2021 Annual Meeting | S12 - Interventional/Observational Studies of Neuromuscular Diseases
Program Materials Program Evaluations
| 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
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| 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, FAAN |
| 04:32 PM - 04:40 PM EDT | Abstract |
004 - SORD Neuropathy: Gene Identification and Proposal for a Follow Up Multi-Centre Natural History Study
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| 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
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| 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. |
| 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. |
| 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. |
| 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. |
| Davide Pareyson, MD | No disclosure on file |
| 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. |
| 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. |
| 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. |
| Tanya Stojkovic | Tanya Stojkovic has nothing to disclose. |
| 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 Hoffmann-La Roche Ltd. 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. The institution of Dr. Oskoui has received research support from Novartis. 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. |
| 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 $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 $5,000-$9,999 for serving as a Consultant for Annexon. Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for amgen. |
| 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. |
| 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. |
| 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. |
| No disclosure on file | |
| 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. |
| 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. |
| 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. Dr. Farmakidis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Muscular Dystrophy Association. |
| 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. |
| Jeff Guptill, MD, FAAN | Dr. Guptill has received personal compensation for serving as an employee of argenx. Dr. Guptill has stock in argenx. |
| 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. |
| Patricio Brand, MD | Dr. Brand has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. |
| Omar Jawdat, MD | Dr. Jawdat has nothing to disclose. |
| Duaa Jabari, MD | Dr. Jabari has nothing to disclose. |
| Chiara Marini-Bettolo | Chiara Marini-Bettolo has nothing to disclose. |
| Fabio Adrian Barroso, MD | Fabio Adrian Barroso, MD has nothing to disclose. |
| Chiseko Ikenaga, MD, PhD | Dr. Ikenaga has nothing to disclose. |
| Shawna Feely | Shawna Feely has nothing to disclose. |
| Timothy R. Fullam, MD, FAAN | Dr. Fullam has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amylyx. Dr. Fullam has received publishing royalties from a publication relating to health care. |
| 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. |
| Alejandro Kohler | No disclosure on file |
| Swathy Chandrashekhar, MD | Dr. Chandrashekhar has received publishing royalties from a publication relating to health care. |
| Juan I. Castiglione, MD | Dr. Castiglione has nothing to disclose. |
| Marta Caballero-Ávila, MD | Marta Caballero-Ávila has nothing to disclose. |
| Sherifa A. Hamed, MD, MBBS | No disclosure on file |
| Henry Houlden | No disclosure on file |
| Adriana Rebelo | No disclosure on file |
| Victoria Hodgkinson | No disclosure on file |
| Pascal Lafort | No disclosure on file |
| Jordi Diaz-Manera | No disclosure on file |