2021 Annual Meeting | S12 - Interventional/Observational Studies of Neuromuscular Diseases

Date Sunday 04/18/21

Time 04:00 PM - 05:00 PM EDT 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) Jeff Guptill, MD, FAAN, Michelle L. Mauermann, MD, FAAN

Topic(s) Neuromuscular and Clinical Neurophysiology (EMG)

CME Available 1.00 CME credit

Program Tracks Advanced Practice Provider

Program Materials Program Evaluations

Event Timeline
04:00 PM - 04:08 PM EDT	Abstract	001 - Long-term remission with rituximab in refractory generalized myasthenia gravis Juan I. Castiglione, MD
04:08 PM - 04:16 PM EDT	Abstract	002 - The Canadian Neuromuscular Disease Registry: A national spinal muscular atrophy (SMA) registry for real world evidence Maryam Oskoui, MD, FAAN
04:16 PM - 04:24 PM EDT	Q&A	Questions and Answers
04:24 PM - 04:32 PM EDT	Abstract	003 - Non-dystrophic Myotonia: 2 year outcomes of objective and patient reported outcomes Timothy R. Fullam, MD
04:32 PM - 04:40 PM EDT	Abstract	004 - SORD Neuropathy: Gene Identification and Proposal for a Follow Up Multi-Centre Natural History Study
04:40 PM - 04:48 PM EDT	Abstract	005 - Description of clinical symptoms of a large international cohort of patients with valosin-containing-protein related disease Marta Caballero-Ávila, MD
04:48 PM - 05:00 PM EDT	Q&A	Questions and Answers

Faculty Disclosures
	No disclosure on file
Lawrence Korngut, MD	Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe. Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche.
Victoria Hodgkinson	No disclosure on file
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.
Juan I. Castiglione, MD	Dr. Castiglione 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.
Chiseko Ikenaga, MD, PhD	Dr. Ikenaga has nothing to disclose.
Sherifa A. Hamed, MD, MBBS	No disclosure on file
Marta Caballero-Ávila, MD	Marta Caballero-Ávila 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.
Patricio Brand, MD	Dr. Brand has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen.
Michela Guglieri	The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne. The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NS Pharma. Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. The institution of Michela Guglieri has received research support from EC - H2020. The institution of Michela Guglieri has received research support from Duchenne UK. The institution of Michela Guglieri has received research support from Sarepta. The institution of Michela Guglieri has received research support from DMD Research Fund.
Timothy R. Fullam, MD	Dr. Fullam has received publishing royalties from a publication relating to health care.
Mamatha Pasnoor, MD, FAAN	Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx BVBA. Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda Pharmaceuticals. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen Pharmaceutical. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Pasnoor 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. Pasnoor has received research support from NIH.
Conrad Weihl, MD, PhD	Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Casma therapeutics. Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abata. Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Weihl has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Acceleron. Dr. Weihl has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Wilson Elser Moskowitz Edelman & Dicker LLP.
Duaa Jabari, MD	Dr. Jabari has nothing to disclose.
Constantine Farmakidis, MD, FAAN	Dr. Farmakidis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Farmakidis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Farmakidis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for J&J.
Davide Pareyson, MD	No disclosure on file
Fabio Adrian Barroso, MD	Fabio Adrian Barroso, MD has nothing to disclose.
David N. Herrmann, MD, FAAN	Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Acceleron. Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint global. Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GLG. Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurogene. Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Herrmann has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regenacy. Dr. Herrmann has received intellectual property interests from a discovery or technology relating to health care.
Swathy Chandrashekhar, MD	Dr. Chandrashekhar has received publishing royalties from a publication relating to health care.
Maryam Oskoui, MD, FAAN	Dr. Oskoui has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for the Association des Neurologues du Quebec. The institution of Dr. Oskoui has received research support from Roche Genetech. The institution of Dr. Oskoui has received research support from Muscular Dystrophy Canada. The institution of Dr. Oskoui has received research support from Canadian Institutes of Health Research. The institution of Dr. Oskoui has received research support from Santhera. Dr. Oskoui has a non-compensated relationship as a Member of the Medical and Scientific Advisory Committee with Muscular Dystrophy Canada that is relevant to AAN interests or activities.
Chiara Marini-Bettolo	Chiara Marini-Bettolo has nothing to disclose.
Henry Houlden	No disclosure on file
Franco Taroni, MD	The institution of Dr. Taroni has received research support from Italian Ministry of Health. The institution of Dr. Taroni has received research support from Fondazione Regionale per la Ricerca Biomedica.
Adriana Rebelo	No disclosure on file
Pascal Lafort	No disclosure on file
Tanya Stojkovic	Tanya Stojkovic has nothing to disclose.
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.
Mary Reilly, MD, FRCP, FRCPI	The institution of Mary Reilly, MD, FRCP, FRCPI has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alynlam. Mary Reilly, MD, FRCP, FRCPI has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AKCEA. Mary Reilly, MD, FRCP, FRCPI has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Inflectis. The institution of Mary Reilly, MD, FRCP, FRCPI has received research support from MRC. The institution of Mary Reilly, MD, FRCP, FRCPI has received research support from NIH. The institution of Mary Reilly, MD, FRCP, FRCPI has received research support from MDA.
Omar Jawdat, MD	Dr. Jawdat has nothing to disclose.
Shawna Feely	Shawna Feely has nothing to disclose.
Jordi Diaz-Manera	No disclosure on file
Craig G. Campbell, MD	Dr. Campbell has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Campbell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid. The institution of Dr. Campbell has received research support from Biogen. The institution of Dr. Campbell has received research support from Genzyme. The institution of Dr. Campbell has received research support from Acceleron, AMO, Biogen, Sarepta, Wave, PTC, Pfizer, Roche.
Maike Dohrn, MD	Dr. Dohrn has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea, Alnylam, Pfizer, Amicus. Dr. Dohrn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus, Akcea. Dr. Dohrn has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Akcea, Alnylam, Pfizer. The institution of Dr. Dohrn has received research support from Pfizer. Dr. Dohrn has received research support from German Research Foundation.
Hugh J. McMillan, MD	Dr. McMillan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis Gene Therapies. Dr. McMillan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis Gene Therapies. The institution of Dr. McMillan has received research support from Roche.
Bernard Brais, MD	Dr. Brais has nothing to disclose.
Jiri Vajsar, MD	Dr. Vajsar 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. Vajsar has received research support from CSL Behring.
Alejandro Kohler	No disclosure on file
Jeff Guptill, MD, FAAN	Dr. Guptill has received personal compensation for serving as an employee of argenx. Dr. Guptill has stock in argenx.
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.