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2020 Annual Meeting | S6 - Neuromuscular and Clinical Neurophysiology (EMG): Genetic Muscle Disorders

Sunday 04/26/20
01:00 PM - 03:00 PM Add To Calendar
This program will be presented both in-person and online
This program is not expected to be available in the meeting's On Demand product.
Scientific Platform Session
Suur Biliciler, MD, Ali A. Habib, MD
Neuromuscular and Clinical Neurophysiology (EMG)
2.00 CME credits
Event Timeline
01:00 PM - 01:12 PM Abstract 001 - ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment
Perry Shieh, MD, PhD, FAAN
01:12 PM - 01:24 PM Abstract 002 - NEO1 and NEO-EXT Studies: Long-Term Safety and Exploratory Efficacy of Repeat Avalglucosidase Alfa Dosing for 5.5 Years in Late-Onset Pompe Disease Patients
Mazen M. Dimachkie, MD, FAAN
01:24 PM - 01:36 PM Abstract 003 - Phase 1 Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD): Safety, Tolerability, and Target Engagement
Michelle L. Mellion, MD
01:36 PM - 01:48 PM Abstract 004 - Design of a Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD): ReDUX4
Alrabi Tawil, MD, FAAN
02:00 PM - 02:12 PM Abstract 006 - Delays in Progression of Duchenne Muscular Dystrophy with Eteplirsen Treatment: Attenuation of Pulmonary Function Decline and Projected Freedom from Continuous Ventilation
02:12 PM - 02:24 PM Abstract 007 - A Biomarker of DUX4 Activity to Evaluate losmapimod Treatment Effect in FSHD Phase 2 Trials
02:24 PM - 02:36 PM Abstract 008 - Thymidine Phosphorylase Intracellular Enzyme Replacement Therapy in a Murine Model of Mitochondrial Neurogastrointestinal Encephalopathy (MNGIE)
Jonathan Shintaku, PhD
02:48 PM - 03:00 PM Abstract 010 - Clinicogenetic Lessons from 370 Patients with Autosomal Recessive Limb-Girdle Muscular Dystrophy
Marcondes C. Franca, Jr., MD
Faculty Disclosures
Suur Biliciler, MD Dr. Biliciler has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Efficient CME. Dr. Biliciler has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenyx. Dr. Biliciler has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for UCB.
Ali A. Habib, MD Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech/Roche. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alpine Immune Sciences. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Arcellx. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizon/ Amgen. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunis Biomedical. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman-La Roche. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from Horizon/Amgen. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Genentech. The institution of Dr. Habib has received research support from Regeneron.
Jaya R. Trivedi, MD, FAAN The institution of Dr. Trivedi has received research support from Avidity.
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.
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 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 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 $5,000-$9,999 for serving on a Speakers Bureau for Argenx. The institution of Dr. Goyal has received research support from Fulcrum. The institution of Dr. Goyal has received research support from Annexon. 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 Transposon. The institution of Dr. Goyal has received research support from Abcuro. The institution of Dr. Goyal has received research support from Calico. The institution of Dr. Goyal has received research support from Clene. The institution of Dr. Goyal has received research support from Janssen. The institution of Dr. Goyal has received research support from PepGen. The institution of Dr. Goyal has received research support from PTC. The institution of Dr. Goyal has received research support from Roche. The institution of Dr. Goyal has received research support from Sanofi. Dr. Goyal has received publishing royalties from a publication relating to health care.
Diego Cadavid, MD, FAAN Dr. Cadavid has received personal compensation for serving as an employee of Verge Genomics. Dr. Cadavid has received personal compensation for serving as an employee of X4 Pharmaceuticals. Dr. Cadavid has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pheno Therapeutics. Dr. Cadavid has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novo Nordisk. Dr. Cadavid has stock in Verge Genomics.
Alan Pestronk, MD, FAAN The institution of Dr. Pestronk has received research support from Jain. The institution of Dr. Pestronk has received research support from Fulcrum. The institution of Dr. Pestronk has received research support from Argenyx. The institution of Dr. Pestronk has received research support from NeuroNext.
Hanns Lochmuller, MD, FAAN Dr. Lochmuller has nothing to disclose.
Kathryn R. Wagner, MD, PhD Dr. Wagner has received personal compensation for serving as an employee of F. Hoffmann-La Roche Ltd. Dr. Wagner has stock in F. Hoffmann-La Roche.
Summer Gibson, MD An immediate family member of Dr. Gibson has received personal compensation for serving as an employee of Recursion Pharmaceuticals. Dr. Gibson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint. An immediate family member of Dr. Gibson has received stock or an ownership interest from Recursion Pharmaceuticals . An immediate family member of Dr. Gibson has received intellectual property interests from a discovery or technology relating to health care. Dr. Gibson has received publishing royalties from a publication relating to health care.
Cornelia Kornblum No disclosure on file
Craig McDonald, MD 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.
Michelle L. Mellion, MD Dr. Mellion has received personal compensation for serving as an employee of PepGen. Dr. Mellion has stock in PepGen.
Carsten G. Bonnemann, MD Dr. Bonnemann has a non-compensated relationship as a Scientific Advisor with RYR1 Foundation that is relevant to AAN interests or activities. Dr. Bonnemann has a non-compensated relationship as a Scientific Advisor with Genethon that is relevant to AAN interests or activities. Dr. Bonnemann has a non-compensated relationship as a Scientific Advisor with Sarepta Therapeutics that is relevant to AAN interests or activities. Dr. Bonnemann has a non-compensated relationship as a Scientific Advisor with Kate Therapeutics that is relevant to AAN interests or activities. Dr. Bonnemann has a non-compensated relationship as a Scientific Advisor with CureCMD that is relevant to AAN interests or activities. Dr. Bonnemann has a non-compensated relationship as a Scientific Advisor with Seal Therapeutics that is relevant to AAN interests or activities.
No disclosure on file
Philip Van Damme The institution of Dr. Van Damme has received research support from CSL Behring.
Shane Raines Shane Raines has received personal compensation for serving as an employee of Praxis Precision Medicines. Shane Raines has received personal compensation for serving as an employee of 2b Analytics.
Baziel G. van Engelen, MD, PhD, MA The institution of Dr. van Engelen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum . The institution of Dr. van Engelen has received research support from Stichting Spieren voor Spieren. The institution of Dr. van Engelen has received research support from Prinses Beatrix Fonds. The institution of Dr. van Engelen has received research support from Dutch FSHD society. Dr. van Engelen has received intellectual property interests from a discovery or technology 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 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 $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vita Therapeutics. 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. 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 Vertex . 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.
Nicholas E. 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 $500-$4,999 for serving as a Consultant for Avidity. Dr. Johnson has received personal compensation in the range of $100,000-$499,999 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 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 Argenx. Dr. Johnson has stock in Angle Therapeutics. Dr. Johnson has stock in Juvena Therapeutics. Dr. Johnson has stock in Myogene Therapies. 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. The institution of Dr. Johnson has received research support from Avidity Biosciences. The institution of Dr. Johnson has received research support from Edgewise. 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. Dr. Subramony has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Subramony has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Subramony has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Fulcrum. 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. The institution of Dr. Subramony has received research support from Avidity. The institution of Dr. Subramony has received research support from PTC. The institution of Dr. Subramony has received research support from Biohaven. The institution of Dr. Subramony has received research support from Fulcrum. The institution of Dr. Subramony has received research support from Vertex. The institution of Dr. Subramony has received research support from Arthrex.
Alrabi Tawil, MD, FAAN Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving as a Consultant for meRicule. 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.
Lawrence J. Hayward, MD, PhD Dr. Hayward has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Myocea, Inc. Dr. Hayward has received personal compensation in the range of $0-$499 for serving as a Consultant for HC Wainwright. Dr. Hayward has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Fulcrum Therapeutics. The institution of Dr. Hayward has received research support from Fulcrum Therapeutics. The institution of Dr. Hayward has received research support from Solve FSHD. The institution of Dr. Hayward has received research support from Myscular Dystrophy Association. The institution of Dr. Hayward has received research support from Swan Bio Therapeutics. The institution of Dr. Hayward has received research support from NIH.
Johanna Hamel, MD Dr. Hamel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharmaceuticals.
John Vissing, MD Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne Therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Solid Biosciences. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AskBio. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity Therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Italfarmaco. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Biopharma. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion Pharmaceuticals. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson & Johnson (Janssen). Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Edgewise Therapeutics. The institution of Prof. Vissing has received research support from Rigshospitalet. The institution of Prof. Vissing has received research support from UCB Pharma. The institution of Prof. Vissing has received research support from Sanofi. The institution of Prof. Vissing has received research support from Roche. The institution of Prof. Vissing has received research support from AnJii.
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.
Nancy L. Kuntz, MD, FAAN Dr. Kuntz 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. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Affinia Therapeutics. Dr. Kuntz has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenyx. Dr. Kuntz has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Audentes Therapeutics. Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Kuntz 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. Kuntz 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. Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta.
Barry Byrne Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rocket Pharma. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving as an officer or member of the Board of Directors for Muscular Dystrophy Association.
Loren D. Pena No disclosure on file
Mazen M. Dimachkie, MD, FAAN Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Octapharma. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medlink. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL Behring. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Janssen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TACT / Treat NMD. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cabaletta Bio. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clinical Neurological Society of America, Inc . Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ig Society, Inc. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abcuro. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amicus. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for EMD Serono. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipsen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nuvig. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fortrea. The institution of Dr. Dimachkie has received research support from NIH. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received publishing royalties from a publication relating to health care.
Jun Lee, MD No disclosure on file
Suyash Prasad, MBBS Dr. Prasad has received personal compensation for serving as an employee of Audentes Therapeutics. Dr. Prasad has received stock or an ownership interest from Audentes.
Laurent Servais Laurent Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Laurent Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. The institution of Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Evox. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sysnav. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BioHaven. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zentech. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MitoRx. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Lupin. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fibrogen. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alltrana. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Illumina. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Laurent Servais has received research support from Roche. The institution of Laurent Servais has received research support from Novartis. The institution of Laurent Servais has received research support from Biogen. The institution of Laurent Servais has received research support from Zentech. The institution of Laurent Servais has received research support from BioHaven. The institution of Laurent Servais has received research support from PerkinHalmers. The institution of Laurent Servais has received research support from Scholar Rock.
Francesco Muntoni, MD Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Biogen. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. The institution of Dr. Muntoni has received research support from European Commission. The institution of Dr. Muntoni has received research support from Medical Research Council. The institution of Dr. Muntoni has received research support from Biogen. The institution of Dr. Muntoni has received research support from Muscular Dystrophy UK. The institution of Dr. Muntoni has received research support from MDA USA. The institution of Dr. Muntoni has received research support from Sarepta. The institution of Dr. Muntoni has received research support from Association Francoise Myopathies. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving as a Clinical expert with UK NICE Committee.
Tina Duong Tina Duong has received personal compensation for serving as an employee of Roche. Tina Duong has received personal compensation for serving as an employee of Novartis. Tina Duong has received personal compensation for serving as an employee of Scholar Rock. Tina Duong has received personal compensation for serving as an employee of Genentech. Tina Duong has received personal compensation for serving as an employee of Novartis. Tina Duong has received personal compensation for serving as an employee of Sarepta. Tina Duong has received personal compensation for serving as an employee of Biogen. Tina Duong has received personal compensation for serving as an employee of Audentes. Tina Duong has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Tina Duong has received personal compensation in the range of $0-$499 for serving as a Consultant for Audentes. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Genentech. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for biogen. The institution of Tina Duong has received research support from biogen. The institution of Tina Duong has received research support from scholar rock.
Pascal Laforet No disclosure on file
Priya Kishnani, MD Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi/Genzyme. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amicus Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Maze Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for JCR Pharmaceutical. Priya Kishnani, MD has received personal compensation in the range of $500,000-$999,999 for serving as a Consultant for Asklepios Biopharmaceutical Inc. (AskBio). Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi/Genzyme. Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Baebies, Inc.. The institution of Priya Kishnani, MD has received research support from Sanofi/Genzyme. Priya Kishnani, MD has received research support from Amicus Therapeutics. Priya Kishnani, MD has received intellectual property interests from a discovery or technology relating to health care. Priya Kishnani, MD has received intellectual property interests from a discovery or technology relating to health care.
Umesh A. Badrising, MD, PhD No disclosure on file
Richard J. Barohn, MD, FAAN Dr. Barohn has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for NuFactor. The institution of Dr. Barohn has received research support from FDA OPD R01. Dr. Barohn has received intellectual property interests from a discovery or technology relating to health care.
Marcondes C. Franca, Jr., MD Dr. Franca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Franca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Dr. Franca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Franca has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. The institution of Dr. Franca has received research support from FARA.
Shafeeq Ladha, MD Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Ladha has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Ladha has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech. Dr. Ladha has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Ladha has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurosense. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rapa Therapeutics. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Sanofi. Dr. Ladha has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Genentech. The institution of Dr. Ladha has received research support from Biogen. The institution of Dr. Ladha has received research support from Sanofi.
Benedikt Schoser Benedikt Schoser has received personal compensation for serving as an employee of Alexion. Benedikt Schoser has received personal compensation for serving as an employee of Argenx. Benedikt Schoser has received personal compensation for serving as an employee of Amicus. Benedikt Schoser has received personal compensation for serving as an employee of Astellas. Benedikt Schoser has received personal compensation for serving as an employee of Denali. Benedikt Schoser has received personal compensation for serving as an employee of Avidity . Benedikt Schoser has received personal compensation for serving as an employee of Sanofi. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Taysha. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astellas. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Encoded. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Kedrion.
Martin K. Childers, DO, PhD No disclosure on file
Ans Van Der Ploeg The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi Genzyme . The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics . The institution of Ans Van Der Ploeg has received research support from Sanofi Genzyme.
Pablo B. Winckler, MD, PhD Dr. Winckler has nothing to disclose.
Jonas A. Saute, MD No disclosure on file
Michio Hirano, MD, FAAN Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB Biopharma SRL. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Precision Biosciences. Dr. Hirano has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Biopharma SRL. Dr. Hirano has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Apollo Communication. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Envision Communications. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for 好色先生. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Cure SMA. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Muscular Dystrophy Association. The institution of Dr. Hirano has received research support from UCB. The institution of Dr. Hirano has received research support from Cyclerion. Dr. Hirano has received research support from Reneo Pharmaceuticals. The institution of Dr. Hirano has received research support from Astellas. Dr. Hirano has received intellectual property interests from a discovery or technology relating to health care. Dr. Hirano has received intellectual property interests from a discovery or technology relating to health care. Dr. Hirano has received personal compensation in the range of $0-$499 for serving as a Study Section Reviewer with NIH. Dr. Hirano has a non-compensated relationship as a Research Advisory Board member with Muscular Dystrophy Association that is relevant to AAN interests or activities. Dr. Hirano has a non-compensated relationship as a Scientific and Medical Advisory Board member with United Mitochondrial Disease Foundation that is relevant to AAN interests or activities. Dr. Hirano has a non-compensated relationship as a Scientific Advisory Board member with Barth Syndrome Foundation that is relevant to AAN interests or activities.
Edmar Zanotelli No disclosure on file
Erik K. Henricson, MPH No disclosure on file
Leo H. 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 $5,000-$9,999 for serving as a Consultant for Avidity. Dr. Wang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB Pharma. Dr. Wang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lupin. Dr. Wang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Wang has received personal compensation in the range of $10,000-$49,999 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.
Jonathan Shintaku, PhD No disclosure on file
Juan J. Vilchez Padilla, MD Dr. Vilchez Padilla has nothing to disclose.
Angela L. Genge, MD Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AL-S Pharma. Dr. Genge has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amylyx. Dr. Genge has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Quralis. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Genge has received personal compensation in the range of $0-$499 for serving as a Consultant for WAVE. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for eikonizo. Dr. Genge has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for pepgen. Dr. Genge has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for rapa.
Eugen Mengel No disclosure on file
Samantha J. LoRusso, MD Dr. LoRusso has nothing to disclose.
Lindsay N. Alfano, PT Ms. Alfano has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ATOM International, Ltd (Amicus Therapeutics, Catabasis, Genethon, Italfarmaco, NS Pharma, Pfizer, PTC Therapeutics). Ms. Alfano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Novartis Gene Therapies. The institution of Ms. Alfano has received research support from Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Audentes Therapeutics/Astellas Gene Therapies. Ms. Alfano has received intellectual property interests from a discovery or technology relating to health care.
James Dowling, MD, PhD Dr. Dowling has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GLG. Dr. Dowling has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ikaika Therapeutics. Dr. Dowling has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bain Consulting. Dr. Dowling has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint. Dr. Dowling has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for RYR1 Foundation. Dr. Dowling has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Dechert LLP. The institution of Dr. Dowling has received research support from Astellas. The institution of Dr. Dowling has received research support from NIH. The institution of Dr. Dowling has received research support from CIHR. The institution of Dr. Dowling has received research support from Astellas. Dr. Dowling has received personal compensation in the range of $5,000-$9,999 for serving as a advisor with Ontario Ministry of Health.
Sarah B. Neuhaus, DO Dr. Neuhaus has nothing to disclose.
Salvador Rico Salvador Rico has received personal compensation for serving as an employee of Audentes Therapeutics. Salvador Rico has received personal compensation for serving as an employee of Encoded Therapeutics. Salvador Rico has received stock or an ownership interest from Audentes Therapeutics.
Kristina an Haack No disclosure on file