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2021 Annual Meeting | S20 - Motor Neuron Disease/Amyotrophic Lateral Sclerosis

Tuesday 04/20/21
02:00 PM - 03:00 PM EDT Add To Calendar
This program will be presented both in-person and online
This program will be available in the meeting's On Demand product.
Scientific Platform Session
Colin Quinn, MD, Namita Goyal, MD, FAAN
Neuromuscular and Clinical Neurophysiology (EMG)
1.00 CME credit

Program Materials Program Evaluations

Event Timeline
02:00 PM - 02:08 PM EDT Abstract 001 - Design of a Phase 3, Randomized, Placebo-controlled Trial of Tofersen Initiated in Clinically Pre-symptomatic SOD1 Mutation Carriers with a Longitudinal Natural History Run-in
Michael G. Benatar, MBChB, DPhil, FAAN
02:08 PM - 02:16 PM EDT Abstract 002 - Clinical Utility of Whole-Genome Sequencing in an ALS Cohort
Adriano Chio, MD, FAAN
02:16 PM - 02:24 PM EDT Q&A Questions and Answers
02:24 PM - 02:32 PM EDT Abstract 003 - Determinants of clinical response to levosimendan in the REFALS phase 3 study in people with ALS
Merit E. Cudkowicz, MD, MSC, FAAN
02:32 PM - 02:40 PM EDT Abstract 004 - Metabolic Brain Changes Across Different Levels Of Cognitive Impairment In ALS: A 18F-FDG-PET Study
Antonio Canosa
02:40 PM - 02:48 PM EDT Abstract 005 - WVE-004, an investigational stereopure antisense oligonucleotide for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)
Yuanjing Liu
02:48 PM - 03:00 PM EDT Q&A Questions and Answers
Faculty Disclosures
No disclosure on file
Chris Garratt Chris Garratt has received personal compensation for serving as an employee of Orion Pharma.
Janice Wong, MD Dr. Wong has received personal compensation for serving as an employee of Janssen Research & Development. Dr. Wong has received personal compensation for serving as an employee of Biogen. Dr. Wong has stock in Biogen. Dr. Wong has stock in Janssen Research & Development.
Joanne Wuu, SCM The institution of Ms. Wuu has received research support from NIH NINDS. The institution of Ms. Wuu has received research support from NIH NCATS/NINDS. The institution of Ms. Wuu has received research support from CDC. The institution of Ms. Wuu has received research support from Target ALS. The institution of Ms. Wuu has received research support from ALS Recovery Fund. The institution of Ms. Wuu has received research support from NIH/NINDS. The institution of Ms. Wuu has received research support from NIH NINDS. The institution of an immediate family member of Ms. Wuu has received research support from NIH NINDS.
Sonja W. Scholz, MD, FAAN Dr. Scholz has received personal compensation for serving as an employee of National Institutes of Health. An immediate family member of Dr. Scholz has received personal compensation for serving as an employee of National Institutes of Health. The institution of Dr. Scholz has received research support from National Institutes of Health. The institution of an immediate family member of Dr. Scholz has received research support from National Institutes of Health. An immediate family member of Dr. Scholz has received intellectual property interests from a discovery or technology relating to health care. Dr. Scholz has a non-compensated relationship as a Scientific Advisory Council Member with Lewy Body Dementia Association that is relevant to AAN interests or activities. Dr. Scholz has a non-compensated relationship as a Editorial Board Member with JAMA Neurology that is relevant to AAN interests or activities. Dr. Scholz has a non-compensated relationship as a Editorial Board Member with Journal of Parkinson's Disease that is relevant to AAN interests or activities. Dr. Scholz has a non-compensated relationship as a Scientific Advisory Board Member with Mission MSA that is relevant to AAN interests or activities. Dr. Scholz has a non-compensated relationship as a Scientific Advisory Board Member with The GBA1 Canada Initiative (G-Can) that is relevant to AAN interests or activities.
Bob Bucelli, MD, PhD Dr. Bucelli has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen . Dr. Bucelli has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Bucelli has stock in Neuroquestions.com. An immediate family member of Dr. Bucelli has stock in Neuroquestions.com. The institution of Dr. Bucelli has received research support from Biogen. The institution of Dr. Bucelli has received research support from Ionis.
Maura Brunetti No disclosure on file
Sandra D'Alfonso No disclosure on file
Toby A. Ferguson, MD Dr. Ferguson has received personal compensation for serving as an employee of Biogen. Dr. Ferguson has received stock or an ownership interest from Biogen.
Adriano Chio, MD, FAAN Dr. Chio has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics. Dr. Chio has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi. Dr. Chio has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen.
Nicholas J. Maragakis, MD, FAAN Dr. Maragakis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UptoDate. Dr. Maragakis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Apellis Pharmaceuticals. Dr. Maragakis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. An immediate family member of Dr. Maragakis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for National Basketball Association. Dr. Maragakis has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Akava. An immediate family member of Dr. Maragakis has received personal compensation in the range of $0-$499 for serving as an officer or member of the Board of Directors for Johns Hopkins Howard County Medical Center. Dr. Maragakis has stock in Akava. The institution of Dr. Maragakis has received research support from NIH/NINDS. The institution of Dr. Maragakis has received research support from Department of Defense. The institution of Dr. Maragakis has received research support from Maryland Stem Cell Research Fund. Dr. Maragakis has received intellectual property interests from a discovery or technology relating to health care.
Angela L. Genge, MD Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AL-S Pharma. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics. Dr. Genge has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amylyx. Dr. Genge has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Quralis. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Genge has received personal compensation in the range of $0-$499 for serving as a Consultant for WAVE.
Ruth Chia No disclosure on file
Stephanie Fradette Stephanie Fradette has received personal compensation for serving as an employee of Biogen. Stephanie Fradette has received stock or an ownership interest from Biogen.
Michael G. Benatar, MBChB, DPhil, FAAN Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. The institution of Dr. Benatar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Arrowhead. Dr. Benatar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UniQure. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cartesian. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eli Lilly. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alector. Dr. Benatar 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. Benatar 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. Benatar has received intellectual property interests from a discovery or technology relating to health care.
Francesca Palumbo No disclosure on file
Bryan Traynor, MD The institution of Dr. Traynor has received research support from ALS Association. The institution of Dr. Traynor has received research support from Merck. The institution of Dr. Traynor has received research support from Myasthenia Gravis Foundation. The institution of Dr. Traynor has received research support from Cerevel Therapeutics. Dr. Traynor has received intellectual property interests from a discovery or technology relating to health care.
Letizia Mazzini No disclosure on file
Merit E. Cudkowicz, MD, MSC, FAAN Dr. Cudkowicz has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for biogen. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for cytokinetics. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 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 denali. The institution of Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for wave. 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 $10,000-$49,999 for serving as a Consultant for Neurosense. 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 $0-$499 for serving as a Consultant for Arrowhead. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Servier. Dr. Cudkowicz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eledon. 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 roche. Dr. Cudkowicz has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for novartis. Dr. Cudkowicz has received personal compensation in the range of $0-$499 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 $10,000-$49,999 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 . An immediate family member of Dr. Cudkowicz has stock in Sqz. The institution of Dr. Cudkowicz has received research support from NINDS. The institution of Dr. Cudkowicz has received research support from ALSA . 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. Dr. Cudkowicz has received publishing royalties from a publication relating to health care.
Ammar Al-Chalabi, PhD, FRCP DipStat The institution of Dr. Al-Chalabi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for OrionPharma. The institution of Dr. Al-Chalabi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics Inc. The institution of Dr. Al-Chalabi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe Pharma. The institution of Dr. Al-Chalabi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. The institution of Dr. Al-Chalabi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Quralis. Dr. Al-Chalabi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for My Name'5 Doddie Foundation. The institution of Dr. Al-Chalabi has received research support from Medical Research Council. The institution of Dr. Al-Chalabi has received research support from NIHR. The institution of Dr. Al-Chalabi has received research support from European Commission. The institution of Dr. Al-Chalabi has received research support from MND Association. The institution of Dr. Al-Chalabi has received research support from My Name'5 Doddie Foundation. Dr. Al-Chalabi has received publishing royalties from a publication relating to health care. Dr. Al-Chalabi has received publishing royalties from a publication relating to health care. Dr. Al-Chalabi has a non-compensated relationship as a SAB Member and Executive Member with TRICALS that is relevant to AAN interests or activities.
Fabiola De Marchi Fabiola De Marchi has nothing to disclose.
Antonio Canosa Antonio Canosa has nothing to disclose.
Yuanjing Liu Yuanjing Liu has received personal compensation for serving as an employee of Wave life sciences . Yuanjing Liu has received stock or an ownership interest from Wave life sciences.
Jinsy Andrews, MD, FAAN Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Wave Life Sciences. Dr. Andrews has received personal compensation in the range of $0-$499 for serving as a Consultant for Cytokinetics. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Apellis . Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Revalasio . Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AL-S Pharma. Dr. Andrews 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. Andrews has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Quralis . Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for Neurosense. Dr. Andrews has received personal compensation in the range of $0-$499 for serving as an officer or member of the Board of Directors for Akava. The institution of Dr. Andrews has received research support from Amylyx. The institution of Dr. Andrews has received research support from Biogen . The institution of Dr. Andrews has received research support from Cytokinetics . The institution of Dr. Andrews has received research support from NIH . The institution of Dr. Andrews has received research support from Biohaven/Platform Trial. The institution of Dr. Andrews has received research support from Clene/Platform Trial . The institution of Dr. Andrews has received research support from Ra Pharma/Platform Trial . The institution of Dr. Andrews has received research support from Prilenia/Platform Trial . Dr. Andrews has received research support from ALS Association . Dr. Andrews has a non-compensated relationship as a Board of Trustee Member with ALS Association that is relevant to AAN interests or activities.
Andrea Calvo, MD, PhD, FAAN Dr. Calvo has nothing to disclose.
Maurizio Grassano, MD Dr. Grassano has received research support from American Brain Foundation, ALS Association and American Academy of Neurology.
Cristina Moglia Cristina Moglia has nothing to disclose.
Leonard H. Van den Berg, MD The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx. The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ferrrer. The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for BMS. The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ArgenX. The institution of Dr. Van den Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Projenx. The institution of Dr. Van den Berg has received research support from Netherlands ALS Foundation.
Barbara Iazzolino No disclosure on file
Umberto Manera, MD Dr. Manera has nothing to disclose.
Rosario Vasta, MD Dr. Vasta has nothing to disclose.
Peter Munch Andersen, MD Prof. Andersen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Prof. Andersen has received personal compensation in the range of $0-$499 for serving as a Consultant for Orphazyme A/S. Prof. Andersen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Prof. Andersen has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Orphazyme A/S. The institution of Prof. Andersen has received research support from Swedish Research Council. The institution of Prof. Andersen has received research support from Swedish Brain Foundation. The institution of Prof. Andersen has received research support from KAW. The institution of Prof. Andersen has received research support from The Ulla-Carin Lindquist patient organization. The institution of Prof. Andersen has received research support from Umea University Research Foundation. The institution of Prof. Andersen has received research support from Swedish Neuro Patient Organization.
Chandra Vargeese No disclosure on file
Lucia Corrado No disclosure on file
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.
Namita Goyal, MD, FAAN Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genzyme. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB . Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astrazenica. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kezar. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abcuro. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL Behring. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx. The institution of Dr. Goyal has received research support from Brainstorm Cell Therapeutics. The institution of Dr. Goyal has received research support from Cytokinetics. The institution of Dr. Goyal has received research support from Fulcrum. The institution of Dr. Goyal has received research support from Kezar. The institution of Dr. Goyal has received research support from Alexion. The institution of Dr. Goyal has received research support from Annexon. The institution of Dr. Goyal has received research support from Annelixis. The institution of Dr. Goyal has received research support from Healey Platform. The institution of Dr. Goyal has received research support from Medicinova. The institution of Dr. Goyal has received research support from Amylyx. The institution of Dr. Goyal has received research support from MT Pharma. The institution of Dr. Goyal has received research support from Transposon. Dr. Goyal has received publishing royalties from a publication relating to health care.