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S23 - Genetic Muscle Disorders

Event Time:	Monday May 6, 2019 3:30 pm to 5:30 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
5:10 PM - 5:30 PM	Q&A Panel with Authors	Faculty

Disclosures
Speaker	Disclosure

Abstract Presenters
Start Time	Pub.	Title	Presenter
3:30 PM	002	Sensitive Assessment of Duchenne Muscular Dystrophy Progression with a Dedicated Electrical Impedance Myography Device	Disclosure: Dr. Rutkove has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Myolex, Biogen, Axcella Health, Merck. Dr. Rutkove has received personal compensation in an editorial capacity for Annals of Neurology. Dr. Rutkove has received compensation for serving on the Board of Directors of Myolex, Inc. Dr. Rutkove holds stock and/or stock options in Myolex, Inc.
3:30 PM	003	Genetic Prevalence of Myotonic Dystrophy Type 1	Disclosure: Dr. Johnson has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Acceleron, Strongbridge, Vertex, Fulcrum, and AveXis. Dr. Johnson has received personal compensation in an editorial capacity for Continuum. Dr. Johnson has received royalty, license fees, or contractual rights payments from University of Rochester. Dr. Johnson has received research support from Acceleron, CSL Behring, Fulcrum, Vertex, and AveXis.
3:30 PM	004	Breaking the sound barrier of randomized, controlled trials in congenital myopathies: results of the N-acetylcysteine trial in RYR1-related disorders	Disclosure: Dr. Todd has nothing to disclose.
3:30 PM	005	An Instrumented Timed Up and Go is Sensitive to Progression in Facioscapulohumeral Dystrophy	Disclosure: Dr. Statland has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Acceleron Pharma, Inc., Expansion Therapeutics, , Fulcrum Therapeutics, Genea Biocells, and Strongbridge Biopharma. Dr. Statland has received research support from FSH Society, Muscular Dystrophy Association, and the National Institutes of Health.
3:30 PM	006	FTX-1821 for Treatment of FSHD at its Root Cause: Results of a Clinical Trial in a Dish	Disclosure: Dr. Mellion has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Fulcrum Therapeutics, Vertex Pharmaceuticals. Dr. Mellion holds stock and/or stock options in Vertex Pharmaceuticals which sponsored research in which Dr. Mellion was involved as an investigator. Dr. Mellion holds stock and/or stock options in Vertex Pharmaceuticals. Dr. Mellion has received research support from Fulcrum Therapeutics, Vertex Pharmaceuticals.
3:30 PM	007	Comparison of Symptom Profile in Patients with SCN4A and CLCN1 Nondystrophic Myotonias	Disclosure: Dr. Meyer has nothing to disclose.
3:30 PM	008	Open-Label Phase 2 Clinical Trial of ManNAc for GNE Myopathy	Disclosure: Dr. Carillo-Carrasco has received research support from Leadiant Biosciences.
3:30 PM	009	Progressive Degenerative Myofibrillar Myopathy in Knock-In Mice Expressing LDB3 p.A165V Protein: A Mouse Model for Markesbery-Griggs Distal Myopathy	Disclosure: Dr. Blech-Hermoni has nothing to disclose.

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