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2024 Annual Meeting | S21 - Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations

Tuesday 04/16/24
01:00 PM - 03:00 PM MDT Add To Calendar
Colorado Convention Center | Four Seasons 1
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)
 A group of abstracts covering a similar topic presented in an oral format. Presentaons are eight minutes in length with four minutes for queson and answer with the author. 
2.00 CME credits

Program Materials Program Evaluations

Event Timeline
01:00 PM - 01:12 PM MDT Abstract 001 - Ataluren Delays Clinically Meaningful Milestones of Decline in 6WMD in Patients with nmDMD from Study 041, a Phase 3, Placebo-controlled Trial
Jeffrey Statland, MD
01:12 PM - 01:24 PM MDT Abstract 002 - Effects of EDG-5506, a Fast Myosin Modulator, on Function and Biomarkers of Muscle Damage in Adults with Becker Muscular Dystrophy
Joanne Donovan, MD,PhD
01:24 PM - 01:36 PM MDT Abstract 003 - Effect Size Analysis of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa in ERT-experienced Adults with Late-onset Pompe Disease in PROPEL
Hani Kushlaf, MD, FAAN
01:36 PM - 01:48 PM MDT Abstract 004 - Long-term Follow-up Deoxynucleoside Therapy for Late-onset Thymidine Kinase 2 Deficiency Patients
Hacer Durmus, MD
01:48 PM - 02:00 PM MDT Abstract 005 - RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data
Aravindhan Veerapandiyan, MD
02:00 PM - 02:12 PM MDT Abstract 006 - REMEDY: A Novel CRISPR-based Allele Specific Approach Corrects VCP Mutations
Eleonora D'Ambrosio, MD
02:12 PM - 02:24 PM MDT Abstract 007 - Peripheral Nervous System Involvement of Hereditary Transthyretin Amyloidosis in the United States: A Multi-center Perspective
Hristelina S. Ilieva, MD
02:24 PM - 02:36 PM MDT Abstract 008 - Peripheral Neuropathy in AL Amyloidosis: Clinical Presentations and Outcomes of Current Therapies
Pitcha Chompoopong, MD
02:36 PM - 02:48 PM MDT Abstract 009 - PAK2 Is Necessary for Myelination in the Peripheral Nerve System
Jun Li, MD, PhD, FAAN
02:48 PM - 03:00 PM MDT Abstract 010 - Improved Neuromuscular Junction in R98C Mpz Heterozygous Mice Treated with IFB-088
Yunhong Bai
Faculty Disclosures
Colin Quinn, MD Dr. Quinn has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx Pharmaceuticals. Dr. Quinn has received personal compensation in the range of $0-$499 for serving as a Consultant for Amicus. Dr. Quinn has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta Therapeutics. Dr. Quinn has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Quinn has received personal compensation in the range of $0-$499 for serving as a Consultant for Clene Nanomedicine. Dr. Quinn has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Quinn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Partners Health Care. Dr. Quinn has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Robins Kaplan.
Eleonora D'Ambrosio, MD Dr. D'Ambrosio has nothing to disclose.
Meisam Naeimi Kararoudi, PhD,Other The institution of Dr. Naeimi Kararoudi has received research support from Metagenomi. Dr. Naeimi Kararoudi has received intellectual property interests from a discovery or technology relating to health care.
Zarife Sahenk, MD, FAAN, PhD Dr. Sahenk has nothing to disclose.
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.
Burcak Ozes, PhD Dr. Ozes has nothing to disclose.
Bo Hu, MD, PhD Dr. Hu has nothing to disclose.
Daniel Moiseev Daniel Moiseev has nothing to disclose.
Isabella Schena, BS Miss B.S. has nothing to disclose.
Bulat Faezov Mr. Faezov has nothing to disclose.
Roland Dunbrack, Jr., PhD Dr. Dunbrack has nothing to disclose.
Jonathan Chernoff Jonathan Chernoff has nothing to disclose.
Jun Li, MD, PhD, FAAN The institution of Dr. Li has received personal compensation in the range of $500-$4,999 for serving as a Consultant for FDA. The institution of Dr. Li has received research support from NIH. Dr. Li has a non-compensated relationship as a Associate Editor of ACTN journal with ANA that is relevant to AAN interests or activities.
Bruce A. C. Cree, MD, PhD, MCR, FAAN The institution of Dr. Cree has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. The institution of Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for EMD Serono. The institution of Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. The institution of Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. The institution of Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for TG Therapeutics. The institution of Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Autobahn. The institution of Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avotres. The institution of Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Horizon. Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Neuron23. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Boston Pharma. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Hexal/Sandoz. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kyverna. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Boston Pharma. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunic AG. The institution of Dr. Cree has received research support from Genentech. Dr. Cree has received publishing royalties from a publication relating to health care.
David Wang, MD Dr. Wang has nothing to disclose.
Xingyao Wu Xingyao Wu has nothing to disclose.
Mason LaMarche, MD Dr. LaMarche has nothing to disclose.
Michael E. Shy, MD, FAAN Dr. Shy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Applied Therapeutics. The institution of Dr. Shy has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for DTx Pharma. The institution of Dr. Shy has received research support from NIH. The institution of Dr. Shy has received research support from Muscular Dystrophy Association. The institution of Dr. Shy has received research support from Charcot Marie Tooth Association. The institution of Dr. Shy has received research support from Applied Therapeutics.
Yunhong *Use 186003 Bai, MD Dr. Bai has nothing to disclose.
Jai *use 131043 Perumal No disclosure on file
Pitcha Chompoopong, MD Dr. Chompoopong has nothing to disclose.
Eli Muchtar, MD Dr. Muchtar has nothing to disclose.
Michelle L. Mauermann, MD, FAAN The institution of Dr. Mauermann has received research support from IONIS. The institution of Dr. Mauermann has received research support from Alnylam. Dr. Mauermann has received publishing royalties from a publication relating to health care.
Xiangyi Meng, PhD Dr. Meng has nothing to disclose.
Hani Kushlaf, MD, FAAN Dr. Kushlaf has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion AstraZeneca Rare Disease. Dr. Kushlaf has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Genzyme. Dr. Kushlaf has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Kushlaf 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. Kushlaf has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Sanofi Genzyme.
Drago Bratkovic Drago Bratkovic has nothing to disclose.
Barry Byrne Barry Byrne has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for AavantiBio. 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 Pfizer. Barry Byrne has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex. Barry Byrne has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Barry Byrne 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. Barry Byrne has received intellectual property interests from a discovery or technology relating to health care.
Jordi Diaz-Manera Jordi Diaz-Manera 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.
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.
Mark Roberts Mark Roberts has nothing to disclose.
Antonio Toscano, MD Dr. Toscano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Genzyme.
Jeff Castelli, PhD Dr. Castelli has received personal compensation for serving as an employee of Amicus Therpeutics.
Frederick Holdbrook Frederick Holdbrook has received personal compensation for serving as an employee of Amicus Therapeutics Inc.. Frederick Holdbrook has stock in Amicus Therapeutics Inc..
Yasmine Wasfi, MD,PhD Dr. Wasfi has received personal compensation for serving as an employee of Amicus Therapeutics. Dr. Wasfi has stock in Amicus Therapeutics.
Benedikt Schoser Benedikt Schoser has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi Genzyme. Benedikt Schoser has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amicus. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bayer. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Taysha. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. 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 Speakers Bureau for Kedrion.
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.
Kristl Claeys, MD, PhD Dr. Claeys has nothing to disclose.
Sheela Sitaraman Das Dr. Das has received personal compensation for serving as an employee of Amicus Therapeutics.
Wael Richeh, MD Dr. Richeh has nothing to disclose.
Hacer Durmus, MD Dr. Durmus has nothing to disclose.
Asuman *Use 388835 Gedikbasi, Sr., MD,PhD Prof. Gedikbasi has nothing to disclose.
Serdar Ceylaner Serdar Ceylaner has nothing to disclose.
ESEN KIYAN, MD Prof. KIYAN has nothing to disclose.
Fatma Yesim Parman, MD Dr. Parman has nothing to disclose.
Jesus F. Lovera, MD Dr. Lovera has nothing to disclose.
Amanda C. Peltier, MD, MS Dr. Peltier has received personal compensation for serving as an employee of Alnylam. Dr. Peltier has received personal compensation for serving as an employee of Akcea. Dr. Peltier has received personal compensation for serving as an employee of CSL Behring. Dr. Peltier has received personal compensation for serving as an employee of Catalyst. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Csl Behring. Dr. Peltier has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Peltier has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Akcea. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for CSL Behring. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for catalyst. The institution of Dr. Peltier has received research support from NIH.
Hristelina S. Ilieva, MD The institution of Dr. Ilieva has received research support from ALSA. The institution of Dr. Ilieva has received research support from Strategius. Dr. Ilieva has received personal compensation in the range of $500-$4,999 for serving as a reviewer with ALS Canada. Dr. Ilieva has received personal compensation in the range of $0-$499 for serving as a reviewer with DOD.
James Eyer Mr. Eyer has nothing to disclose.
Urvi G. Desai, MD, FAAN Dr. Desai has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Desai 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. Desai 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. Desai 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. Desai 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. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Desai 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. Desai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx.
Raisa Coral Martinez, MD No disclosure on file
Samuel Anthony Collins, MBBS,PhD Dr. Collins has received personal compensation for serving as an employee of Edgewise Therapeutics.
Han Phan, MD Dr. Phan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Wave Biosciences. Dr. Phan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stealth.
Alan J. Russell, PhD Mr. Russell has received personal compensation for serving as an employee of Edgewise Therapeutics. Mr. Russell has received personal compensation in the range of $0-$499 for serving as an officer or member of the Board of Directors for Edgewise Therapeutics. Mr. Russell has stock in Edgewise Therapeutics. Mr. Russell has received intellectual property interests from a discovery or technology relating to health care.
Ben Barthel, PhD Dr. Barthel has received personal compensation for serving as an employee of Edgewise Therapeutics. Dr. Barthel has stock in Edgewise Therapeutics.
Liz Thaler Mrs. Thaler has nothing to disclose.
Nicole Kilburn Ms. Kilburn has received personal compensation for serving as an employee of Edgewise Therapeutics. Ms. Kilburn has stock in Edgewise Therapeutics.
Maria Mancini No disclosure on file
James MacDougall, PhD Mr. MacDougall has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Actinium Pharmaceuticals. Mr. MacDougall has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Aura Biosciences. Mr. MacDougall has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vigil Neuro. Mr. MacDougall has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Catabasis Pharmaceuticals. Mr. MacDougall has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Edgewise Therapeutics. Mr. MacDougall has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Enterin . Mr. MacDougall has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Neurogastrx, Inc.. Mr. MacDougall has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Promentis. Mr. MacDougall has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Synlogic Therapeutics. Mr. MacDougall has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Cogent Biosciences. Mr. MacDougall has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Point Biopharma. Mr. MacDougall has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ultragenyx.
Joanne Donovan, MD,PhD Dr. Donovan has received personal compensation for serving as an employee of Edgewise Therapeutics. Dr. Donovan has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Edgewise Therapeutics. Dr. Donovan has stock in Edgewise Therapeutics.
Joseph Hentz No disclosure on file
Shiwen Wu, MD Dr. Wu has nothing to disclose.
Sheffali Gulati, MD, FAMS, FIAP, FIMS Prof. Gulati has nothing to disclose.
Hirofumi Komaki Hirofumi Komaki has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Nippon Shinyaku. Hirofumi Komaki has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. The institution of Hirofumi Komaki 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 Inc. Hirofumi Komaki has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Chugai .
Rosa Escobar Cedillo Rosa Escobar Cedillo has nothing to disclose.
Anna Kostera-Pruszczyk, MD, PhD No disclosure on file
Jin-Hong Shin, MD,PhD Dr. Shin has nothing to disclose.
Kazuhiro Haginoya, MD Dr. Haginoya has nothing to disclose.
Vinay Penematsa Vinay Penematsa has nothing to disclose.
Connie Chou Connie Chou has stock in PTC Therapeutics.
Paula Williams, Other Mrs. Williams has nothing to disclose.
Christian Werner Christian Werner has received personal compensation for serving as an employee of PTC Therapeutics .
Jeffrey Statland, MD Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MT Pharma. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA.
Aravindhan Veerapandiyan, MD Dr. Veerapandiyan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AMO Pharma, AveXis, Biogen, Edgewise Therapeutics, FibroGen, Novartis, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Inc., UCB Pharma, Catalyst, Entrada, Scholar Rock, Catalyst, Lupin. Dr. Veerapandiyan has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MedLink Neurology. The institution of Dr. Veerapandiyan has received research support from AMO Pharma, Capricor Therapeutics, Edgewise Therapeutics, FibroGen, Muscular Dystrophy Association, Novartis, Parent Project Muscular Dystrophy, Pfizer, RegenxBio and Sarepta Therapeutics. Dr. Veerapandiyan has received personal compensation in the range of $5,000-$9,999 for serving as a MD with PPMD, MDA.
Vamshi Rao, MD No disclosure on file
Janannaz Dastgir, DO Dr. Dastgir has received personal compensation for serving as an employee of Regenxbio.
Paulo Falabella Paulo Falabella has received personal compensation for serving as an employee of REGENXBIO Inc.
Steve Pakola No disclosure on file
Sarang Rastogi, Other Dr. Rastogi has received personal compensation for serving as an employee of REGENXBIO. Dr. Rastogi has stock in REGENXBIO.
Dawn Phillips Dawn Phillips has received personal compensation for serving as an employee of Regenxbio.
Catherine Wilson Mrs. Wilson has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Regenxbio. Mrs. Wilson has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Sanofi.
Nidal Boulos, PhD Dr. Boulos has received personal compensation for serving as an employee of REGENXBIO. Dr. Boulos has stock in REGENXBIO.
Michelle Gilmor Michelle Gilmor has received personal compensation for serving as an employee of REGENXBIO. Michelle Gilmor has stock in REGENXBIO.
Lin Yang, PhD Dr. Yang has nothing to disclose.
Hiren Patel Dr. Patel has stock in Regenxbio Inc.
Michele Fiscella Mr. Fiscella has received personal compensation for serving as an employee of REGENXBIO. Mr. Fiscella has stock in REGENXBIO.
Olivier Danos, PhD Dr. Danos has received personal compensation for serving as an employee of REGENXBIO. Dr. Danos has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vor Bio. Dr. Danos 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 Coave Therapeutics. Dr. Danos has stock in REGENXBIO. Dr. Danos has stock in Coave Therapeutics. Dr. Danos has stock in Rocket Therapeutics.
Yunhong Bai Dr. Bai has nothing to disclose.