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2022 Annual Meeting | S23 - Therapeutics for Muscle Diseases

Tuesday 04/05/22
03:30 PM - 05:30 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
03:30 PM - 03:30 PM PDT Speaker Walk-in Slides
Erin Jackson
03:42 PM - 03:54 PM PDT Abstract 002 - Phase 1/2a Trial of SRP-9001 in Patients with Duchenne Muscular Dystrophy: 3-Year Safety and Functional Outcomes
Jerry R. Mendell, MD, FAAN
03:54 PM - 04:06 PM PDT Abstract 003 - Antibody-Oligonucleotide Conjugates (AOCs) Demonstrate Potent and Durable Exon Skipping and Dystrophin Restoration in a Mouse Model of Duchenne Muscular Dystrophy
Georgios Karamanlidis, PhD
04:06 PM - 04:18 PM PDT Abstract 004 - A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of SRP-9001 for Treating Patients with Duchenne Muscular Dystrophy
Jerry R. Mendell, MD, FAAN
04:18 PM - 04:30 PM PDT Abstract 005 - Safety, ß-Sarcoglycan Expression, and Functional Outcomes From Systemic Gene Transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4
Jacob S. Elkins, MD
04:30 PM - 04:42 PM PDT Abstract 006 - Study Design of AOC 1001-CS1, a Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA)
Nicholas Elwood Johnson, MD, FAAN
04:42 PM - 04:54 PM PDT Abstract 007 - A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD: ReDUX4
Christopher Morabito, MD
04:54 PM - 05:06 PM PDT Abstract 008 - An Open-Label Study of Losmapimod to Evaluate the Safety, Tolerability, and Biomarker and Clinical Outcome Assessment Changes in Subjects with FSHD1
Jennifer *Use 325910 Shoskes, PharmD
05:06 PM - 05:18 PM PDT Abstract 009 - Whole Body MRI Quantitative muscle analysis to evaluate Efficacy of Losmapimod in a Phase 2 Placebo-Controlled Study in Subjects with FSHD (ReDUX4)
Christopher Morabito, MD
05:18 PM - 05:30 PM PDT Abstract 010 - A Randomized, Double-Blind, Placebo-Controlled Study of Arimoclomol in Patients with Inclusion Body Myositis
Mazen M. Dimachkie, MD, FAAN
Faculty Disclosures
Thomas E. Lloyd, MD, PhD Dr. Lloyd has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier.
Claus Sundgreen Claus Sundgreen has received personal compensation for serving as an employee of Orphazyme A/S.
Loretta Staudt Loretta Staudt has nothing to disclose.
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.
Karina Phonekeo, Other Mrs. Phonekeo has nothing to disclose.
Nicol Voermans, MD The institution of Mrs. Voermans has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dynacure. The institution of Mrs. Voermans has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH. The institution of Mrs. Voermans has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Neuromuscular disorder.
Charles A. Thornton, MD, FAAN Dr. Thornton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity. Dr. Thornton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Thornton has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Ionis. Dr. Thornton has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne. Dr. Thornton has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pepgen. The institution of Dr. Thornton has received research support from Marigold Foundation. The institution of Dr. Thornton has received research support from NIH. The institution of Dr. Thornton has received research support from Muscular Dystrophy Association . The institution of Dr. Thornton has received research support from Dyne. The institution of Dr. Thornton has received research support from Avidity. The institution of Dr. Thornton has received research support from Ionis. Dr. Thornton has received intellectual property interests from a discovery or technology relating to health care.
Hanns Lochmuller, MD, FAAN Dr. Lochmuller has nothing to disclose.
Tyler Albin, PhD Dr. Albin has received personal compensation for serving as an employee of Avidity Biosciences. Dr. Albin has received stock or an ownership interest from Avidity Biosciences.
Payam Soltanzadeh, MD The institution of Dr. Soltanzadeh has received research support from Ra Pharma (UCB).
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.
Jeremy Woods Jeremy Woods has nothing to disclose.
Sarah M. Jones, MD No disclosure on file
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.
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.
Olecya Tyaglo Ms. Tyaglo has received personal compensation for serving as an employee of Avidity Biosciences. Ms. Tyaglo has received stock or an ownership interest from Avidity Biosciences.
William D. Arnold, MD Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for La Hoffmann Roche. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cadent Therapeutics . Dr. Arnold has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. The institution of Dr. Arnold has received research support from NIH. The institution of Dr. Arnold has received research support from NMD Pharma. The institution of Dr. Arnold has received research support from Gilead Sciences. The institution of Dr. Arnold has received research support from CureSMA. Dr. Arnold has received intellectual property interests from a discovery or technology relating to health care.
Jyoti Malhotra Jyoti Malhotra has nothing to disclose.
Carrie Nease Carrie Nease has nothing to disclose.
Rachael Potter Rachael Potter has received stock or an ownership interest from Sarepta Therapeutics.
Craig M. Zaidman, MD Dr. Zaidman has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for sarepta. The institution of Dr. Zaidman has received research support from Washington University in St Louis. The institution of Dr. Zaidman has received research support from Novartis.
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.
Michael George Hanna, MD, MBBS No disclosure on file
Aziz I. Shaibani, MD, FAAN Dr. Shaibani has nothing to disclose.
Sarah Berten Neuhaus, DO Dr. Neuhaus has nothing to disclose.
Elizabeth Jane Ackermann, PhD Dr. Ackermann has received personal compensation for serving as an employee of Avidity Biosciences.
Robert Gould, PhD Dr. Gould has received personal compensation for serving as an employee of Fulcrum Therapeutics. Dr. Gould has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Fulcrum Therapeutics. Dr. Gould 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 Fulcrum Therapeutics. Dr. Gould has received stock or an ownership interest from Fulcrum Therapeutics.
Tianle Hu Tianle Hu has received personal compensation for serving as an employee of Sarepta Therapeutics. Tianle Hu has received stock or an ownership interest from Sarepta Therapeutics.
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.
Juan Jesus Vilchez Padilla, MD Dr. Vilchez Padilla has nothing to disclose.
David Reyes Leiva, MD Dr. Leiva has nothing to disclose.
Mazen M. Dimachkie, MD, FAAN Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EcoR1 Capital, LLC. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cello Health BioConsulting, previously Defined Health. 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 CSL Behring. 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 $5,000-$9,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for RAF-5. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Houston NeuroCare. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Shire. Dr. Dimachkie has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kezar. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for RaPharma/UCB. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 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 Sanofi. An immediate family member of Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kezar. 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 SCHOLAR Rock. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jenssen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amazentis / Vandria. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roivant. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SANEM. 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 $5,000-$9,999 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 Third Rock. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Orphazyme . Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 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 Roivant . 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 $500-$4,999 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 $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Covance / Labcorp. The institution of Dr. Dimachkie has received research support from CSL Behring. The institution of Dr. Dimachkie has received research support from Orphazyme. The institution of Dr. Dimachkie has received research support from FDA-OOPD. 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 publishing royalties from a publication relating to health care.
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.
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.
Anders Noerkaer Joergensen, PhD Mr. Joergensen has received personal compensation for serving as an employee of Orphazyme A/S.
John Jiang, PhD Dr. Jiang has received personal compensation for serving as an employee of Fulcrum Therapeutics.
Jennifer *Use 325910 Shoskes, PharmD Dr. Shoskes has received personal compensation for serving as an employee of Fulcrum Therapeutics. An immediate family member of Dr. Shoskes has received personal compensation for serving as an employee of Exact Science.
Sameer Upadhyay Sameer Upadhyay has received personal compensation for serving as an employee of Sarepta Therapeutics. Sameer Upadhyay has received stock or an ownership interest from Sarepta Therapeutics. Sameer Upadhyay has received stock or an ownership interest from Vertex Pharmaceuticals.
Kellie Lemoine Ms. Lemoine has received personal compensation for serving as an employee of Avidity Biosciences. Ms. Lemoine has received stock or an ownership interest from Avidity Biosciences.
Li-Jung Tai, MD, PhD Li-Jung Tai has received personal compensation for serving as an employee of Avidity Biosciences. Li-Jung Tai has received stock or an ownership interest from Avidity.
Thomas Blaettler No disclosure on file
Samantha Jo LoRusso, MD Dr. LoRusso has nothing to disclose.
Alissa N. Peters Mrs. Peters has received personal compensation for serving as an employee of Avidity Biosciences. Mrs. Peters has received stock or an ownership interest from Avidity Biosciences .
Thomas Carstensen No disclosure on file
Kelly Ditrapani Kelly Ditrapani has received personal compensation for serving as an employee of Avidity Biosciences. Kelly Ditrapani has received personal compensation for serving as an employee of Iovance Biotherapeutics. An immediate family member of Kelly Ditrapani has received personal compensation for serving as an employee of Jazz Pharmaceuticals. Kelly Ditrapani has stock in Iovance Biotherapeutics. Kelly Ditrapani has stock in Avidity Biosciences. Kelly Ditrapani has stock in Aravive Inc.. An immediate family member of Kelly Ditrapani has stock in Jazz Pharmaceuticals. Kelly Ditrapani has stock in Abbvie.
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 $500-$4,999 for serving as a Consultant for Biogen. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics. 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 Alexion. 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.
Howard Mao Howard Mao has nothing to disclose.
Kelly Marshall Ms. Marshall has nothing to disclose.
Erica Koenig Erica Koenig has nothing to disclose.
Verna Zhao, Other Ms. Zhao has received personal compensation for serving as an employee of Avidity.
Kelly Lehman, MSN, CNP Ms. Lehman has nothing to disclose.
Arthur A. Levin, PhD,PT Dr. Levin has received personal compensation for serving as an employee of Avidity Biosciences. Dr. Levin has received stock or an ownership interest from Avidity Biosciences.
Richard Shell Richard Shell has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avexis. Richard Shell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen.
Maitea Guridi, PhD Dr. Guridi has received personal compensation for serving as an employee of Hoffmann-La Roche.
Miriam L. Freimer, MD, FAAN Dr. Freimer has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Freimer 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. Freimer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for J and J. The institution of Dr. Freimer has received research support from Alnylum. The institution of Dr. Freimer has received research support from UCB. The institution of Dr. Freimer has received research support from NIH. The institution of Dr. Freimer has received research support from Janssen. Dr. Freimer has received research support from Avidity. Dr. Freimer has received research support from Fulcrum. Dr. Freimer has received personal compensation in the range of $10,000-$49,999 for serving as a presenations/teaching with UCB.
Anthony Accorsi Anthony Accorsi has received personal compensation for serving as an employee of Fulcrum Therapeutics. Anthony Accorsi has received personal compensation in the range of $0-$499 for serving as a Consultant for Faze Therapeutics.
Kathleen Church Kathleen Church has nothing to disclose.
Kathryn Anne Giblin, MD Dr. Giblin has received personal compensation for serving as an employee of Sarepta Therapeutics. Dr. Giblin has received stock or an ownership interest from Sarepta Therapeutics. Dr. Giblin has received intellectual property interests from a discovery or technology relating to health care.
Eric Pozsgai Eric Pozsgai has received personal compensation for serving as an employee of Sarepta Therapeutics. Eric Pozsgai has received stock or an ownership interest from Sarepta Therapeutics.
Natalie F. Reash, PT Dr. Reash has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ATOM International. Dr. Reash has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Casimir, LLC. The institution of Dr. Reash has received research support from Sarepta. Dr. Reash has received intellectual property interests from a discovery or technology relating to health care. Dr. Reash has received intellectual property interests from a discovery or technology relating to health care.
Carrie Heusner, PhD Dr. Heusner has received personal compensation for serving as an employee of Avidity Biosciences.
Raghav Bhardwaj Mr. Bhardwaj has received personal compensation for serving as an employee of Avidity Biosciences. Mr. Bhardwaj has received stock or an ownership interest from Avidity Biosciences.
Christopher Moxham, PhD Dr. Moxham has received personal compensation for serving as an employee of Fulcrum Therapeutics.
Chao-Yin Chen, PhD Dr. Chen has received personal compensation for serving as an employee of Avidity Biosciences.
Jorge Alonso-Perez, MD Dr. Alonso-Perez has nothing to disclose.
Alan Pestronk, MD, FAAN The institution of Dr. Pestronk has received research support from RA. The institution of Dr. Pestronk has received research support from Fulcrum. The institution of Dr. Pestronk has received research support from Acceleron. The institution of Dr. Pestronk has received research support from Biogen. The institution of Dr. Pestronk has received research support from Jain. The institution of Dr. Pestronk has received research support from Sanofi. The institution of Dr. Pestronk has received research support from Muscular Dystrophy Association. Dr. Pestronk has received intellectual property interests from a discovery or technology relating to health care.
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.
Megan Iammarino, PT Dr. Iammarino has nothing to disclose.
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.
Pedro Machado No disclosure on file
Sarah E. Lewis Mrs. Lewis has received personal compensation for serving as an employee of Sarepta Therapeutics . Mrs. Lewis has stock in Sarepta Therapeutics.
Xiaoxi Li, PhD Dr. Li has received personal compensation for serving as an employee of Sarepta Therapeutics. Dr. Li has received stock or an ownership interest from Sarepta Therapeutics.
Louise R. Rodino-Klapac Ms. Rodino-Klapac has received personal compensation for serving as an employee of Sarepta Therapeutics, Inc.. Ms. Rodino-Klapac has received stock or an ownership interest from Sarepta Therapeutics. Ms. Rodino-Klapac has received intellectual property interests from a discovery or technology relating to health care.
Lucienne Ronco No disclosure on file
Michael Cochran, PhD Dr. Cochran has received personal compensation for serving as an employee of Avidity Biosciences.
Brenna Powers, PT Dr. Powers has nothing to disclose.
Joost Kools, MD The institution of Mr. Kools has received research support from Fulcrum Therapeutics.
Aaron Meadows Aaron Meadows has received stock or an ownership interest from Sarepta Therapeutics.
Alejandro Rojas Alejandro Rojas has received personal compensation for serving as an employee of Fulcrum Therapeutics.
William M. Flanagan, PhD Dr. Flanagan has received personal compensation for serving as an employee of Avidity Biosciences. Dr. Flanagan has received stock or an ownership interest from Avidity Biosciences.
Jordan Vaiea No disclosure on file
Philip Kovach, Jr. Mr. Kovach has received personal compensation for serving as an employee of Avidity Biosciences. Mr. Kovach has received stock or an ownership interest from Avidity Biosciences.
Hae Won Kwon, Other Ms. Kwon has received personal compensation for serving as an employee of Avidity Biosciences. Ms. Kwon has received stock or an ownership interest from Avidity Biosciences.
Shufang Wang Shufang Wang has received personal compensation for serving as an employee of Sarepta Therapeutics INC. Shufang Wang has received stock or an ownership interest from Sarepta Therapeutics INC.
Zarife Sahenk, MD, FAAN, PhD Dr. Sahenk has nothing to disclose.
Husam Younis, PhD Mr. Younis has received personal compensation for serving as an employee of Avidity Bioscience. Mr. Younis has received stock or an ownership interest from Avidity Bioscience.
Sarah Lewis Sarah Lewis has received stock or an ownership interest from Sarepta.
Todd D. Levine, MD Dr. Levine has received personal compensation for serving as an employee of CND life sciences . Dr. Levine has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Nufactor. Dr. Levine has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Grifols. Dr. Levine has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for PNA. Dr. Levine has stock in CND Life Sciences. Dr. Levine has stock in Corinthian reference lab.
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.
Jerry R. Mendell, MD, FAAN Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vertex. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics . The institution of Dr. Mendell has received research support from Sarepta.
Laura Herbelin No disclosure on file
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.
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.
Maria Azzurra Missinato, PhD Dr. Missinato has received personal compensation for serving as an employee of Avidity Biosciences. An immediate family member of Dr. Missinato has received stock or an ownership interest from Avidity Biosciences.
Craig McDonald, MD Dr. McDonald has received personal compensation in the range of $10,000-$49,999 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 $500-$4,999 for serving as a Consultant for Astellas. Dr. McDonald has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Capricor Therapeutics. Dr. McDonald has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Catabasis. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epirium Bio. Dr. McDonald has received personal compensation in the range of $0-$499 for serving as a Consultant for Italfarmaco. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Santhera Pharmaceuticals. 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 $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Capricor. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis Pharmaceuticals. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Santhera Pharmaceuticals. 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 Santhera Pharmaceuticals. The institution of Dr. McDonald has received research support from Astellas. The institution of Dr. McDonald has received research support from Capricor Therapeutics. The institution of Dr. McDonald has received research support from Catabasis. The institution of Dr. McDonald has received research support from Epirium Bio. 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 Pfizer. The institution of Dr. McDonald has received research support from NIH (NINDS). The institution of Dr. McDonald has received research support from NIDILRR. The institution of Dr. McDonald has received research support from Department of Defense. 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.
Per Wildholm Per Wildholm has received personal compensation for serving as an employee of AMRA Medical.
Nuria Muelas Nuria Muelas has nothing to disclose.
Tejdip Singh Tejdip Singh has received personal compensation for serving as an employee of Sarepta Therapeutics . Tejdip Singh has received stock or an ownership interest from Sarepta Therapeutics.
Isaac Marks, PhD Dr. Marks has received personal compensation for serving as an employee of Avidity Biosciences. Dr. Marks has received stock or an ownership interest from Avidity Biosciences.
Emma Ciafaloni, MD, FAAN Dr. Ciafaloni has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx, Alexion, Sarepta, UCB, Hoffman-LaRoche, Biogen. Dr. Ciafaloni 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, AnnJi Pharmaceutical, ML-BIO, Avidity. The institution of Dr. Ciafaloni has received research support from CDC, CureSMA, FDA, Orphazyme, Sarepta, PCORI, Neurogene. Dr. Ciafaloni has received publishing royalties from a publication relating to health care.
Jordi Diaz Manera No disclosure on file
Baziel G M van Engelen, MD, MA, PhD The institution of Dr. van Engelen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Facio. 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.
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.
Matthew Diaz Mr. Diaz has received personal compensation for serving as an employee of Avidity Biosciences. Mr. Diaz has received stock or an ownership interest from Avidity Biosciences.
Varun Goel, PhD Dr. GOEL has received personal compensation for serving as an employee of Avidity Biosciences. An immediate family member of Dr. GOEL has received personal compensation for serving as an employee of PTC Therapeutics. An immediate family member of Dr. GOEL has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for PTC Therapeutics. Dr. GOEL has received stock or an ownership interest from Alnylam Pharmaceuticals. An immediate family member of Dr. GOEL has received stock or an ownership interest from PTC Therapeutics. Dr. GOEL has received intellectual property interests from a discovery or technology relating to health care.
Ben Knisely Mr. Knisely has nothing to disclose.
Mohita Kumar, MD Dr. Kumar has received personal compensation for serving as an employee of Avidity Biosciences. Dr. Kumar has received stock or an ownership interest from Avidity Biosciences.
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.
Olof Dahlqvist Leinhard Olof Dahlqvist Leinhard has received personal compensation for serving as an employee of AMRA Medical AB. Olof Dahlqvist Leinhard has received stock or an ownership interest from AMRA Medical AB.
Karen Bonefeld Karen Bonefeld has received personal compensation for serving as an employee of Orphazyme. Karen Bonefeld has received personal compensation for serving as an employee of Novo Nordisk .
Andrew Heim Andrew Heim has nothing to disclose.
Larisa Collins Miss Collins has received personal compensation for serving as an employee of Sarepta Therapeutics. Miss Collins has received stock or an ownership interest from Sarepta Therapeutics.
Linda Pax Lowes, PT PhD The institution of Ms. Lowes has received research support from Sarepta Therapeutics.
William Tracewell William Tracewell has received personal compensation for serving as an employee of Nuventra LLC.
Adefowope Odueyungbo, PhD Dr. Odueyungbo has received personal compensation for serving as an employee of Fulcrum Therapeutics.
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.
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.
Christy Skura Christy Skura has nothing to disclose.
Christopher Morabito, MD Dr. Morabito has received personal compensation for serving as an employee of Fulcrum Therapeutics. Dr. Morabito has stock in Fulcrum Therapeutics.
Danielle A. Griffin Ms. Griffin has stock in Sarepta Therapeutics. Ms. Griffin has received intellectual property interests from a discovery or technology relating to health care.
Mark Hogan Mark Hogan has nothing to disclose.
Kristi R. R Ms. R has received personal compensation for serving as an employee of Avidity Biosciences. Ms. R has received stock or an ownership interest from Avidity Biosciences.
Anthony A. Amato, MD, FAAN Dr. Amato has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson & Johnson. Dr. Amato 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. Amato has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abscuro. Dr. Amato 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. Amato has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Caballeta. Dr. Amato has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Amato has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for EMD Serono. Dr. Amato has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Neurology. Dr. Amato has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for legal firms. The institution of Dr. Amato has received research support from NIH. Dr. Amato 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 X4 Pharma. Dr. Cadavid has received personal compensation for serving as an employee of Fulcrum Therapeutics. Dr. Cadavid has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arvinas. Dr. Cadavid has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abata Therapeutics. Dr. Cadavid has received stock or an ownership interest from X4 Pharma. Dr. Cadavid has received stock or an ownership interest from Fulcrum Therapeutics.
Usue Etxaniz, PhD Mrs. Etxaniz has received personal compensation for serving as an employee of Avidity Biosciences. Mrs. Etxaniz has received stock or an ownership interest from Avidity Biosciences.
Aaron George Anderson, Other Mr. Anderson has received personal compensation for serving as an employee of Avidity Biosciences. Mr. Anderson has received stock or an ownership interest from Avidity Biosciences.
Michelle L. Mellion, MD Dr. Mellion has received personal compensation for serving as an employee of PepGen. Dr. Mellion has stock in PepGen.
Diana Castro, MD Dr. Castro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Castro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Castro has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC. Dr. Castro 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. Castro 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. Castro has received research support from Biogen. The institution of Dr. Castro has received research support from Sarepta. The institution of Dr. Castro has received research support from Fibrogen .
Matthew Harms, MD Dr. Harms has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Harms has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Muscular Dystrophy Association. Dr. Harms has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Invitae. Dr. Harms has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Variant Bio. Dr. Harms 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. Harms has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx. Dr. Harms has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for uniQure. Dr. Harms has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Littlepage Booth. Dr. Harms has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for O'Connor First. Dr. Harms has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Searcy Denney. Dr. Harms has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Ford, Parshall & Baker LLC . The institution of Dr. Harms has received research support from ALS Association. The institution of Dr. Harms has received research support from Ionis. The institution of Dr. Harms has received research support from ALS Finding a Cure. The institution of Dr. Harms has received research support from Target ALS.
Georgios Karamanlidis, PhD Dr. Karamanlidis has received personal compensation for serving as an employee of Avidity Biosciences. Dr. Karamanlidis has stock in Avidity Biosciences.
Erin Jackson Ms. Jackson has nothing to disclose.
Jacob S. Elkins, MD No disclosure on file