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Abstract Details

Deramiocel Significantly Slows Upper Limb Functional Decline in Duchenne Muscular Dystrophy: Skeletal Muscle Outcomes from the Phase 3 HOPE-3 Trial
Child Neurology and Developmental Neurology
LS2 - Late-breaking Science 2 (5:57 PM-6:03 PM)
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Authors/Disclosures
Aravindhan Veerapandiyan, MD (Arkansas Childrens Hospital/UAMS)
PRESENTER
Dr. Veerapandiyan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen, Novartis,Edgewise Therapeutics, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Inc., UCB Pharma, Catalyst, Entrada, Lupin, Percheron, ITF. Dr. Veerapandiyan has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MedLink Neurology. Dr. Veerapandiyan has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Muscle and Nerve. The institution of Dr. Veerapandiyan has received research support from AMO Pharma, Capricor Therapeutics, Edgewise Therapeutics, FibroGen, Muscular Dystrophy Association, Novartis, Parent Project Muscular Dystrophy, Pfizer, RegenxBio, SolodBio and Sarepta Therapeutics. Dr. Veerapandiyan has received personal compensation in the range of $5,000-$9,999 for serving as a MD with PPMD, MDA.
Craig McDonald, MD (UC Davis Dept. of PM&R) Dr. McDonald has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Sarepta Therapeutics. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for PTC Therapeutics. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Solid Biosciences. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta Therapeutics. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Biosciences. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Edgewise Therapeutics. The institution of Dr. McDonald has received research support from Sarepta Therapeutics. The institution of Dr. McDonald has received research support from PTC Therapeutics. The institution of Dr. McDonald has received research support from Edgewise Therapeutics. The institution of Dr. McDonald has received research support from Capricor Therapeutics. The institution of Dr. McDonald has received research support from Italfarmaco. Dr. McDonald has received research support from NS Pharma. The institution of Dr. McDonald has received research support from NIH (NINDS). The institution of Dr. McDonald has received research support from Parent Project Muscular Dystrophy. The institution of Dr. McDonald has received research support from Muscular Dystrophy Association. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Member National Advisory Board for Medical Rehabilitation Research with NIH.
Susan T. Iannaccone, MD, FAAN (Department of Pediatrics) Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AveXis. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. The institution of Dr. Iannaccone has received research support from AveXis. The institution of Dr. Iannaccone has received research support from Biogen. The institution of Dr. Iannaccone has received research support from Sarepta. The institution of Dr. Iannaccone has received research support from PTC Therapeutics. The institution of Dr. Iannaccone has received research support from FibroGen. The institution of Dr. Iannaccone has received research support from ReveraGen. The institution of Dr. Iannaccone has received research support from MDA. The institution of Dr. Iannaccone has received research support from PPMD. The institution of Dr. Iannaccone has received research support from NIH. Dr. Iannaccone has received personal compensation in the range of $0-$499 for serving as a grant reviewer with NIH.
Arun Varadhachary, MD, PhD (Washington University in St Louis) Dr. Varadhachary has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Varadhachary has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Varadhachary has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. Dr. Varadhachary has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for legal firms .
Leigh Ramos-Platt, MD, MBA (USC/Children'S Hospital of Los Angeles) Dr. Ramos-Platt has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint. The institution of Dr. Ramos-Platt has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Ramos-Platt has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cure sma. Dr. Ramos-Platt has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Ramos-Platt has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Catalyst. Dr. Ramos-Platt has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MyTomorrows.
Katheryn Gambetta, MD Dr. Gambetta has nothing to disclose.
Russell Butterfield, MD, PhD, BS (University of Utah) Dr. Butterfield has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Butterfield has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Dr. Butterfield has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Reata. The institution of Dr. Butterfield has received research support from NIH. The institution of Dr. Butterfield has received research support from Ionis. The institution of Dr. Butterfield has received research support from CDC.
Mindy J. Leffler Mr. Leffler has received personal compensation for serving as an employee of Emmes. Mr. Leffler has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Capricor.
Kati Maharry, PhD Dr. Maharry has received personal compensation for serving as an employee of Capricor. Dr. Maharry has or had stock in Capricor.
Nathaniel J. Hogan, PhD Dr. Hogan has received personal compensation for serving as an employee of Capricor. Dr. Hogan has or had stock in Capricor.
Susan Apkon, MD Dr. Apkon has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Italifarmaco. Dr. Apkon has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Halls Booth Smith. The institution of Dr. Apkon has received research support from Capricor. The institution of Dr. Apkon has received research support from Dyne. The institution of Dr. Apkon has received research support from Sarepta.
Cuixia Tian, MD (Cincinnati Childrens Hospital Medical Center) Dr. Tian has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Tian has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Tian has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Itf. Dr. Tian has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regenxbio. Dr. Tian has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Bioscience. The institution of Dr. Tian has received research support from MDA. The institution of Dr. Tian has received research support from NIH.
Katherine D. Mathews, MD, FAAN (University of Iowa - Dept of Pediatrics) Dr. Mathews has received personal compensation for serving as an employee of Avidity Bioscience. The institution of Dr. Mathews has received research support from NIH. The institution of Dr. Mathews has received research support from Centers for Disease Control and Prevention. The institution of Dr. Mathews has received research support from Muscular Dystrophy Association . The institution of Dr. Mathews has received research support from Friedreich's Ataxia Research Alliance . The institution of Dr. Mathews has received research support from Sarepta . The institution of Dr. Mathews has received research support from Pfizer. The institution of Dr. Mathews has received research support from Reata . The institution of Dr. Mathews has received research support from PTC Therapeutics, Inc. The institution of Dr. Mathews has received research support from Italfarmaco . The institution of Dr. Mathews has received research support from AMO. The institution of Dr. Mathews has received research support from FibroGen. The institution of Dr. Mathews has received research support from Capricor. The institution of Dr. Mathews has received research support from edgewise. The institution of Dr. Mathews has received research support from biogen.
Nancy E. Bass, MD (Children's Wisconsin) Dr. Bass has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elseviert.
Chamindra G. Laverty, MD (UC San Diego) The institution of Dr. Laverty has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sarepta . Dr. Laverty has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Laverty has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Catalyst. Dr. Laverty has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Solid Biosciences. Dr. Laverty has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. The institution of Dr. Laverty has received research support from Sarepta. The institution of Dr. Laverty has received research support from Novartis Gene Therapies. The institution of Dr. Laverty has received research support from Italfarmaco. The institution of Dr. Laverty has received research support from Fibrogen. The institution of Dr. Laverty has received research support from Avidity. The institution of Dr. Laverty has received research support from Dyne.
Han Phan, MD (Rare Disease Research, LLC) Dr. Phan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Wave Biosciences. Dr. Phan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stealth.
Partha S. Ghosh, MD, FAAN (Boston Children'S Hospital) Dr. Ghosh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Ghosh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen.
Seth J. Perlman, MD (Seattle Children's Hospital) Dr. Perlman has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Perlman has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Biosciences. Dr. Perlman has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. The institution of Dr. Perlman has received research support from Scholar Rock. The institution of Dr. Perlman has received research support from Pfizer. The institution of Dr. Perlman has received research support from Sarepta. The institution of Dr. Perlman has received research support from ReveraGen. The institution of Dr. Perlman has received research support from FibroGen. The institution of Dr. Perlman has received research support from PTC Therapeutics. The institution of Dr. Perlman has received research support from CSL Behring. Dr. Perlman has a non-compensated relationship as a Registry Committee Member with Cure SMA that is relevant to AAN interests or activities. Dr. Perlman has a non-compensated relationship as a Assessment Program Pilot Committee Member with American Board of Psychiatry & Neurology that is relevant to AAN interests or activities.
Rebecca J. Scharf, MD Dr. Scharf has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for RegenXBio. The institution of Dr. Scharf has received research support from Capricor. The institution of Dr. Scharf has received research support from Biohaven. The institution of Dr. Scharf has received research support from Sarepta. The institution of Dr. Scharf has received research support from Genentech. The institution of Dr. Scharf has received research support from Novartis. The institution of Dr. Scharf has received research support from Biogen. The institution of Dr. Scharf has received research support from Catalyst.
Kathryn Mosher, MD Dr. Mosher has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Mosher has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Mosher has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Catalyst.
Saunder Bernes, MD (Phoenix Childrens Hospital) Dr. Bernes has nothing to disclose.
Jane Anne Emerson, MD Dr. Emerson has nothing to disclose.
Edward C. Smith, MD (Rare Disease Research - NC, LLC) Dr. Smith has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Avidity Biosciences. Dr. Smith has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Bioscences. Dr. Smith has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Catalyst.
Michael Binks, MD Dr. Binks has received personal compensation for serving as an employee of Capricor Therapeutics Inc.. The institution of Dr. Binks has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Capricor Therapeutics Inc.
Mark S. Awadalla Mr. Awadalla has received personal compensation for serving as an employee of Capricor.
Linda Marban, PhD Dr. Marban has received personal compensation for serving as an employee of Capricor Therapeutics. Dr. Marban has or had stock in Capricor Therapeutics.Dr. Marban has received intellectual property interests from a discovery or technology relating to health care.