN 2867 02:11:53,880 --> 02:11:54,240 CONDITIONS. 2868 02:11:54,240 --> 02:11:57,720 IT AFFECTS AS MANY AS 1/2100 2869 02:11:57,720 --> 02:12:01,680 PEOPLE ALTHOUGH MOST ARE NOT 2870 02:12:01,680 --> 02:12:02,280 DIAGNOSED. 2871 02:12:02,280 --> 02:12:03,680 THERE ARE TWO TYPES, TYPE ONE IS 2872 02:12:03,680 --> 02:12:08,160 A RESULT OF A MUTATION IN DMPK 2873 02:12:08,160 --> 02:12:09,640 GENE AND TYPE TWO THE RESULT OF 2874 02:12:09,640 --> 02:12:12,680 MUTATION IN THE CNBP GENE. 2875 02:12:12,680 --> 02:12:16,200 AND IT IS THE MOST COMMON FORM 2876 02:12:16,200 --> 02:12:17,240 OF ADULT MUSCULAR DYSTROPHIES 2877 02:12:17,240 --> 02:12:21,040 BUT SYMPTOMS CAN PRESENT AT ANY 2878 02:12:21,040 --> 02:12:21,480 AGE. 2879 02:12:21,480 --> 02:12:22,800 IT EFFECTS EVERYBODY'S SYSTEM 2880 02:12:22,800 --> 02:12:25,200 AND THE SYMPTOMS ARE DIFFERENT 2881 02:12:25,200 --> 02:12:25,880 IN EACH INDIVIDUAL, EVEN WITHIN 2882 02:12:25,880 --> 02:12:27,520 THE SAME FAMILY, THE SYMPTOMS 2883 02:12:27,520 --> 02:12:31,440 CAN LOOK VERY, VERY DIFFERENT. 2884 02:12:31,440 --> 02:12:34,080 TYPICALLY, THIS DISEASE 2885 02:12:34,080 --> 02:12:34,920 EXPERIENCES ANTICIPATION WHICH 2886 02:12:34,920 --> 02:12:37,960 IS THE SYMPTOMS ARE MORE SEVERE 2887 02:12:37,960 --> 02:12:39,360 WITH EACH GENERATION. 2888 02:12:39,360 --> 02:12:41,160 THERE'S NO CURE. 2889 02:12:41,160 --> 02:12:44,240 CURRENTLY NO FDA APPROVED 2890 02:12:44,240 --> 02:12:45,080 TREATMENTS. 2891 02:12:45,080 --> 02:12:49,880 SO JUST A FI QUICK SNAPSHOT OF R 2892 02:12:49,880 --> 02:12:50,320 PRIMARY INITIATIVES. 2893 02:12:50,320 --> 02:12:51,440 WE ARE BRINGING TOGETHER THE 2894 02:12:51,440 --> 02:12:54,920 WORLD'S LEADING EXPERTS TO 2895 02:12:54,920 --> 02:12:55,720 CREATE CLINICAL CARE GUIDELINES 2896 02:12:55,720 --> 02:12:56,880 AND RECOMMENDATIONS. 2897 02:12:56,880 --> 02:12:59,680 WE HAVE TOOL KITS TO HELP FOLKS 2898 02:12:59,680 --> 02:13:00,880 FIND A DOCTOR. 2899 02:13:00,880 --> 02:13:02,440 WE HAVE A LOT OF RESOURCE AND 2900 02:13:02,440 --> 02:13:04,000 INFORMATION ON OUR WEBSITE. 2901 02:13:04,000 --> 02:13:06,720 WE HAVE OVER 30 INTERNATIONAL 2902 02:13:06,720 --> 02:13:07,320 SUPPORT GROUPS AND THEY'RE 2903 02:13:07,320 --> 02:13:08,880 OFFERED IN THREE DIFFERENT 2904 02:13:08,880 --> 02:13:10,080 LANGUAGES AND THEY'RE BOTH 2905 02:13:10,080 --> 02:13:12,320 GEOGRAPHY AND TOPIC BASED. 2906 02:13:12,320 --> 02:13:14,640 WE HAVE WHAT WE CALL A WARN LINE 2907 02:13:14,640 --> 02:13:15,840 OR A CALL LINE. 2908 02:13:15,840 --> 02:13:18,600 A HELP LINE FOR FAMILIES AND 2909 02:13:18,600 --> 02:13:20,480 CLINICIANS WHO ARE LOOKING FOR 2910 02:13:20,480 --> 02:13:22,280 RESOURCES AND SUPPORT. 2911 02:13:22,280 --> 02:13:24,040 WE INVEST SIGNIFICANTLY IN 2912 02:13:24,040 --> 02:13:26,440 GRANTS AND IN FELLOWSHIPS, 2913 02:13:26,440 --> 02:13:30,640 CURRENTLY WE HAVE INVESTED OVER 2914 02:13:30,640 --> 02:13:31,520 $10 MILLION IN MYOTONIC 2915 02:13:31,520 --> 02:13:32,320 DYSTROPHY RESEARCH. 2916 02:13:32,320 --> 02:13:35,280 WE PROVIDE AN ANNUAL DM ONLY 2917 02:13:35,280 --> 02:13:36,400 CONFERENCE LARGEST ONE IN THE 2918 02:13:36,400 --> 02:13:36,880 WORLD. 2919 02:13:36,880 --> 02:13:39,840 OUR WHOLE ECOSYSTEM COMES 2920 02:13:39,840 --> 02:13:40,280 TOGETHER. 2921 02:13:40,280 --> 02:13:44,120 WE ARE HOSTING THE MYOTONIC 2922 02:13:44,120 --> 02:13:46,480 DYSTROPHY DIGITAL ACADEMY, THE 2923 02:13:46,480 --> 02:13:48,840 LARGEST DIGITAL LIBRARY FOR 2924 02:13:48,840 --> 02:13:50,160 MYOTONIC DYSTROPHY IN THE WORLD 2925 02:13:50,160 --> 02:13:54,840 OVER 170 VIDEOS, 130 HOURS OF 2926 02:13:54,840 --> 02:13:55,360 CONTENT. 2927 02:13:55,360 --> 02:13:57,360 WE WORK WITH INDUSTRY AND OUR 2928 02:13:57,360 --> 02:13:58,440 STUDY AND TRIAL SITES AROUND THE 2929 02:13:58,440 --> 02:13:59,920 GLOBE TO SUPPORT CLINICAL TRIALS 2930 02:13:59,920 --> 02:14:02,360 IN THE STUDIES. 2931 02:14:02,360 --> 02:14:06,040 AND WE DOLL SIGNIFICANT ADVOCACY 2932 02:14:06,040 --> 02:14:07,080 FOR OTHER WARENESS RAISING IN 2933 02:14:07,080 --> 02:14:08,960 GENERAL AND IMPROVED AND 2934 02:14:08,960 --> 02:14:11,440 INCREASED RESEARCH FUNDING. 2935 02:14:11,440 --> 02:14:14,880 A BIT ABOUT OUR STRATEGIC 2936 02:14:14,880 --> 02:14:15,440 PLANNING BACKGROUND, WE WERE 2937 02:14:15,440 --> 02:14:17,040 FOUNDED IN 2007 BY SHANNON LORD 2938 02:14:17,040 --> 02:14:18,960 AND FAMILIES WHO WERE LOOKING 2939 02:14:18,960 --> 02:14:21,640 FOR ANSWERS AND RESOURCES. 2940 02:14:21,640 --> 02:14:24,720 THAT'S WHEN OUR FIRST MISSION 2941 02:14:24,720 --> 02:14:26,240 STATEMENT WAS DEVELOPED. 2942 02:14:26,240 --> 02:14:28,400 THE STRATEGIES FOCUSED ON 2943 02:14:28,400 --> 02:14:29,280 DEVELOPING RESPONSIVE PROGRAMS 2944 02:14:29,280 --> 02:14:31,240 AND FINDING THE REVENUE TO FUND 2945 02:14:31,240 --> 02:14:31,720 THOSE PROGRAMS. 2946 02:14:31,720 --> 02:14:33,720 WE THEN MADE STRATEGIC 2947 02:14:33,720 --> 02:14:37,840 INVESTMENTS TO CREATE THE 2948 02:14:37,840 --> 02:14:42,280 MYOTONIC DYSTROPHY REGISTRY THAT 2949 02:14:42,280 --> 02:14:43,080 HAS 2,400 PEOPLE REGISTERED 2950 02:14:43,080 --> 02:14:45,000 N-2015, WE CREATED A PLAN FOR 2951 02:14:45,000 --> 02:14:47,360 OUR CURE NOT INVESTMENTS AND 2952 02:14:47,360 --> 02:14:48,480 RESEARCH INVESTMENTS AND IT WAS 2953 02:14:48,480 --> 02:14:50,320 VERY CLEAR THAT WE NEED TODAY DO 2954 02:14:50,320 --> 02:14:51,840 MORE TO HELP ELIMINATE THE 2955 02:14:51,840 --> 02:14:56,600 BARRIERS TO DRUG DEVELOPMENT. 2956 02:14:56,600 --> 02:14:57,720 IN 2020, WE HAD AN OPPORTUNITY 2957 02:14:57,720 --> 02:14:59,840 TO REALLY DO AN ASSESSMENT OF 2958 02:14:59,840 --> 02:15:02,000 OUR COMMUNITY NEEDS AND DEVELOP 2959 02:15:02,000 --> 02:15:06,160 RESPONSIVE GOALS AND OBJECTIVES. 2960 02:15:06,160 --> 02:15:12,680 SO, IN 2015, WHICH THE LAST 2961 02:15:12,680 --> 02:15:14,760 ACTION PLAN WAS 2015 AND I 2962 02:15:14,760 --> 02:15:16,000 WANTED TO LOOK WHAT WAS 2963 02:15:16,000 --> 02:15:17,120 HAPPENING AT THAT POINT AND TIME 2964 02:15:17,120 --> 02:15:20,280 AND THERE WAS ONLY ONE COMPANY 2965 02:15:20,280 --> 02:15:22,680 FOCUSED ON MYOTONIC DYSTROPHY. 2966 02:15:22,680 --> 02:15:23,720 TODAY WE HAVE THREE DOZEN 2967 02:15:23,720 --> 02:15:26,640 COMPANIES LOOKING AT MYOTONIC 2968 02:15:26,640 --> 02:15:27,960 DYSTROPHY AND INSTITUTIONS 2969 02:15:27,960 --> 02:15:29,000 FOCUSED ON THE DISEASE. 2970 02:15:29,000 --> 02:15:31,200 THERE ARE FOUR ACTIVE CLINICAL 2971 02:15:31,200 --> 02:15:33,760 TRIALS AND WE KNOW OF THREE THAT 2972 02:15:33,760 --> 02:15:35,600 ARE GOING TO BE ESTIMATED TO 2973 02:15:35,600 --> 02:15:37,000 LAUNCH IN THE NEXT YEAR BUT WE 2974 02:15:37,000 --> 02:15:43,200 HOPE THAT THERE ARE MORE. 2975 02:15:43,200 --> 02:15:47,080 SEW WHAT DID WE DO IN 2015 TO HE 2976 02:15:47,080 --> 02:15:48,640 CAN SELL RATE THE DRUG 2977 02:15:48,640 --> 02:15:49,360 DEVELOPMENT PIPELINE. 2978 02:15:49,360 --> 02:15:51,240 WE MADE A 5 MILLION-DOLLAR 2979 02:15:51,240 --> 02:15:51,720 INVESTMENT. 2980 02:15:51,720 --> 02:15:53,640 FIRST WE HELD THE PATIENT 2981 02:15:53,640 --> 02:15:57,640 FOCUSED DRUG DEVELOPMENT SESSION 2982 02:15:57,640 --> 02:15:58,920 WITH THE F.A.A. AND WE HAD A 2983 02:15:58,920 --> 02:16:07,400 PATIENT REPORT THAT CAME OUT IN 2984 02:16:07,400 --> 02:16:09,040 2017. 2985 02:16:09,040 --> 02:16:10,840 AND THE STUDIES INVOLVED IN IT 2986 02:16:10,840 --> 02:16:12,880 AND WE EXPANDED OUR RESEARCH 2987 02:16:12,880 --> 02:16:13,360 FELLOW SHOP PROGRAM. 2988 02:16:13,360 --> 02:16:18,000 FELL SHIP PROGRAM. 2989 02:16:18,000 --> 02:16:20,840 Dr. BIANCHI MENTIONED THE GAB 2990 02:16:20,840 --> 02:16:23,440 A RECEPTORS AND THE DOCTOR WHO 2991 02:16:23,440 --> 02:16:26,160 WAS ACTUALLY ONE OF THE FELLOWS 2992 02:16:26,160 --> 02:16:28,320 FOR MDF WHILE SHE WAS WORKING ON 2993 02:16:28,320 --> 02:16:30,960 THAT PROJECT AND Dr. ERIC 2994 02:16:30,960 --> 02:16:35,160 WAYNE WHO ALSO COMMENTED ON THAT 2995 02:16:35,160 --> 02:16:40,080 PROJECT, HE WAS AN PDF FELLOW SO 2996 02:16:40,080 --> 02:16:50,520 WE'RE HOPING TO IN MY WITH 2997 02:17:09,560 --> 02:17:10,880 CLINICAL CARE RECOMMENDATION AND 2998 02:17:10,880 --> 02:17:15,600 GUIDELINES FOR DM1, 2, 2999 02:17:15,600 --> 02:17:17,000 CARDIOLOGY AND ANESTHESIAIA, 3000 02:17:17,000 --> 02:17:19,240 PULL MINUTOLOGY AND OTHERS. 3001 02:17:19,240 --> 02:17:21,960 AND WE WORKED TO HELP EXPAND THE 3002 02:17:21,960 --> 02:17:24,720 NATURAL HISTORY STUDY FROM 3003 02:17:24,720 --> 02:17:35,200 MYOTONIC DYSTROPHY TYPE ONE. 3004 02:17:43,560 --> 02:17:45,600 AND THAT WAS OVER ABOUT FIVE 3005 02:17:45,600 --> 02:17:46,320 YEARS OF INVESTMENTS IN 3006 02:17:46,320 --> 02:17:47,840 DIFFERENT PROJECTS AND IN THE 3007 02:17:47,840 --> 02:17:50,800 SPRING OF 2021, WE EMBARKED ON A 3008 02:17:50,800 --> 02:17:53,160 NINE-MONTH LONG STRATEGIC 3009 02:17:53,160 --> 02:17:54,920 PLANNING PROCESS TO ENVISION THE 3010 02:17:54,920 --> 02:17:56,240 NEAR TERM FUTURE OF THE 3011 02:17:56,240 --> 02:17:59,920 ORGANIZATION OVER THE NEXT THREE 3012 02:17:59,920 --> 02:18:04,840 YEARS. 3013 02:18:04,840 --> 02:18:11,640 IT WAS PEOPLE TYPE ONE, TYPE TWO 3014 02:18:11,640 --> 02:18:18,000 AND INTERNATIONAL PARTNERS AND 3015 02:18:18,000 --> 02:18:20,960 OUR BOARD MEMBERS AND STAFF AND 3016 02:18:20,960 --> 02:18:22,440 CONSULTANTS. 3017 02:18:22,440 --> 02:18:26,040 AND PROCESS WAS THREE PHASES. 3018 02:18:26,040 --> 02:18:31,320 LEARNING IDEATION AND ITERATIVE 3019 02:18:31,320 --> 02:18:32,040 DESIGN. 3020 02:18:32,040 --> 02:18:32,880 WE'RE VERY PROUD OF THE 3021 02:18:32,880 --> 02:18:36,480 COMMUNITY ENGAGEMENT THAT WE 3022 02:18:36,480 --> 02:18:38,880 WORKED VERY, VERY HARD AND TO 3023 02:18:38,880 --> 02:18:40,640 CREATE AND TO ENCOURAGE AND WE 3024 02:18:40,640 --> 02:18:46,960 HAD OVER 800 MEMBERS OF THE 3025 02:18:46,960 --> 02:18:48,040 COMMUNITY PARTICIPATING AND 3026 02:18:48,040 --> 02:18:49,480 SHARING THEIR EXPERIENCE AND 713 3027 02:18:49,480 --> 02:18:52,200 WERE SURVEY PARTICIPANTS AND WE 3028 02:18:52,200 --> 02:18:53,960 HAD 39 ONE-ON-ONE INTERVIEW WITH 3029 02:18:53,960 --> 02:18:55,600 STAKEHOLDERS AND D.N. EXPERT 3030 02:18:55,600 --> 02:18:58,240 INTERVIEWS AND 18 FOCUS GROUPS 3031 02:18:58,240 --> 02:19:03,360 WITH 71 TOTAL PARTICIPANTS. 3032 02:19:03,360 --> 02:19:04,760 SO THE COMMUNITY ENGAGEMENT. 3033 02:19:04,760 --> 02:19:06,840 WHO ARE ALL THOSE 800 PEOPLE? 3034 02:19:06,840 --> 02:19:09,640 THEY WERE AFFECTED BY MYOTONIC 3035 02:19:09,640 --> 02:19:11,680 DYSTROPHY AND CAREGIVERS. 3036 02:19:11,680 --> 02:19:14,120 OUR IS SUPPORT GROUP 3037 02:19:14,120 --> 02:19:16,120 FACILITIESORS, CLINICIANS, 3038 02:19:16,120 --> 02:19:17,920 PARTNERS, RESEARCHERS, BOARD 3039 02:19:17,920 --> 02:19:19,440 MEMBERS, WE HAD OUR DONORS 3040 02:19:19,440 --> 02:19:23,560 INVOLVED IN IS THIS AND OR 3041 02:19:23,560 --> 02:19:25,000 SCIENTIFIC ADVISORY AND OTHER 3042 02:19:25,000 --> 02:19:28,640 RARE DISEASE PATIENT ADVOCACY 3043 02:19:28,640 --> 02:19:29,360 ORGANIZATIONS. 3044 02:19:29,360 --> 02:19:32,560 AND ULTIMATELY THAT PLAN CREATED 3045 02:19:32,560 --> 02:19:35,040 A VISION STATEMENT, UPDATED 3046 02:19:35,040 --> 02:19:37,280 MISSION STATEMENT, OUR CORE 3047 02:19:37,280 --> 02:19:38,000 VALUES, THE OVERARCHING GOALS 3048 02:19:38,000 --> 02:19:40,080 FOR THE NEXT THREE YEARS AND 3049 02:19:40,080 --> 02:19:41,880 THOSE ALSO INCLUDED STRATEGIES 3050 02:19:41,880 --> 02:19:45,120 AND MEDICINE TRICKS AND A 3051 02:19:45,120 --> 02:19:48,320 TIMELINE AND BUDGET WITH THOSE 3052 02:19:48,320 --> 02:19:49,640 ACTIVITIES. 3053 02:19:49,640 --> 02:19:55,280 WHAT WERE THOSE THREE OVER THE 3054 02:19:55,280 --> 02:19:56,840 GOALS FOR THIS PROJECT AND THIS 3055 02:19:56,840 --> 02:19:59,200 PLAN AND COMMUNITY CARE, CURE 3056 02:19:59,200 --> 02:20:00,440 AND ORGANIZATIONAL STRENGTH. 3057 02:20:00,440 --> 02:20:03,000 IT WAS REALLY CLEAR THAT THE 3058 02:20:03,000 --> 02:20:09,040 VOICE OF THE COMMUNITY AND OUR 3059 02:20:09,040 --> 02:20:10,720 ECOSYSTEM TOLD US WE NEED TO 3060 02:20:10,720 --> 02:20:12,360 IMPROVE AND EXPAND ACCESS TO 3061 02:20:12,360 --> 02:20:14,040 HEALTHCARE AND RESOURCES TO MEET 3062 02:20:14,040 --> 02:20:15,560 THE NEEDS OF PEOPLE LIVING WITH 3063 02:20:15,560 --> 02:20:18,400 DISEASE AND THEIR FAMILIES. 3064 02:20:18,400 --> 02:20:20,520 WE NEEDED TO ELIMINATE BARRIERS 3065 02:20:20,520 --> 02:20:21,560 FOR DRUG DEVELOPMENT AND TO 3066 02:20:21,560 --> 02:20:26,280 BUILD A STRONG AND SUSTAINABLE 3067 02:20:26,280 --> 02:20:27,480 ORGANIZATION SO WE'RE NOW IN 3068 02:20:27,480 --> 02:20:30,200 YEAR TWO OF OUR STRATEGIC PLAN. 3069 02:20:30,200 --> 02:20:37,240 AND NOT SOMETHING THAT WE JUST 3070 02:20:37,240 --> 02:20:39,240 COMPLETED, THIS WEEK, WE 3071 02:20:39,240 --> 02:20:41,240 COMPLETED OUR MYOTONIC DYSTROPHY 3072 02:20:41,240 --> 02:20:43,840 RESEARCH LANDSCAPE ASSESSMENT 3073 02:20:43,840 --> 02:20:45,160 INCLUDING 91 INDIVIDUALS, 49 3074 02:20:45,160 --> 02:20:48,160 INTERVIEWS AND FOCUS GROUPS, 39 3075 02:20:48,160 --> 02:20:50,640 INSTITUTIONS, AND NINE 3076 02:20:50,640 --> 02:20:50,920 COUNTRIES. 3077 02:20:50,920 --> 02:20:53,360 WE WERE LOOKING FOR THE GAPS AND 3078 02:20:53,360 --> 02:20:55,360 BARRIERS THAT EXIST FROM 3079 02:20:55,360 --> 02:20:57,240 MYOTONIC DYSTROPHY RESEARCH AND 3080 02:20:57,240 --> 02:20:59,000 BARRIERS TO ACTIVATING AND 3081 02:20:59,000 --> 02:21:00,680 MAINTAINING HIGH FUNCTIONING 3082 02:21:00,680 --> 02:21:04,960 RESEARCH SITES AND WELL ENROLLED 3083 02:21:04,960 --> 02:21:06,360 STUDIES AND TRIALS AND WE WANTED 3084 02:21:06,360 --> 02:21:11,240 TO KNOW HOW AND COULD FURTHER 3085 02:21:11,240 --> 02:21:13,720 SUPPORT PROGRESS IN THE FIELD, 3086 02:21:13,720 --> 02:21:16,440 WE HAVE RELEASED THE MYOTONIC 3087 02:21:16,440 --> 02:21:19,040 RESEARCH MAP SO THIS IS AN 3088 02:21:19,040 --> 02:21:20,880 INTERACTIVE VISUAL BASE OF OUR 3089 02:21:20,880 --> 02:21:26,800 ECOSYSTEM AND IT HAS OVER 68,000 3090 02:21:26,800 --> 02:21:37,240 DATA POINTS AND WE HOPE TO 3091 02:21:54,160 --> 02:21:56,000 RELEASE IN THE NEXT SIX MONTHS. 3092 02:21:56,000 --> 02:21:58,000 IN THE LAST YEAR AS PART OF THIS 3093 02:21:58,000 --> 02:21:59,320 PLAN, WE'VE HELPED TO LAUNCH A 3094 02:21:59,320 --> 02:22:02,760 GLOBAL ALLIANCE FOR MYOTONIC 3095 02:22:02,760 --> 02:22:04,120 DYSTROPHY AWARENESS AND THIS IS 3096 02:22:04,120 --> 02:22:06,000 ON FIVE CONTINENTS REPRESENTING 3097 02:22:06,000 --> 02:22:08,360 OUR ENTIRE ECOSYSTEM AND A 3098 02:22:08,360 --> 02:22:09,640 PATIENT ADVOCACY ORGANIZATION 3099 02:22:09,640 --> 02:22:11,920 AND RESEARCHES IN ACADEMIA AND 3100 02:22:11,920 --> 02:22:14,640 HOSPITALS AND CLINICS, SCHOOLS, 3101 02:22:14,640 --> 02:22:18,760 BIOTECH AND PHARMA AND WE WORK 3102 02:22:18,760 --> 02:22:23,680 TO DESIGNATE SEPTEMBER 15th AS 3103 02:22:23,680 --> 02:22:25,480 INTERNATIONAL MYOTONICEL 3104 02:22:25,480 --> 02:22:26,080 DYSTROPHY AWARENESS DAY. 3105 02:22:26,080 --> 02:22:28,320 WE HAVE MONTHLY MEET THE DM DRUG 3106 02:22:28,320 --> 02:22:29,760 DEVELOPER SESSIONS AND IT WAS 3107 02:22:29,760 --> 02:22:34,080 CLEAR FROM OUR COMMUNITY THAT 3108 02:22:34,080 --> 02:22:35,520 THEY DIDN'T UNDERSTAND THE DRUG 3109 02:22:35,520 --> 02:22:36,640 DEVELOPMENT PROCESS WAS AND WHAT 3110 02:22:36,640 --> 02:22:37,800 WAS GOING TO HAPPEN EX HOW THEY 3111 02:22:37,800 --> 02:22:39,400 WERE INVOLVED AND THEY WANTED TO 3112 02:22:39,400 --> 02:22:41,080 ASK QUESTIONS. 3113 02:22:41,080 --> 02:22:47,160 SO, OUR INDUSTRY PARTNERS HAVE 3114 02:22:47,160 --> 02:22:54,160 AGREED TO AND IT'S BEEN VERY, 3115 02:22:54,160 --> 02:22:55,240 VERY SUCCESSFUL AND APPRECIATED 3116 02:22:55,240 --> 02:22:56,880 BY THE COMMUNITY. 3117 02:22:56,880 --> 02:22:58,120 WE'RE ALSO HELPING TO FUND 3118 02:22:58,120 --> 02:23:00,120 FURTHER EXPANSION OF OUR 3119 02:23:00,120 --> 02:23:02,000 MYOTONIC CLINICAL RESEARCH 3120 02:23:02,000 --> 02:23:02,440 NETWORK. 3121 02:23:02,440 --> 02:23:04,480 WE'RE NOW MAINTAINING THE PUBLIC 3122 02:23:04,480 --> 02:23:07,440 VERSION OF THE DM DRUG 3123 02:23:07,440 --> 02:23:09,520 DEVELOPMENT PIPELINE. 3124 02:23:09,520 --> 02:23:12,520 WE SERVE AS A LIAISON BETWEEN 3125 02:23:12,520 --> 02:23:13,840 INDUSTRY AND THE DM COMMUNITY 3126 02:23:13,840 --> 02:23:15,360 AND THIS IS NOW OUR SECOND YEAR 3127 02:23:15,360 --> 02:23:19,400 OF PROVIDING EARLY CAREER 3128 02:23:19,400 --> 02:23:21,640 RESEARCH WHICH ARE 200,000 3129 02:23:21,640 --> 02:23:23,400 GRANTS WHICH WE HAVE NOT DONE 3130 02:23:23,400 --> 02:23:26,960 PRIOR TO THIS STRATEGIC PLAN. 3131 02:23:26,960 --> 02:23:27,880 ADDITIONALLY, WE HAVE EXPANDED 3132 02:23:27,880 --> 02:23:30,920 THE FELLOWSHIP PROGRAM. 3133 02:23:30,920 --> 02:23:34,800 WE NOW HAVE 53 RESEARCH FELLOWS 3134 02:23:34,800 --> 02:23:36,480 SINCE 2009 AND THAT COVERS 19 3135 02:23:36,480 --> 02:23:38,520 INSTITUTIONS AND FIVE COUNTRIES 3136 02:23:38,520 --> 02:23:41,200 AND ABOUT 90% STAY IN THE FIELD 3137 02:23:41,200 --> 02:23:43,400 OF MYOTONIC DYSTROPHY AND DATA 3138 02:23:43,400 --> 02:23:44,800 FROM THE FELLOWSHIP PROGRAMS 3139 02:23:44,800 --> 02:23:47,960 HAVE BEEN USED TO LEVERAGE 3140 02:23:47,960 --> 02:23:50,120 LARGER FEDERAL AND OTHER GRANTS, 3141 02:23:50,120 --> 02:23:52,480 OFTEN PILOT PROGRAMS THAT SORT 3142 02:23:52,480 --> 02:23:54,720 OF JUST LAUNCH INTO THESE HUGE 3143 02:23:54,720 --> 02:23:56,040 MANY GREAT FEDERAL GRANTS WHICH 3144 02:23:56,040 --> 02:24:01,640 HAVE BEEN REALLY FANTASTIC SEE. 3145 02:24:01,640 --> 02:24:03,160 WE HAVE ADDITIONAL LANGUAGES AS 3146 02:24:03,160 --> 02:24:07,480 WE HAVE CLEARLY LEARNED FROM OUR 3147 02:24:07,480 --> 02:24:10,320 COMMUNITY THAT THERE'S NOT A LOT 3148 02:24:10,320 --> 02:24:12,560 OUT THERE AND NOT IN ENGLISH AND 3149 02:24:12,560 --> 02:24:14,520 WE ARE DEVELOPING NEW PATIENT 3150 02:24:14,520 --> 02:24:24,800 FACING RESOURCES. 3151 02:24:26,760 --> 02:24:28,320 EARLY TESTING AND DIAGNOSTIC 3152 02:24:28,320 --> 02:24:29,280 INITIATIVES. 3153 02:24:29,280 --> 02:24:31,280 WE HAVE RESEARCH IN PROGRAM 3154 02:24:31,280 --> 02:24:32,920 INVESTMENTS AND DECISIONS THAT 3155 02:24:32,920 --> 02:24:34,200 ARE MADE IN THE NEXT COUPLE OF 3156 02:24:34,200 --> 02:24:36,640 MONTHS AND WE ARE HOPING THAT 3157 02:24:36,640 --> 02:24:38,360 THE RESEARCH LANDSCAPE ANALYSIS 3158 02:24:38,360 --> 02:24:40,600 WILL HELP US TO MAKE SOME MORE 3159 02:24:40,600 --> 02:24:44,720 OF THOSE DECISIONS. 3160 02:24:44,720 --> 02:24:48,120 THAT'S IT. 3161 02:24:48,120 --> 02:24:52,360 THANK YOU. 3162 02:24:52,360 --> 02:24:54,720 THANK YOU Dr. STEVENSON. 3163 02:24:54,720 --> 02:24:56,880 AGAIN, ANOTHER VERY CLEAR AND 3164 02:24:56,880 --> 02:24:58,400 WELL ORGANIZED PRESENTATION. 3165 02:24:58,400 --> 02:25:08,960 DO WE HAVE QUESTIONS FOR TANYA? 3166 02:25:11,840 --> 02:25:13,280 >>THANK YOU MANY OF THAT WAS A 3167 02:25:13,280 --> 02:25:13,680 NICE PRESENTATION. 3168 02:25:13,680 --> 02:25:17,160 I WAS CURIOUS ABOUT THE MYOTONIC 3169 02:25:17,160 --> 02:25:18,600 DYSTROPHY RESEARCH MAP THAT YOU 3170 02:25:18,600 --> 02:25:21,280 WERE DESCRIBING WITH 68,000 DATA 3171 02:25:21,280 --> 02:25:21,760 POINTS. 3172 02:25:21,760 --> 02:25:23,520 CAN YOU GIVE SOME EXAMPLES OF 3173 02:25:23,520 --> 02:25:26,240 WHAT ARE THE DATA POINTS AND 3174 02:25:26,240 --> 02:25:28,960 POSSIBLE USES OF THAT MAP? 3175 02:25:28,960 --> 02:25:30,840 >>YES. 3176 02:25:30,840 --> 02:25:36,000 SO, THE DATA POINTS LOOK AT 3177 02:25:36,000 --> 02:25:38,240 EVERY PUBLICATION THAT IS OUT 3178 02:25:38,240 --> 02:25:40,400 THERE AND HOW IT CONNECTS TO WHO 3179 02:25:40,400 --> 02:25:42,160 THE RESEARCHERS AND THE AUTHORS 3180 02:25:42,160 --> 02:25:44,080 ARE AND WHERE THEIR INSTITUTIONS 3181 02:25:44,080 --> 02:25:46,080 ARE AND WHAT KIND OF STUDIES ARE 3182 02:25:46,080 --> 02:25:47,600 BEING DONE AND HOW THAT MIGHT OR 3183 02:25:47,600 --> 02:25:49,720 MIGHT NOT CONNECT TO ALL OF THE 3184 02:25:49,720 --> 02:25:51,360 DRUGS THAT ARE IN DEVELOPMENT, 3185 02:25:51,360 --> 02:25:52,800 THE DIFFERENT COMPANIES THAT ARE 3186 02:25:52,800 --> 02:25:55,800 DEVELOPING THOSE DRUGS AND IT'S 3187 02:25:55,800 --> 02:25:58,240 THIS FANTASTIC INTERACTIVE MAP 3188 02:25:58,240 --> 02:26:00,320 WHERE YOU CAN SEE WHAT THOSE 3189 02:26:00,320 --> 02:26:01,360 CONNECTIONS ARE ALL AROUND THE 3190 02:26:01,360 --> 02:26:03,600 GLOBE SO IF YOU REALLY WANT TO 3191 02:26:03,600 --> 02:26:09,480 SEE WHO IS FOCUSED ON MYOTONIC 3192 02:26:09,480 --> 02:26:10,920 TYPE ONE TO SEE WHO IS INVOLVED 3193 02:26:10,920 --> 02:26:13,520 IN PUBLICATIONS AND HOW THEY'RE 3194 02:26:13,520 --> 02:26:14,000 CONNECTED. 3195 02:26:14,000 --> 02:26:17,480 IT'S A PRETTY PHENOMENAL MAP AND 3196 02:26:17,480 --> 02:26:19,480 I'M HAPPY TO SHARE THE LINK AND 3197 02:26:19,480 --> 02:26:21,480 I'M SORRY I DIDN'T THINK TO 3198 02:26:21,480 --> 02:26:23,080 SHARE THE LINK BUT IT'S ON OUR 3199 02:26:23,080 --> 02:26:23,560 WEBSITE. 3200 02:26:23,560 --> 02:26:25,720 I WOULD BE HAPPY TO SEND THAT 3201 02:26:25,720 --> 02:26:26,480 OVER. 3202 02:26:26,480 --> 02:26:28,120 >>Diana Bianchi: I WONDER IF 3203 02:26:28,120 --> 02:26:31,840 NEXT YEAR WE'LL BE SHOWING SOME 3204 02:26:31,840 --> 02:26:36,360 GPT4 VERSIONS OF EVERYTHING OUT 3205 02:26:36,360 --> 02:26:36,640 THERE. 3206 02:26:36,640 --> 02:26:45,080 IT WAS AMAZING. 3207 02:26:45,080 --> 02:26:47,720 SO ERIC LANG HAS PUT UP THE MAP 3208 02:26:47,720 --> 02:26:48,920 AND WE CAN CLICK ON IT AND IN 3209 02:26:48,920 --> 02:26:52,720 THE MEANTIME, IF THERE ARE NO 3210 02:26:52,720 --> 02:26:53,600 OTHER QUESTIONS WE'LL MOVE TO 3211 02:26:53,600 --> 02:26:56,920 THE THIRD AND LAST PRESENTER WHO 3212 02:26:56,920 --> 02:27:03,440 IS MARIELENA MCGWIRE 3213 02:27:03,440 --> 02:27:04,760 REPRESENTING THE CONGRESSLY 3214 02:27:04,760 --> 02:27:05,920 DIRECTED MEDICAL RESEARCH 3215 02:27:05,920 --> 02:27:08,160 PROGRAM OF THE U.S. ARMY MEDICAL 3216 02:27:08,160 --> 02:27:09,360 RESEARCH AND DEVELOPMENT 3217 02:27:09,360 --> 02:27:09,960 COMMAND. 3218 02:27:09,960 --> 02:27:13,200 THAT'S Dr. MCGWIRE. 3219 02:27:13,200 --> 02:27:16,440 SO, OVER TO YOU. 3220 02:27:16,440 --> 02:27:19,520 >>THANK YOU, VERY MUCH, Dr. 3221 02:27:19,520 --> 02:27:20,240 BIANCHI. 3222 02:27:20,240 --> 02:27:22,400 AND I WANT TO THANK THE 3223 02:27:22,400 --> 02:27:25,080 COMMITTEE FOR THE OPPORTUNITY TO 3224 02:27:25,080 --> 02:27:25,840 PRESENT TODAY. 3225 02:27:25,840 --> 02:27:28,040 IT'S BEEN A WHILE SINCE WE'VE 3226 02:27:28,040 --> 02:27:29,280 HAD AN OPPORTUNITY OR I'VE HAD 3227 02:27:29,280 --> 02:27:33,760 AN OPPORTUNITY TO PRESENT. 3228 02:27:33,760 --> 02:27:35,920 JUST FOR FUTURE REFERENCE, THE 3229 02:27:35,920 --> 02:27:37,520 CONGRESSLY DIRECTED MEDICAL 3230 02:27:37,520 --> 02:27:39,800 RESEARCH PROGRAMS, I'LL ABOUT BY 3231 02:27:39,800 --> 02:27:45,280 AT ACRONYM, CCDMDRP AND I'LL 3232 02:27:45,280 --> 02:27:50,400 TELL YOU THE PROGRAM UNDER THE 3233 02:27:50,400 --> 02:28:00,520 CDMRP. 3234 02:28:03,040 --> 02:28:13,120 SIDDIQUI 3235 02:28:15,920 --> 02:28:18,120 >>BEFORE I START TALKING ABOUT 3236 02:28:18,120 --> 02:28:23,400 THE DESCHENN, SPECIFICALLY ABOUT 3237 02:28:23,400 --> 02:28:28,720 OUR DESCHENN PROGRAM, SO CDMRP 3238 02:28:28,720 --> 02:28:30,320 IS TRANSFORMING HEALTHCARE 3239 02:28:30,320 --> 02:28:34,000 THROUGH INNOVATIVE AND IMPACTFUL 3240 02:28:34,000 --> 02:28:36,400 RESEARCH. 3241 02:28:36,400 --> 02:28:38,280 CDMRP STARTED BY CONGRESS ALL 3242 02:28:38,280 --> 02:28:42,720 THE WAY BACK IN 1992 WHEN BREAST 3243 02:28:42,720 --> 02:28:43,800 CANCER ADVOCATES PUSHED FOR MORE 3244 02:28:43,800 --> 02:28:46,120 FUNDING TO SUPPORT SPECIFICALLY 3245 02:28:46,120 --> 02:28:48,160 BREAST CANCER RESEARCH. 3246 02:28:48,160 --> 02:28:50,000 SINCE THAT PROGRAM WAS INITIATED 3247 02:28:50,000 --> 02:28:53,200 AND IT WAS VERY SUCCESSFUL 3248 02:28:53,200 --> 02:28:54,960 PROGRAM, CDMRP HAS ACTUALLY 3249 02:28:54,960 --> 02:28:59,640 GROWN TO NOW SUPPORT 35 TARGETED 3250 02:28:59,640 --> 02:29:01,000 PROGRAMS COVERING A WIDE ARRAY 3251 02:29:01,000 --> 02:29:04,520 OF BIOMEDICAL RESEARCH AREAS. 3252 02:29:04,520 --> 02:29:06,520 SO, WHAT ARE WE FUNDING? 3253 02:29:06,520 --> 02:29:07,680 WELL WE FUND ALL OF OUR PROGRAMS 3254 02:29:07,680 --> 02:29:10,080 ARE REALLY FOCUSED ON FUNDING 3255 02:29:10,080 --> 02:29:12,520 INNOVATIVE AND IMPACTFUL 3256 02:29:12,520 --> 02:29:15,920 BIOMEDICAL RESEARCH. 3257 02:29:15,920 --> 02:29:17,680 WE DO THAT IN TERMS OF THAT WE 3258 02:29:17,680 --> 02:29:20,160 WILL FUND RESEARCH ALL OVER THE 3259 02:29:20,160 --> 02:29:22,560 WORLD AND IN FACT IN THE PAST 3260 02:29:22,560 --> 02:29:24,880 THREE YEARS, WE HAVE FUNDED 3261 02:29:24,880 --> 02:29:26,360 RESEARCH IN 23 OTHER COUNTRIES 3262 02:29:26,360 --> 02:29:29,080 OUTSIDE OF THE UNITED STATES. 3263 02:29:29,080 --> 02:29:32,160 AND WE DO THIS USING IN TERMS OF 3264 02:29:32,160 --> 02:29:35,240 HOW WE CARRY OUT THIS WORK IS WE 3265 02:29:35,240 --> 02:29:36,200 USE A TWO-TIER REVIEW PROCESS 3266 02:29:36,200 --> 02:29:41,120 FOR THE REVIEW OF THE PROPOSALS, 3267 02:29:41,120 --> 02:29:43,400 APPLICATIONS AND THIS TWO-TIER 3268 02:29:43,400 --> 02:29:45,600 REVIEW PROCESS INVOLVES NOT ONLY 3269 02:29:45,600 --> 02:29:47,960 SCIENTIST AND CLINICIANS BUT 3270 02:29:47,960 --> 02:29:50,400 ALSO INVOLVES CONSUMERS. 3271 02:29:50,400 --> 02:29:53,560 THE INDIVIDUALS, PATIENTS, 3272 02:29:53,560 --> 02:29:55,560 SURVIVORS, PARENTS, CAREGIVERS, 3273 02:29:55,560 --> 02:29:57,640 AND THE LIKE WHO ARE ACTUALLY 3274 02:29:57,640 --> 02:29:59,800 LIVING OR WORKING WITH 3275 02:29:59,800 --> 02:30:01,240 INDIVIDUALS WITH THE PARTICULAR 3276 02:30:01,240 --> 02:30:04,760 DISEASE OR CONDITION OR INJURY. 3277 02:30:04,760 --> 02:30:07,200 AND BEHIND ALL OF THIS RESEARCH, 3278 02:30:07,200 --> 02:30:12,040 OUR GOAL IS TO REALLY ACCELERATE 3279 02:30:12,040 --> 02:30:13,000 RESEARCH SOL WE CAN MOVE IT 3280 02:30:13,000 --> 02:30:15,280 FASTER TO ACHIEVE CURES AND 3281 02:30:15,280 --> 02:30:18,400 BREAKTHROUGHS THAT WILL IMPROVE 3282 02:30:18,400 --> 02:30:22,120 LIVES. 3283 02:30:22,120 --> 02:30:25,960 SO, MOVING ON THERE ARE KEY 3284 02:30:25,960 --> 02:30:29,640 HALLMARKS ABOUT CDMRP THAT I'D 3285 02:30:29,640 --> 02:30:31,200 LIKE TO TOUCH UPON. 3286 02:30:31,200 --> 02:30:33,720 THE FIRST IS THAT CONGRESS ADDS 3287 02:30:33,720 --> 02:30:36,800 TARGETED RESEARCH FUNDS TO THE 3288 02:30:36,800 --> 02:30:37,640 DOD BUDGET. 3289 02:30:37,640 --> 02:30:38,920 THE MONEY FOR OUR PROGRAMS ARE 3290 02:30:38,920 --> 02:30:42,280 NOT PART OF THE PRESIDENT'S 3291 02:30:42,280 --> 02:30:42,640 BUDGET. 3292 02:30:42,640 --> 02:30:44,000 THEY'RE ADDED ON AFTERWARDS WHEN 3293 02:30:44,000 --> 02:30:46,880 THE BUDGET IS SENT TO CONGRESS 3294 02:30:46,880 --> 02:30:51,880 AND THE DOD WE DO NOT ASK FOR O 3295 02:30:51,880 --> 02:30:53,040 WHY THE FUNDS NOR DO THE FUNDS 3296 02:30:53,040 --> 02:30:55,320 THAT GO TO SUPPORT ALL OF THESE 3297 02:30:55,320 --> 02:30:59,760 PROGRAMS THAT THEY DON'T 3298 02:30:59,760 --> 02:31:01,640 TAKEAWAY FROM ANY OTHER DoD 3299 02:31:01,640 --> 02:31:02,440 RESEARCH AREAS. 3300 02:31:02,440 --> 02:31:08,000 CONGRESS AND ADDING THESE FUNDS 3301 02:31:08,000 --> 02:31:09,200 SPECIFIES THE AMOUNT AND 3302 02:31:09,200 --> 02:31:10,200 PROVIDES DIRECTION IF THERE'S 3303 02:31:10,200 --> 02:31:11,720 ANY SPECIFIC FOCUS OR INTENT 3304 02:31:11,720 --> 02:31:13,760 THAT THEY WOULD LIKE TO SEE WITH 3305 02:31:13,760 --> 02:31:18,800 THE EXPENDITUTHE EXPENDITURE OF. 3306 02:31:18,800 --> 02:31:21,000 WE USE A TWO-TIER PROCESS 3307 02:31:21,000 --> 02:31:22,240 RECOMMENDED BY THE NATIONAL 3308 02:31:22,240 --> 02:31:26,960 ACADEMY OF MEDICINE AND PART OF 3309 02:31:26,960 --> 02:31:31,560 THIS WAS RECOMMENDATIONS TO FOR 3310 02:31:31,560 --> 02:31:33,840 CONSUMERS WHO ARE REALLY THE 3311 02:31:33,840 --> 02:31:37,440 TRUE NORTH FOR OUR PROGRAMS. 3312 02:31:37,440 --> 02:31:40,320 THEY PARTICIPATE THROUGHOUT THE 3313 02:31:40,320 --> 02:31:41,480 REVIEW PROCESS AS WELL AS BEING 3314 02:31:41,480 --> 02:31:43,640 INVOLVED WHEN PROGRAMS ARE 3315 02:31:43,640 --> 02:31:45,040 DETERMINING WHAT THEIR GOALS ARE 3316 02:31:45,040 --> 02:31:47,440 GOING TO BE AND FOCUS AREAS AND 3317 02:31:47,440 --> 02:31:50,000 INVESTMENTS STRATEGIES SO, THEY 3318 02:31:50,000 --> 02:31:51,640 REALLY ARE CONSUMERS, 3319 02:31:51,640 --> 02:31:53,920 PARTICIPATION IS REALLY SORT OF 3320 02:31:53,920 --> 02:31:56,560 A FOUNDATION BEDROCK TO OUR 3321 02:31:56,560 --> 02:31:57,280 PROGRAMS. 3322 02:31:57,280 --> 02:31:59,320 ALSO, I WOULD SAY A UNIQUE 3323 02:31:59,320 --> 02:32:01,240 BENEFIT TO THE TYPES OF 3324 02:32:01,240 --> 02:32:02,200 APPROPRIATIONS THAT WE RECEIVED 3325 02:32:02,200 --> 02:32:06,760 TO SUPPORT THESE PROGRAMS IS IT 3326 02:32:06,760 --> 02:32:08,760 ALLOWS FOR US TO RESPOND VERY 3327 02:32:08,760 --> 02:32:10,600 QUICKLY TO THE CHANGING NEEDS IN 3328 02:32:10,600 --> 02:32:13,080 A PARTICULAR FIELD. 3329 02:32:13,080 --> 02:32:14,720 ALTHOUGH ALL OF OUR PROGRAMS 3330 02:32:14,720 --> 02:32:21,080 WILL SET LONGER TERM GOALS, WE 3331 02:32:21,080 --> 02:32:25,800 DO EVALUATE VAL ANNUALLY, EVERY, 3332 02:32:25,800 --> 02:32:27,840 WHAT IS HAPPENING AND CAN ADJUST 3333 02:32:27,840 --> 02:32:31,680 OUR INVESTMENT STRATEGIES BASED 3334 02:32:31,680 --> 02:32:36,480 ONTO THE CURRENTS SITUATIONS AND 3335 02:32:36,480 --> 02:32:37,080 FOR DUCHENE MUSCULAR DYSTROPHY 3336 02:32:37,080 --> 02:32:39,480 PROGRAM IT START INSIDE FISCAL 3337 02:32:39,480 --> 02:32:41,360 YEAR 201 AND YOU CAN SEE IN THE 3338 02:32:41,360 --> 02:32:43,560 BAR GRAPH THE APPROPRIATIONS THE 3339 02:32:43,560 --> 02:32:45,200 PROGRAM HAS RECEIVED OVER THE 3340 02:32:45,200 --> 02:32:51,200 YEARS AND WE SAW A TRIPLING OF 3341 02:32:51,200 --> 02:32:54,720 OUR FUFUNDS FROM $3 MILLION TO 3342 02:32:54,720 --> 02:32:56,280 $10 MILLION IN FM20 THAT MADE A 3343 02:32:56,280 --> 02:32:59,680 DIFFERENCE IN TERMS OF THE 3344 02:32:59,680 --> 02:33:00,720 NUMBER AVENUE WARDS AND THE 3345 02:33:00,720 --> 02:33:04,960 TYPES OF AWARDS THAT ARE ABLE TO 3346 02:33:04,960 --> 02:33:07,600 OFFER AND SO, CDMRP, WE'VE 3347 02:33:07,600 --> 02:33:10,240 DEVELOPED AND USED A COMMON 3348 02:33:10,240 --> 02:33:11,560 PROGRAM MANAGEMENT APPROACH FOR 3349 02:33:11,560 --> 02:33:15,200 ALL OF OUR PROGRAMS AND HOWEVER 3350 02:33:15,200 --> 02:33:17,800 EACH PROGRAM WILL DEVELOP IT'S 3351 02:33:17,800 --> 02:33:19,960 OWN TAILORED GOALS AND UNIQUE 3352 02:33:19,960 --> 02:33:21,600 VISION BASED ON WHAT IS NEEDED 3353 02:33:21,600 --> 02:33:24,000 IN THE RESEARCH FIELD. 3354 02:33:24,000 --> 02:33:25,640 SO, WHEN THE CYCLE BEGINS IN 3355 02:33:25,640 --> 02:33:29,160 TERMS OF RECEIVING AN 3356 02:33:29,160 --> 02:33:30,840 APPROPRIATION, THE PROGRAM AND 3357 02:33:30,840 --> 02:33:32,280 SOMETIMES ARE MORE ESTABLISHED 3358 02:33:32,280 --> 02:33:33,960 PROGRAMS, WILL HOLD OF A 3359 02:33:33,960 --> 02:33:36,560 STAKEHOLDERS MEETING TO GATHER 3360 02:33:36,560 --> 02:33:39,520 INPUT FROM BOTH THE RESEARCH AND 3361 02:33:39,520 --> 02:33:42,480 ADVOCACY CONSUMER COMMUNITY AND 3362 02:33:42,480 --> 02:33:47,360 DETERMINE WHAT IS NEEDED OUT IN 3363 02:33:47,360 --> 02:33:49,960 THE FIELD AND WHAT ARE THE 3364 02:33:49,960 --> 02:33:50,960 RECOMMENDATIONS FOR ESTABLISHING 3365 02:33:50,960 --> 02:33:55,600 THE RESEARCH PROGRAM. 3366 02:33:55,600 --> 02:33:57,840 SO, FOR DUCHENES BACK IN 2011, 3367 02:33:57,840 --> 02:34:00,840 WE HELD A STAKEHOLDERS' MEETING 3368 02:34:00,840 --> 02:34:07,080 AND FOR THAT MEETING, WE INVITED 3369 02:34:07,080 --> 02:34:08,040 REPRESENTATIVES FROM GOVERNMENT 3370 02:34:08,040 --> 02:34:13,120 FUNDING AGENCIES AND THE CDC AND 3371 02:34:13,120 --> 02:34:17,320 NINDS WELLS NIAMS AND WE ALSO 3372 02:34:17,320 --> 02:34:20,400 INVITED ACADEMIC CLINICAL 3373 02:34:20,400 --> 02:34:23,520 RESEARCHERS IN THE FIELD OF 3374 02:34:23,520 --> 02:34:29,920 DUCHENE'S RESEARCH AND ALSO 3375 02:34:29,920 --> 02:34:32,400 REPRESENTATIVES AND YOU CAN SEE 3376 02:34:32,400 --> 02:34:34,280 THE ORGANIZATIONS THAT WERE 3377 02:34:34,280 --> 02:34:37,920 REPRESENTED AT THAT MEETING AND 3378 02:34:37,920 --> 02:34:42,480 WE ALSO FELT IT WAS IMPORTANT TO 3379 02:34:42,480 --> 02:34:43,880 INCLUDE REPRESENTATION FROM 3380 02:34:43,880 --> 02:34:45,160 INDUSTRY AND WE HAD ONE 3381 02:34:45,160 --> 02:34:46,320 INDIVIDUAL FROM INDUSTRY ATTEND 3382 02:34:46,320 --> 02:34:48,320 A STAKEHOLDERS MEETING SO OUR 3383 02:34:48,320 --> 02:34:51,160 TAKE HOLDERS MEETING, WE HAD 3384 02:34:51,160 --> 02:34:53,560 SENT OUT AN RFI TO THE 3385 02:34:53,560 --> 02:34:55,400 PARTICIPANTS AND ASKING THEM TO 3386 02:34:55,400 --> 02:34:58,400 IDENTIFY WHAT THEY SAW AS GAPS 3387 02:34:58,400 --> 02:35:03,080 IN BASIC RESEARCH AS WELL AS 3388 02:35:03,080 --> 02:35:05,040 THERAPEUTIC DEVELOPMENT AND JUST 3389 02:35:05,040 --> 02:35:06,840 TRANSLATIONAL RESEARCH IN 3390 02:35:06,840 --> 02:35:07,600 GENERAL. 3391 02:35:07,600 --> 02:35:09,840 WE ALSO ASKED THE STAKEHOLDERS 3392 02:35:09,840 --> 02:35:13,520 TO IDENTIFY WHAT THEY SAW AS 3393 02:35:13,520 --> 02:35:16,000 OPPORTUNITIES AND THE DMD 3394 02:35:16,000 --> 02:35:19,760 RESEARCH AND PROVIDE 3395 02:35:19,760 --> 02:35:20,920 RECOMMENDATIONS ON PERHAPS OUR 3396 02:35:20,920 --> 02:35:22,360 INVESTMENTS FOCUS FOR TREATING 3397 02:35:22,360 --> 02:35:25,960 AND MANAGING DMD FOR THIS 3398 02:35:25,960 --> 02:35:27,360 PROGRAM. 3399 02:35:27,360 --> 02:35:30,480 SO, THE OUT PUTS FROM THAT 3400 02:35:30,480 --> 02:35:34,000 STAKEHOLDERS' MEETING WERE THEN 3401 02:35:34,000 --> 02:35:35,400 USED TO INFORM THE NEXT STEP 3402 02:35:35,400 --> 02:35:37,280 WHICH IS A VISION SETTING 3403 02:35:37,280 --> 02:35:39,240 MEETING AND WE ACTUALLY HOLD 3404 02:35:39,240 --> 02:35:40,840 57th YEAR THESE MEETING WHEN 3405 02:35:40,840 --> 02:35:42,800 WE RECEIVED A CONGRESSIONAL 3406 02:35:42,800 --> 02:35:43,720 APPROPRIATION, WE'RE GOING TO 3407 02:35:43,720 --> 02:35:49,680 HOLD A VISION SETTING MEETING 3408 02:35:49,680 --> 02:35:57,360 WITH WITH OUR STANDING PANEL AND 3409 02:35:57,360 --> 02:35:58,040 THIS PAP HE WILL WILL ESTABLISH 3410 02:35:58,040 --> 02:35:59,280 THE GOALS FOR THE PROGRAM FOR 3411 02:35:59,280 --> 02:36:01,360 THAT YEAR AND WHAT ARE OUR 3412 02:36:01,360 --> 02:36:04,720 RESEARCH PRIORITIES AND GOING TO 3413 02:36:04,720 --> 02:36:13,600 BE AND DETERMINE WHAT IS THE 3414 02:36:13,600 --> 02:36:14,960 FUNDING STRATEGY AND FOR THE 3415 02:36:14,960 --> 02:36:16,200 PROGRAM THAT YOU ARE PROVIDING 3416 02:36:16,200 --> 02:36:19,760 THOSE RECOMMENDATIONS AND SO THE 3417 02:36:19,760 --> 02:36:24,720 PANEL FOR THE DMDC IS COMPLIES 3418 02:36:24,720 --> 02:36:26,160 AND WE RESEARCHERS AND 3419 02:36:26,160 --> 02:36:29,520 CLINICIANS AND REPRESENTATIONS 3420 02:36:29,520 --> 02:36:30,720 ON INDUSTRY AND CONSUMERS FROM 3421 02:36:30,720 --> 02:36:34,880 THE FIELD OF THE MUSCULAR 3422 02:36:34,880 --> 02:36:37,880 DYSTROPHY AND ALSO FROM OUR 3423 02:36:37,880 --> 02:36:40,320 ADVOCACY GROUPS AND MANY OF 3424 02:36:40,320 --> 02:36:42,120 THESE INDIVIDUALS ACTUALLY 3425 02:36:42,120 --> 02:36:44,320 PARTICIPATED IN THE ORIGINAL 3426 02:36:44,320 --> 02:36:48,120 STAKEHOLDERS MEETING AND SO 3427 02:36:48,120 --> 02:36:49,640 DURING OUR VISION SETTING 3428 02:36:49,640 --> 02:36:54,760 MEETING, ON THE SLIDE, WE TAKE 3429 02:36:54,760 --> 02:36:55,400 INTO CONSIDERATION, WE ALWAYS 3430 02:36:55,400 --> 02:36:56,760 LOOK AND CONSIDER ANY 3431 02:36:56,760 --> 02:36:57,840 CONGRESSIONAL LANGUAGE THAT HAS 3432 02:36:57,840 --> 02:36:59,480 BEEN PROVIDED SO THAT WE MAKE 3433 02:36:59,480 --> 02:37:06,520 SURE WE STAY TRUE TO THE INTENT 3434 02:37:06,520 --> 02:37:16,960 AND WE ALSO WILL AND HOLD 3435 02:37:31,040 --> 02:37:32,160 DISCUSSION TO IDENTIFY OKAY 3436 02:37:32,160 --> 02:37:35,840 WHERE ARE OUR RESEARCH GAPS OUR 3437 02:37:35,840 --> 02:37:39,160 CAPABILITY GAPS AS WELL AS NOT 3438 02:37:39,160 --> 02:37:49,680 ONLY IN FOR THE PROGRAM AND THEN 3439 02:38:01,240 --> 02:38:03,840 ULTIMATELY WHAT WHAT THE 'EM 3440 02:38:03,840 --> 02:38:05,280 PACT IS GOING TO BE AND WITHIN 3441 02:38:05,280 --> 02:38:06,360 OUR PROGRAMS WE WANT TO ACHIEVE 3442 02:38:06,360 --> 02:38:11,000 THE LARGEST IMPACT WITH THE 3443 02:38:11,000 --> 02:38:18,200 FUNDS THAT WE HAVE AVAILABLE AND 3444 02:38:18,200 --> 02:38:20,200 SO WHEN THIS PROGRAM STARTED UP 3445 02:38:20,200 --> 02:38:23,120 THE DIRECTION FOR THE DMDRP HAS 3446 02:38:23,120 --> 02:38:26,520 BEEN TO SUPPORT TRANSLATIONAL 3447 02:38:26,520 --> 02:38:28,640 RESEARCH AND TO FACILITATE 3448 02:38:28,640 --> 02:38:36,360 THERAPEUTICS FOR DMD AND YOU MAY 3449 02:38:36,360 --> 02:38:42,320 REMEMBER WAS AROUND $3 MILLION 3450 02:38:42,320 --> 02:38:44,760 TO INVEST IN RESEARCH SO WE 3451 02:38:44,760 --> 02:38:46,920 WANTED TO BE ABLE TO LEVERAGE 3452 02:38:46,920 --> 02:38:48,960 THOSE FUNDS AND WITH ALL THE 3453 02:38:48,960 --> 02:38:51,400 OTHER GROUPS THAT ARE FUNDING 3454 02:38:51,400 --> 02:38:53,200 RESEARCH AND HAVE OUR BIGGEST 3455 02:38:53,200 --> 02:38:55,440 IMPACT AND IT WAS FELT THAT 3456 02:38:55,440 --> 02:38:57,000 SUPPORTING THERAPEUTIC 3457 02:38:57,000 --> 02:38:58,440 DEVELOPMENTS CERTAINLY IN THAT 3458 02:38:58,440 --> 02:38:59,840 TIMEFRAME WAS AN AREA THAT WE 3459 02:38:59,840 --> 02:39:02,600 COULD HAVE OUR BIGGEST IMPACT, 3460 02:39:02,600 --> 02:39:04,000 PARTICULARLY IF WE WERE LOOKING 3461 02:39:04,000 --> 02:39:14,480 TO TRY AND SUPPORT THAT ARE 3462 02:39:20,160 --> 02:39:27,040 PRECLINICAL AND READINESS AND 3463 02:39:27,040 --> 02:39:30,000 WHAT ARE THE SUPPORT AND IN THAT 3464 02:39:30,000 --> 02:39:31,120 SPACE. 3465 02:39:31,120 --> 02:39:37,120 CURRENTLY, THE STRATEGIC 3466 02:39:37,120 --> 02:39:37,880 PRIORITIES ARE WE HAVE THREE 3467 02:39:37,880 --> 02:39:40,880 POINTS HERE AND THE FIRST IS TO 3468 02:39:40,880 --> 02:39:41,600 ACCELERATE DISCOVERY AND 3469 02:39:41,600 --> 02:39:43,040 DEVELOPMENT OF THERAPEUTICS AND 3470 02:39:43,040 --> 02:39:45,320 ALL LOOKING WITH A PATH INTO 3471 02:39:45,320 --> 02:39:54,160 CLINICAL APPLICATIONS DID SO 3472 02:39:54,160 --> 02:39:56,240 BOTH VERY EARLY AGE ALL THE WAY 3473 02:39:56,240 --> 02:40:01,120 THROUGH THE OLDER AGE AND 3474 02:40:01,120 --> 02:40:02,760 INDIVIDUAL AND OUR SECOND POINT 3475 02:40:02,760 --> 02:40:04,440 IS WE ARE INTERESTED IN THIS IS 3476 02:40:04,440 --> 02:40:05,920 SOMETHING THAT WE HAVE HE CAN 3477 02:40:05,920 --> 02:40:08,640 PANNEDDED INTO MORE RECENTLY AS 3478 02:40:08,640 --> 02:40:09,960 ADVANCED UNDERSTANDING OF THE 3479 02:40:09,960 --> 02:40:12,080 EFFECTIVE DUCHENNE ON NOT ONLY 3480 02:40:12,080 --> 02:40:14,600 ON SKELETAL MUSCLE AND HEART BUT 3481 02:40:14,600 --> 02:40:19,640 THE BONE CENTRAL NERVOUS SYSTEM 3482 02:40:19,640 --> 02:40:20,840 GASTROEN AT THE SCENE TINNAL 3483 02:40:20,840 --> 02:40:24,040 SYSTEM ACROSS THE LIFESPAN OF 3484 02:40:24,040 --> 02:40:24,760 THE MUSCULAR DYSTROPHY AND THEN 3485 02:40:24,760 --> 02:40:26,840 FINALLY ANOTHER AREA THAT WE 3486 02:40:26,840 --> 02:40:36,080 REALLY TRY TO SUPPORT THE MORE 3487 02:40:36,080 --> 02:40:37,200 RESEARCH WE CAN BRING INTO THE 3488 02:40:37,200 --> 02:40:41,560 FIELD WE CAN INCREASE OUR 3489 02:40:41,560 --> 02:40:44,640 OPPORTUNITY OF BRINGING NEW 3490 02:40:44,640 --> 02:40:46,040 IDEAS INTO RESEARCH AND BRING IN 3491 02:40:46,040 --> 02:40:47,400 NEW TREATMENTS AND OUT COME 3492 02:40:47,400 --> 02:40:50,280 MEASURES AND THE LIKE FOR 3493 02:40:50,280 --> 02:40:50,680 DUCHENES. 3494 02:40:50,680 --> 02:40:53,600 TO SUPPORT THESE RESEARCH 3495 02:40:53,600 --> 02:40:55,720 PRIORITIES, WE HAVE I WOULD 3496 02:40:55,720 --> 02:40:58,000 ACTUALLY SAY FIVE MAIN FOCUS 3497 02:40:58,000 --> 02:41:01,320 AREAS AND THE FIRST THAT WE 3498 02:41:01,320 --> 02:41:05,120 STARTED FOUR YEARS AGO, THAT IS 3499 02:41:05,120 --> 02:41:07,800 FOCUSING ON ADDRESSING 3500 02:41:07,800 --> 02:41:09,600 OPPORTUNITIES AND CHALLENGES AND 3501 02:41:09,600 --> 02:41:11,960 DEVELOPING SAFE AND EFFECTIVE 3502 02:41:11,960 --> 02:41:16,440 MACRO MOLECULAR CELLS CELLULAR 3503 02:41:16,440 --> 02:41:18,200 THERAPIES SO I WOULD SAY WE'RE 3504 02:41:18,200 --> 02:41:21,800 LUCKY AT THIS POINT THAT 3505 02:41:21,800 --> 02:41:22,880 DUCHENES THERE ARE TREATMENTS 3506 02:41:22,880 --> 02:41:24,920 OUT THERE IN CLINICAL TRIAL 3507 02:41:24,920 --> 02:41:26,960 TESTING OR ON THE VERGE OF 3508 02:41:26,960 --> 02:41:30,120 MOVING FORWARD INTO CLINICAL 3509 02:41:30,120 --> 02:41:37,240 TRIALS AND WHAT WE'VE LEARNED TO 3510 02:41:37,240 --> 02:41:47,760 DATE AND THE IMMUNE THEY HAVE 3511 02:41:48,440 --> 02:41:50,480 THEIR CHALLENGES OR NEED 3512 02:41:50,480 --> 02:41:52,720 IMPROVEMENTS SO WE'RE STARTING 3513 02:41:52,720 --> 02:41:56,880 ASKING FOR SORT OF NEW IDEAS AND 3514 02:41:56,880 --> 02:41:58,760 INNOVATIVE IDEAS ON HOW TO 3515 02:41:58,760 --> 02:42:03,680 ADDRESS THOSE CHALLENGES FOR 3516 02:42:03,680 --> 02:42:14,120 THERAPIES AND WHEN WE LOOK 3517 02:42:29,320 --> 02:42:31,160 TOWARDS CLINICAL TRIALS LOOKING 3518 02:42:31,160 --> 02:42:34,280 TO SUPPORT IND ENABLING OR 3519 02:42:34,280 --> 02:42:36,000 CLINICAL STUDIES THAT ARE 3520 02:42:36,000 --> 02:42:38,280 DESIGNED TO IMPROVE CARE AND 3521 02:42:38,280 --> 02:42:39,080 QUALITY OF LIFE. 3522 02:42:39,080 --> 02:42:41,800 WE'RE LOOKING AS I MENTIONED 3523 02:42:41,800 --> 02:42:43,440 BEFORE, TO LOOKING AT ASSESSMENT 3524 02:42:43,440 --> 02:42:47,400 OF CLINICAL TRIAL TOOLS AND 3525 02:42:47,400 --> 02:42:48,800 OUTCOMES AND IN PARTICULAR, 3526 02:42:48,800 --> 02:42:53,040 LOOKING WITH A FOCUS ON UNDER 3527 02:42:53,040 --> 02:42:54,240 STUDIED SYSTEMS SO AGAIN 3528 02:42:54,240 --> 02:42:55,920 EXAMPLES LOOKING AT THE 3529 02:42:55,920 --> 02:42:59,080 COGNITIVE CARDIAC OR GI SYSTEMS 3530 02:42:59,080 --> 02:43:00,840 AND AS WELL AS AGE RANGES FROM 3531 02:43:00,840 --> 02:43:04,280 INFANTS, TODDLERS TO OUR NON 3532 02:43:04,280 --> 02:43:07,400 AMBULATORY POPULATION. 3533 02:43:07,400 --> 02:43:09,600 FINALLY OUR MOST RECENT FOCUS 3534 02:43:09,600 --> 02:43:15,160 AREA IS LOOKING TO SUPPORT 3535 02:43:15,160 --> 02:43:16,800 NATIONAL -- NATURAL HISTORY 3536 02:43:16,800 --> 02:43:18,440 STUDIES IN UNDER STUDY SYSTEMS 3537 02:43:18,440 --> 02:43:21,040 AND IN PARTICULAR GOING BACK TO 3538 02:43:21,040 --> 02:43:22,600 THE COGNITIVARD YACK AND G.I. 3539 02:43:22,600 --> 02:43:26,240 SYSTEM AND OR ACROSS THE AGE 3540 02:43:26,240 --> 02:43:29,920 RANGES ALL WITH THE NAME TOWARDS 3541 02:43:29,920 --> 02:43:37,040 CLINICAL TRIAL READINESS SO, OUR 3542 02:43:37,040 --> 02:43:39,120 INVESTMENT STRATEGY TO FOCUS 3543 02:43:39,120 --> 02:43:41,040 AREAS WE HAVE NOW OFFERED TWO 3544 02:43:41,040 --> 02:43:43,400 DIFFERENT FUNDING MECHANISMS AND 3545 02:43:43,400 --> 02:43:47,920 ONE GOING FROM BENCH TO BEDSIDE 3546 02:43:47,920 --> 02:43:49,640 AND WE HAVE AN IDEA DEVELOPMENT 3547 02:43:49,640 --> 02:43:53,080 AWARD AND THE I.D.A. THAT IS 3548 02:43:53,080 --> 02:43:56,960 SPECIFICALLY USED TO ADDRESS THE 3549 02:43:56,960 --> 02:43:58,760 FOCUS AREA ABOUT CHARGES AND 3550 02:43:58,760 --> 02:44:02,880 BEING SAFE AND EFFECTIVE 3551 02:44:02,880 --> 02:44:04,520 THERAPIES SO THIS IS THE AWARD 3552 02:44:04,520 --> 02:44:06,000 MECHANISMS AND WE'RE LOOKING AT 3553 02:44:06,000 --> 02:44:09,080 NEW IDEAS AND INNOVATIVE IDEAS 3554 02:44:09,080 --> 02:44:11,520 THAT ARE TRYING TO LOOK AT AND 3555 02:44:11,520 --> 02:44:13,320 COME UP WITH WAYS TO GET AROUND 3556 02:44:13,320 --> 02:44:15,400 THE CURRENT PROBLEMS THAT WE ARE 3557 02:44:15,400 --> 02:44:17,720 FINDING WITH THE THERAPIES THAT 3558 02:44:17,720 --> 02:44:19,080 ARE OUT THERE. 3559 02:44:19,080 --> 02:44:21,560 WITH THIS MECHANISM, WE ARE ALSO 3560 02:44:21,560 --> 02:44:26,320 TRYING TO TARGET EXPANDING OUR 3561 02:44:26,320 --> 02:44:27,160 RESEARCH COMMUNITY BY OFFERING 3562 02:44:27,160 --> 02:44:31,400 DIFFERENT CATEGORIES AND WE HAVE 3563 02:44:31,400 --> 02:44:35,520 AN EARLY STAGE INVESTIGATOR 3564 02:44:35,520 --> 02:44:37,120 CATEGORY AND A INDIVIDUAL COMING 3565 02:44:37,120 --> 02:44:40,200 FROM ANOTHER FIELD FROM BESIDES 3566 02:44:40,200 --> 02:44:41,040 THE MUSCULAR DYSTROPHIES AND 3567 02:44:41,040 --> 02:44:43,520 BRINGING HIS OR HER SPECIFIC 3568 02:44:43,520 --> 02:44:46,120 DISCIPLINE, TALENTS AND TO APPLY 3569 02:44:46,120 --> 02:44:50,720 AND USE IN STUDYING DUCHENE 3570 02:44:50,720 --> 02:44:51,360 MUSCULAR DYSTROPHY. 3571 02:44:51,360 --> 02:44:53,400 O WE HAVE USED THOSE VARIOUS 3572 02:44:53,400 --> 02:44:54,120 OPTIONS. 3573 02:44:54,120 --> 02:44:56,240 AND WHEN WE REVIEWED THE 3574 02:44:56,240 --> 02:45:02,280 APPLICATIONS, WE REVIEWED AND 3575 02:45:02,280 --> 02:45:03,160 COMPARE IN THOSE STAGES. 3576 02:45:03,160 --> 02:45:04,680 WE DON'T HAVE IT COMPETED 3577 02:45:04,680 --> 02:45:06,880 AGAINST OUR MORE WELL 3578 02:45:06,880 --> 02:45:09,840 ESTABLISHED INVESTIGATORS. 3579 02:45:09,840 --> 02:45:11,880 OUR OTHER MECHANISM THAT WE ZOOS 3580 02:45:11,880 --> 02:45:13,800 A TRANSLATIONAL RESEARCH AWARD 3581 02:45:13,800 --> 02:45:17,080 AND THE TRA MECHANISM AND IT IS 3582 02:45:17,080 --> 02:45:19,400 USED TO ADDRESS ALL OF OUR OTHER 3583 02:45:19,400 --> 02:45:22,880 FOCUS AREAS AND THIS IS A 3584 02:45:22,880 --> 02:45:24,520 MECHANISM THAT AGAIN LOOKING FOR 3585 02:45:24,520 --> 02:45:27,840 MORE ADVANCED SORT OF 3586 02:45:27,840 --> 02:45:29,280 TRANSLATIONAL TYPE RESEARCH 3587 02:45:29,280 --> 02:45:32,000 PROJECTS AND ONE THING THAT WE 3588 02:45:32,000 --> 02:45:34,000 AGAIN ADDED JUST LAST YEAR, 3589 02:45:34,000 --> 02:45:36,240 AGAIN SUPPORTING BRINGING 3590 02:45:36,240 --> 02:45:37,680 RESEARCHERS INTO THE FIELD IS AN 3591 02:45:37,680 --> 02:45:40,320 EARLY CAREER PARTNERING P.I. 3592 02:45:40,320 --> 02:45:42,200 OPTION AND SO THAT ENCOURAGES 3593 02:45:42,200 --> 02:45:46,640 WITH PROVIDING EXTRA FUNDING TO 3594 02:45:46,640 --> 02:45:48,680 HAVE A COLLABORATING P.I. 3595 02:45:48,680 --> 02:45:50,800 PARTNERING P.I. THAT IS IN THE 3596 02:45:50,800 --> 02:45:53,120 EARLY STAGES OF THEIR CAREER 3597 02:45:53,120 --> 02:45:57,080 WITHIN 10 YEARS OF THEIR FIRST 3598 02:45:57,080 --> 02:46:00,840 FACULTY APPOINTMENT AND THAT 3599 02:46:00,840 --> 02:46:03,360 INDIVIDUAL THAT PARTNERING P.I. 3600 02:46:03,360 --> 02:46:05,000 PLAN WILL HAVE THEIR OWN AWARD 3601 02:46:05,000 --> 02:46:07,640 SO AGAIN THAT SUPPORTS TO THE 3602 02:46:07,640 --> 02:46:10,000 ESTABLISHING THEIR CAREER. 3603 02:46:10,000 --> 02:46:11,600 SO THE NEXT TWO SLIDES I WAS 3604 02:46:11,600 --> 02:46:14,440 GOING TO PRESENT JUST SOME BASIC 3605 02:46:14,440 --> 02:46:17,720 INFORMATION ABOUT SOME OF THE 3606 02:46:17,720 --> 02:46:19,760 PROJECTS THAT ARE CURRENTLY IN 3607 02:46:19,760 --> 02:46:21,760 OUR PORTFOLIO WITHIN THESE TWO 3608 02:46:21,760 --> 02:46:23,920 DIFFERENT AWARD CATEGORIES SO, 3609 02:46:23,920 --> 02:46:25,720 LOOKING AT EARLY STAGE RESEARCH 3610 02:46:25,720 --> 02:46:27,720 AND THIS IS THE I.D.A. MECHANISM 3611 02:46:27,720 --> 02:46:29,840 AND LOOKING AT NOVEL TREATMENT 3612 02:46:29,840 --> 02:46:32,200 STRATEGIES AND WE SEE WE HAVE 3613 02:46:32,200 --> 02:46:34,120 FOUR MAIN AREAS THAT WE HAVE 3614 02:46:34,120 --> 02:46:35,400 PROJECTS IN AT THIS POINT AND 3615 02:46:35,400 --> 02:46:45,960 OBVIOUSLY LOOKING AT LOOKING AT 3616 02:46:48,680 --> 02:46:51,080 GENE THERAPY WE HAVE 1 AWARDS 3617 02:46:51,080 --> 02:47:01,600 ADDRESSING VERY NOVEL AND THIS 3618 02:47:03,320 --> 02:47:05,520 CARGO SIZE LIMITATIONS WITH THE 3619 02:47:05,520 --> 02:47:06,920 VECTOR AND LIVER TOXICITY AND AS 3620 02:47:06,920 --> 02:47:11,360 WELL AS MUSCLE UPTAKE AND I HAVE 3621 02:47:11,360 --> 02:47:13,440 ONE EXAMPLE ON THE SLIDE THERE 3622 02:47:13,440 --> 02:47:16,040 THAT AN AWARD THAT IS USING A 3623 02:47:16,040 --> 02:47:21,240 NANO ENGINEERED EXOSOME FOR 3624 02:47:21,240 --> 02:47:24,160 DELIVERING OF DNA OR RNA 3625 02:47:24,160 --> 02:47:31,400 PROTEINS TO REPLENISH THE 3626 02:47:31,400 --> 02:47:35,200 MISSING DYSTROPHINE AND WE HAVE 3627 02:47:35,200 --> 02:47:37,760 AWARDS LOOKING AT GENE EDITING 3628 02:47:37,760 --> 02:47:40,040 TECHNIQUES THAT WOULD 3629 02:47:40,040 --> 02:47:41,440 PERMANENTLY CORRECT THE DISEASE 3630 02:47:41,440 --> 02:47:43,760 CAUSING MUTATIONS. 3631 02:47:43,760 --> 02:47:45,560 AND IN THIS CASE, WE SEE ONE 3632 02:47:45,560 --> 02:47:51,920 WHERE WE'RE USING A PIN POINT 3633 02:47:51,920 --> 02:47:58,200 EDITING SYSTEM WHERE THEY'RE IT 3634 02:47:58,200 --> 02:48:01,760 MODIFYING THE NUCLEOTIDE AND TO 3635 02:48:01,760 --> 02:48:04,280 STIPULATE THE HARBORING TO THE 3636 02:48:04,280 --> 02:48:06,360 DMD MUTATIONS AT THE DNA LEVEL 3637 02:48:06,360 --> 02:48:09,080 AND THE OTHER PROJECT IS 3638 02:48:09,080 --> 02:48:17,440 ACTUALLY USING OUR STUDY USI 3639 02:48:17,440 --> 02:48:19,960 USING -- ACTIVITY ON DOUBLE 3640 02:48:19,960 --> 02:48:25,440 STRANDED RNA TO CORRECT NONSENSE 3641 02:48:25,440 --> 02:48:29,720 MUTATIONS AND ALLOW TRANSLATION 3642 02:48:29,720 --> 02:48:33,720 AND SO EX ON SKIPPING CHALLENGES 3643 02:48:33,720 --> 02:48:38,240 HERE FALLEN TO LOW EFFICACY AND 3644 02:48:38,240 --> 02:48:41,080 SHORT HALF LIFE SO EFFORTS TO 3645 02:48:41,080 --> 02:48:46,720 INCREASE THE MUSCLE TROUPISM AND 3646 02:48:46,720 --> 02:48:48,640 WE HAVE PROJECTS WHICH ARE 3647 02:48:48,640 --> 02:48:56,280 LOOKING AT MODIFICATIONS OF THE 3648 02:48:56,280 --> 02:48:57,160 ANTI-(INAUDIBLE) AS WELL AS 3649 02:48:57,160 --> 02:49:03,000 OTHER PROJECTS THAT ARE LOOKING 3650 02:49:03,000 --> 02:49:12,600 AT USING IN COMBINATIONS WITH 3651 02:49:12,600 --> 02:49:14,400 NUCLEOTIDES TO INCREASE THEIR 3652 02:49:14,400 --> 02:49:15,000 EXPRESSION. 3653 02:49:15,000 --> 02:49:17,080 WHEN IT COMES TO SELL BASED 3654 02:49:17,080 --> 02:49:19,760 THERAPIES, WE HAVE SOME PROJECTS 3655 02:49:19,760 --> 02:49:21,200 LOOKING AT STUDYING AND 3656 02:49:21,200 --> 02:49:24,960 TARGETING MUSCLE STEM CELL 3657 02:49:24,960 --> 02:49:29,600 ACTIVATION OR MACROPHAGE 3658 02:49:29,600 --> 02:49:31,200 TARGETED NUCLEOTIDES AND WE HAVE 3659 02:49:31,200 --> 02:49:39,120 TWO AWARDS THAT ARE LOOKING TO 3660 02:49:39,120 --> 02:49:43,400 BOOST MUSTEL USING BY PIN ONE OR 3661 02:49:43,400 --> 02:49:47,400 BOOSTING MUSCLE STRENGTH WITH 3662 02:49:47,400 --> 02:49:48,560 EROCIN. 3663 02:49:48,560 --> 02:49:51,240 AND NOW JUST MOVE TO OUR OTHER 3664 02:49:51,240 --> 02:49:54,520 AWARD CATEGORY AND WHICH IS THE 3665 02:49:54,520 --> 02:49:58,080 TRANSLATIONAL RESEARCH AWARD AND 3666 02:49:58,080 --> 02:50:00,960 IT'S LOOKING AT PROJECTS AND 3667 02:50:00,960 --> 02:50:02,720 THREE MAIN AREAS THERE'S GETTING 3668 02:50:02,720 --> 02:50:05,160 CLINICAL READINESS AND CLINICAL 3669 02:50:05,160 --> 02:50:07,400 APPLICATIONS AND THEN FINALLYEL 3670 02:50:07,400 --> 02:50:10,000 CLINICAL BIOMARKERS AND WE HAVE 3671 02:50:10,000 --> 02:50:12,480 A NOVEL PROJECT VERY EXCITING 3672 02:50:12,480 --> 02:50:15,200 PROJECT THAT IS USING A NON 3673 02:50:15,200 --> 02:50:22,160 VIRAL GENE THERAPY PLATFORM AND 3674 02:50:22,160 --> 02:50:26,520 THAT FULL LENGTH WITHOUT USING 3675 02:50:26,520 --> 02:50:30,760 THE AAV SECTOR AND WE HAVE TWO 3676 02:50:30,760 --> 02:50:31,840 DIFFERENT PROJECTS THAT ARE 3677 02:50:31,840 --> 02:50:34,000 LOOKING AT ANY FIBROSIS ONE THAT 3678 02:50:34,000 --> 02:50:38,760 MANY OF OF YOU MAY BE FAMILIAR 3679 02:50:38,760 --> 02:50:43,440 WITH AND IS THE LTB4 AND 3680 02:50:43,440 --> 02:50:45,600 SUPPORTED SOME DEVELOPMENT OF 3681 02:50:45,600 --> 02:50:48,840 HUMANIZED ANTIBODIES TO LTBB4 3682 02:50:48,840 --> 02:50:52,840 AND IT'S PROVIDED SOME PROOF OF 3683 02:50:52,840 --> 02:50:56,720 CONCEPT WORK THAT IT IS 3684 02:50:56,720 --> 02:51:00,400 PROVIDING STRONG ANTI-FIBROSIS 3685 02:51:00,400 --> 02:51:02,640 EFFICACY AND THESE PRECLINICAL 3686 02:51:02,640 --> 02:51:05,760 STUDIES HAVE NOW SUPPORTED THE 3687 02:51:05,760 --> 02:51:08,000 INVESTIGATOR Dr. McNALLY 3688 02:51:08,000 --> 02:51:10,720 GETTING NIH FOLLOW ON FUNDING TO 3689 02:51:10,720 --> 02:51:11,920 CONTINUE THIS STUDY HERE IN 3690 02:51:11,920 --> 02:51:14,400 ORDER TO GET THIS ANTIBODY READY 3691 02:51:14,400 --> 02:51:18,160 FOR CLINICAL TRIALS. 3692 02:51:18,160 --> 02:51:20,280 WHEN IT IT COMES TO LOOKING AT 3693 02:51:20,280 --> 02:51:23,480 CLINICAL APPLICATIONS, WE HAVE A 3694 02:51:23,480 --> 02:51:28,520 STUDY THAT IS LOOKING AT 3695 02:51:28,520 --> 02:51:29,120 LOWERING THE STEROID TREATMENT 3696 02:51:29,120 --> 02:51:31,240 DOSE IN COMBINATION WITH 3697 02:51:31,240 --> 02:51:34,160 EXERCISE AND THAT IS BEEN SHOWN 3698 02:51:34,160 --> 02:51:36,240 TO SEE IMPROVED MUSCLE GROWTH 3699 02:51:36,240 --> 02:51:38,960 AND FUNCTION WITH NO OBVIOUS 3700 02:51:38,960 --> 02:51:42,960 SIDE EFFECTS AND WITH USING A 3701 02:51:42,960 --> 02:51:46,680 TWICE A WEEK REGIMENT OF 3702 02:51:46,680 --> 02:51:48,760 STEROIDS AND THEN ANOTHER TWO 3703 02:51:48,760 --> 02:51:56,200 OTHER STUDIES THAT ARE TESTING 3704 02:51:56,200 --> 02:51:57,760 USING REMOTE MONITORING FOR 3705 02:51:57,760 --> 02:51:59,200 MOTOR FUNCTION ASSESSMENT IN 3706 02:51:59,200 --> 02:52:01,120 PATIENTS WITH DMD. 3707 02:52:01,120 --> 02:52:04,080 AND FINALLY, AS WE'VE HEARD FROM 3708 02:52:04,080 --> 02:52:06,360 ALL OF OUR OTHER PRESENTERS, 3709 02:52:06,360 --> 02:52:07,880 THERE'S ALWAYS A NEED FOR 3710 02:52:07,880 --> 02:52:11,720 CLINICAL BIOMARKERS AND 3711 02:52:11,720 --> 02:52:13,040 PARTICULARLY FOR OUR CLINICAL 3712 02:52:13,040 --> 02:52:16,160 TRIALS AND MONITORING NOT SO WE 3713 02:52:16,160 --> 02:52:19,560 HAVE STUDY THAT IS USED BONE 3714 02:52:19,560 --> 02:52:21,240 MRIs AND A BIOMEDICAL MARKER 3715 02:52:21,240 --> 02:52:23,400 AND ANOTHER STUDY THAT IS 3716 02:52:23,400 --> 02:52:25,800 LOOKING AT CARDIA ARRHYTHMIA 3717 02:52:25,800 --> 02:52:28,480 RISK ANALYSIS WITH ECG AND MRI 3718 02:52:28,480 --> 02:52:35,120 AND THEN FINALLY, A EXOSOME RNA 3719 02:52:35,120 --> 02:52:36,560 THAT'S BEING CAPTURED FROM BLOOD 3720 02:52:36,560 --> 02:52:39,040 OR URINE IF PATIENTS WITH 3721 02:52:39,040 --> 02:52:40,480 DUCHENES AND USING IT AS A 3722 02:52:40,480 --> 02:52:43,360 BIOMEDICAL MARKER FOR EVALUATING 3723 02:52:43,360 --> 02:52:45,000 DISEASE SEVERITY AND TREATMENT 3724 02:52:45,000 --> 02:52:47,680 EFFICACY AFTER EX ON SKIPPING 3725 02:52:47,680 --> 02:52:48,440 TREATMENT. 3726 02:52:48,440 --> 02:52:51,080 SO THESE ARE JUST SAMPLING OF 3727 02:52:51,080 --> 02:52:53,960 SOME OF THE PROJECTS WE HAVE IN 3728 02:52:53,960 --> 02:52:55,600 THE PORTFOLIO AND I'M HAPPY TO 3729 02:52:55,600 --> 02:52:57,520 AT THIS POINT ANSWER ANY 3730 02:52:57,520 --> 02:52:59,280 SPECIFIC QUESTIONS YOU MAY HAVE. 3731 02:52:59,280 --> 02:53:01,960 OR JUST JOIN IN THE 3732 02:53:01,960 --> 02:53:02,360 CONVERSATION. 3733 02:53:02,360 --> 02:53:03,840 >>Diana Bianchi: THANK YOU, 3734 02:53:03,840 --> 02:53:07,920 VERY MUCH, MARIELENA. 3735 02:53:07,920 --> 02:53:11,120 IT WAS CERTAINLY NEWS TO ME THAT 3736 02:53:11,120 --> 02:53:12,840 THE CONGRESSIONALLY DIRECTED 3737 02:53:12,840 --> 02:53:14,160 MEDICAL RESEARCH PROGRAMS WERE 3738 02:53:14,160 --> 02:53:16,360 SO INVOLVED AND MOST OF THEIR 3739 02:53:16,360 --> 02:53:19,400 RESEARCH SO THIS WAS A VERY, 3740 02:53:19,400 --> 02:53:20,480 VERY HELPFUL PRESENTATION. 3741 02:53:20,480 --> 02:53:24,880 THE FLOOR IS OPEN NOW FOR 3742 02:53:24,880 --> 02:53:30,480 QUESTIONS. 3743 02:53:30,480 --> 02:53:30,680 GLEN. 3744 02:53:30,680 --> 02:53:31,360 >>Glen Nuckolls: THANK YOU FOR 3745 02:53:31,360 --> 02:53:35,160 A GREAT PRESENTATION. 3746 02:53:35,160 --> 02:53:38,080 SO, AS I RECALL, YOU KNOW, THE 3747 02:53:38,080 --> 02:53:40,800 BUDGET INCREASE IN THE PROGRAM 3748 02:53:40,800 --> 02:53:42,440 IN RECENT YEARS SO I'M CURIOUS 3749 02:53:42,440 --> 02:53:44,880 IF YOU LOOK BACK AT KIND OF THE 3750 02:53:44,880 --> 02:53:46,760 APPLICATIONS THAT WE RECEIVED 3751 02:53:46,760 --> 02:53:49,960 AND THOSE THAT WERE FUNDED, DID 3752 02:53:49,960 --> 02:53:53,080 THAT INCREASE IN BUDGET RESULT 3753 02:53:53,080 --> 02:53:55,400 IN KIND OF DEEPER INTO THE 3754 02:53:55,400 --> 02:53:56,600 TOPICS THAT YOU WERE ALREADY 3755 02:53:56,600 --> 02:53:58,400 WORKING ON OR DID IT RESULT IN 3756 02:53:58,400 --> 02:54:00,640 KIND OF A EXPANSION INTO 3757 02:54:00,640 --> 02:54:03,840 ADDITIONAL TOPICS? 3758 02:54:03,840 --> 02:54:06,240 >>I AM GOING TO SAY IT PROBABLY 3759 02:54:06,240 --> 02:54:07,400 A LITTLE BIT OF BOTH BUT 3760 02:54:07,400 --> 02:54:10,560 CERTAINLY BEING ABLE TO EXPAND 3761 02:54:10,560 --> 02:54:12,320 IN PART BECAUSE WE WERE REALLY 3762 02:54:12,320 --> 02:54:15,640 ABLE TO NOW FUND MORE PROJECTS 3763 02:54:15,640 --> 02:54:16,800 AND THAT WAS ALWAYS, YOU MAY 3764 02:54:16,800 --> 02:54:17,760 REMEMBER, WHEN YOU WERE SERVING 3765 02:54:17,760 --> 02:54:20,960 ON THE PROBLEMATIC PANEL, WE 3766 02:54:20,960 --> 02:54:23,240 OFTEN TIMES HAD A MUCH BIGGER 3767 02:54:23,240 --> 02:54:25,720 LIST OF PROJECTS WE WOULD LIKE 3768 02:54:25,720 --> 02:54:28,120 TO SUPPORT AND THEN THERE'S ONLY 3769 02:54:28,120 --> 02:54:32,720 SO MUCH MONEY AND I THINK IN 3770 02:54:32,720 --> 02:54:35,400 PARTICULAR, BEING ABLE TO START 3771 02:54:35,400 --> 02:54:36,640 FUNDING PROJECTS MUCH EARLIER 3772 02:54:36,640 --> 02:54:39,280 STAGE STILL TRANSLATIONAL FOCUS 3773 02:54:39,280 --> 02:54:42,200 THERAPEUTIC FOCUS BUT THERE'S 3774 02:54:42,200 --> 02:54:43,760 REALLY EARLY KNEW IDEAS THAT 3775 02:54:43,760 --> 02:54:46,120 SOMEONE HAS REALLY TRYING TO GET 3776 02:54:46,120 --> 02:54:51,120 MORE INNOVATION AND COMING UP 3777 02:54:51,120 --> 02:54:53,440 WELCOME BACK HOW DO WE GET 3778 02:54:53,440 --> 02:54:55,120 AROUND THE KNOWN PROBLEMS WHERE 3779 02:54:55,120 --> 02:54:56,880 WE'RE SEEING NOW WITH THE 3780 02:54:56,880 --> 02:54:59,040 VARIOUS TYPES OF TREATMENT THAT 3781 02:54:59,040 --> 02:55:00,360 WE REALLY WERE NOT WORKING IN 3782 02:55:00,360 --> 02:55:01,520 THAT SPACE AT ALL. 3783 02:55:01,520 --> 02:55:03,840 WE'RE JUST GOING TO END OUR WITH 3784 02:55:03,840 --> 02:55:05,520 THE INCREASED FUNDING THE NUMBER 3785 02:55:05,520 --> 02:55:08,440 OF AWARDS THAT ACTUALLY HALF THE 3786 02:55:08,440 --> 02:55:11,040 AWARDS IN THE PORTFOLIO ARE WITH 3787 02:55:11,040 --> 02:55:12,840 THESE IDA AWARDS AND THEY'RE 3788 02:55:12,840 --> 02:55:17,120 SHORT AND THEY'RE TWO YEAR AWARD 3789 02:55:17,120 --> 02:55:18,880 MECHANISM AND MANY OF THE 3790 02:55:18,880 --> 02:55:19,920 PROJECTS ARE SHOWING PROMISING 3791 02:55:19,920 --> 02:55:22,480 IN THAT YOU KNOW, GETTING SOME 3792 02:55:22,480 --> 02:55:24,120 PUBLICATIONS IN AND I THINK 3793 02:55:24,120 --> 02:55:25,720 PARTICULARLY THEY'RE ABLE TO 3794 02:55:25,720 --> 02:55:27,600 ACTUALLY GET FOLLOW ON FUNDING 3795 02:55:27,600 --> 02:55:30,920 MUCH TIMES FROM THE NHL TO 3796 02:55:30,920 --> 02:55:32,120 CONTINUE THOSE PROJECTS THAT ARE 3797 02:55:32,120 --> 02:55:33,680 SHOWING PROMISE WHICH IS REALLY 3798 02:55:33,680 --> 02:55:36,160 WHAT WE HOPE TO DO. 3799 02:55:36,160 --> 02:55:38,400 IT'S LIKE OKAY, GET A GOOD IDEA 3800 02:55:38,400 --> 02:55:41,240 STARTED AND THEN HOPEFULLY THE 3801 02:55:41,240 --> 02:55:42,920 INVESTORS ARE ABLE TO CONTINUE 3802 02:55:42,920 --> 02:55:46,800 BY GETTING FUNDING FROM OTHER 3803 02:55:46,800 --> 02:55:47,960 AGENCIES TOO CONTINUE A 3804 02:55:47,960 --> 02:55:51,480 PROMISING PROJECT. 3805 02:55:51,480 --> 02:55:52,080 >>Glen Nuckolls: THANK YOU. 3806 02:55:52,080 --> 02:55:58,760 >>I HAVE A QUESTION. 3807 02:55:58,760 --> 02:56:03,840 >>THANK YOU FOR THE 3808 02:56:03,840 --> 02:56:05,800 CLARIFICATIONS ON YOUR 3809 02:56:05,800 --> 02:56:07,880 CONGRESSIONAL DIRECTION AND 3810 02:56:07,880 --> 02:56:08,440 FUTURE. 3811 02:56:08,440 --> 02:56:11,160 THANK YOU FOR THE EXCELLENT 3812 02:56:11,160 --> 02:56:12,200 PRESENTATION. 3813 02:56:12,200 --> 02:56:20,920 MY QUESTION IS, ALLOWING 3814 02:56:20,920 --> 02:56:23,400 DISEASES MYOTONIC, DUCHENE, 3815 02:56:23,400 --> 02:56:26,920 THEY'RE ALL STRUGGLING WITH THE 3816 02:56:26,920 --> 02:56:29,880 SAME ACCESS WITH THE SAME 3817 02:56:29,880 --> 02:56:35,880 ISSUES, LIKE TOXICITY, FAILURE 3818 02:56:35,880 --> 02:56:44,880 OF CLINICAL TRIALS AND THE 3819 02:56:44,880 --> 02:56:50,640 STROKE AND DU DUCHENE AND THE 3820 02:56:50,640 --> 02:56:51,640 MYOTONIC AREA. 3821 02:56:51,640 --> 02:56:56,080 THE QUESTION IS ARE THESE 3822 02:56:56,080 --> 02:56:57,720 CONCLUDED OTHER AND EVEN THOUGH 3823 02:56:57,720 --> 02:57:05,160 YOU ARE WORKING ON DUCHENE, AND 3824 02:57:05,160 --> 02:57:13,000 ARE THERE LOOKING AT MYOTONIC IN 3825 02:57:13,000 --> 02:57:19,760 THE CONTEXT OF DUCHENE OR IS 3826 02:57:19,760 --> 02:57:21,240 THAT A POSSIBILITY? 3827 02:57:21,240 --> 02:57:25,640 CAN WE LEARN THINGS? 3828 02:57:25,640 --> 02:57:28,960 SOME BIOMARKERS RATHER THAN 3829 02:57:28,960 --> 02:57:35,600 WORKING IN SILENCE AND AT THIS 3830 02:57:35,600 --> 02:57:39,680 POINT TO CONGRESS AND PROGRAM 3831 02:57:39,680 --> 02:57:44,880 COULD HAVE THE TRIAL THAT DOES 3832 02:57:44,880 --> 02:57:49,080 INCLUDE DUCHENE AND OTHER 3833 02:57:49,080 --> 02:57:52,320 MYOTONIC AND WHAT IT NOW. 3834 02:57:52,320 --> 02:57:57,200 >>THANK YOU, DA, FOR ME THE 3835 02:57:57,200 --> 02:57:57,640 QUESTION. 3836 02:57:57,640 --> 02:58:01,040 YOU NOTED, THAT WE ARE, EACH OF 3837 02:58:01,040 --> 02:58:03,760 OUR PROGRAMS DO HAVE A MANDATE 3838 02:58:03,760 --> 02:58:14,320 OR DIRECTION INTENT AND THAT IS 3839 02:58:17,640 --> 02:58:20,400 PROVIDED AND THAT AND COULD SHOW 3840 02:58:20,400 --> 02:58:27,200 AND COM ANALITY BETWEEN DISEASES 3841 02:58:27,200 --> 02:58:29,440 AND THAT IS WORTH VALUE OF 3842 02:58:29,440 --> 02:58:31,440 STUDIES AND AS LONG AS THE 3843 02:58:31,440 --> 02:58:36,800 PROJECT DOES INCLUDE AND IT HAS 3844 02:58:36,800 --> 02:58:39,360 SIGNIFICANT FOCUS IN DUCHENE IT 3845 02:58:39,360 --> 02:58:42,360 SHOULD BE CONSIDERED FOR THIS 3846 02:58:42,360 --> 02:58:48,080 PROGRAM AND I DO KNOW THAT FOR 3847 02:58:48,080 --> 02:58:50,680 EXAMPLE, IT WAS MENTIONED 3848 02:58:50,680 --> 02:58:52,960 PREVIOUSLY WITH THE MYOTONIC 3849 02:58:52,960 --> 02:58:54,360 ORGANIZATION THAT WE HAVE 3850 02:58:54,360 --> 02:58:55,920 ANOTHER RESEARCH PROGRAM PER 3851 02:58:55,920 --> 02:58:59,400 REVIEW MEDICAL IT'S A MULTI 3852 02:58:59,400 --> 02:59:01,040 TOPIC PROGRAM AND MY DYSTROPHY 3853 02:59:01,040 --> 02:59:04,760 IS A TOPIC AND THAT PROGRAM AND 3854 02:59:04,760 --> 02:59:07,400 WE DO TAKE A LOOK AND THEY DO 3855 02:59:07,400 --> 02:59:08,880 GET FUNDING, THEY DO HAVE A 3856 02:59:08,880 --> 02:59:11,240 GRANTS AWARDED USUALLY EVERY 3857 02:59:11,240 --> 02:59:12,760 YEAR AT LEAST ONE OR TWO GRANTS 3858 02:59:12,760 --> 02:59:15,960 ARE BEING AWARDED AND THERE WAS 3859 02:59:15,960 --> 02:59:17,760 A TOPIC A COUPLE YEARS AGO WAS 3860 02:59:17,760 --> 02:59:27,720 FOCUSED ON CARDIO MY ON AGENT' O 3861 02:59:27,720 --> 02:59:31,560 THERE IS THE ABILITY TO 3862 02:59:31,560 --> 02:59:34,080 POTENTIALLY LOOK AT OTHER 3863 02:59:34,080 --> 02:59:37,880 MUSCULAR DYSTROPHIES BEFORE THE 3864 02:59:37,880 --> 02:59:39,240 DUCHENNE PROGRAM AND THERE NEEDS 3865 02:59:39,240 --> 02:59:41,280 TO BE A STRONG PRO PHONE NOT IN 3866 02:59:41,280 --> 02:59:48,200 THE STUDY THAT WILL ON THE 3867 02:59:48,200 --> 02:59:49,120 MUSCULAR DYSTROPHY FOR IT TO BE 3868 02:59:49,120 --> 02:59:50,080 CONSIDERED AND AGAIN IT'S TIED 3869 02:59:50,080 --> 02:59:52,680 IN WITH THE CONGRESSIONAL INTENT 3870 02:59:52,680 --> 02:59:56,480 THAT GOES WITH EVERY SINGLE 3871 02:59:56,480 --> 03:00:06,640 PROGRAM. 3872 03:00:15,160 --> 03:00:17,560 >>FOLLOWING UP ON COMMENTS FROM 3873 03:00:17,560 --> 03:00:19,040 DAN, I THINK IT'S AN INTERESTING 3874 03:00:19,040 --> 03:00:20,440 POINT. 3875 03:00:20,440 --> 03:00:23,360 BECAUSE, MUCH OF FUNDING AND 3876 03:00:23,360 --> 03:00:25,720 RESEARCHERS FOCUSED ON DISEASE 3877 03:00:25,720 --> 03:00:27,280 AREAS AND IT IT'S VERY 3878 03:00:27,280 --> 03:00:31,920 INTERESTING TO LOOK ACROSS THE 3879 03:00:31,920 --> 03:00:33,880 MUSQUEAM AND IT STRIKES ME THAT 3880 03:00:33,880 --> 03:00:36,520 THE EXPERTISE IN THE FIELD OF 3881 03:00:36,520 --> 03:00:38,400 THE INVESTIGATOR STUDY IN 3882 03:00:38,400 --> 03:00:43,400 DISEASES IT'S REALLY DICTATED BY 3883 03:00:43,400 --> 03:00:46,680 THE UNDERLINING MOLECULAR 3884 03:00:46,680 --> 03:00:48,080 DISEASE AND IT HAS HARD RNA 3885 03:00:48,080 --> 03:00:51,480 PROCESSING EXPERTISE AND THE 3886 03:00:51,480 --> 03:00:53,920 FHSA FIELD HAS EXPERTISE AND THE 3887 03:00:53,920 --> 03:00:55,480 DUCHENE FIELD HAS STRONG 3888 03:00:55,480 --> 03:00:59,080 MEMBRANE BIOLOGY AND OTHER 3889 03:00:59,080 --> 03:01:02,680 RELATED AREAS AND THE 3890 03:01:02,680 --> 03:01:04,680 TECHNOLOGIES AND TOOLS THAT ARE 3891 03:01:04,680 --> 03:01:07,720 REQUIRED TO DEVELOP TREATMENTS 3892 03:01:07,720 --> 03:01:09,920 ARE MUCH MORE FUNDAMENTAL OFTEN 3893 03:01:09,920 --> 03:01:12,320 AND THEY CROSS DISEASE AREAS AND 3894 03:01:12,320 --> 03:01:16,840 SO IT'S SORT OF A BIGGER 3895 03:01:16,840 --> 03:01:19,000 CONVERSATION ABOUT HOW TO 3896 03:01:19,000 --> 03:01:19,480 FACILITATE THAT. 3897 03:01:19,480 --> 03:01:22,320 I MEAN, IT'S A WAY TO THINK 3898 03:01:22,320 --> 03:01:23,800 ABOUT HOW TO SUPPORT RESEARCH 3899 03:01:23,800 --> 03:01:24,720 AND I DON'T KNOW WHAT THE ANSWER 3900 03:01:24,720 --> 03:01:27,320 TO THAT IS S YOU NEED TO DO BOTH 3901 03:01:27,320 --> 03:01:28,920 OBVIOUSLY BUT I THINK IT'S 3902 03:01:28,920 --> 03:01:38,280 REALLY IMPORTANT POINT. 3903 03:01:38,280 --> 03:01:41,360 >>DO YOU WANT TO COMMENT ON 3904 03:01:41,360 --> 03:01:41,880 THAT? 3905 03:01:41,880 --> 03:01:44,520 >>I THINK WHAT I WOULD SAY TO 3906 03:01:44,520 --> 03:01:47,400 SOMETHING LIKE THAT AS YOU POINT 3907 03:01:47,400 --> 03:01:52,240 THE OUT, THE RESEARCH TECHNIQUES 3908 03:01:52,240 --> 03:01:56,960 OR DISCIPLINES THAT ARE NEEDED 3909 03:01:56,960 --> 03:01:58,280 COME FROM DIFFERENT DISEASE 3910 03:01:58,280 --> 03:02:00,360 AREAS IN THIS CASE AND THAT IS 3911 03:02:00,360 --> 03:02:02,320 IN PART WHEN WE, I WOULD SAY, 3912 03:02:02,320 --> 03:02:04,080 THIS PAST YEAR WHEN WE OFFERED 3913 03:02:04,080 --> 03:02:07,360 AND WE'VE ALSO TRIED IN PREVIOUS 3914 03:02:07,360 --> 03:02:09,640 YEARS A WHAT WE'RE CALLING OUR 3915 03:02:09,640 --> 03:02:11,560 TRANSITIONING INVESTIGATORS TO 3916 03:02:11,560 --> 03:02:13,960 ENCOURAGE AND TRY TO BRING INTO 3917 03:02:13,960 --> 03:02:16,400 THE RESEARCH PROGRAMS 3918 03:02:16,400 --> 03:02:18,480 INVESTIGATORS FROM OTHER 3919 03:02:18,480 --> 03:02:20,040 DISCIPLINES THAT HAVE NOT 3920 03:02:20,040 --> 03:02:21,800 TRADITIONALLY WORKING ON DUCHENE 3921 03:02:21,800 --> 03:02:23,960 BUT BRING THAT EXPERTISE AND 3922 03:02:23,960 --> 03:02:25,400 CONSIDER AND BRING THEIR 3923 03:02:25,400 --> 03:02:31,400 EXPERTISE TO STUDY DUCHENES 3924 03:02:31,400 --> 03:02:33,320 QUESTION CHALLENGE AND WHAT NOT 3925 03:02:33,320 --> 03:02:34,880 SO THAT'S WHAT WE HAVE BEEN 3926 03:02:34,880 --> 03:02:38,160 TRYING TO DO WITH THE PAST BY 3927 03:02:38,160 --> 03:02:41,040 OFFERING A MULTI-DISCIPLINARY 3928 03:02:41,040 --> 03:02:42,720 PARTNERSHIP AND THIS 3929 03:02:42,720 --> 03:02:46,200 TRANSITIONING INVESTIGATOR AND I 3930 03:02:46,200 --> 03:02:48,520 WOULD SAY WE WERE ACTUALLY 3931 03:02:48,520 --> 03:02:51,480 RATHER SUCCESSFUL WITH THIS 3932 03:02:51,480 --> 03:02:53,040 TRANSITIONING INVESTIGATOR AND I 3933 03:02:53,040 --> 03:02:54,960 CAN'T SAY A LOT MORE ABOUT IT 3934 03:02:54,960 --> 03:02:58,200 BECAUSE WE HAVEN'T RELEASED OUR 3935 03:02:58,200 --> 03:03:00,440 AWARDEES YET FOR FY '22 AND THEY 3936 03:03:00,440 --> 03:03:02,560 WILL BE COMING OUT SHORTLY AND 3937 03:03:02,560 --> 03:03:05,280 WHAT I CAN SAY IS WE HAD SOME 3938 03:03:05,280 --> 03:03:09,600 SUCCESS AND THAT AREA. 3939 03:03:09,600 --> 03:03:14,680 >>IF I CAN ADD TO ERIC'S POINT, 3940 03:03:14,680 --> 03:03:25,080 THAT I KNOW, I MEAN, OTHER NIH 3941 03:03:25,080 --> 03:03:28,840 AGENCIES HAVE SOME FORMS THAT 3942 03:03:28,840 --> 03:03:30,800 ARE ESPECIALLY FOR THAT TO TO 3943 03:03:30,800 --> 03:03:38,360 CREATE TOOLS THAT CAN BE USED 3944 03:03:38,360 --> 03:03:46,080 FOR DIFFERENT, IN THIS CASE, 3945 03:03:46,080 --> 03:03:50,160 DYSTROPHIES LIKE THE IS PART OF 3946 03:03:50,160 --> 03:03:52,800 THE AND THINGS LIKE THAT. 3947 03:03:52,800 --> 03:03:57,240 IN OUR PATIENT ADVOCACY 3948 03:03:57,240 --> 03:03:58,000 ORGANIZATION WE'VE BEEN TALKING 3949 03:03:58,000 --> 03:04:01,040 FOR A FEW YEARS NOW ABOUT HOW TO 3950 03:04:01,040 --> 03:04:03,040 INCREASE THE NUMBER OF THIS TYPE 3951 03:04:03,040 --> 03:04:06,320 OF PLATFORMS BECAUSE AS HE SAID 3952 03:04:06,320 --> 03:04:09,640 AS I MENTIONED, MANY OF THE 3953 03:04:09,640 --> 03:04:11,680 TOOLS WILL BE AS IMPORTANT OR 3954 03:04:11,680 --> 03:04:17,920 MORE THAN NOT THE MECHANISMS. 3955 03:04:17,920 --> 03:04:20,440 SO IT'S SOMETHING MAYBE TO 3956 03:04:20,440 --> 03:04:28,000 DISCUSS IN THE PLAN FOR 2025. 3957 03:04:28,000 --> 03:04:29,840 HOW TO ACCESS OR HOW TO CREATE 3958 03:04:29,840 --> 03:04:40,280 MORE OF THESE PLATFORMS. 3959 03:04:44,960 --> 03:04:55,480 DISEASE TECHNOLOGIES BECAUSE OF 3960 03:05:09,360 --> 03:05:11,400 IT'S A DISEASE DIAGNOSTIC THAT 3961 03:05:11,400 --> 03:05:15,320 WE HAVE TO WORK TOGETHER AGAIN 3962 03:05:15,320 --> 03:05:19,320 AND JUST EXACTLY AND THE MEETING 3963 03:05:19,320 --> 03:05:22,440 DISEASE CONTEXT AND ONE COULD 3964 03:05:22,440 --> 03:05:32,960 GAIN AN IN SIGHT THAT AND THEN 3965 03:05:34,200 --> 03:05:41,120 VICE VESA AND SO, I JUST SAW THE 3966 03:05:41,120 --> 03:05:45,240 DEFINITION AND SO THANKS. 3967 03:05:45,240 --> 03:05:46,520 >>I DEFINITELY HEARD SOME 3968 03:05:46,520 --> 03:05:49,480 SIMILAR THEMES ACROSS THE THREE 3969 03:05:49,480 --> 03:05:50,680 PRESENTATIONS TODAY. 3970 03:05:50,680 --> 03:05:54,640 SO, ONE THING TO CONSIDER WHEN 3971 03:05:54,640 --> 03:05:55,720 THE STRATEGIC PLAN IS BEING 3972 03:05:55,720 --> 03:05:59,400 WORKED ON IS WHETHER YOU WANT TO 3973 03:05:59,400 --> 03:06:00,840 DEVELOP CROSS CUTTING THEMES 3974 03:06:00,840 --> 03:06:02,000 JUST FOR EXAMPLE BY OWE MARKERS 3975 03:06:02,000 --> 03:06:03,600 AND THE NEED FOR BIOMARKERS 3976 03:06:03,600 --> 03:06:05,520 MIGHT BE A CROSS CUTTING THEME 3977 03:06:05,520 --> 03:06:09,640 FOR AWFUL THE CONDITIONS BEING 3978 03:06:09,640 --> 03:06:13,320 STUDIED AND IT'S SOMETHING TO 3979 03:06:13,320 --> 03:06:18,360 CONSIDER. 3980 03:06:18,360 --> 03:06:20,160 SO, AS WE MENTIONED BEFORE, WE 3981 03:06:20,160 --> 03:06:22,680 PLAN TO CONTINUE THIS SESSION -- 3982 03:06:22,680 --> 03:06:22,920 WALTER. 3983 03:06:22,920 --> 03:06:26,480 DID YOU WANT TO ADD SOMETHING? 3984 03:06:26,480 --> 03:06:31,320 >>JUST NOTE THE PLATFORMS DAN 3985 03:06:31,320 --> 03:06:32,600 WAS TALKING ABOUT IS SOMETHING 3986 03:06:32,600 --> 03:06:34,880 IT KEEP OUR EYE ON IS CALLED THE 3987 03:06:34,880 --> 03:06:37,960 BEE SPOKE GENE THERAPY PROGRAM 3988 03:06:37,960 --> 03:06:39,520 AND IT'S AN ACCELERATED MEDICINE 3989 03:06:39,520 --> 03:06:41,800 PARTNERSHIP WITH INDUSTRY AND 3990 03:06:41,800 --> 03:06:43,680 NIH FOLKS WHO IS ACTUALLY PETER 3991 03:06:43,680 --> 03:06:46,000 MARKS IS ONE OF THE INSTIGATORS 3992 03:06:46,000 --> 03:06:49,240 AND IT'S ACTUALLY TO BUILD A 3993 03:06:49,240 --> 03:06:52,320 GENE THERAPY PLATFORM WHERE THE 3994 03:06:52,320 --> 03:07:00,400 FDA WOULD LIKE IS TO SEE SOME 3995 03:07:00,400 --> 03:07:04,520 TYPE O OF THAT A PLATFORM IS SAE 3996 03:07:04,520 --> 03:07:06,560 AND THE VECTOR AND AV THAT 3997 03:07:06,560 --> 03:07:08,960 THEY'RE DEALING WITH YOU CAN 3998 03:07:08,960 --> 03:07:10,400 JUST PLUG IN DIFFERENT GENE AND 3999 03:07:10,400 --> 03:07:13,520 THE PATHWAY TO APPROVAL WOULD 4000 03:07:13,520 --> 03:07:18,240 THEN BE MUCH MORE STREAM LINED 4001 03:07:18,240 --> 03:07:21,240 SO THAT IS CURRENTLY UNDERWAY 4002 03:07:21,240 --> 03:07:24,960 AND COUPLE PILOT PROJECTS AND IF 4003 03:07:24,960 --> 03:07:27,800 IT WORKS, THEN IT COULD BE 4004 03:07:27,800 --> 03:07:32,240 USEFUL TO MANY DIFFERENT RARE 4005 03:07:32,240 --> 03:07:32,800 DISEASES. 4006 03:07:32,800 --> 03:07:34,480 I'LL PUT THE LINK IN THE CHAT. 4007 03:07:34,480 --> 03:07:37,920 >>Diana Bianchi: THANK YOU, 4008 03:07:37,920 --> 03:07:42,520 WALTER. 4009 03:07:42,520 --> 03:07:46,280 >>THERE A SIMILAR PROGRAM WHERE 4010 03:07:46,280 --> 03:07:49,200 REBUILDING MUSCLE? 4011 03:07:49,200 --> 03:07:51,520 THE GENE THERAPY IN THERE AND 4012 03:07:51,520 --> 03:07:54,760 THEY HAVE ACCESS TO GENE 4013 03:07:54,760 --> 03:07:55,600 THERAPY? 4014 03:07:55,600 --> 03:07:57,440 REMAIN VERY NEUTRAL AND 4015 03:07:57,440 --> 03:07:59,600 CONSIDERING HOW TO REGENERATE 4016 03:07:59,600 --> 03:08:07,320 AND RECREATE, GROW MUSCLE CELLS? 4017 03:08:07,320 --> 03:08:10,720 DO YOU HAVE SIMILAR PROGRAMS IN 4018 03:08:10,720 --> 03:08:11,560 THAT DIRECTION AS WELL NOT JUST 4019 03:08:11,560 --> 03:08:13,680 GENE THERAPY? 4020 03:08:13,680 --> 03:08:18,600 THE CELL THERAPY AND 4021 03:08:18,600 --> 03:08:23,520 REGENERATIVE MEDICINE? 4022 03:08:23,520 --> 03:08:25,760 >>I GUESS THERE IS A 4023 03:08:25,760 --> 03:08:31,960 REGENERATIVE MEDICINE PROGRAM IT 4024 03:08:31,960 --> 03:08:35,160 WAS RUN OUT OF NHLBI AND I DON'T 4025 03:08:35,160 --> 03:08:41,360 KNOW IF IT WAS TRANSITIONING. 4026 03:08:41,360 --> 03:08:43,360 THEY HAD CALLS FOR DIFFERENT 4027 03:08:43,360 --> 03:08:47,720 CELL THERAPY AND THEY FUNDED A 4028 03:08:47,720 --> 03:08:49,640 COUPLE AND AGAIN THEY WERE 4029 03:08:49,640 --> 03:08:52,720 LOOKING TO KIND OF STREAMLINE 4030 03:08:52,720 --> 03:08:55,560 THINGS AND WITH THE FDA TO MOVE 4031 03:08:55,560 --> 03:08:57,120 CELL THERAPY MOVE THROUGH 4032 03:08:57,120 --> 03:09:01,720 QUICKLY AND I HAVE NOT BEEN 4033 03:09:01,720 --> 03:09:03,120 INVOLVED IN THOSE. 4034 03:09:03,120 --> 03:09:05,040 I CAN TRY AND FIND THAT LINK AND 4035 03:09:05,040 --> 03:09:08,880 PUT IT IN THE CHAT AS WELL. 4036 03:09:08,880 --> 03:09:10,880 IT'S MUCH SMALLER PROGRAM AND 4037 03:09:10,880 --> 03:09:12,440 IT'S BEEN GOING ON FOR AT LEAST 4038 03:09:12,440 --> 03:09:19,720 FIVE YEARS NOW I THINK. 4039 03:09:19,720 --> 03:09:22,440 >>IF I COULD JUST ADD AN FINAL 4040 03:09:22,440 --> 03:09:23,760 ANALOGY TO BRING THIS HOME AND 4041 03:09:23,760 --> 03:09:26,800 MAYBE EVERYBODY GETS IT. 4042 03:09:26,800 --> 03:09:28,440 AN IMAGE CAME TO MIND WHEN WE 4043 03:09:28,440 --> 03:09:29,800 WERE TALKING ABOUT THIS. 4044 03:09:29,800 --> 03:09:31,400 WHEN WE'RE TRYING TO CURE A 4045 03:09:31,400 --> 03:09:34,600 CERTAIN DISEASE OR PROVIDE 4046 03:09:34,600 --> 03:09:36,040 TREATMENTS IT'S LIKE YOU NEED TO 4047 03:09:36,040 --> 03:09:36,720 COOK DINNER. 4048 03:09:36,720 --> 03:09:38,280 YOU NEED TO GET ALL THE 4049 03:09:38,280 --> 03:09:39,080 INGREDIENTS TOGETHER AND THERE'S 4050 03:09:39,080 --> 03:09:40,880 A LOT OF DIFFERENT WAYS TO DO 4051 03:09:40,880 --> 03:09:41,480 IT. 4052 03:09:41,480 --> 03:09:47,520 SO IT'S VERY POPULAR TO GET ALL 4053 03:09:47,520 --> 03:09:48,960 INGREDIENTS IN A GROUP SHIPPED 4054 03:09:48,960 --> 03:09:50,720 TO YOUR HOUSE IF YOU ARE BUSY OR 4055 03:09:50,720 --> 03:09:52,320 GO TO THE GROCERY STORE AND IN 4056 03:09:52,320 --> 03:09:53,880 THE GROCERY STORE, EVERYTHING IS 4057 03:09:53,880 --> 03:09:56,360 KIND OF ORGANIZED BY CATEGORY 4058 03:09:56,360 --> 03:09:57,280 AND YOU HAVE SOMETHING IN 4059 03:09:57,280 --> 03:09:59,040 BETWEEN LIKE IF YOU LIVE IN 4060 03:09:59,040 --> 03:10:00,600 EUROPE YOU GO TO THE MARKET AND 4061 03:10:00,600 --> 03:10:04,880 YOU CAN PICK UP EVERYTHING AND 4062 03:10:04,880 --> 03:10:07,680 IT'S A HYBRID. 4063 03:10:07,680 --> 03:10:09,280 IT'S A KIT OR YOU HAVE TO DO 4064 03:10:09,280 --> 03:10:11,840 EVERYTHING AND SO, CHANCES THAT 4065 03:10:11,840 --> 03:10:16,320 YOU ARE THE BEST AT SAY LIKE 4066 03:10:16,320 --> 03:10:18,400 WASHING A HEAD OF LETTUCE OR 4067 03:10:18,400 --> 03:10:19,880 FINDING GOOD WAY TO SCALE A 4068 03:10:19,880 --> 03:10:22,280 SPECIFIC INGREDIENT IF IF YOU 4069 03:10:22,280 --> 03:10:22,920 ARE DOING EVERYTHING ELSE IT'S 4070 03:10:22,920 --> 03:10:24,120 NOT THE BEST. 4071 03:10:24,120 --> 03:10:25,640 IT DOESN'T MAKE FOR A EASY STORY 4072 03:10:25,640 --> 03:10:27,760 TO TELL OR A WAY TO SAY 4073 03:10:27,760 --> 03:10:29,400 SOMETHING VERY EXCITING BUT I 4074 03:10:29,400 --> 03:10:31,640 THINK THAT WAY OF THINKING GO 4075 03:10:31,640 --> 03:10:32,840 PLATFORMS AND TOOLS IS VALUABLE 4076 03:10:32,840 --> 03:10:34,800 BECAUSE IT'S REALLY FOCUSING 4077 03:10:34,800 --> 03:10:36,320 DIRECTLY ON THE THINGS THAT 4078 03:10:36,320 --> 03:10:46,760 WE'LL PUT TOGETHER LATER. 4079 03:10:52,840 --> 03:10:54,960 >>IT SEEMS IT WOK MORE 4080 03:10:54,960 --> 03:10:57,440 ATTRACTIVE FOR SOME PHARMA 4081 03:10:57,440 --> 03:10:58,320 COMPANIES TOO. 4082 03:10:58,320 --> 03:11:01,160 THEY COULD -- IF THEY HAVE THIS 4083 03:11:01,160 --> 03:11:04,480 PLATFORMS THAT THEY COULD ATTACK 4084 03:11:04,480 --> 03:11:08,920 OUREL WORK ON MORE DISEASES AND 4085 03:11:08,920 --> 03:11:10,720 MORE CHANCE TO SUCCEED IN IN 4086 03:11:10,720 --> 03:11:18,160 SOME OF THEM AT LEAST. 4087 03:11:18,160 --> 03:11:21,000 >>WOULD AGREE. 4088 03:11:21,000 --> 03:11:25,680 I THINK SYMPTOMS ARE MISSING. 4089 03:11:25,680 --> 03:11:29,680 THEY'RE MISSING INFORMATION. 4090 03:11:29,680 --> 03:11:32,680 WE NEED OF BETTER DATA AND 4091 03:11:32,680 --> 03:11:41,880 BETTER INFORMATION OR BETTER NEW 4092 03:11:41,880 --> 03:11:47,480 INSIGHTS ON THE SYSTEM AND THE 4093 03:11:47,480 --> 03:11:58,040 BIOMEDICAL RESEARCH AND OR MORE 4094 03:11:59,320 --> 03:12:00,920 NEEDS TO BE DONE WITH MORE 4095 03:12:00,920 --> 03:12:07,480 COMMON IN THE SITUATION WITH THE 4096 03:12:07,480 --> 03:12:17,800 EXCELLENT COMMENTS. 4097 03:12:18,640 --> 03:12:19,080 >>Diana Bianchi: OKAY. 4098 03:12:19,080 --> 03:12:20,520 AGAIN, AS WE MENTIONED EARLIER, 4099 03:12:20,520 --> 03:12:22,040 WE PLAN TO CONTINUE THIS SESSION 4100 03:12:22,040 --> 03:12:23,600 FOR OUR FALL MEETING. 4101 03:12:23,600 --> 03:12:26,760 AND WE'D LIKE TO INCLUDE OTHER 4102 03:12:26,760 --> 03:12:28,240 MUSCULAR DYSTROPHY PATIENT 4103 03:12:28,240 --> 03:12:32,200 ADVOCACY ORGANIZATIONS WHO WISH 4104 03:12:32,200 --> 03:12:33,360 TO PRESENT. 4105 03:12:33,360 --> 03:12:36,480 PLEASE CONTACT GLEN IN YOUR 4106 03:12:36,480 --> 03:12:37,720 ORGANIZATION HAS A MUSCULAR 4107 03:12:37,720 --> 03:12:38,320 DYSTROPHY SPECIFIC PLAN THAT 4108 03:12:38,320 --> 03:12:40,240 DEFINES GOALS AND OCTAVES FOR 4109 03:12:40,240 --> 03:12:42,520 RESEARCH OR PATIENT ACCESS TO 4110 03:12:42,520 --> 03:12:46,960 CARE OR SERVICES. 4111 03:12:46,960 --> 03:12:48,480 A LITTLE BIT TIME AT END OF THE 4112 03:12:48,480 --> 03:12:53,400 AGENDA TO TALK ABOUT HOW THIS 4113 03:12:53,400 --> 03:12:56,040 NEW 2025MDCC ACTION PLAN CAN 4114 03:12:56,040 --> 03:12:57,680 BUILD UPON AND INTEGRATE THE 4115 03:12:57,680 --> 03:12:59,080 STRATEGIC PLANS WE'VE HEARD 4116 03:12:59,080 --> 03:12:59,280 ABOUT. 4117 03:12:59,280 --> 03:13:01,480 WE DISCUSSED IT A LITTLE BIT. 4118 03:13:01,480 --> 03:13:03,280 BEFORE WE GO THERE FOR FINAL 4119 03:13:03,280 --> 03:13:05,400 COMMENTS, I DID WANT TO 4120 03:13:05,400 --> 03:13:08,720 ACKNOWLEDGE A COMMENT FROM JUNE 4121 03:13:08,720 --> 03:13:11,360 WHO HAD WRITTEN EARLIER ABOUT AN 4122 03:13:11,360 --> 03:13:13,880 OUR AGO, THAT LACK OF INSURANCE 4123 03:13:13,880 --> 03:13:15,800 REIMBURSEMENT FOR GENETIC 4124 03:13:15,800 --> 03:13:17,560 COUNSELING AND GENETIC TESTING 4125 03:13:17,560 --> 03:13:18,960 IS A MAJOR ISSUE. 4126 03:13:18,960 --> 03:13:23,360 CAN THE MDCC SUGGEST ACTIONS ON 4127 03:13:23,360 --> 03:13:26,160 THIS ISSUE AND I AM A MEDICAL 4128 03:13:26,160 --> 03:13:28,440 GENETIC SIS AND I AGREE WHOLE 4129 03:13:28,440 --> 03:13:29,360 HEARDEDLY, ONE OF THE THINGS I 4130 03:13:29,360 --> 03:13:31,160 DID NOT FIND APPEALING ABOUT 4131 03:13:31,160 --> 03:13:33,480 GENETICS IS THE AMOUNT OF TIME 4132 03:13:33,480 --> 03:13:35,520 WE HAD TO SPEND TO GET 4133 03:13:35,520 --> 03:13:40,320 PRE-APPROVAL FOR GENETIC 4134 03:13:40,320 --> 03:13:50,480 TESTING. 4135 03:13:55,560 --> 03:13:59,880 >>THIS IS A COMPLICATED COMMENT 4136 03:13:59,880 --> 03:14:00,880 BECAUSE OFTEN TIMES INSURANCE 4137 03:14:00,880 --> 03:14:03,320 AND IT VARIES, YOU KNOW, THERE 4138 03:14:03,320 --> 03:14:04,640 ARE DIFFERENT KINDS OF INSURANCE 4139 03:14:04,640 --> 03:14:08,840 MEDICARE AND MARKETPLACE AND 4140 03:14:08,840 --> 03:14:11,280 MEDICAID AND ALL INSURERS ARE 4141 03:14:11,280 --> 03:14:13,680 PRETTY MUCH REQUIRED TO DO TESTS 4142 03:14:13,680 --> 03:14:16,720 THAT ARE IN THE U.S. 4143 03:14:16,720 --> 03:14:18,520 PREVENTATIVE TASK FORCE LIST BUT 4144 03:14:18,520 --> 03:14:20,200 AS YOU KNOW THAT'S NOT MOST 4145 03:14:20,200 --> 03:14:23,440 GENETIC SCREENING AND MOST 4146 03:14:23,440 --> 03:14:24,840 INSURERS WILL COVER THINGS THAT 4147 03:14:24,840 --> 03:14:26,400 ARE NECESSARY FOR THE DIAGNOSIS 4148 03:14:26,400 --> 03:14:29,200 AND TREATMENT OF A CONDITION SO 4149 03:14:29,200 --> 03:14:33,000 THAT CAN COME UP IN THE CASE OF 4150 03:14:33,000 --> 03:14:34,160 DIAGNOSIS OF SOMETHING BUT STILL 4151 03:14:34,160 --> 03:14:36,640 CAN BE A CHALLENGE TO GET IT 4152 03:14:36,640 --> 03:14:37,320 APPROVED. 4153 03:14:37,320 --> 03:14:40,240 SOMEONE THINKS THEY MIGHT 4154 03:14:40,240 --> 03:14:43,760 HAVE -- THERE WILL BE CRITERIA 4155 03:14:43,760 --> 03:14:47,360 FOR CYSTIC FIBROSIS OR I ASSUME 4156 03:14:47,360 --> 03:14:48,680 MUSCULAR DYSTROPHY, NOT DETAILED 4157 03:14:48,680 --> 03:14:49,040 THERE. 4158 03:14:49,040 --> 03:14:56,720 I THINK THE PLACES TO WORK ON IT 4159 03:14:56,720 --> 03:14:59,240 ARE THINKING ABOUT THE FUTURE 4160 03:14:59,240 --> 03:15:07,840 AND FOR PEDIATRIC AND SCREENING 4161 03:15:07,840 --> 03:15:10,640 AND A BIGGER LIFT I THINK AND SO 4162 03:15:10,640 --> 03:15:13,720 THAT IS KIND OF WHAT I HAVE TO 4163 03:15:13,720 --> 03:15:16,000 OFFER THAT IS SOMETHING THAT 4164 03:15:16,000 --> 03:15:17,680 COULD BE KIND OF PUT ON THE 4165 03:15:17,680 --> 03:15:20,320 RADAR IN AN ACTION PLAN. 4166 03:15:20,320 --> 03:15:25,960 BUT IT IS A LONG-TERM RADAR TYPE 4167 03:15:25,960 --> 03:15:27,160 OF THING. 4168 03:15:27,160 --> 03:15:29,560 >>THANK YOU FOR THAT COMMENT. 4169 03:15:29,560 --> 03:15:32,960 THE HOPE IS THAT SOME DAY, YOU 4170 03:15:32,960 --> 03:15:36,480 KNOW, ALL NEWBORNS WILL BE 4171 03:15:36,480 --> 03:15:39,640 SEQUENCED AND SO THE STATES WILL 4172 03:15:39,640 --> 03:15:41,960 PAY FOR THAT AND THEN THAT WILL 4173 03:15:41,960 --> 03:15:44,360 ALLOW US TO KNOW WHAT TO EXPECT 4174 03:15:44,360 --> 03:15:45,320 IN THOSE INFANTS. 4175 03:15:45,320 --> 03:15:47,960 I MEAN IT'S ONE OF THE -- 4176 03:15:47,960 --> 03:15:49,600 >>AND THEY WON'T BE 4177 03:15:49,600 --> 03:15:50,640 DISCRIMINATED ON THE BASIS OF 4178 03:15:50,640 --> 03:15:52,760 WHAT THE SEQUENCES SHOW AND I 4179 03:15:52,760 --> 03:15:54,080 KNOW WE HAVE SOME FEDERAL 4180 03:15:54,080 --> 03:15:55,720 PROTECTIONS AGAINST THAT BUT I 4181 03:15:55,720 --> 03:15:57,840 KNOW SOME PEOPLE AVOID THEIR 4182 03:15:57,840 --> 03:15:58,520 INSURANCE COMPANIES FOR 4183 03:15:58,520 --> 03:16:02,760 SCREENING BECAUSE THEY DON'T 4184 03:16:02,760 --> 03:16:04,960 WANT THAT. 4185 03:16:04,960 --> 03:16:07,160 OF THE ACT PROVIDES THAT 4186 03:16:07,160 --> 03:16:07,440 PROTECTION. 4187 03:16:07,440 --> 03:16:08,280 >>Diana Bianchi: THAT'S BEEN 4188 03:16:08,280 --> 03:16:09,760 AROUND FOR A LONG TIME. 4189 03:16:09,760 --> 03:16:12,800 PRESIDENT BUSH, THE YOUNGER 4190 03:16:12,800 --> 03:16:15,720 PRESIDENT BUSH SIGNED THAT LAW 4191 03:16:15,720 --> 03:16:17,960 AND YOU REALLY SHOULDN'T BE 4192 03:16:17,960 --> 03:16:19,400 DISCRIMINATED AGAINST BUT I 4193 03:16:19,400 --> 03:16:23,880 THINK PART OF IT MANY PHYSICIANS 4194 03:16:23,880 --> 03:16:31,400 DON'T HAVE GENETIC EDUCATION OR 4195 03:16:31,400 --> 03:16:32,760 THE INSURANCE COMPANY DON'T 4196 03:16:32,760 --> 03:16:35,520 UNDERSTAND THE POTENTIAL FOR THE 4197 03:16:35,520 --> 03:16:36,120 DIAGNOSTIC ODYSSEY AND IN THE 4198 03:16:36,120 --> 03:16:38,480 END OF IT IMPORTANTLY PROVIDE 4199 03:16:38,480 --> 03:16:40,800 BETTER PATIENT CARE AND MORE 4200 03:16:40,800 --> 03:16:41,520 TARGETED THERAPY BUT IT SAVES 4201 03:16:41,520 --> 03:16:45,200 MONEY AND IT'S BEEN WELL 4202 03:16:45,200 --> 03:16:47,440 DEMONSTRATED IT SAVES MONEY. 4203 03:16:47,440 --> 03:16:51,320 >>THE BEST CASE IS WHEN THERE'S 4204 03:16:51,320 --> 03:16:55,400 A DIAGNOSTIC ODYSSEY GOING ON 4205 03:16:55,400 --> 03:16:59,680 AND IT'S HARD TO WORK THE 4206 03:16:59,680 --> 03:17:04,720 INSURANCE COMPANIES PROCESS OF 4207 03:17:04,720 --> 03:17:06,520 APPEALS AND CONSIDERATIONS AND 4208 03:17:06,520 --> 03:17:11,240 THE LIKE AND MEDICARE WE HAVE A 4209 03:17:11,240 --> 03:17:13,000 GOOD POINT HOW FAR THEY DON'T 4210 03:17:13,000 --> 03:17:16,240 COVER LIFE INSURANCE AND 4211 03:17:16,240 --> 03:17:19,400 DISABILITY INSURANCE BUT YOU 4212 03:17:19,400 --> 03:17:23,240 KNOW WE DO HAVE SOME ADJUSTMENTS 4213 03:17:23,240 --> 03:17:25,360 THAT WE'RE BEING DONE THAT ABOUT 4214 03:17:25,360 --> 03:17:26,640 PRIOR AUTHORIZATION AND WE'LL 4215 03:17:26,640 --> 03:17:29,160 SEE HOW THOSE HELP BOTH IN THE 4216 03:17:29,160 --> 03:17:30,440 MEDICARE VANTAGE ROLE WHICH IS 4217 03:17:30,440 --> 03:17:40,960 FOR MEDICARE AND ALSO IN THE THE 4218 03:17:47,920 --> 03:17:49,720 POINT OF ASKING BETTER SCIENCE 4219 03:17:49,720 --> 03:17:50,680 FOR THOSE PRIOR AUTHORIZATION 4220 03:17:50,680 --> 03:17:54,720 AND TRANSPARENCY AND THOSE PRIOR 4221 03:17:54,720 --> 03:17:57,600 AUTHORIZATION RULES AND 4222 03:17:57,600 --> 03:17:58,520 PROCESSES. 4223 03:17:58,520 --> 03:18:03,360 IT'S A LONG HALL. 4224 03:18:03,360 --> 03:18:04,720 >>JUST A COMMENT. 4225 03:18:04,720 --> 03:18:09,600 I THINK WE'RE MISSING SOME 4226 03:18:09,600 --> 03:18:12,440 IMPORTANT WE NEED OUR POSITION. 4227 03:18:12,440 --> 03:18:19,040 FIRSTLY, WE CAN CREATE BETTER 4228 03:18:19,040 --> 03:18:24,840 DIAGNOSTIC TALKS WITHOUT THE 4229 03:18:24,840 --> 03:18:30,240 ADJUSTED OR'S YEAR TO USE AND 4230 03:18:30,240 --> 03:18:35,400 THE GENETIC LANDSCAPE DRIVERS 4231 03:18:35,400 --> 03:18:38,040 THE CLINICAL PROCESS SO WE 4232 03:18:38,040 --> 03:18:42,960 REALLY NEED TO GET A HANDLE ON 4233 03:18:42,960 --> 03:18:49,560 THE GENETIC COMPLEXION OF 4234 03:18:49,560 --> 03:18:52,280 ESPECIALLY WITH DISEASE LIKE 4235 03:18:52,280 --> 03:18:53,440 ALZHEIMER'S WHERE YOU HAVE A LOT 4236 03:18:53,440 --> 03:18:55,960 OF ACTIVE DISEASE TRIALS GOING 4237 03:18:55,960 --> 03:18:59,400 ON EARLIER AND UNSEEN AND OUT OF 4238 03:18:59,400 --> 03:19:02,840 SIGHT AND THEN YOU ALSO HAVE 4239 03:19:02,840 --> 03:19:06,320 GENETIC SITUATIONS OF THE 4240 03:19:06,320 --> 03:19:09,480 DISEASE. 4241 03:19:09,480 --> 03:19:14,520 IT'S IN AND OUT SYSTEMS AND 4242 03:19:14,520 --> 03:19:20,720 BASED ON A SO IT'S A DISEASE 4243 03:19:20,720 --> 03:19:25,720 LIKE FSH WHERE THEY CLAIM IT AND 4244 03:19:25,720 --> 03:19:28,280 YOU ARE MISSING THE POINT. 4245 03:19:28,280 --> 03:19:31,520 IN THE BODY I WANT TO KNOW IT 4246 03:19:31,520 --> 03:19:42,040 AND THIS IS A VERY DYNAMIC WE 4247 03:19:55,440 --> 03:19:58,920 REALLY HAVE TO WAKE UP. 4248 03:19:58,920 --> 03:20:08,840 AND AND TRUSTING AND THE AND 4249 03:20:08,840 --> 03:20:14,600 WITHOUT AND WE HE HAVE TO WAKE 4250 03:20:14,600 --> 03:20:17,720 UP. 4251 03:20:17,720 --> 03:20:19,480 >>I THINK ANOTHER MODEL THAT IS 4252 03:20:19,480 --> 03:20:21,840 NOT A SHORT TERM MODEL IS YOU 4253 03:20:21,840 --> 03:20:25,920 KNOW, THE BREAST CANCER GENETIC 4254 03:20:25,920 --> 03:20:28,040 TESTING IS PART OF THE U.S. 4255 03:20:28,040 --> 03:20:31,360 PREVENTATIVE SERVICES TASK FORCE 4256 03:20:31,360 --> 03:20:34,080 LIST OF ITEMS THAT BY-LAW 4257 03:20:34,080 --> 03:20:36,000 THERE'S SOME CHALLENGES TO IT 4258 03:20:36,000 --> 03:20:37,320 BUT SOME LEGAL CHALLENGES ARE 4259 03:20:37,320 --> 03:20:40,920 GOING ON NOW BUT BY-LAW THOSE 4260 03:20:40,920 --> 03:20:43,560 NEED TO BE COVERED BITCH PLANS 4261 03:20:43,560 --> 03:20:48,040 NOT AND IN OF THE TESTING, YOU 4262 03:20:48,040 --> 03:20:51,360 KNOW, THE SCIENCE COULD SUPPORT 4263 03:20:51,360 --> 03:20:53,600 CONCEIVABLY THE TESTING FOR 4264 03:20:53,600 --> 03:20:57,600 PEOPLE WITH A FAMILY HISTORY SAY 4265 03:20:57,600 --> 03:20:59,200 IN THAT KIND OF A MODEL SO 4266 03:20:59,200 --> 03:21:00,320 THAT'S ANOTHER THING TO PURSUE. 4267 03:21:00,320 --> 03:21:04,040 IT US DIDN'T HAPPEN QUICKLY BUT 4268 03:21:04,040 --> 03:21:05,640 IT IS SOMETHING SOMETHING WORTH 4269 03:21:05,640 --> 03:21:06,480 PURSUING. 4270 03:21:06,480 --> 03:21:09,120 AGAIN, THOSE THINGS DRIVE HOW 4271 03:21:09,120 --> 03:21:15,360 INSURANCE COMPANY AND CMS COVER 4272 03:21:15,360 --> 03:21:18,040 THINGS. 4273 03:21:18,040 --> 03:21:20,880 >>Diana Bianchi: SO THERE ARE A 4274 03:21:20,880 --> 03:21:23,240 COUPLE OF COMMENTS UNTIL THE 4275 03:21:23,240 --> 03:21:25,720 CHAT BOX AND A LOT OF REACTIONS 4276 03:21:25,720 --> 03:21:27,040 SOMETHING DONOVAN DECKER 4277 03:21:27,040 --> 03:21:29,880 MENTIONED SO I'M GOING TO HANDLE 4278 03:21:29,880 --> 03:21:30,440 THAT. 4279 03:21:30,440 --> 03:21:34,320 LET ME READ WHAT DONOVAN'S OTHER 4280 03:21:34,320 --> 03:21:36,720 COMMENT IS IF I GO BACK. 4281 03:21:36,720 --> 03:21:39,480 I SAYS, WHAT CAN BE DONE WHEN A 4282 03:21:39,480 --> 03:21:40,800 LARGE COMPANY BUYS THE RESEARCH 4283 03:21:40,800 --> 03:21:42,320 IN YOUR DISEASE AND THEN THEY 4284 03:21:42,320 --> 03:21:44,400 DON'T DO ANYTHING WITH IT? 4285 03:21:44,400 --> 03:21:49,040 WE, THE PATIENTS, SUFFER. 4286 03:21:49,040 --> 03:21:51,800 AND THEN GUSTAVO ASKED FOR A 4287 03:21:51,800 --> 03:21:54,680 SPECIFIC EXAMPLE AND ERIC, DO 4288 03:21:54,680 --> 03:21:58,840 YOU WANT TO COME OFF MUTE AND 4289 03:21:58,840 --> 03:22:02,240 MAYBE REPEAT YOUR ANSWER TO 4290 03:22:02,240 --> 03:22:02,440 THEM. 4291 03:22:02,440 --> 03:22:04,080 >>CORRECT ME IF I'VE WRONG BUT 4292 03:22:04,080 --> 03:22:05,840 YOU ARE REFERRING TO INSTANCES 4293 03:22:05,840 --> 03:22:09,120 WHERE COMPANY LICENSE IP FROM AN 4294 03:22:09,120 --> 03:22:09,760 INCONVENIENT ERIN INVESTIGATOR 4295 03:22:09,760 --> 03:22:12,000 THAT IS AIMED AT TREATING 4296 03:22:12,000 --> 03:22:14,720 DISEASE BUT THE COMPANY FOR A 4297 03:22:14,720 --> 03:22:15,880 VARIETY OF REASONS MIGHT NOT 4298 03:22:15,880 --> 03:22:17,200 MOVE FORWARD WITH IT AND MIGHT 4299 03:22:17,200 --> 03:22:19,600 NOT MOVE FORWARD QUICKLY WITH IT 4300 03:22:19,600 --> 03:22:22,000 AND USUALLY THERE ARE TIME LINES 4301 03:22:22,000 --> 03:22:23,240 AND DILIGENCE MILESTONES WITH 4302 03:22:23,240 --> 03:22:26,440 THE LICENSES BUT IT DEFINITELY 4303 03:22:26,440 --> 03:22:28,440 CAN DELAY PROGRESS EX PREVENT 4304 03:22:28,440 --> 03:22:29,720 OTHERS FROM WANT TO GO GET INTO 4305 03:22:29,720 --> 03:22:31,280 THE SPACE BECAUSE THEY CAN'T BE 4306 03:22:31,280 --> 03:22:32,440 PROTECTED SO IT'S AN ISSUE AND 4307 03:22:32,440 --> 03:22:38,120 THIS IS AN INSTANCE IN WHICH 4308 03:22:38,120 --> 03:22:38,960 LIKE IN THE PAST THERE'S 4309 03:22:38,960 --> 03:22:43,560 LEGISLATION TO HE HE CAN SELL E 4310 03:22:43,560 --> 03:22:46,160 MOVEMENT INTO THE PRIVATE SECTOR 4311 03:22:46,160 --> 03:22:48,320 WHERE NIH CAN HAVE AN IMPACT. 4312 03:22:48,320 --> 03:22:50,200 >>YEAH. 4313 03:22:50,200 --> 03:22:51,600 THERE SHOULD BE TWO PARALLEL 4314 03:22:51,600 --> 03:22:53,920 CONVERSATIONS GOING ON SO ONE IS 4315 03:22:53,920 --> 03:22:58,640 RELATED TO THE INTELLECTUAL 4316 03:22:58,640 --> 03:23:00,240 PROPERTY AND HOW IT ADVANCES OR 4317 03:23:00,240 --> 03:23:02,160 PUTS A BARRIER IN FRONT OF 4318 03:23:02,160 --> 03:23:02,880 ADVANCING TO TREATMENT AND 4319 03:23:02,880 --> 03:23:05,680 THERE'S A PARALLEL CONVERSATION 4320 03:23:05,680 --> 03:23:08,560 GOING ON RELATED TO THE GENETIC 4321 03:23:08,560 --> 03:23:11,920 COUNSELING AND AT T TESTING 4322 03:23:11,920 --> 03:23:13,160 REIMBURSEMENT SO JUNE WRITES IT 4323 03:23:13,160 --> 03:23:15,360 WOULD BE HELPFUL IF THE NDCC 4324 03:23:15,360 --> 03:23:19,120 COULD PROVIDE A CONSENSUS ACTION 4325 03:23:19,120 --> 03:23:20,560 THAT WE CAN ALL GET BEHIND 4326 03:23:20,560 --> 03:23:22,080 WITHOUT A CLEAR CALL TO ACTION 4327 03:23:22,080 --> 03:23:26,160 IT'S HARD TO ACT. 4328 03:23:26,160 --> 03:23:29,760 FROM THE NIH PERSPECTIVE, WE CAN 4329 03:23:29,760 --> 03:23:33,520 FUND RESEARCH THAT PROVIDES 4330 03:23:33,520 --> 03:23:34,160 EVIDENCE. 4331 03:23:34,160 --> 03:23:36,680 TYPICALLY, A PROFESSIONAL 4332 03:23:36,680 --> 03:23:38,320 RECOMMENDATION FOR CARE COMES 4333 03:23:38,320 --> 03:23:40,840 FROM PROFESSIONAL SOCIETY SO I 4334 03:23:40,840 --> 03:23:44,640 MEAN, I THINK IT COULD GO ON THE 4335 03:23:44,640 --> 03:23:46,320 AGENDA FOR DISCUSSIONS DURING 4336 03:23:46,320 --> 03:23:48,400 THE STRATEGIC PLANNING BUT I 4337 03:23:48,400 --> 03:23:54,200 DON'T THINK THAT THE MDCC -- I 4338 03:23:54,200 --> 03:23:58,040 MEAN, WE CAN DISCUSS WHAT OUR 4339 03:23:58,040 --> 03:24:00,960 SWIM LANES ARE IN TERMS OF HOW 4340 03:24:00,960 --> 03:24:05,160 FAR FAR WE CAN GET INTO MAKING 4341 03:24:05,160 --> 03:24:08,440 ACTUAL RECOMMENDATIONS FOR 4342 03:24:08,440 --> 03:24:18,680 PATIENT CARE? 4343 03:24:26,760 --> 03:24:28,200 WHAT ARE THE CONDITIONS THAT 4344 03:24:28,200 --> 03:24:29,760 WOULD MANDATE TESTING AND BE 4345 03:24:29,760 --> 03:24:40,280 REPORTED AS A SECONDARY FINDING. 4346 03:24:42,280 --> 03:24:46,040 WE HAVE A LITTLE -- THERE'S 4347 03:24:46,040 --> 03:24:56,560 ANOTHER COMMENT AND THERE'S NO 4348 03:25:02,760 --> 03:25:02,960 CURE. 4349 03:25:02,960 --> 03:25:04,360 THIS IS WHAT I WAS GETTING AT 4350 03:25:04,360 --> 03:25:07,200 WHERE THERE'S AN EDUCATION GAP. 4351 03:25:07,200 --> 03:25:08,560 MANY PHYSICIANS ARE LACKING 4352 03:25:08,560 --> 03:25:09,840 INFORMATION ABOUT REGISTRIES AND 4353 03:25:09,840 --> 03:25:11,760 CLINICAL STUDIES AND EXISTING 4354 03:25:11,760 --> 03:25:15,280 TREATMENTS FOR CONDITIONS LIKE 4355 03:25:15,280 --> 03:25:16,840 POMPEII AND SPINAL MUSCULAR 4356 03:25:16,840 --> 03:25:17,160 ASTRO FEE. 4357 03:25:17,160 --> 03:25:19,480 IT'S UP TO THE PATIENT TO PUSH 4358 03:25:19,480 --> 03:25:21,520 TO GET THE TESTING, WELL, YEAH, 4359 03:25:21,520 --> 03:25:27,360 BUT SORRY, OKAY. 4360 03:25:27,360 --> 03:25:31,040 ANDREA KLEIN, THANK YOU. 4361 03:25:31,040 --> 03:25:33,520 PATIENTS NEED TO ADVOCATE FOR 4362 03:25:33,520 --> 03:25:33,960 THEMSELVES. 4363 03:25:33,960 --> 03:25:35,400 IT'S NOT EASY. 4364 03:25:35,400 --> 03:25:42,840 LIKE I SAID, HAVING BEEN IN THE 4365 03:25:42,840 --> 03:25:44,640 PRACTICE OF PROVIDING AND WE'VE 4366 03:25:44,640 --> 03:25:48,040 HAD THE TECHNOLOGY FOR A LONG 4367 03:25:48,040 --> 03:25:50,960 TIME AND IT'S A SHAME THAT IT 4368 03:25:50,960 --> 03:25:58,400 HASN'T BECOME EASIER TO GET THE 4369 03:25:58,400 --> 03:25:59,080 TESTING. 4370 03:25:59,080 --> 03:26:01,520 >>I DON'T KNOW IF THAT'S IN 4371 03:26:01,520 --> 03:26:06,240 MARITMDCCSWIM LANE BUT EFFORTS E 4372 03:26:06,240 --> 03:26:08,960 SURE THAT CLINICAL PARTNERS KIND 4373 03:26:08,960 --> 03:26:11,400 OF UNDERSTAND SORT OF WHERE WE 4374 03:26:11,400 --> 03:26:14,920 ARE WITH THE CONDITION BECAUSE 4375 03:26:14,920 --> 03:26:17,520 THIS KIND OF QUICKLY EVOLVING 4376 03:26:17,520 --> 03:26:20,600 AND 15 YEARS AGO, THAT WAS A 4377 03:26:20,600 --> 03:26:23,280 TRUE STATEMENT AND I THINK, BUT 4378 03:26:23,280 --> 03:26:25,480 NOW IT'S NOT BUT IT MAY NOT BE 4379 03:26:25,480 --> 03:26:28,680 SOMETHING THAT IS DIFFUSED INTO 4380 03:26:28,680 --> 03:26:32,080 THE COMMUNITY AND IT'S NOT 4381 03:26:32,080 --> 03:26:32,560 TAKING CARE OF MUSCULAR 4382 03:26:32,560 --> 03:26:34,080 DYSTROPHY. 4383 03:26:34,080 --> 03:26:34,840 SO THAT COULD BE IT. 4384 03:26:34,840 --> 03:26:37,400 >>MY GUESS IS THAT, YOU KNOW, 4385 03:26:37,400 --> 03:26:39,360 ONE OF THE WAYS YOU COULD GET 4386 03:26:39,360 --> 03:26:41,800 AROUND IT IS THAT YOU INSTEAD OF 4387 03:26:41,800 --> 03:26:45,800 SAYING EVERY PATIENT WITH A 4388 03:26:45,800 --> 03:26:46,400 MUSCULAR DYSTROPHY SHOULD HAVE 4389 03:26:46,400 --> 03:26:49,400 GENETIC TESTING, WE COULD 4390 03:26:49,400 --> 03:26:53,240 RECOMMEND AFTER EXISTING 4391 03:26:53,240 --> 03:26:56,400 LITERATURE AND EXAM WHAT IS TO 4392 03:26:56,400 --> 03:26:59,800 COME BUT SAY SOMETHING TO THE 4393 03:26:59,800 --> 03:27:02,200 EFFECT OF KNOWING A PATIENT'S 4394 03:27:02,200 --> 03:27:06,080 SPECIFIC MUTATIONS MAY HELP TO 4395 03:27:06,080 --> 03:27:10,880 GUIDE THERAPIES OR COUNSELING 4396 03:27:10,880 --> 03:27:12,800 AND OR PROGNOSIS SO YOU ARE NOT 4397 03:27:12,800 --> 03:27:14,320 COMING OUT AND SAYING YOU HAVE 4398 03:27:14,320 --> 03:27:15,920 TO HAVE GENETIC TESTING BUT IF 4399 03:27:15,920 --> 03:27:18,320 YOU KNOW THE GENETIC TESTING YOU 4400 03:27:18,320 --> 03:27:19,520 ARE IMPLYING IT'S IMPROVING THE 4401 03:27:19,520 --> 03:27:29,880 CARE FOR THE PATIENT. 4402 03:27:30,960 --> 03:27:35,600 >>THAT'S IMPORTANT WITHEN 4403 03:27:35,600 --> 03:27:37,400 TENSION AND IT'S ALSO GOING TO 4404 03:27:37,400 --> 03:27:41,320 DICTATE THE DIRECTION OF 4405 03:27:41,320 --> 03:27:42,560 WHATEVER RESEARCH AS WELL AND 4406 03:27:42,560 --> 03:27:50,520 IT'S NOT JUST A DECISION IT'S 4407 03:27:50,520 --> 03:27:52,400 ALSO PRE DOM I AM APTLY WORKING 4408 03:27:52,400 --> 03:27:58,840 ON THIS KIND OF DIRECT CONSTRUCT 4409 03:27:58,840 --> 03:28:01,840 AND RESEARCH STRUCTURE AS WELL. 4410 03:28:01,840 --> 03:28:02,600 >>Diana Bianchi: YEAH, SINCE 4411 03:28:02,600 --> 03:28:04,080 THERE ARE SUCH FUNDAMENTAL 4412 03:28:04,080 --> 03:28:05,760 DIFFERENT MECHANISMS IN THE 4413 03:28:05,760 --> 03:28:07,920 CONDITIONS THAT WE'VE BEEN 4414 03:28:07,920 --> 03:28:09,200 DISCUSSING, I MEAN IT MAY BE 4415 03:28:09,200 --> 03:28:11,280 IMPORTANT TO INCLUDE, AS PART OF 4416 03:28:11,280 --> 03:28:13,640 THE STRATEGIC PLAN, THAT AGAIN, 4417 03:28:13,640 --> 03:28:15,880 IMPLYING IF YOU DO THE TESTING 4418 03:28:15,880 --> 03:28:18,040 AND YOU KNOW THE MUTATION OR YOU 4419 03:28:18,040 --> 03:28:20,320 KNOW THE UNDERLYING MECHANISMS 4420 03:28:20,320 --> 03:28:22,480 THAT IS GOING TO RESULT IN MUCH 4421 03:28:22,480 --> 03:28:25,240 MORE PRECISE THERAPIES SO REALLY 4422 03:28:25,240 --> 03:28:31,720 TAKING A PRECISION MEDICINE 4423 03:28:31,720 --> 03:28:32,200 APPROACH. 4424 03:28:32,200 --> 03:28:34,240 WALTER IS ASKING IS THERE A 4425 03:28:34,240 --> 03:28:35,840 CLEANED GENE PANEL AND HE MEANS 4426 03:28:35,840 --> 03:28:46,240 AN N FDA CLEARED PANEL? 4427 03:28:46,720 --> 03:28:48,320 I CAN'T ANSWER THAT BECAUSE I 4428 03:28:48,320 --> 03:28:49,640 HAVEN'T PRACTICED GENETICS IN 4429 03:28:49,640 --> 03:28:51,560 THE LAST SIX OR SEVEN YEARS SO 4430 03:28:51,560 --> 03:28:54,480 THERE MAY BE. 4431 03:28:54,480 --> 03:29:05,040 >>WHAT IS REFERRED DEFINITION? 4432 03:29:06,040 --> 03:29:08,840 >>Diana Bianchi: YEAH, SO YOU 4433 03:29:08,840 --> 03:29:12,440 KNOW, I THINK WALTER NOWADAYS 4434 03:29:12,440 --> 03:29:14,560 IT'S ALMOST MORE -- IT'S LESS 4435 03:29:14,560 --> 03:29:15,960 EXPENSIVE BECAUSE OF YOUR FIRST 4436 03:29:15,960 --> 03:29:17,760 PANEL COMES OUT NEGATIVE THEN 4437 03:29:17,760 --> 03:29:22,080 YOU END UP LOOKING AT OTHER 4438 03:29:22,080 --> 03:29:23,360 PANELS. 4439 03:29:23,360 --> 03:29:25,320 IT MAY BE EITHER NOW OR SOON 4440 03:29:25,320 --> 03:29:27,680 THAT PEOPLE ARE GOING TO GO 4441 03:29:27,680 --> 03:29:29,960 DIRECTLY TO EITHER A WHOLE EX ON 4442 03:29:29,960 --> 03:29:40,440 OR A WHOLE GENOME IT'S LOOKING 4443 03:29:41,840 --> 03:29:52,320 AT REPEAT LINKS AND SO IT'S NOT 4444 03:29:54,520 --> 03:29:55,160 AND FSHD. 4445 03:29:55,160 --> 03:29:56,800 THERE ARE DIFFERENT TESTS FOR 4446 03:29:56,800 --> 03:29:58,760 DIFFERENT DYSTROPHIES IT US 4447 03:29:58,760 --> 03:30:00,640 DIDMENT ALL NICELY FIT ON ONE 4448 03:30:00,640 --> 03:30:00,960 PANEL. 4449 03:30:00,960 --> 03:30:01,640 >>RIGHT. 4450 03:30:01,640 --> 03:30:04,400 BUT PRESUMABLY YOU WOULD HAVE 4451 03:30:04,400 --> 03:30:05,680 SOME CLINICAL SUSPICION THAT 4452 03:30:05,680 --> 03:30:07,080 MIGHT DIRECT YOU IN THE 4453 03:30:07,080 --> 03:30:11,480 DIRECTION THAT YOU WOULD LIKE TO 4454 03:30:11,480 --> 03:30:12,080 GO? 4455 03:30:12,080 --> 03:30:15,400 I THINK BECAUSE THERE'SES A LOT 4456 03:30:15,400 --> 03:30:18,360 OF INTEREST IN GENETICS AND 4457 03:30:18,360 --> 03:30:22,760 UNDERLYING MECHANISMS WE NEED TE 4458 03:30:22,760 --> 03:30:24,200 MECHANISMS THEME WASN'T IT, 4459 03:30:24,200 --> 03:30:27,520 GLEN, IN THE PROPOSED STRATEGIC 4460 03:30:27,520 --> 03:30:29,040 PLAN SO -- 4461 03:30:29,040 --> 03:30:29,560 >>Glen Nuckolls: YEAH. 4462 03:30:29,560 --> 03:30:30,240 >>Diana Bianchi: MAYBE THAT'S 4463 03:30:30,240 --> 03:30:34,800 WHERE GENETICS SHOULD BE 4464 03:30:34,800 --> 03:30:37,280 INCLUDED. 4465 03:30:37,280 --> 03:30:37,880 >>Glen Nuckolls: SO THERE'S 4466 03:30:37,880 --> 03:30:42,920 ALSO A COMMENT FROM THERESA SAN 4467 03:30:42,920 --> 03:30:46,320 AGO STEEN CONSIDERED TO ADD 4468 03:30:46,320 --> 03:30:48,320 STRATEGY BEYOND MEDICAL STRATEGY 4469 03:30:48,320 --> 03:30:51,400 SUCH AS SCHOOL LIVING, 4470 03:30:51,400 --> 03:30:55,600 INDEPENDENTLY AND GAINFUL 4471 03:30:55,600 --> 03:30:57,880 EMPLOYMENT AND THERESA WAS 4472 03:30:57,880 --> 03:30:59,320 INVOLVED IN THE 2015 ACTION PLAN 4473 03:30:59,320 --> 03:31:02,920 THAT HAS OBJECTIVES ON 4474 03:31:02,920 --> 03:31:07,360 TRANSITION TO -- FROM SCHOOL 4475 03:31:07,360 --> 03:31:10,800 INTO THE WORKFORCE INDEPENDENT 4476 03:31:10,800 --> 03:31:12,160 LIVING, YOU KNOW, SO I THINK 4477 03:31:12,160 --> 03:31:13,360 THAT THESE WILL CONTINUE TO BE 4478 03:31:13,360 --> 03:31:16,800 IMPORTANT TOPICS FOR THE 2015 4479 03:31:16,800 --> 03:31:18,080 RENEWAL. 4480 03:31:18,080 --> 03:31:18,800 >>Diana Bianchi: WELL, WOULDN'T 4481 03:31:18,800 --> 03:31:20,000 THAT GO UNDER THE QUALITY OF 4482 03:31:20,000 --> 03:31:21,360 LIFE? 4483 03:31:21,360 --> 03:31:22,040 >>Glen Nuckolls: YEAH. 4484 03:31:22,040 --> 03:31:24,920 >>Diana Bianchi: THEME. 4485 03:31:24,920 --> 03:31:25,720 >>Glen Nuckolls: RIGHT. 4486 03:31:25,720 --> 03:31:29,880 >>Diana Bianchi: OKAY. 4487 03:31:29,880 --> 03:31:31,400 >>Glen Nuckolls: SO, IF I COULD 4488 03:31:31,400 --> 03:31:33,760 TAKE A MINUTE AND COMMENT THAT I 4489 03:31:33,760 --> 03:31:35,680 THINK WITH THE REALLY GREAT 4490 03:31:35,680 --> 03:31:38,640 TALKS THAT WE'VE HEARD THIS 4491 03:31:38,640 --> 03:31:40,560 AFTERNOON, IT'S CLEAR THAT THERE 4492 03:31:40,560 --> 03:31:45,600 ARE VERY STRONG EFFORTS IN 4493 03:31:45,600 --> 03:31:46,600 VARIOUS DIFFERENT MUSCULAR 4494 03:31:46,600 --> 03:31:48,480 DYSTROPHIES TO REACH OUT TO 4495 03:31:48,480 --> 03:31:51,800 EXPERTS AND IDENTIFY GAPS AND 4496 03:31:51,800 --> 03:31:52,480 OPPORTUNITIES. 4497 03:31:52,480 --> 03:31:54,960 IN THE RENEWAL OF THE ACTION 4498 03:31:54,960 --> 03:31:56,400 PLAN, FIRST OF ALL, THE PEOPLE 4499 03:31:56,400 --> 03:31:58,080 THAT WE WOULD GET TO PARTICIPATE 4500 03:31:58,080 --> 03:32:00,680 IN THAT ARE PROBABLY THE SAME 4501 03:32:00,680 --> 03:32:02,600 PEOPLE THAT ARE ALREADY 4502 03:32:02,600 --> 03:32:04,320 PARTICIPATING IN 4503 03:32:04,320 --> 03:32:05,920 DISEASE-SPECIFIC GAPS AND 4504 03:32:05,920 --> 03:32:07,080 OPPORTUNITIES IDENTIFICATION AND 4505 03:32:07,080 --> 03:32:09,000 THAT WE'RE NOT GOING TO FIND NEW 4506 03:32:09,000 --> 03:32:10,760 GAPS AND OPPORTUNITIES FOR 4507 03:32:10,760 --> 03:32:12,160 INDIVIDUAL DISEASES AND SO WHERE 4508 03:32:12,160 --> 03:32:14,320 WE REALLY DO NEED TO BE FOCUSING 4509 03:32:14,320 --> 03:32:17,480 THE EFFORT OF THE RENEWAL OF THE 4510 03:32:17,480 --> 03:32:19,400 ACTION PLAN, IS THAT CROSS 4511 03:32:19,400 --> 03:32:24,560 CUTTING ASPECT AND IT'S WHAT ARE 4512 03:32:24,560 --> 03:32:27,280 THE ABOVE LEVEL GAPS AND 4513 03:32:27,280 --> 03:32:29,080 OPPORTUNITIES THAT WOULD HAVE 4514 03:32:29,080 --> 03:32:34,920 THE MOST EFFECT ON PULL POLL 4515 03:32:34,920 --> 03:32:39,480 DYSTROPHIES. 4516 03:32:39,480 --> 03:32:44,800 WE HAVE WORKING GROUPS AND WHO 4517 03:32:44,800 --> 03:32:48,320 HAVE ALREADY CONTRIBUTED TO 4518 03:32:48,320 --> 03:32:52,200 STRATEGIC PLANS FOR INDIVIDUAL 4519 03:32:52,200 --> 03:32:53,800 TYPES AND IT CAN CUT ACROSS TO 4520 03:32:53,800 --> 03:32:58,240 ANOTHER TYPE OF DYSTROPHY. 4521 03:32:58,240 --> 03:32:58,840 >>Diana Bianchi: YEAH, I 4522 03:32:58,840 --> 03:32:59,320 DEFINITELY AGREE. 4523 03:32:59,320 --> 03:33:04,280 WE USE THE CONCEPT IN OUR NICHD 4524 03:33:04,280 --> 03:33:06,160 STRATEGIC PLAN OF THE WEST SO 4525 03:33:06,160 --> 03:33:07,560 THERE WERE CERTAIN SCIENTIFIC 4526 03:33:07,560 --> 03:33:10,520 THINGS AND THE CROSS CUTTING 4527 03:33:10,520 --> 03:33:12,520 THINGS AS IF YOU WERE WEAVING A 4528 03:33:12,520 --> 03:33:14,680 PIECE OF FABRIC. 4529 03:33:14,680 --> 03:33:24,080 IT MIGHT BE HELPFUL TOO TO CROSS 4530 03:33:24,080 --> 03:33:25,800 FERTILIZE AND PUTTING THEM ON A 4531 03:33:25,800 --> 03:33:27,000 DIFFERENT GROUP SO THEY CAN BE 4532 03:33:27,000 --> 03:33:28,440 THINKING ABOUT WHAT IS THE SAME 4533 03:33:28,440 --> 03:33:31,440 AND WHAT IS DIFFERENT FOR THIS 4534 03:33:31,440 --> 03:33:32,840 OTHER MUSCULAR DYSTROPHY THEY'RE 4535 03:33:32,840 --> 03:33:43,320 NOT A SUBJECT MATTER EXPERT. 4536 03:33:47,960 --> 03:33:49,520 WE'RE GETTING NEAR 5:00 WHICH IS 4537 03:33:49,520 --> 03:33:50,600 A GREAT OPPORTUNITY FOR PEOPLE 4538 03:33:50,600 --> 03:33:52,280 TO SPEAK UP IF THERE'S ANYTHING 4539 03:33:52,280 --> 03:33:56,200 ELSE THAT THEY WOULD LIKE TO 4540 03:33:56,200 --> 03:34:01,840 ADD. 4541 03:34:01,840 --> 03:34:04,840 I THINK IT'S BEEN VERY 4542 03:34:04,840 --> 03:34:05,880 INTERESTING AND ENGAGING 4543 03:34:05,880 --> 03:34:06,440 MEETING. 4544 03:34:06,440 --> 03:34:09,600 I'M VERY EXCITING ABOUT 4545 03:34:09,600 --> 03:34:11,040 PARTICIPATING IN THE NEW 4546 03:34:11,040 --> 03:34:14,280 STRATEGIC PLAN AND REALLY 4547 03:34:14,280 --> 03:34:15,640 REFRESHING GOALS AND THE 4548 03:34:15,640 --> 03:34:18,200 OBJECTIVES AND REALLY 4549 03:34:18,200 --> 03:34:19,960 INCORPORATING THEM AND THE 4550 03:34:19,960 --> 03:34:22,080 SCIENCE THAT HAS ADVANCED IN THE 4551 03:34:22,080 --> 03:34:23,680 LAST 10 YEARS AND MAKING 4552 03:34:23,680 --> 03:34:24,600 PREDICTIONS ABOUT WHERE WE WOULD 4553 03:34:24,600 --> 03:34:29,440 BE IN 2035 AND IS IS HE THIS A 4554 03:34:29,440 --> 03:34:31,640 GREAT START TO THAT DISCUSSION 4555 03:34:31,640 --> 03:34:34,640 AND WE'RE GOING TO BALANCE 4556 03:34:34,640 --> 03:34:36,120 WHATEVER SOCIETIES THAT HAVE 4557 03:34:36,120 --> 03:34:38,040 ALSO DONE SOME STRATEGIC 4558 03:34:38,040 --> 03:34:40,040 PLANNING WITH AGAIN THE 4559 03:34:40,040 --> 03:34:43,200 DISCUSSION OF WHETHER TO INCLUDE 4560 03:34:43,200 --> 03:34:45,880 NEWBORN SCREENING FOR DUCHENE 4561 03:34:45,880 --> 03:34:47,080 MUSCULAR DYSTROPHIES AS PART OF 4562 03:34:47,080 --> 03:34:49,040 THE RECOMMENDED UNIFORM 4563 03:34:49,040 --> 03:34:51,720 SCREENING PANELS SO WE LOOK 4564 03:34:51,720 --> 03:34:54,200 FORWARD TO THAT DISCUSSION. 4565 03:34:54,200 --> 03:34:56,720 I'D LIKE TO THANK ALL THE 4566 03:34:56,720 --> 03:35:00,760 PRESENTERS AS WELL AS THE EMPTY 4567 03:35:00,760 --> 03:35:03,800 CCT TEAM PARTICULARLY GLEN WHO 4568 03:35:03,800 --> 03:35:09,040 WAS SO ORGANIZED AND KEEPS US ON 4569 03:35:09,040 --> 03:35:10,320 TRACK AS WELL AS THE I.T. 4570 03:35:10,320 --> 03:35:11,480 SUPPORT SEW WITH THAT I'M GOING 4571 03:35:11,480 --> 03:35:14,240 TO HAND IT BACK OVER TO GLEN FOR 4572 03:35:14,240 --> 03:35:17,880 SOME FINAL COMMENTS AND THEN WE 4573 03:35:17,880 --> 03:35:21,720 WILL CALL IT THE END OF THE 4574 03:35:21,720 --> 03:35:23,760 MEETING. 4575 03:35:23,760 --> 03:35:25,040 >>Glen Nuckolls: THANK YOU FOR 4576 03:35:25,040 --> 03:35:28,000 MANAGING A COMPLICATED HYBRID 4577 03:35:28,000 --> 03:35:30,760 MEETING WITH UNTIL THE POLL 4578 03:35:30,760 --> 03:35:35,400 TMULTIPLE THREADS OFCONVERSATIOE 4579 03:35:35,400 --> 03:35:35,680 CHAT. 4580 03:35:35,680 --> 03:35:37,600 IF IT WORKS WELL FOR PEOPLE'S 4581 03:35:37,600 --> 03:35:39,200 SCHEDULES IS FOR THE FALL 4582 03:35:39,200 --> 03:35:42,680 MEETING TO TRY AND GO BACK TO A 4583 03:35:42,680 --> 03:35:46,480 FULL DAY IN-PERSON MEETING OR 4584 03:35:46,480 --> 03:35:47,440 PREDOMINANTLY IN-PERSON. 4585 03:35:47,440 --> 03:35:52,680 I SEE THE VALUE OF PEOPLE COMING 4586 03:35:52,680 --> 03:35:54,080 TOGETHER AND HAVING SIDE BAR 4587 03:35:54,080 --> 03:35:55,360 CONVERSATIONS AT BREAK TIMES AND 4588 03:35:55,360 --> 03:35:56,720 STUFF LIKE THAT AND IT SOUNDS 4589 03:35:56,720 --> 03:35:58,960 LIKE WE'VE GOT A COUPLE OF 4590 03:35:58,960 --> 03:36:00,720 PRETTY BEEFY TOPICS TO DEAL WITH 4591 03:36:00,720 --> 03:36:02,760 FOR THE FALL MEETING AND NEWBORN 4592 03:36:02,760 --> 03:36:04,920 SCREENING AND THEN A NUMBER OF 4593 03:36:04,920 --> 03:36:06,440 OTHER ADVOCACY GROUPS THAT MAY 4594 03:36:06,440 --> 03:36:10,120 WANT TO TELL US ABOUT THEIR 4595 03:36:10,120 --> 03:36:10,520 STRATEGIC PLAN. 4596 03:36:10,520 --> 03:36:11,560 WE HAVE SOME IDEAS FOR 4597 03:36:11,560 --> 03:36:13,360 ADDITIONAL TOPICS BEYOND THAT SO 4598 03:36:13,360 --> 03:36:15,080 WE MIGHT HAVE TO DO SOME 4599 03:36:15,080 --> 03:36:15,600 WEEDING. 4600 03:36:15,600 --> 03:36:16,880 SO THAT'S OUR THINKING AT THIS 4601 03:36:16,880 --> 03:36:19,480 TIME IS TO TRY AND HAVE, GO BACK 4602 03:36:19,480 --> 03:36:23,200 TO A PREDOMINANTLY IN-PERSON 4603 03:36:23,200 --> 03:36:28,320 9:00 A.M. TO 4:00 P.M. MEETING 4604 03:36:28,320 --> 03:36:30,520 HERE AROUND BETHESDA FOR THE 4605 03:36:30,520 --> 03:36:31,280 FALL. 4606 03:36:31,280 --> 03:36:33,000 HOPE THAT EVERYONE IS ABLE TO 4607 03:36:33,000 --> 03:36:36,520 COME AND PARTICIPATE IN THAT. 4608 03:36:36,520 --> 03:36:40,240 I SEE ONE THUMBS UP FOR THAT. 4609 03:36:40,240 --> 03:36:41,560 SO, YEAH, I AGREE. 4610 03:36:41,560 --> 03:36:43,680 THANK YOU TO ALL THE GREAT 4611 03:36:43,680 --> 03:36:45,440 PRESENTATIONS TODAY. 4612 03:36:45,440 --> 03:36:47,320 AND ALL THE HELP THAT I HAD FROM 4613 03:36:47,320 --> 03:36:48,240 VARIETY OF PEOPLE TRYING TO 4614 03:36:48,240 --> 03:36:51,560 ORGANIZE THIS MEETING. 4615 03:36:51,560 --> 03:36:52,080 >>Diana Bianchi: GREAT. 4616 03:36:52,080 --> 03:36:54,120 SO WITH THAT, WE'RE GOING TO LET 4617 03:36:54,120 --> 03:36:55,880 YOU ENJOY THIS BEAUTIFUL DAY AND 4618 03:36:55,880 --> 03:37:00,840 IT LOOKS LIKE IT'S 77° IN 4619 03:37:00,840 --> 03:37:02,320 BETHESDA OUTSIDE SO -- 4620 03:37:02,320 --> 03:37:03,000 >>THANK YOU. 4621 03:37:03,000 --> 03:37:04,800 >>TAKE CARE EVERYBODY. 4622 03:37:04,800 --> 00:00:00,000 >>THANK YOU.