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S58 - Therapeutics in Neuromuscular Disorders

Event Time:	Friday May 10, 2019 1:00 pm to 3:00 pm
Topic(s):	Neuromuscular and Clinical Neurophysiology (EMG)
Director(s):
Description:
Completion Message:
CME Credits:	2
Core Competencies:

Program Speakers - Tentative
Start/End Time	Title	Faculty
2:40 PM - 3:00 PM	Q&A Panel with Authors	Faculty

Disclosures
Speaker	Disclosure

Abstract Presenters
Start Time	Pub.	Title	Presenter
1:00 PM	001	Intravenous Immunoglobulin for Treatment of Demyelinating Polyneuropathy in Patients with Diabetes Mellitus: A Randomized, Double-Blinded, Placebo-Controlled Crossover Trial	Disclosure: Dr. Breiner has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Pfizer, CSL, Allergan, Akcea. Dr. Breiner has received research support from Grifols, Inc..
1:00 PM	002	Prevalence and Associations of Peripheral Neuropathy at Disease Onset in ANCA-Associated Vasculitides: Insights from the DCVAS Study	Disclosure: Dr. Bischof has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen Idec, Actelion.
1:00 PM	003	Immune-mediated rippling muscle disease, associated neoplasia and response to therapy.	Disclosure: Dr. Triplett has nothing to disclose.
1:00 PM	004	Gene therapy after onset of neuropathy provides therapeutic benefit in a model of CMT1X	Disclosure: Dr. Kleopa has nothing to disclose.
1:00 PM	005	RNAi/gene therapy combined approach as therapeutic strategy for Charcot-Marie-Tooth 2A	Disclosure: Dr. Rizzo has nothing to disclose.
1:00 PM	006	Results of the Dose-Escalation Portion of a Phase 2 Study of ACE-083, a Local Muscle Therapeutic, in Patients with Charcot-Marie-Tooth (CMT) Disease	Disclosure: Dr. Thomas has received personal compensation for consulting serving on a scientific advisory board speaking or other activities with Acceleron Genentech Novartis Pharnext Sanofi. Dr. Thomas has received personal compensation in an editorial capacity for Journal of Spinal Cord Medicine.
1:00 PM	007	Nusinersen Efficacy in Adults with Spinal Muscular Atrophy	Disclosure: Dr. Day has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AMO, Audentes, AveXis, Biogen, Cytokinetics, Santhera, and Sarepta. Dr. Day has received research support from AveXis, Biogen, Cytokinetics, Genzyme, Ionis, Roche, Santhera, and Sarepta.
1:00 PM	008	The Transplantation of a Specific iPSC-Derived Neural Stem Cells Subpopulation Improves the Pathological Hallmarks of Spinal Muscular Atrophy with Respiratory Distress Type 1 in Mice.	Disclosure: Dr. Forotti has nothing to disclose.
1:00 PM	009	Kinase Inhibitors Improve Neurofilament Distribution in CMT2E Human Motor Neuron Axons	Disclosure: Dr. Saporta has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Alnylam, Strongbridge, Biogen, Acceleron, Sarepta, Stealth, and Neurogene.

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