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2022 Annual Meeting | S29 - Preclinical and Observational Studies of Neuromuscular Diseases

Wednesday 04/06/22
01:00 PM - 03:00 PM PDT Add To Calendar
This program will be presented both in-person and online
This program will be available in the meeting's On Demand product.
Scientific Platform Session
Neuromuscular and Clinical Neurophysiology (EMG)
2.00 CME credits

Program Materials Program Evaluations

Event Timeline
01:00 PM - 01:00 PM PDT Speaker Walk-in Slides
Erin Jackson
01:00 PM - 01:12 PM PDT Abstract 001 - PROMISE-MG: A Multicenter Prospective Observational Comparative Effectiveness Study of Myasthenia Gravis Treatments
Pushpa Narayanaswami, MBBS, MD, FAAN
01:12 PM - 01:24 PM PDT Abstract 002 - Preclinical Pharmacology and Toxicology of intrathecally infused AAV9-hABCD1, a gene therapy candidate for AMN, in Non-Human Primates
Vidyullatha Vasireddy, PhD
01:24 PM - 01:36 PM PDT Abstract 003 - INSPIRE-IBM: An NIH-funded, two-year, multicenter, observational study in inclusion body myositis (IBM)
Marie Wencel, CCRP
01:48 PM - 02:00 PM PDT Abstract 005 - Repeat expansion size predicts age of onset in RFC1 CANVAS and disease spectrum
Andrea *Use 181908 Cortese, Md, PhD
02:00 PM - 02:12 PM PDT Abstract 006 - Inflammasome inhibitors for the treatment of muscular dystrophies
Nicolas Dubuisson, MD
02:12 PM - 02:24 PM PDT Abstract 007 - Motor Outcomes to Validate Evaluations in Facioscapulohumeral muscular dystrophy (MOVE FSHD): Protocol for an observational study.
Michaela Walker, MPH, CCRP
02:24 PM - 02:36 PM PDT Abstract 008 - Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): Baseline characteristics
Jeffrey Statland, MD
02:36 PM - 02:48 PM PDT Abstract 009 - Bioassay-confirmed Pathogenic De Novo ATP1A1 Variants Cause a Complex Neurodevelopmental Syndrome
Maike Dohrn, MD
02:48 PM - 03:00 PM PDT Abstract 010 - Ataxia And Cough Are Indicative For Biallelic RFC1 Repeat Expansions In Hereditary Sensory And Autonomic Neuropathy
Danique Beijer, PhD
Faculty Disclosures
Charlotte J. Sumner, MD, FAAN Dr. Sumner has received personal compensation for serving as an employee of Biogen. Dr. Sumner has received personal compensation for serving as an employee of Avexis. Dr. Sumner has received personal compensation for serving as an employee of Roche/Genentech. Dr. Sumner has received personal compensation for serving as an employee of Ionis Pharmaceuticals. Dr. Sumner has received personal compensation for serving as an employee of Sarepta. Dr. Sumner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Sumner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avexis/Novartis. Dr. Sumner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche/Genentech. Dr. Sumner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Sumner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Sumner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Proneurotech. Dr. Sumner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CMTRF. Dr. Sumner has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Journal Clinical Investigation. Dr. Sumner has received research support from Roche. Dr. Sumner has received intellectual property interests from a discovery or technology relating to health care. Dr. Sumner has received publishing royalties from a publication relating to health care.
Mary Reilly, MD, FRCP, FRCPI The institution of Mary Reilly, MD, FRCP, FRCPI has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alynlam. Mary Reilly, MD, FRCP, FRCPI has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AKCEA. Mary Reilly, MD, FRCP, FRCPI has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Inflectis. The institution of Mary Reilly, MD, FRCP, FRCPI has received research support from MRC. The institution of Mary Reilly, MD, FRCP, FRCPI has received research support from NIH. The institution of Mary Reilly, MD, FRCP, FRCPI has received research support from MDA.
Camille Selvais Miss Selvais has nothing to disclose.
Karen Kozarsky, PhD Dr. Kozarsky has received personal compensation for serving as an employee of SwanBio Therapeutics. Dr. Kozarsky has received stock or an ownership interest from SwanBio Therapeutics.
Charles Karan, Jr., PhD Dr. Karan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Darwin Health. Dr. Karan has received stock or an ownership interest from Darwin Health. The institution of Dr. Karan has received research support from Bristol Myers Squibb.
Richard Roxburgh, MD, FRACP, PhD Dr. Roxburgh has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Roxburgh has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CHDI. The institution of Dr. Roxburgh has received research support from Biogen. The institution of Dr. Roxburgh has received research support from Roche.
Siddharth Srivastava, MD Dr. Srivastava has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint.
Jaya Raghav Trivedi, MD, FAAN Dr. Trivedi has nothing to disclose.
Henry Houlden Henry Houlden has nothing to disclose.
Tanya Stojkovic, MD Dr. STOJKOVIC has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for BIOGEN. Dr. STOJKOVIC has received research support from BIOGEN. Dr. STOJKOVIC has received research support from SANOFI. An immediate family member of Dr. STOJKOVIC has received research support from Ultragenyx. Dr. STOJKOVIC has received research support from LFB. Dr. STOJKOVIC has received research support from CSL.
Rishi Desai No disclosure on file
Fan Li No disclosure on file
David William Anderson, PhD Dr. Anderson has received personal compensation for serving as an employee of SwanBio Therapeutics Inc. Dr. Anderson has received personal compensation for serving as an employee of Spark Therapeutics Inc. An immediate family member of Dr. Anderson has received personal compensation for serving as an employee of GSK. An immediate family member of Dr. Anderson has received personal compensation for serving as an employee of Novartis. Dr. Anderson has stock in SwanBio Therapeutics Inc. An immediate family member of Dr. Anderson has stock in GSK. Dr. Anderson has stock in Roche . An immediate family member of Dr. Anderson has stock in Novartis.
Valeria A. Sansone, MD Dr. Sansone has nothing to disclose.
Rinze F Neuteboom The institution of Rinze F Neuteboom has received research support from MS research foundation The Netherlands.
Danique Beijer, PhD Dr. Beijer has nothing to disclose.
Maria America Davis-López de Carrizosa, PhD Dr. Davis-López de Carrizosa has nothing to disclose.
William Martens William Martens has nothing to disclose.
Jonathan Baets Jonathan Baets has nothing to disclose.
Romain Versèle, PhD Dr. Versèle has nothing to disclose.
Sonia Brichard Prof. Brichard has nothing to disclose.
Fiore Manganelli Fiore Manganelli has nothing to disclose.
Alka Malhotra, PhD Dr. Malhotra has received personal compensation for serving as an employee of Illumina, Inc. Dr. Malhotra has received stock or an ownership interest from Illumina, Inc.
Michel Abou Samra, PhD Dr. SAMRA has nothing to disclose.
Donald B. Sanders, MD, FAAN Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Accordant Health Services. Dr. Sanders has received personal compensation in the range of $0-$499 for serving as a Consultant for Cabaletta BIO. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kashiv Biosciences. Dr. Sanders has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Momenta Pharmaceuticals. Dr. Sanders has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen RnD. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi-aventis. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for VielaBio. Dr. Sanders has received publishing royalties from a publication relating to health care.
Perry Shieh, MD, PhD, FAAN Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for CSL Behring. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Grifols. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Shieh has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Biogen. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Genentech. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Catalyst.
Seth Friedman Seth Friedman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Fulcrum Therapeutics. Seth Friedman has received research support from NIH.
Mark Emory Cartwright, PhD,Other Dr. Cartwright has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for SwanBio.
Franco Taroni, MD The institution of Dr. Taroni has received research support from Italian Ministry of Health. The institution of Dr. Taroni has received research support from Fondazione Regionale per la Ricerca Biomedica.
Nicolas Dubuisson, MD Dr. Dubuisson has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for ArgenX. The institution of Dr. Dubuisson has received research support from Le Fonds de la Recherche Scientifique (FNRS).
Peter Lawrence Jones, PhD Dr. Jones has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Fulcrum Therapeutics. Dr. Jones has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Jones has stock in Fulcrum Therapeutics. Dr. Jones has stock in Renogenyx, Inc. An immediate family member of Dr. Jones has stock in Renogenyx, Inc. The institution of Dr. Jones has received research support from FSHD Canada Research Foundation. The institution of Dr. Jones has received research support from Friends of FSH Research. The institution of Dr. Jones has received research support from The Chris Carrino Foundation for FSHD. The institution of Dr. Jones has received research support from FSHD Global Research Foundation. The institution of Dr. Jones has received research support from The National Institutes of Health. Dr. Jones has received intellectual property interests from a discovery or technology relating to health care. Dr. Jones has received intellectual property interests from a discovery or technology relating to health care. Dr. Jones has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Jones has received intellectual property interests from a discovery or technology relating to health care.
Nicholas Elwood Johnson, MD, FAAN Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta Therapuetics. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne Therapeutics. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharma. Dr. Johnson has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for ML BIo . Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity. Dr. Johnson has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Arthex. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Juvena. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Entrada. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Rgenta. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Angle Therapeutics. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AskBio. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum. Dr. Johnson has stock in Angle Therapeutics. Dr. Johnson has stock in Juvena Therapeutics. The institution of Dr. Johnson has received research support from Novartis. The institution of Dr. Johnson has received research support from Takeda . The institution of Dr. Johnson has received research support from AMO Pharma. The institution of Dr. Johnson has received research support from Sarepta. The institution of Dr. Johnson has received research support from Dyne Therapeutics. The institution of Dr. Johnson has received research support from AskBio. The institution of Dr. Johnson has received research support from Pepgen. The institution of Dr. Johnson has received research support from ML Bio. The institution of Dr. Johnson has received research support from Vertex. The institution of Dr. Johnson has received research support from Arthex. Dr. Johnson has received intellectual property interests from a discovery or technology relating to health care.
S H. Subramony, MBBS, FAAN Dr. Subramony has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity. Dr. Subramony has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne. Dr. Subramony has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lupin. Dr. Subramony has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Fallonmedica. The institution of Dr. Subramony has received research support from Reata. The institution of Dr. Subramony has received research support from Retrotope. The institution of Dr. Subramony has received research support from Acceleron. The institution of Dr. Subramony has received research support from Biohaven. The institution of Dr. Subramony has received research support from Pharnext. The institution of Dr. Subramony has received research support from Fulcrum. The institution of Dr. Subramony has received research support from National Ataxia Foundation. The institution of Dr. Subramony has received research support from Friedreich Ataxia Research Alliance. The institution of Dr. Subramony has received research support from Muscular Dystrophy Association. The institution of Dr. Subramony has received research support from Univ of Rochester, MDA. The institution of Dr. Subramony has received research support from Virginia Commonwealth Univ (FDA, Wyck Foundation)). The institution of Dr. Subramony has received research support from Children's Hospital, Philadelphia (FDA). The institution of Dr. Subramony has received research support from Houston Methodist (NIH). The institution of Dr. Subramony has received research support from NIHR01 AR076060-01A1 . The institution of Dr. Subramony has received research support from NIH2R42HD089804-04 . The institution of Dr. Subramony has received research support from NIH R01 AR056973 . The institution of Dr. Subramony has received research support from FSHD Society. The institution of Dr. Subramony has received research support from AAVANTI BIO. The institution of Dr. Subramony has received research support from COHAV FL State Dept of Health.
Stephan Zuchner, MD, FAAN Dr. Zuchner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Applied Therapeutics. The institution of Dr. Zuchner has received research support from Muscular Dystrophy Association. The institution of Dr. Zuchner has received research support from CMT Association. Dr. Zuchner has received intellectual property interests from a discovery or technology relating to health care.
Robert Smigiel, MD Prof. Smigiel has nothing to disclose.
Pushpa Narayanaswami, MBBS, MD, FAAN Dr. Narayanaswami has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx USA. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Muscle and Nerve, Wiley. Dr. Narayanaswami has stock in Moderna. Dr. Narayanaswami has stock in Dr. Reddys laboratories. Dr. Narayanaswami has stock in Pfizer. Dr. Narayanaswami has stock in Viatris. Dr. Narayanaswami has stock in Doximity. The institution of Dr. Narayanaswami has received research support from Alexion. Dr. Narayanaswami has a non-compensated relationship as a Member, Medical and Scientific Advisory Board with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Narayanaswami has a non-compensated relationship as a Member, Board of Directors with AANEM that is relevant to AAN interests or activities.
Nigel G. Laing Nigel G. Laing has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elslevier. The institution of Nigel G. Laing has received research support from Australian National Health and Medical Research Council. The institution of Nigel G. Laing has received research support from Australian Medical Research Future Fund. Nigel G. Laing has received publishing royalties from a publication relating to health care.
Peter Y.K. Van Den Bergh, MD, PhD, FAAN Dr. Van den Bergh has nothing to disclose.
Katy Eichinger, PT,PhD, DPT, NCS Dr. Eichinger has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Fulcrum. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Avidity. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for DyneTherapeutic. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Eichinger has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for FSHD Society. The institution of Dr. Eichinger has received research support from Charcot Marie Tooth Association.
Leo Hong-Li Wang, MD, PhD, FAAN Dr. Wang has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AskBio. Dr. Wang has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Fulcrum Therapeutics. Dr. Wang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Wang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe. Dr. Wang has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Wang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity. Dr. Wang has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. The institution of Dr. Wang has received research support from National Institute of Health. Dr. Wang has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant with US Government.
Peter De Jonghe, MD,PhD Prof. De Jonghe has nothing to disclose.
Andrea *Use 181908 Cortese, Md, PhD Dr. Cortese has nothing to disclose.
Kiley Higgs Kiley Higgs has nothing to disclose.
Jurgen Venitz No disclosure on file
Chiara Briani, MD Dr. Briani has nothing to disclose.
Riccardo Curro, MD Dr. Curro has nothing to disclose.
Gorka Fernandez-Eulate, MD Dr. Fernandez-Eulate has received publishing royalties from a publication relating to health care.
Jeffrey Statland, MD Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MT Pharma. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA.
FILIPPO M. SANTORELLI, MD,PhD Dr. SANTORELLI has nothing to disclose.
on Behalf of the RFC Study Group No disclosure on file
Dennis W. Shaw, MD The institution of Dennis W. Shaw, MD has received research support from Friends of FSHD Research.
Gabriella Silvestri, MD Prof. Silvestri has nothing to disclose.
Matthis Synofzik Matthis Synofzik has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis Pharmaceuticals. Matthis Synofzik has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen Pharmaceuticals. Matthis Synofzik has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Orphayzme Pharmaceuticals. The institution of Matthis Synofzik has received research support from EJPRD.
Annette *use 386619 Hartmann No disclosure on file
Jonathan *Use 391772 De Winter Mr. De Winter has nothing to disclose.
Davide Pareyson, MD No disclosure on file
Kathie Marie Bibeau Mrs. Bibeau has nothing to disclose.
Nicola Brunetti Pierri, MD Prof. Pierri has nothing to disclose.
Vidyullatha Vasireddy, PhD Dr. Vasireddy has stock in SwanBio Therapeutics.
Elena Carraro No disclosure on file
Leann Lewis Leann Lewis has nothing to disclose.
Maike Dohrn, MD Dr. Dohrn has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea, Alnylam, Pfizer, Amicus. Dr. Dohrn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus, Akcea. Dr. Dohrn has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Akcea, Alnylam, Pfizer. The institution of Dr. Dohrn has received research support from Pfizer. Dr. Dohrn has received research support from German Research Foundation.
Sean W. Clark, PhD Dr. Clark has received personal compensation for serving as an employee of SwanBio Therapeutics. Dr. Clark has received personal compensation for serving as an employee of Amicus Therapeutics. Dr. Clark has received stock or an ownership interest from Amicus Therapeutics. Dr. Clark has received intellectual property interests from a discovery or technology relating to health care. Dr. Clark has received intellectual property interests from a discovery or technology relating to health care.
Natalia Dominik Miss Dominik has nothing to disclose.
Russell Butterfield, MD, PhD, BS 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 Avexis. 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 Biogen. 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 Aavanti Bio. 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 FSHD Society. The institution of Dr. Butterfield has received research support from NIH. The institution of Dr. Butterfield has received research support from Ionis.
Karlien Mul The institution of Karlien Mul has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity Biosciences.
Laurence Noel, Other Mrs. Noel has nothing to disclose.
Irina Utkina-Susunova Irina Utkina-Susunova has nothing to disclose.
Donald Basel, MBBS Prof. Basel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for IQVIA. Prof. Basel has received publishing royalties from a publication relating to health care.
Adriana Rebelo Adriana Rebelo has nothing to disclose.
Doris G. Leung, MD The institution of Dr. Leung has received research support from US Department of Defense. The institution of Dr. Leung has received research support from Fulcrum Therapeutics. The institution of Dr. Leung has received research support from ML Bio Solutions, Inc.. The institution of Dr. Leung has received research support from Sarepta Therapeutics. The institution of Dr. Leung has received research support from Cumberland Pharmaceuticals. The institution of Dr. Leung has received research support from FibroGen Inc.. The institution of Dr. Leung has received research support from Astellas Pharma. The institution of Dr. Leung has received research support from University of Kansas Medical Center Research Institute. The institution of Dr. Leung has received research support from Pfizer, Inc.. The institution of Dr. Leung has received research support from Genethon. The institution of Dr. Leung has received research support from Harmony Biosciences. The institution of Dr. Leung has received research support from Edgewise Therapeutics.
Marie Wencel, CCRP Ms. Wencel has nothing to disclose.
PROMISE-MG Study Group No disclosure on file
Gauthier Remiche, MD Dr. Remiche has nothing to disclose.
Marina Kennerson Marina Kennerson has nothing to disclose.
Sabrina Sacconi, MD Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for LUPIN. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SANOFI. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for BIOGEN. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AMICUS. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Pharma. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ALEXION. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for FULCRUM THERAPEUTICS. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for AXELYS. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for OLOGY. The institution of Dr. Sacconi has received research support from Solve FSHD.
Bo Liu, Other Mr. Liu has nothing to disclose.
Bakri Elsheikh, MD, FAAN Dr. Elsheikh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen . Dr. Elsheikh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argnex . The institution of Dr. Elsheikh has received research support from Biogen. The institution of Dr. Elsheikh has received research support from Cure SMA.
Sarah Fazal Ms. Fazal has nothing to disclose.
Hai Li, PhD Dr. Li has nothing to disclose.
Gerarda Cappuccio, MD,PhD Dr. Cappuccio has nothing to disclose.
Nazli Basak Nazli Basak has nothing to disclose.
Namita Goyal, MD, FAAN Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genzyme. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB . Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astrazenica. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kezar. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abcuro. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL Behring. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx. The institution of Dr. Goyal has received research support from Brainstorm Cell Therapeutics. The institution of Dr. Goyal has received research support from Cytokinetics. The institution of Dr. Goyal has received research support from Fulcrum. The institution of Dr. Goyal has received research support from Kezar. The institution of Dr. Goyal has received research support from Alexion. The institution of Dr. Goyal has received research support from Annexon. The institution of Dr. Goyal has received research support from Annelixis. The institution of Dr. Goyal has received research support from Healey Platform. The institution of Dr. Goyal has received research support from Medicinova. The institution of Dr. Goyal has received research support from Amylyx. The institution of Dr. Goyal has received research support from MT Pharma. The institution of Dr. Goyal has received research support from Transposon. Dr. Goyal has received publishing royalties from a publication relating to health care.
John W. Day, MD, PhD Dr. Day has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Affinia Therapeutics. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapy. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche/Genentech. Dr. Day 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. Day 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. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for Muscular Dystrophy Association. The institution of Dr. Day has received research support from Audentes. The institution of Dr. Day has received research support from Novartis Gene Therapy. The institution of Dr. Day has received research support from Biogen. The institution of Dr. Day has received research support from Cytokinetics. The institution of Dr. Day has received research support from Roche/Genentech. The institution of Dr. Day has received research support from Sanofi/Genzyme. The institution of Dr. Day has received research support from Sarepta. The institution of Dr. Day has received research support from Scholar Rock. Dr. Day has received intellectual property interests from a discovery or technology relating to health care.
Jon Infante Ceberio No disclosure on file
Matthew P. Wicklund, MD, FAAN Dr. Wicklund has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus Therapeutics. Dr. Wicklund has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi-Genzyme. Dr. Wicklund 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 Therapeutics. Dr. Wicklund has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Spark Therapeutics. Dr. Wicklund has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ultragenyx. Dr. Wicklund has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex Pharmaceuticals. The institution of Dr. Wicklund has received research support from Orphazyme. The institution of Dr. Wicklund has received research support from Muscular Dystrophy Association. The institution of Dr. Wicklund has received research support from Coalition to Cure Calpain 3. The institution of Dr. Wicklund has received research support from Sarepta Therapeutics. The institution of Dr. Wicklund has received research support from ML Bio. The institution of Dr. Wicklund has received research support from Alexion. The institution of Dr. Wicklund has received research support from Acceleron. The institution of Dr. Wicklund has received research support from Edgewise. The institution of Dr. Wicklund has received research support from Fulcrum. The institution of Dr. Wicklund has received research support from Roche-Genentech. The institution of Dr. Wicklund has received research support from Avidity. Dr. Wicklund has received research support from Harmony Bioscience.
Anna Faino Anna Faino has nothing to disclose.
Michaela Walker, MPH, CCRP Miss Walker has nothing to disclose.
Serge Przedborski, MD, PhD Dr. Przedborski has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for eLife. The institution of Dr. Przedborski has received research support from NIH. The institution of Dr. Przedborski has received research support from DoD. The institution of Dr. Przedborski has received research support from Ludwig Foudantion. The institution of Dr. Przedborski has received research support from CZI.
Ingrid M.B. Van de Laar No disclosure on file
Alrabi Tawil, MD, FAAN Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving as a Consultant for DYNE Therapeutics. Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead Pharma. Dr. Tawil has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Acceleron Pharma. Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MT Pharma. Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche Pharma. Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for miRecule. The institution of Dr. Tawil has received research support from Friends of FSH Research. The institution of Dr. Tawil has received research support from FSH Society. The institution of Dr. Tawil has received research support from NIH. The institution of Dr. Tawil has received research support from Fulcrum. Dr. Tawil has received intellectual property interests from a discovery or technology relating to health care. Dr. Tawil has received publishing royalties from a publication relating to health care.
Mattia Gentile, MD Dr. GENTILE has nothing to disclose.
Paola Mandich No disclosure on file
Manu Jokela Manu Jokela has nothing to disclose.
Alfredo Brusco Alfredo Brusco has nothing to disclose.
Michael McDermott Michael McDermott has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NeuroDerm, Ltd.. Michael McDermott has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Eli Lilly and Company. Michael McDermott has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis Pharmaceuticals, Inc.. Michael McDermott has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vaccinex, Inc.. Michael McDermott has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurocrine Biosciences, Inc.. Michael McDermott has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Prilenia Therapeutics Development, Inc.. Michael McDermott has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ReveraGen BioPharma, Inc.. Michael McDermott has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NS Pharma, Inc.. The institution of Michael McDermott has received research support from NIH. The institution of Michael McDermott has received research support from FDA. The institution of Michael McDermott has received research support from Cure SMA.
Sinead Murphy Sinead Murphy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Sinead Murphy has received research support from Novartis.
Gian Maria Fabrizi No disclosure on file
Andrew C. Krueger Andrew C. Krueger has received personal compensation for serving as an employee of Accordant.
Jeff Guptill, MD, FAAN Dr. Guptill has received personal compensation for serving as an employee of argenx. Dr. Guptill has stock in argenx.
Marios Hadjivassiliou Marios Hadjivassiliou has nothing to disclose.
Samantha Jo LoRusso, MD Dr. LoRusso has nothing to disclose.
Tahseen Mozaffar, MD, FAAN Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Modis Therapeutics. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB/ Ra Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Maze Therapeutics. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Mozaffar 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. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for National Institutes of Health. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for Neuromuscular Disease Foundation. The institution of Dr. Mozaffar has received research support from NIH. The institution of Dr. Mozaffar has received research support from Muscular Dystrophy Association. The institution of Dr. Mozaffar has received research support from Sanofi-Genzyme. The institution of Dr. Mozaffar has received research support from Argenx. The institution of Dr. Mozaffar has received research support from Amicus Therapeutics. The institution of Dr. Mozaffar has received research support from Spark Therapeutics. The institution of Dr. Mozaffar has received research support from Audentes Therapeutics. The institution of Dr. Mozaffar has received research support from Cartesian. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.
The INSPIRE-IBM Study Group No disclosure on file
Filippo Santorelli, MD No disclosure on file
Conrad Weihl, MD, PhD Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Casma therapeutics. Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abata. Dr. Weihl 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. Weihl 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. Weihl has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Acceleron. Dr. Weihl has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Wilson Elser Moskowitz Edelman & Dicker LLP.
Elisa Vegezzi, MD Dr. Vegezzi has nothing to disclose.
Mamatha Pasnoor, MD, FAAN Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx BVBA. Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda Pharmaceuticals. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen Pharmaceutical. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for catalyst pharmaceuticals. Dr. Pasnoor has received research support from NIH.
Erin Jackson Ms. Jackson has nothing to disclose.