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2018 Annual Meeting | S31 - Therapeutics, Phenotypes, and Biomarkers in Neuromuscular Disorders

Wednesday 04/25/18
01:00 PM - 03:00 PM Add To Calendar
This program will be presented both in-person and online
This program is not expected to be available in the meeting's On Demand product.
Scientific Platform Session
Neuromuscular and Clinical Neurophysiology (EMG)
2.00 CME credits
Event Timeline
01:00 PM - 03:00 PM Abstract 001 - The natural history of impairments and disabilities in Oculopharyngeal Muscular Dystrophy: a large retrospective study
Jean-Denis Brisson, MD
01:00 PM - 03:00 PM Abstract 002 - Clinical Phenotypes of Pure Primary Lateral Sclerosis
Shivam O. Mittal, MD
01:00 PM - 03:00 PM Abstract 003 - Evaluation of Quality of Life and Disability in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Following Treatment with Patisiran, An Investigational RNAi Therapeutic: Results from the Phase 3 APOLLO Study
01:00 PM - 03:00 PM Abstract 004 - More than just fun and games: ACTIVE Workspace volume video game quantifies upper extremity function in individuals with neuromuscular disease
Lindsay N. Alfano, PT
01:00 PM - 03:00 PM Abstract 005 - Sporadic Late-Onset Nemaline Myopathy
Elie Naddaf, MD
01:00 PM - 03:00 PM Abstract 006 - RA101495, A Subcutaneously Administered Peptide Inhibitor of Complement Component 5 (C5) for the Treatment of Generalized Myasthenia Gravis (gMG): Phase 1 Results and Phase 2 Design
James F. Howard, Jr., MD, FAAN
01:00 PM - 03:00 PM Abstract 007 - Autologous Hematopoietic Stem Cell Transplantation May Be Highly Effective Treatment for Severe, Treatment Refractory Stiff Person Syndrome
01:00 PM - 03:00 PM Abstract 008 - Small Fiber Polyneuropathy in Patients with Neurofibromatosis, Type 1
Vera Bril, MD
02:35 PM - 03:00 PM Discussion Discussion
Faculty Disclosures
Lauren Phillips No disclosure on file
Srikanth Muppidi, MD, FAAN Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizont Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care.
Lauren Phillips, MD Dr. Phillips has nothing to disclose.
No disclosure on file
Michael D. Weiss, MD, FAAN Dr. Weiss has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB-RA. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Soleo. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi.
Gloria Von Geldern, MD, FAAN The institution of Dr. Von Geldern has received research support from Novartis. The institution of Dr. Von Geldern has received research support from National MS Society. Dr. Von Geldern has received personal compensation in the range of $0-$499 for serving as a DSMB member with NIH, NINDS. Dr. Von Geldern has a non-compensated relationship as a editorial board member with MS and Related Disorders Journal that is relevant to AAN interests or activities.
Michael L. Pearlman, MD, PhD No disclosure on file
Keith A. Josephs, Jr., MD, FAAN Dr. Josephs has nothing to disclose.
Alon Abraham, MD No disclosure on file
Peter J. Dyck, MD, FAAN Dr. Dyck has nothing to disclose.
Ramin Farzaneh-Far No disclosure on file
Marianne T. Sweetser, MD, PhD Dr. Sweetser has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Dr. Sweetser has received stock or an ownership interest from Alnylam Pharmaceuticals.
Lindsay N. Alfano, PT Ms. Alfano has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ATOM International, Ltd (Amicus Therapeutics, Catabasis, Genethon, Italfarmaco, NS Pharma, Pfizer, PTC Therapeutics). Ms. Alfano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Novartis Gene Therapies. The institution of Ms. Alfano has received research support from Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Audentes Therapeutics/Astellas Gene Therapies. Ms. Alfano has received intellectual property interests from a discovery or technology relating to health care.
Annette Wundes, MD, FAAN The institution of Dr. Wundes has received research support from Benaroya Research Institute .
George H. Kraft, MD No disclosure on file
Megan Iammarino, PT Dr. Iammarino has nothing to disclose.
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.
Linda P. Lowes, PT PhD The institution of Ms. Lowes has received research support from Sarepta Therapeutics.
Jared Gollob, MD No disclosure on file
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 .
J. E. Ahlskog, MD, PhD Dr. Ahlskog has received publishing royalties from a publication relating to health care.
Samiah A. Al-Zaidy, MD Dr. Al-Zaidy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AveXis Inc.
Henry J. Kaminski, MD, FAAN Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Kaminski has received personal compensation in the range of $0-$499 for serving as a Consultant for Cabaletta Bio. The institution of Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Dr. Kaminski has stock in mimivax. The institution of Dr. Kaminski has received research support from National Institutes of Health. Dr. Kaminski has received publishing royalties from a publication relating to health care.
Gil I. Wolfe, MD, FAAN Dr. Wolfe has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Wolfe has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Wolfe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ArgenX. Dr. Wolfe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Wolfe has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB/Ra. Dr. Wolfe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Wolfe has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Wolfe has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Grifols. The institution of Dr. Wolfe has received research support from UCB/Ra. The institution of Dr. Wolfe has received research support from ArgenX. Dr. Wolfe has received personal compensation in the range of $0-$499 for serving as a Advisor with FDA Advisory Panel for Cellular, Tissue and Genetic Therapies.
Teresa Coelho, MD Dr. Coelho has nothing to disclose.
Shivam O. Mittal, MD No disclosure on file
Anhar Hassan, MBBCH, FAAN The institution of Dr. Hassan has received research support from Intrabio . Dr. Hassan has received personal compensation in the range of $500-$4,999 for serving as a Invited speaker with Korean Movement Disorders Society.
Richard J. Nowak, MD Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Viela Bio. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cour Pharma. The institution of Dr. Nowak has received research support from Ra Pharma. The institution of Dr. Nowak has received research support from Alexion . The institution of Dr. Nowak has received research support from Momenta . The institution of Dr. Nowak has received research support from Immunovant . Dr. Nowak has received research support from argenx. Dr. Nowak has received research support from Viela Bio . Dr. Nowak has a non-compensated relationship as a Member of the Board of Directors with Myasthenia Gravis Foundation of America (MGFA) that is relevant to AAN interests or activities.
Bernard Brais, MD Dr. Brais has nothing to disclose.
Taro Yamashita, MD, PhD Dr. Yamashita has nothing to disclose.
James F. Howard, Jr., MD, FAAN Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for arenx . Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ra Pharma (now UCB Biosciences). Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon Therapeutics. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi US. Dr. Howard 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. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Academic CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for PeerView CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Platform Q CME. Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Zai Laboratories. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Medscape CME. Dr. Howard has stock in General Electric dividends. Dr. Howard has stock in Johnson & Johnson dividends. Dr. Howard has stock in Pfizer dividends. An immediate family member of Dr. Howard has stock in GlaxoSmithKline dividends. An immediate family member of Dr. Howard has stock in GE Healthcare dividends. The institution of Dr. Howard has received research support from Alexion Pharmaceuticals. The institution of Dr. Howard has received research support from argenx . The institution of Dr. Howard has received research support from UCB Biosciences. The institution of Dr. Howard has received research support from NIH. The institution of Dr. Howard has received research support from Centers for Disease Control/Research Triangle Institute. The institution of Dr. Howard has received research support from Duke University (DCRI). The institution of Dr. Howard has received research support from Cartestian Therapeutics. Dr. Howard has a non-compensated relationship as a Scientific Advisiory Board member, Committee member with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities.
Ole Suhr The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam . The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Prothena. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Intellia. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akces. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alnylam. Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Akcea. The institution of Ole Suhr has received research support from Swedish Heart and Lung Foundation.
Eric J. Sorenson, MD, FAAN The institution of Dr. Sorenson has received research support from Biogen.
William T. Hu, MD, PhD, FAAN No disclosure on file
James D. Bowen, MD Dr. Bowen has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen IDEC. Dr. Bowen has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for BristolMyers Squibb. Dr. Bowen has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Genentech. Dr. Bowen has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Novartis. The institution of Dr. Bowen has received research support from Biogen IDEC. The institution of Dr. Bowen has received research support from Genentech. The institution of Dr. Bowen has received research support from Genzyme. The institution of Dr. Bowen has received research support from Novartis. The institution of Dr. Bowen has received research support from Roche.
John T. Kissel, MD, FAAN Dr. Kissel has nothing to disclose.
Margherita Milone, MD, FAAN Dr. Milone 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. Milone has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Neurology Genetics, AAN. The institution of Dr. Milone has received research support from Mayo Clinic, CCaTS-CBD. The institution of Dr. Milone has received research support from Mayo Clinic, SGP Award. The institution of Dr. Milone has received research support from MDA for Care Center grant. The institution of Dr. Milone has received research support from Regenerative medicine Minnesota.
Kevin M. Flanigan, MD, FAAN Dr. Flanigan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Apic Bio. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AavantiBio. Dr. Flanigan has stock in 4D Molecular Therapeutics. The institution of Dr. Flanigan has received research support from Abeona Therapeutics. The institution of Dr. Flanigan has received research support from Sarepta Therapeutics. The institution of Dr. Flanigan has received research support from Astellas Therapeutics. Dr. Flanigan has received intellectual property interests from a discovery or technology relating to health care.
Chih-Chao Yang, MD No disclosure on file
John L. Berk John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca/IONIS. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eidos/BridgBio. John L. Berk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Intellia Therapeutics. John L. Berk has received research support from Alnylam . John L. Berk has received research support from Ionis. John L. Berk has received research support from Eidos/Bridgbio.
Carolina Barnett Tapia, MD Dr. Barnett Tapia has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Barnett Tapia has received personal compensation in the range of $0-$499 for serving as a Consultant for Sanofi. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. The institution of Dr. Barnett Tapia has received research support from Department of defense. The institution of Dr. Barnett Tapia has received research support from Muscular dystrophy canada. The institution of Dr. Barnett Tapia has received research support from MgNet. The institution of Dr. Barnett Tapia has received research support from NIH. Dr. Barnett Tapia has received intellectual property interests from a discovery or technology relating to health care.
Petra W. Duda, MD, PhD Dr. Duda has received personal compensation for serving as an employee of UCB. Dr. Duda has stock in UCB.
Jerry R. Mendell, MD, FAAN Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vertex. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics . The institution of Dr. Mendell has received research support from Sarepta.
Jean-Denis Brisson, MD No disclosure on file
Jean Mathieu, MD No disclosure on file
Vera Bril, MD Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octapharma. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Powell Mansfield. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta (now J&J). Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NovoNordisk. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Bril 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. Bril has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Bril 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. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL. Dr. Bril 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. Bril has received research support from UCB. The institution of Dr. Bril has received research support from Argenx. The institution of Dr. Bril has received research support from Momenta (now J&J). The institution of Dr. Bril has received research support from Immunovant. The institution of Dr. Bril has received research support from Alexion. The institution of Dr. Bril has received research support from Takeda. The institution of Dr. Bril has received research support from Grifols. The institution of Dr. Bril has received research support from CSL. The institution of Dr. Bril has received research support from Octapharma. The institution of Dr. Bril has received research support from Akcea. The institution of Dr. Bril has received research support from Ionis. The institution of Dr. Bril has received research support from Viela Bio (now Horizon). Dr. Bril has received intellectual property interests from a discovery or technology relating to health care.
Catherine Maurice, MD No disclosure on file