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

Date Tuesday 04/16/24

Time 01:00 PM - 03:00 PM MDT Add To Calendar

Session Format This program will be presented both in-person and online

On Demand This program will be available in the meeting's On Demand product.

Event Type Scientific Platform Session

Moderator(s) Colin Quinn, MD, Michelle L. Mauermann, MD, FAAN

Topic(s) Neuromuscular and Clinical Neurophysiology (EMG)

Learning Objectives 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.

CME Available 2.00 CME credits

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
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.
Zarife Sahenk, MD, PhD, FAAN	Dr. Sahenk 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.
Mazen M. Dimachkie, MD, FAAN	Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Octapharma. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medlink. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL Behring. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Janssen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TACT / Treat NMD. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cabaletta Bio. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clinical Neurological Society of America, Inc . Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ig Society, Inc. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abcuro. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amicus. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for EMD Serono. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipsen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nuvig. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fortrea. The institution of Dr. Dimachkie has received research support from NIH. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received publishing royalties from a publication relating to health care.
Tahseen Mozaffar, MD, FAAN	Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 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 $10,000-$49,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas Gene Therapy. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics. 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. 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 Astellas Gene Therpay. The institution of Dr. Mozaffar has received research support from Cartesian. The institution of Dr. Mozaffar has received research support from Cabaletta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.
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.
Kazuhiro Haginoya, MD	Dr. Haginoya 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.
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.
Bruce A. Cree, MD, PhD, MAS, 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 $10,000-$49,999 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 on a Scientific Advisory or Data Safety Monitoring board for Immunic AG. The institution of Dr. Cree has received research support from Genentech. The institution of Dr. Cree has received research support from Kyverna. Dr. Cree has received publishing royalties from a publication relating to health care.
Jesus Lovera, MD	Dr. Lovera has nothing to disclose.
Amanda C. Peltier, MD, MS	Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Peltier has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for astrazeneca. Dr. Peltier has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for CSL Behring. 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 $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for alnylam. Dr. Peltier has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for astrazeneca. Dr. Peltier has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Goldman Ismail. The institution of Dr. Peltier has received research support from NIH. The institution of Dr. Peltier has received research support from CSL Behring.
Steve Pakola	No disclosure on file
Janannaz Dastgir, DO	Dr. Dastgir has received personal compensation for serving as an employee of Regenxbio. Dr. Dastgir has a non-compensated relationship as a Scientific Advisory Board with Team Titin that is relevant to AAN interests or activities. Dr. Dastgir has a non-compensated relationship as a Scientific Advisory Board with Hannah's Hope Fund / Gengan Therapeutics that is relevant to AAN interests or activities.
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.
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.
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.
Joseph Hentz	No disclosure on file
Vamshi Rao, MD	No disclosure on file
Jeffrey Statland, MD	Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vita Therapeutics. Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex . The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA.
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 Amgen. 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 Catalyst Pharmaceuticals. 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 Scientific Advisory or Data Safety Monitoring board for Sarepta Therapeutics . Dr. Kushlaf has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amicus Therapeutics .
Wael Richeh, MD	Dr. Richeh has nothing to disclose.
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.
Hacer Durmus, MD	Dr. Durmus has nothing to disclose.
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.
Raisa C. Martinez, MD	No disclosure on file
Yunhong Bai	Dr. Bai has nothing to disclose.
	No disclosure on file
Sheffali Gulati, MD, FAMS, FIAP, FNASc, FIANs	Prof. Gulati has nothing to disclose.
Aravindhan Veerapandiyan, MD	Dr. Veerapandiyan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen, Novartis,Edgewise Therapeutics, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Inc., UCB Pharma, Catalyst, Entrada, Lupin, Percheron, ITF. Dr. Veerapandiyan has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MedLink Neurology. Dr. Veerapandiyan has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Muscle and Nerve. The institution of Dr. Veerapandiyan has received research support from AMO Pharma, Capricor Therapeutics, Edgewise Therapeutics, FibroGen, Muscular Dystrophy Association, Novartis, Parent Project Muscular Dystrophy, Pfizer, RegenxBio, SolodBio and Sarepta Therapeutics. Dr. Veerapandiyan has received personal compensation in the range of $5,000-$9,999 for serving as a MD with PPMD, MDA.
Xiangyi Meng, PhD	Dr. Meng has nothing to disclose.
Fatma Yesim Parman, MD	Dr. Parman has nothing to disclose.
Pitcha Chompoopong, MD	Dr. Chompoopong has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astrazeneca. Dr. Chompoopong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alnylam.
Kristl Claeys, MD, PhD	Dr. Claeys has nothing to disclose.
Daniel Moiseev	Daniel Moiseev has nothing to disclose.
Vinay Penematsa	Vinay Penematsa has nothing to disclose.
Eleonora D'Ambrosio, MD	Dr. D'Ambrosio has nothing to disclose.
David Wang, MD	Dr. Wang has nothing to disclose.
Michelle Gilmor	Michelle Gilmor has received personal compensation for serving as an employee of REGENXBIO. Michelle Gilmor has stock in REGENXBIO.
Rosa Escobar Cedillo	Rosa Escobar Cedillo has nothing to disclose.
James Eyer, MD	Mr. Eyer has nothing to disclose.
Anna Kostera-Pruszczyk, MD, PhD	No disclosure on file
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.
Bo Hu, MD, PhD	Dr. Hu has nothing to disclose.
Christian Werner	Christian Werner has received personal compensation for serving as an employee of PTC Therapeutics. Christian Werner has stock in PTC Therapeutics.
Alan J. Russell	No disclosure on file
Jonathan Chernoff	No disclosure on file
Barry Byrne	Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rocket Pharma. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving as an officer or member of the Board of Directors for Muscular Dystrophy Association.
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.
Benedikt Schoser	Benedikt Schoser has received personal compensation for serving as an employee of Alexion. Benedikt Schoser has received personal compensation for serving as an employee of Argenx. Benedikt Schoser has received personal compensation for serving as an employee of Amicus. Benedikt Schoser has received personal compensation for serving as an employee of Astellas. Benedikt Schoser has received personal compensation for serving as an employee of Denali. Benedikt Schoser has received personal compensation for serving as an employee of Avidity . Benedikt Schoser has received personal compensation for serving as an employee of Sanofi. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Taysha. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astellas. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Encoded. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Kedrion.
Xingyao Wu	No disclosure on file
Yunhong Bai	No disclosure on file
Hirofumi Komaki	No disclosure on file
Maria Mancini	No disclosure on file
Drago Bratkovic	No disclosure on file
Mark Roberts	Mark Roberts has nothing to disclose.
Serdar Ceylaner	Serdar Ceylaner has nothing to disclose.
Connie Chou	No disclosure on file
Jordi Diaz-Manera	No disclosure on file
Dawn Phillips	Dawn Phillips has received personal compensation for serving as an employee of REGENXBIO. Dawn Phillips has stock in REGENXBIO.
Paulo Falabella	No disclosure on file
Jeff Castelli	No disclosure on file
Yasmine Wasfi	No disclosure on file
Paula Williams	No disclosure on file
Shiwen Wu	No disclosure on file
James MacDougall	No disclosure on file
Jin-Hong Shin	No disclosure on file
Olivier Danos	Olivier Danos has received personal compensation for serving as an employee of regenxbio. Olivier 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. Olivier Danos has stock in regenxbio. Olivier Danos has received intellectual property interests from a discovery or technology relating to health care.
Nicole Kilburn	No disclosure on file
Ben Barthel	No disclosure on file
Liz Thaler	No disclosure on file
Samuel Collins	No disclosure on file
Bulat Faezov	No disclosure on file
Isabella Schena	No disclosure on file
Roland Dunbrack	No disclosure on file
Esen Kiyan	ESEN KIYAN has nothing to disclose.
Asuman Gedikbasi	No disclosure on file
Sheela Das	No disclosure on file
Burcak Ozes	No disclosure on file
Meisam Naeimi Kararoudi	No disclosure on file
Nidal Boulos	Nidal Boulos has received personal compensation for serving as an employee of REGENXBIO.
Hiren Patel	Hiren Patel has received personal compensation for serving as an employee of Regenxbio. Hiren Patel has stock in Regenxbio.
Sarang Rastogi	No disclosure on file
Eli Muchtar	No disclosure on file
Mason LaMarche	No disclosure on file
Michele Fiscella	No disclosure on file
Catherine Wilson	Catherine Wilson has received personal compensation for serving as an employee of REGENXBIO. Catherine Wilson has stock in REGENXBIO. Catherine Wilson has received personal compensation in the range of $100,000-$499,999 for serving as a Contractor with REGENXBIO.
Lin Yang	No disclosure on file

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