2024 Annual Meeting | S5 - ALS and CMT: New Therapeutic Approaches

Date Sunday 04/14/24

Time 03:30 PM - 05:30 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) Elie Naddaf, MD, Christina N. Fournier, MD, MSc

Topic(s) Neuromuscular and Clinical Neurophysiology (EMG)

Learning Objectives A group of abstracts covering a similar topic will be presented in an eight-minute format followed by four minutes of question and answer with the author.

CME Available 2.00 CME credits

Program Evaluations

Event Timeline
03:30 PM - 03:54 PM MDT	Speaker	2024 Sheila Essey Award: An Award for ALS Research: This award recognizes an individual who has made significant research contributions in the search for the cause, prevention of, and cure for amyotrophic lateral sclerosis (ALS). Eva Feldman, MD, PhD, FAAN
03:54 PM - 04:06 PM MDT	Abstract	003 - Pridopidine for the Treatment of ALS—Results from the Phase 2 Healey ALS Platform Trial Jeremy M. Shefner, MD, PhD, FAAN
04:06 PM - 04:18 PM MDT	Abstract	004 - Results from a Phase 2 Clinical Trial of Repeated Intrathecal Autologous Adipose-derived MSCs in ALS Nathan P. Staff, MD, PhD, FAAN
04:18 PM - 04:30 PM MDT	Abstract	005 - Value of FVC and its Rate of Decline for Predicting Survival in ALS Muhannad Seyam, MD
04:30 PM - 04:42 PM MDT	Abstract	006 - Next Generation Prodrug Troriluzole: Increased Bioavailability of Riluzole with No Food Effect in Healthy Subjects Heather Sevinsky
04:42 PM - 04:54 PM MDT	Abstract	007 - Inhibition of MicroRNA-183 Attenuates Pathological TDP-43 Aggregation via SQSTM1/p62 in ALS Liang Lu, MD, FAAN
04:54 PM - 05:06 PM MDT	Abstract	008 - Pre-clinical Antisense Oligonucleotide Treatment of CMT2E in a Human Induced Pluripotent Stem Cell (iPSC)-derived Motor Neuron Model Mario A. Saporta, MD, PhD, FAAN
05:06 PM - 05:18 PM MDT	Abstract	009 - Potential ASO-based Personalized Treatment for Charcot-Marie-Tooth Disease Type 2S Sandra Smieszek
05:18 PM - 05:30 PM MDT	Abstract	010 - Discovering and Rescuing Toxic Autophagy Derangements in CMT4B3 Using a Novel iPSC-derived Peripheral Nervous System Model of MTMR5 Biology Jason Chua, MD, PhD

Faculty Disclosures
Eva Feldman, MD, PhD, FAAN	The institution of Dr. Feldman has received research support from National Institutes of Health. The institution of Dr. Feldman has received research support from Centers for Disease Control and Prevention / Agency for Toxic Substances and Disease Registry. The institution of Dr. Feldman has received research support from Juvenile Diabetes Research Foundation. Dr. Feldman has received intellectual property interests from a discovery or technology relating to health care. Dr. Feldman has a non-compensated relationship as a Editorial Board Member with Journal of the Peripheral Nervous System, Experimental Neurology, Nature Neurology Reviews, Neurobiology of Disease, NeuroRx, Journal of Neurology Neurosurgery and Psychiatry, Scientific Reports, Lancet Neurology, Med (Cell Press Journal) that is relevant to AAN interests or activities. Dr. Feldman has a non-compensated relationship as a Editor-in-Chief with Oxford University Press Contemporary Neurology Series that is relevant to AAN interests or activities. Dr. Feldman has a non-compensated relationship as a Membership Committee Member, Strategic Planning Committee Member, and Chair of Section 07 with National Academy of Medicine that is relevant to AAN interests or activities. Dr. Feldman has a non-compensated relationship as a Associate Editor with Annals of Neurology that is relevant to AAN interests or activities. Dr. Feldman has a non-compensated relationship as a Governance Committee Chair with American Neurological Association that is relevant to AAN interests or activities. Dr. Feldman has a non-compensated relationship as a Membership Committee Member, Strategic Planning Committee Member, Chair of Section 07, Neuroscience Forum, Council Member with National Academy of Medicine that is relevant to AAN interests or activities. Dr. Feldman has a non-compensated relationship as a Committee on Making ALS a Livable Disease Member with National Academy of Sciences, Engineering, and Medicine that is relevant to AAN interests or activities. Dr. Feldman has a non-compensated relationship as a Member, Blueprint Neurotherapeutics Network for Biologics (BPN-Biologics) External Oversight Committee with National Institutes of Health that is relevant to AAN interests or activities.
Jeremy M. Shefner, MD, PhD, FAAN	Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Uniqure. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clene. Dr. Shefner has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cytokinetics. Dr. Shefner has received personal compensation in the range of $0-$499 for serving as a Consultant for Mitsubishi Tanabe Pharma America. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for annexon. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurosense. Dr. Shefner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neuvivo. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nura BIo. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Prilenia. Dr. Shefner has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for swanbio. Dr. Shefner has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for vertex. Dr. Shefner 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. Shefner has received personal compensation in the range of $50,000-$99,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for UpToDate. The institution of Dr. Shefner has received research support from NIH. The institution of Dr. Shefner has received research support from Mitsubishi Tanabe Pharma America. The institution of Dr. Shefner has received research support from Amylyx. The institution of Dr. Shefner has received research support from BIogen. The institution of Dr. Shefner has received research support from Cytokinetics. The institution of Dr. Shefner has received research support from IONIS. The institution of Dr. Shefner has received research support from Healey Center for ALS. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a grant review chair with DOD- General Dynamics.
Rup Tandan, MD, FRCP, FAAN	Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Apellis. Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amylyx. The institution of Dr. Tandan has received research support from Alexion. The institution of Dr. Tandan has received research support from Apellis. The institution of Dr. Tandan has received research support from Cytokinetics.
Anthony J. Windebank, MD, FAAN	Dr. Windebank has nothing to disclose.
Peter H. King, MD	The institution of Dr. King has received research support from Department of Veterans Affairs. The institution of Dr. King has received research support from NIH.
Ted M. Dawson, MD, PhD, FAAN	Dr. Dawson has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Valted Seq Inc. An immediate family member of Dr. Dawson 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 Valted Seq Inc. An immediate family member of Dr. Dawson has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for eNeuro. Dr. Dawson has stock in Valted Seq. Dr. Dawson has stock in American Gene Technologies International Inc. Dr. Dawson has stock in Inhibikase Therapeutics, Inc. Dr. Dawson has stock in D&D Pharmatech. Dr. Dawson has stock in Abbvie. An immediate family member of Dr. Dawson has stock in Valted Seq Inc. An immediate family member of Dr. Dawson has stock in Inhibikase Therapeutics. An immediate family member of Dr. Dawson has stock in D&D Pharmatech. The institution of Dr. Dawson has received research support from Sun Pharmaceutical Industries Ltd. The institution of Dr. Dawson has received research support from Aevum. Dr. Dawson has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Dawson has received intellectual property interests from a discovery or technology relating to health care. Dr. Dawson has received personal compensation in the range of $100,000-$499,999 for serving as a Advisor with Mitokinin.
Merit E. Cudkowicz, MD, MSC, FAAN	Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for biogen. Dr. Cudkowicz has received personal compensation in the range of $0-$499 for serving as a Consultant for cytokinetics. Dr. Cudkowicz has received personal compensation in the range of $0-$499 for serving as a Consultant for immunitypharm. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for transposon. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Quralis. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Regeneron. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for vectorY. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for inflectis. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for novartis. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ono. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for otsuka. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for coya. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ac immune. Dr. Cudkowicz has received personal compensation in the range of $0-$499 for serving as a Consultant for pontifax. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Inflectis. Dr. Cudkowicz has received personal compensation in the range of $0-$499 for serving as a Consultant for ab science. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for aclipse. Dr. Cudkowicz has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Locust Walk. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pasithea. Dr. Cudkowicz has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Praxis Precision Medicine. Dr. Cudkowicz 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 Annals of Neurology . The institution of Dr. Cudkowicz has received research support from NINDS. The institution of Dr. Cudkowicz has received research support from ALSFAC. The institution of Dr. Cudkowicz has received research support from UCB ra. The institution of Dr. Cudkowicz has received research support from Biohaven. The institution of Dr. Cudkowicz has received research support from Clene nanomedicine. The institution of Dr. Cudkowicz has received research support from prilenia. The institution of Dr. Cudkowicz has received research support from ALS one. The institution of Dr. Cudkowicz has received research support from Seelos. The institution of Dr. Cudkowicz has received research support from Calico. The institution of Dr. Cudkowicz has received research support from denali. The institution of Dr. Cudkowicz has received research support from ITB. The institution of Dr. Cudkowicz has received research support from PharmAust. The institution of Dr. Cudkowicz has received research support from woolsley. Dr. Cudkowicz has received research support from lilly. The institution of Dr. Cudkowicz has received research support from J& J. Dr. Cudkowicz has received publishing royalties from a publication relating to health care.
Marwan N. Sabbagh, MD, FAAN	Dr. Sabbagh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Eisai. Dr. Sabbagh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genetech=Roche. Dr. Sabbagh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novo Nordisk. Dr. Sabbagh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lilly. Dr. Sabbagh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Synaptogenix. Dr. Sabbagh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Signant Health. Dr. Sabbagh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Anavex. Dr. Sabbagh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cognito Therapeutics. Dr. Sabbagh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GSK. Dr. Sabbagh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbvie. Dr. Sabbagh 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 CervoMed. Dr. Sabbagh has stock in Neurotau. Dr. Sabbagh has stock in Seq Biomarque. Dr. Sabbagh has stock in uMethod Health. Dr. Sabbagh has stock in Athira. Dr. Sabbagh has stock in Lighthouse Pharmaceuticals. Dr. Sabbagh has stock in Alzheon. The institution of Dr. Sabbagh has received research support from NIH. The institution of Dr. Sabbagh has received research support from ADDF.
Waqar Waheed, MD, MBBS	Dr. Waheed has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB.
Matilde Inglese, MD, PhD	Dr. Inglese 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. Dr. Inglese 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. Inglese has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NOVARTIS. Dr. Inglese has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for MERCK-SERONO. Dr. Inglese has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ROCHE. Dr. Inglese has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MS Journal.
Liang Lu, MD, FAAN	Dr. Lu has nothing to disclose.
Bjorn E. Oskarsson, MD, FAAN	Dr. Oskarsson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. The institution of Dr. Oskarsson has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Oskarsson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AnnJi. The institution of Dr. Oskarsson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi. Dr. Oskarsson has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Tsumura. The institution of Dr. Oskarsson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MediciNova. The institution of Dr. Oskarsson has received research support from Biogen. The institution of Dr. Oskarsson has received research support from Medicinova. The institution of Dr. Oskarsson has received research support from Cytokinetics. The institution of Dr. Oskarsson has received research support from Calico. The institution of Dr. Oskarsson has received research support from Mitsubishi. The institution of Dr. Oskarsson has received research support from Tsumura. The institution of Dr. Oskarsson has received research support from Sanofi. The institution of Dr. Oskarsson has received research support from AZTherapeutics. The institution of Dr. Oskarsson has received research support from Orion. The institution of Dr. Oskarsson has received research support from Esaii.
Erika Driver-Dunckley, MD, FAAN	Dr. Driver-Dunckley has nothing to disclose.
Mario A. Saporta, MD, PhD, FAAN	Dr. Saporta has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for SwanBio. Dr. Saporta has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Pharnext. Dr. Saporta has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Applied Therapeutics.
Irfan Qureshi, MD	Dr. Qureshi has received personal compensation for serving as an employee of Biohaven. Dr. Qureshi has stock in Biohaven Pharmaceuticals.
Stephan Zuchner, MD, FAAN	Dr. Zuchner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Applied Therapeutics. The institution of Dr. Zuchner has received research support from Muscular Dystrophy Association. The institution of Dr. Zuchner has received research support from CMT Association. Dr. Zuchner has received intellectual property interests from a discovery or technology relating to health care.
Valina Dawson, PhD	Dr. Dawson has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Valted Seq Inc. An immediate family member of Dr. Dawson has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Valted Seq Inc. Dr. Dawson has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for eNeuro. Dr. Dawson has stock in Valted Seq Inc. An immediate family member of Dr. Dawson has stock in Valted Seq Inc. Dr. Dawson has stock in D&D Pharmatech. An immediate family member of Dr. Dawson has stock in D&D Pharmatech. Dr. Dawson has stock in Inhibikase Therapeutics, Inc. An immediate family member of Dr. Dawson has stock in Inhibikase Therapeutics, Inc,. An immediate family member of Dr. Dawson has stock in American Gene Technologies International. An immediate family member of Dr. Dawson has stock in Abbvie. The institution of an immediate family member of Dr. Dawson has received research support from Sun Pharmaceutical Industries Ltd. The institution of an immediate family member of Dr. Dawson has received research support from Sebum. Dr. Dawson has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Dawson has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Dawson has received personal compensation in the range of $100,000-$499,999 for serving as a Advisor with Mitokini.
Nathan P. Staff, MD, PhD, FAAN	Dr. Staff has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Stem Cell Research & Therapy. Dr. Staff has received research support from National Institutes of Health.
Jayawant N. Mandrekar, PhD	Dr. Mandrekar has nothing to disclose.
James D. Berry, MD MPH	Dr. Berry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Berry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clene Nanomedicine. Dr. Berry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MT Pharma Holdings of America. Dr. Berry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Regeneron. Dr. Berry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Berry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Berry has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Berry has stock in ReactNeuro. The institution of Dr. Berry has received research support from Biogen. The institution of Dr. Berry has received research support from MT Pharma of America. The institution of Dr. Berry has received research support from Rapa Therapeutics. The institution of Dr. Berry has received research support from Brainstorm Cell Therapeutics. The institution of Dr. Berry has received research support from Alexion. The institution of Dr. Berry has received research support from nQ Medical. The institution of Dr. Berry has received research support from ALSA. The institution of Dr. Berry has received research support from MDA. The institution of Dr. Berry has received research support from Amylyx. The institution of Dr. Berry has received research support from Transposon. Dr. Berry has a non-compensated relationship as a Scientific Advisor with Everything ALS that is relevant to AAN interests or activities.
Iryna Muzyka, MD	Dr. Muzyka has nothing to disclose.
Melanie Leitner, PhD	Dr. Leitner has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Prilenia. Dr. Leitner has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for ALS Investment Fund. Dr. Leitner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Fundamental Pharma. Dr. Leitner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Caraway. Dr. Leitner has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Affinia Therapeutics. Dr. Leitner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Jannsen. Dr. Leitner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Tolerion/Alpha-5 Integrin. Dr. Leitner has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Verge Genomics. Dr. Leitner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Tranquis. Dr. Leitner has stock in Bristol Myers Squibb. Dr. Leitner has stock in Editas. Dr. Leitner has stock in Provention Bio. Dr. Leitner has stock in Sanofi. Dr. Leitner has stock in Vertex. Dr. Leitner has stock in GSK. Dr. Leitner has stock in Eledon. Dr. Leitner has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant with ALS Finding a Cure. Dr. Leitner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant with Charley's Fund.
Christina N. Fournier, MD, MSc	Dr. Fournier has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Fournier has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roon. Dr. Fournier has received personal compensation in the range of $500-$4,999 for serving as a Consultant for QurAlis. Dr. Fournier has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Springer Journals. The institution of Dr. Fournier has received research support from Veterans Affairs. The institution of Dr. Fournier has received research support from Department of Defense. The institution of Dr. Fournier has received research support from Amylyx. The institution of Dr. Fournier has received research support from Biogen. The institution of Dr. Fournier has received research support from MT Pharma. The institution of Dr. Fournier has received research support from Columbia University. The institution of Dr. Fournier has received research support from Denali.
Vladimir Coric	Vladimir Coric has received personal compensation for serving as an employee of Biohaven. Vladimir Coric has received personal compensation in the range of $1,000,000+ for serving as an officer or member of the Board of Directors for Bioahven. Vladimir Coric has stock in Biohaven. Vladimir Coric has received intellectual property interests from a discovery or technology relating to health care.
Nicolas Madigan, MD	Dr. Madigan has nothing to disclose.
Eric A. Macklin, PhD	The institution of Dr. Macklin has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AI Therapeutics. Dr. Macklin has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Chase Therapeutics. Dr. Macklin has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bial Biotech. Dr. Macklin 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. Macklin has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stoparkinson Healthcare LLC. Dr. Macklin has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Macklin 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. Macklin has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. The institution of Dr. Macklin has received research support from Biohaven. The institution of Dr. Macklin has received research support from Clene Nanomedicine. The institution of Dr. Macklin has received research support from Mitsubishi Tanabe Pharmaceuticals America. The institution of Dr. Macklin has received research support from Prilenia. The institution of Dr. Macklin has received research support from UCB Ra Pharma. The institution of Dr. Macklin has received research support from Revalesio. The institution of Dr. Macklin has received research support from Seelos. The institution of Dr. Macklin has received research support from Calico. The institution of Dr. Macklin has received research support from Denali. The institution of Dr. Macklin has received research support from NeuroDex. The institution of Dr. Macklin has received research support from Alector. The institution of Dr. Macklin has received research support from ITB-Med.
Sabrina Paganoni, MD, PhD	Dr. Paganoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics. Dr. Paganoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jannsen. Dr. Paganoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Paganoni has received personal compensation in the range of $0-$499 for serving as a Consultant for Sola. Dr. Paganoni has received personal compensation in the range of $0-$499 for serving as a Consultant for Frequency Therapeutics. The institution of Dr. Paganoni has received research support from Amylyx. The institution of Dr. Paganoni has received research support from Revalesio. The institution of Dr. Paganoni has received research support from Alector. The institution of Dr. Paganoni has received research support from UCB. The institution of Dr. Paganoni has received research support from Biohaven. The institution of Dr. Paganoni has received research support from Clene. The institution of Dr. Paganoni has received research support from Prilenia. The institution of Dr. Paganoni has received research support from Seelos. The institution of Dr. Paganoni has received research support from Calico. The institution of Dr. Paganoni has received research support from Denali. The institution of Dr. Paganoni has received research support from Anelixis. The institution of Dr. Paganoni has received research support from Cytokinetics.
Elie Naddaf, MD	Dr. Naddaf has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Expert Connect. The institution of Dr. Naddaf has received research support from NIAMS. The institution of Dr. Naddaf has received research support from American Neuromuscular Foundation. The institution of Dr. Naddaf has received research support from Fulcrum therapeutics. The institution of Dr. Naddaf has received research support from Abcuro. The institution of Dr. Naddaf has received research support from Cabaletta .
Ron Hashmonay, MD	No disclosure on file
Asim Mahmood	No disclosure on file
	No disclosure on file
Yael Cohen	Yael Cohen has nothing to disclose.
Paul Goldberg	Paul Goldberg has received personal compensation for serving as an employee of Prilenia. Paul Goldberg has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Ionis Pharmaceuticals. Paul Goldberg has stock in Prilenia.
Jason Chua, MD, PhD	Dr. Chua has nothing to disclose.
Muhannad Seyam, MD	Dr. Seyam has nothing to disclose.
Richard Bertz	Richard Bertz has received personal compensation for serving as an employee of Biohaven Pharmaceuticals. Richard Bertz has stock in Biohaven Pharmaceuticals.
Elizabeth H. Jacobs	An immediate family member of Ms. Jacobs has received personal compensation for serving as an employee of Frida. The institution of Ms. Jacobs has received research support from National Institutes of Health.
Michael Hayden	Michael Hayden has received personal compensation for serving as an employee of Prilenia Therapeutics. Michael Hayden has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CAMP4. Michael Hayden has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for AbCellera Biologics Inc.. Michael Hayden has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Ionis Pharmaceuticals . Michael Hayden 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 89 Bio. Michael Hayden has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Oxford Biomedica . Michael Hayden has stock in Ionis Pharmaceuticals. Michael Hayden has stock in AbCellera Biologics Inc.. Michael Hayden has stock in 89 Bio. The institution of Michael Hayden has received research support from Canadian Institutes of Health Research. The institution of Michael Hayden has received research support from National Research Council Canada. The institution of Michael Hayden has received research support from Huntington Society of Canada. The institution of Michael Hayden has received research support from Bev Hartig Huntington Disease Foundation. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care. Michael Hayden has received intellectual property interests from a discovery or technology relating to health care.
Christos Polymeropoulos	No disclosure on file
Heather Sevinsky	Ms. Sevinsky has received personal compensation for serving as an employee of Biohaven Pharmaceuticals. Ms. Sevinsky has stock in Biohaven Pharmaceuticals.
Sandra Smieszek	Dr. Smieszek has received personal compensation for serving as an employee of Vanda Pharmaceuticals Inc.. Dr. Smieszek has stock in Vanda Pharmaceuticals Inc..
Lori Chibnik	No disclosure on file
Adriana Rebelo	No disclosure on file
Yan Zhang	No disclosure on file
Diantha Howard	No disclosure on file
Allan Dietz	No disclosure on file
Han-Cheon Kim	No disclosure on file
Stephen Kaplita	No disclosure on file
Kimberly Gentile	No disclosure on file
Alicia Algeciras-Schimnich	Alicia Algeciras-Schimnich has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche Diagnostics. Alicia Algeciras-Schimnich has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fujirebio Diagnostics. Alicia Algeciras-Schimnich has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche Diagnostics.
Matt Danzi	Matt Danzi has nothing to disclose.
Joan Skelly	Joan Skelly has nothing to disclose.
Jo Ann Malatesta	Jo Ann Malatesta has nothing to disclose.
Lindsay Pothier	No disclosure on file
Michal Geva	Michal Geva has received personal compensation for serving as an employee of Prilenia Therapeutics. Michal Geva has received intellectual property interests from a discovery or technology relating to health care.
Gunther Birznieks	Gunther Birznieks has received personal compensation for serving as an employee of Vanda Pharmaceuticals. Gunther Birznieks has received intellectual property interests from a discovery or technology relating to health care.
Mihael Polymeropoulos	No disclosure on file
Carly Mester	No disclosure on file
Pranathi Madde	No disclosure on file
Timothy Wiltshire	No disclosure on file
Michael Deeds	No disclosure on file
Michelle Turner	No disclosure on file
Daniel Figdore	Daniel Figdore has nothing to disclose.
Lisa Thuro	No disclosure on file
Christina Tyner	Christina Tyner has received personal compensation for serving as an employee of Vanda Pharmaceuticals Inc. Christina Tyner has stock in Vanda Pharmaceuticals Inc..
Caroline Johnson	Caroline Johnson has received personal compensation for serving as an employee of Vanda Pharmaceuticals, Inc.. Caroline Johnson has stock in Vanda Pharmaceuticals, Inc..
Bharat Awsare	Bharat Awsare has received personal compensation for serving as an employee of Biohaven. Bharat Awsare has stock in Biohaven.
Jessica Medina	No disclosure on file
Tracy Nepomuceno	No disclosure on file
Eric Ashbrenner	Eric Ashbrenner has received personal compensation for serving as an employee of Biohaven. Eric Ashbrenner has stock in Biohaven. An immediate family member of Eric Ashbrenner has stock in Biohaven.
Rachel Rozakis	No disclosure on file
Richard Hubbell	No disclosure on file
Betty Hussey	No disclosure on file
Kelly Chen	Kelly Chen has received personal compensation for serving as an employee of Prilenia Therapeutics.
Thomas Osgood	No disclosure on file
Sarah Stumpf	No disclosure on file
Bart Przychodzen	No disclosure on file

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