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Plenary Sessions

PL 1 – Genetics – Wednesday, July 6

PL 2 – Hot Topics – Thursday, July 7

PL 3 – Muscular Dystrophy – Friday, July 8

PL 4 – Motor Neuron Disease – Saturday, July 9



Keynote Speaker: Daniel MacArthur
Massachusetts General Hospital Research Institute


PL 1.1 Genomic Approaches to Diagnosis of Rare Muscle Disease

  • Wednesday, July 6, 8:00 – 9:00

About Daniel MacArthur

Daniel MacArthur is an Assistant Professor at Harvard Medical School and Massachusetts General Hospital and the Associate Director of Medical and Population Genetics at the Broad Institute of Harvard and MIT. MacArthur’s research is focused on the use of genomic approaches to uncover the functional impact of human genetic variation, and especially its role in causing severe Mendelian diseases. He coordinated the functional analysis of protein-coding genetic variants for the 1000 Genomes Project pilot studies, published in 2010, and subsequently led a working group investigating the impact of protein-coding loss-of-function variants in the human genome (MacArthur et al. Science 2012). He currently coordinates the Exome Aggregation Consortium (ExAC), which has aggregated and jointly processed exome sequence data from over 92,000 individuals. He also leads the Genotyping Working Group of the Genotype-Tissue Expression (GTEx) Project, and two consortia focused on the genomic diagnosis of rare neuromuscular diseases. He serves on the Program Committee for the American Society of Human Genetics, and in 2012 co-chaired the NHGRI Working Group on Implicating Sequence Variants in Human Disease. MacArthur’s group also applies a variety of genomic technologies, including exome, whole-genome, and transcriptome sequencing, to improve the diagnosis of patients with rare disease. His clinical focus is on rare neuromuscular disorders such as muscular dystrophies and congenital myopathies. To date his lab’s analyses have led to genetic diagnoses for over 250 families suffering from these diseases. MacArthur received his BMedSc and PhD from the University of Sydney, Australia, and completed postdoctoral training at the Wellcome Trust Sanger Institute in Cambridge, UK before beginning his current position in 2012.


Stephan Züchner 
University of Miami Miller School of Medicine

PL 1.2 Gene Discovery in Charcot-Marie-Tooth Neuropathies

  • Wednesday, July 6, 9:00 – 9:30

About Stephan Züchner

Stephan Züchner is interested in studying the genomic underpinnings of disease. HIs lab takes several approaches to achieve this goal ranging from large-scale genomic studies that require bioinformatics expertise, proteomics and biochemistry as well as modeling of genes and genomic variation in cells and animals (yeast, zebrafish, mice). The latter especially require imaging studies and behavioral studies. Over the years, they have had a particular focus on mitochondrial diseases, axonopathies, and synaptic biology. They have contributed significantly with novel genes and pathways of human disease, from neurodegenerative disease to psychiatric phenotypes. Over the past 10 years they have discovered dozens of disease genes that are now used for routine diagnostics and development of targeted drug therapy. This includes genes for axonopathies, dementia, spastic paraplegia, muscular diseases, ataxias, and others.

James Dowling
Hospital for Sick Children (SickKids)

PL 1.3 RNA Sequence and RNA Analysis

  • Wednesday, July 6, 9:30 – 10:00

About James Dowling

Dr. Dowling is a clinician-scientist who is focused on gene discovery and therapy development for childhood muscle diseases. Dowling received his BSc and MSc from Yale University and his MD/PhD from the University of Chicago. His PhD work was performed in the laboratory of Elaine Fuchs. He did his residency in child neurology at Children’s Hospital of Philadelphia and completed postdoctoral research with Jeff Golden (UPenn) and Eva Feldman (University of Michigan). Until recently, he was an assistant professor at the University of Michigan. His clinical expertise is in childhood neuromuscular disorders and he is considered one of the leading authorities on the diagnosis and management of congenital myopathies. Dowling’s research has examined questions of disease pathogenesis and therapy development for congenital myopathies and childhood muscular dystrophies. His laboratory has helped pioneer the use of the zebrafish model for these disorders. He has authored or co-authored more than 60 peer reviewed manuscripts and been fortunate to enjoy external funding from several sources (including NIH).


Eva L. Feldman
University of Michigan

PL 2.1 Stem Cell Therapy in ALS

  • Thursday, July 7, 8:00 – 8:15

About Eva Feldman

Dr. Feldman received her MD/PhD from the University of Michigan (U-M), completed neurology residency at Johns Hopkins, and returned to U-M for a Neuromuscular fellowship prior to joining faculty at U-M. In addition to her clinical practice and position as the Russell N. DeJong Professor of Neurology at U-M, Dr. Feldman is Director of the A. Alfred Taubman Medical Research Institute and Research Director of the U-M ALS Clinic. She also runs her own 30-scientist laboratory, the Program for Neurology Research & Discovery, and is the Principal Investigator of the first-ever FDA-approved human clinical trials of an intraspinal stem cell therapy for ALS. Dr. Feldman has published over 400 manuscripts and book chapters and is currently PI or co-PI of 8 NIH grants and multiple foundation grants. She is Past President of the Peripheral Nerve Society and served as President of the American Neurological Association from 2011 to 2013. She has been listed in Best Doctors of America for over a decade. Among her greatest accomplishments is her training of scientists and neurologists; 9 scientists have received their PhD degrees under her supervision, over 50 postdoctoral fellows have trained in her laboratory to become neuroscientists, and over 50 neurologists have trained under her to specialize in the understanding and treatment of neuromuscular and neurodegenerative diseases, with an emphasis on ALS and neuropathy. Among her numerous awards, she received both the Early and Distinguished Career Development Awards from U-M, and she was admitted into the National Academy of Medicine in 2014.

Gil I. Wolfe

University at Buffalo/SUNY School of Medicine and Biomedical Sciences

PL 2.2 Results of the Thymectomy Trial in Myasthenia Gravis

  • Thursday, July 7, 8:15 – 8:30

James F. Howard, Jr. 
The University of North Carolina

PL 2.3 Regain: A Randomized, Double-Blind, Placebo-Controlled Multi-Center Phase 3 Study Of The Safety And Efficacy Of Eculizumab In Subjects With Refractory Generalized Myasthenia Gravis

  • Thursday, July 7, 8:30 – 8:45

About James F. Howard, Jr.

James F. Howard, Jr, MD, is Chief of the Neuromuscular Disorders Section in the Department of Neurology, Distinguished Professor of Neuromuscular Disease and Professor of Neurology, Medicine and Allied Health at the University of North Carolina School of Medicine in Chapel Hill. He earned a medical degree at the University of Vermont School of Medicine in Burlington, completed an internship in internal medicine at Albany Medical Center Hospital in New York, and completed residency and fellowship training in neurology, neuromuscular disease and EMG at the University of Virginia Hospital in Charlottesville. He is board-certified in Psychiatry and Neurology and in Electrodiagnostic Medicine. Dr Howard’s research has centered in the immunology and physiology of synaptic transmission in normal and its associated disease states, and in clinical neuromuscular disease, myasthenia gravis (MG), and Duchenne muscular dystrophy (DMD). He has lectured nationally and internationally on MG and DMD, and is the author of more than 65 abstracts, more than 150 articles and book chapters, and 3 books on these subjects.


Richard Barohn
University of Kansas Medical Center

PL 2.4 Approach to Patient-Centered Outcomes Research

  • Thursday, July 7, 8:45 – 9:00

About Richard Barohn

Dr. Richard J. Barohn is Chairman of the Department of Neurology and Vice Chancellor for Research at the University of Kansas Medical Center in Kansas City. He is also the Gertrude and Dewey Ziegler Professor of Neurology, and University Distinguished Professor, and the President of the Research Institute. Dr. Barohn is a graduate of the University of Missouri – Kansas City School of Medicine. He completed a neurology residency at Wilford Hall USAF Medical Center and then completed a neuromuscular fellowship at Ohio State University under the mentorship of Dr. Jerry Mendell. Dr. Barohn is board certified in adult neurology by the American Board of Psychiatry and Neurology with added qualifications in clinical neurophysiology and neuromuscular medicine. He is also certified by the American Board of Electrodiagnostic Medicine. Dr. Barohn holds membership in many professional societies, including the American Academy of Neurology (fellow status), the American Neurological Association, and the Association of University Professors of Neurology. Dr. Barohn has served on the national medical advisory boards for the Myasthenia Gravis Foundation and the Guillain-Barre Syndrome Foundation International and The Myositis Association. He was the recipient of the 2000 Alumni Achievement Award for Medicine from the University of Missouri – Kansas City and the University of Kansas Chancellor’s Club Research Award in 2012.He became a KU University Distinguished Professor in 2014. He was appointed Vice Chancellor for Research for KUMC and President of the Research Institute.In this role he is the chief administrator for all KUMC grants (Schools of Medicine, Nursing and Health Professions), and he controls over 120 million dollars annually of research funds. As chair of the Department of Neurology at KUMC, he also serves as the CEO of Kansas University Neurological Foundation and manages a financial portfolio that involves research and clinical revenue of more than $20 million annually. His present research focuses on myopathies (such as polymyositis and muscular dystrophy), motor neuron disease (such as amyotrophic lateral sclerosis), peripheral neuropathies, and myasthenia gravis. He has developed a number of endpoint scales in neuromuscular disease that have been used in many clinical trials. He was the lead principal investigator of the multi-center study of mexiletine in nondystrophic myotonia that was published in JAMA. He has led or leads several multicenter international trials (methotrexate for myasthenia gravis, rasagiline for ALS, memantine for ALS and arimoclomol for IBM). He leads a 40 site comparative effectiveness study of drugs for painful neuropathy funded by PCORI and he is the leader of the rare disease program in the Greater Plains Collaborative PCORnet. Dr. Barohn is the author of more than 260 journal publications and over 50 book chapters on various aspects of neuromuscular disease. He is the principal investigator on the NIH Clinical and Translational Science Award and the NeuroNEXT grant at KUMC. He is the Director of Frontiers: The Heartland Institute for Clinical and Translational Research. He has served on the editorial board for Neurology and is the Associate Editor for the Journal of Clinical Neuromuscular Disorders. He was the Founding Chair of the Section of Neuromuscular Disease in the American Academy of Neurology.

Carsten G. Bonnemann
National Institute of Neurological Disorders and Stroke/NIH Porter Neuroscience Research Center

PL 2.5 Do We Still Need Muscle Biopsy in the Era of Ultrasound?

  • Thursday, July 7, 9:00 – 9:15

Mazen Dimachkie
University of Kansas Medical Center

PL 2.6 Therapeutic Approaches to Inclusion Body Myositis

  • Thursday, July 7, 9:15 – 9:30

About Mazen Dimachkie

Mazen M. Dimachkie, MD, FAAN, FANA Professor of Neurology & Director of Neuromuscular Division Vice Chair for Research Program, Department of Neurology University of Kansas Medical Center, Kansas City, Kansas, USA Mazen M. Dimachkie, MD, FAAN, FANA, is Professor of Neurology, Director of the Neuromuscular Division at the University of Kansas Medical Center (KUMC) in Kansas City, where he is also Director of the Clinical Neurophysiology and Neuromuscular Medicine Fellowships and Vice Chair for Research in the Neurology Department. As the director of KUMC Neuromuscular Research, he manages one of the busiest neuromuscular clinical trials units in North America. He is also Associate Director of the KUMC site participating in the NINDS Network for Excellence in Neuroscience Clinical Trials known as NeuroNEXT. He received his medical degree from the American University of Beirut in Lebanon before completing an internship in Internal Medicine at Saint Agnes Hospital in Baltimore, Maryland. Dr. Dimachkie then completed a residency in Neurology at UT-Houston from July 1, 1990 to June 30, 1993 and fellowship in electromyography & Neuromuscular Disease from July 1, 1993 to June 30, 1994, both at The University of Texas Health Science Center at Houston (UT-Houston). He is ABPN board-certified in Neurology, Clinical Neurophysiology, and Neuromuscular Medicine and holds a UCNS certificate in Clinical Neuromuscular Pathology. He was on faculty at the UT-Houton Neurology Department from 1995-2007 before being recruited to lead the prolific neuromuscular Section and now Division at the KUMC. He participates in a wide variety of federally-funded neuromuscular research studies and industry-sponsored studies, nationally and internationally. Dr. Dimachkie is a member of the American Association of Neuromuscular & Electrodiagnostic Medicine, the Peripheral Nerve Society, The Muscle Study Group, and sits on the MSABs of the Myasthenia Gravis Foundation of America and on The Myositis Association. He was elected as fellow member of the American Academy of Neurology and of the American Neurological Association. He is a frequently invited lecturer in the fields of myositis, myasthenia gravis and other neuromuscular topics. He has authored or co-authored more than 200 abstracts, articles, and book chapters.


David Adams
CHU Bicêtre, APHP, Univ Paris Sud, FILNEMUS

PL 2.7 Treatment of Amyloid Neuropathy

  • Thursday, July 7, 9:30 – 9:45

Small fiber polyneuropathy : the model of amyloid polyneuropathy. Amyloid polyneuropathies are disabling and fatal diseases due to endoneurial amyloid deposits leading to a progressive peripheral and autonomic neuropathy associated with multisystemic involvement. Familial Amyloid Polyneuropathy (FAP) are usually due to mutation of transthyretin gene with an autosomal dominant transmission. The most common variant is Val30Met with endemic areas including Portugal and Japan. The age of onset is early before 50 years. Small fiber neuropathy is common and a hallmark of the disease. Inaugural manifestations are distal paresthesia, digestive complaints, erectile dysfunction. On examination, there is a predominant involvement of pain and thermal sensations in a length dependent distribution. Skin denervation is common in Val30Met TTR and other variants. Degeneration of cutaneous nerve terminals is correlated with the severity of clinical phenotypes and the level of CSF protein in Ala97Ser. Congo positive amyloid deposits are present in dermis in 67% patients including in 100% of EO Val30Met. Skin denervation seems to be an early event as indicated with the reduction of Intra Epidermal Nerve Fiber Density at distal leg in 59 % of carriers of TTR mutation. There is also pathological evidence of sudomotor denervation in FAP with functional correlation with autonomic symptoms, autonomic tests, ambulation status, and progression of disability. Iodine-123 metaiodobenzylguanidine (MIBG) imaging for heart allows to assess sympathetic cardiac denervation without correlation with conduction disturbances.

About David Adams

David Adams is Head of the French Reference Centre for Familial Amyloidotic Polyneuropathy (FAP), a position held since 2005, and has been the Coordinator of the French Network for FAP since 2010 and built the European Network for ATTR amyloidosis in 2015. He was made a Professor of Neurology at the Université Paris Sud, France in 1996. In 2009, he became Head of the Department of Neurology Centre Hospitalier Universitaire Bicêtre, at Assistance Publique-Hôpitaux de Paris. His main areas of expertise are peripheral neuropathies, including FAP and other rare peripheral neuropathies. He has been particularly involved in the evaluation of the effects of liver transplantation on neuropathy in FAP patients in France since 1993, and the neurological risks of domino liver transplantation. He is Principal Investigator in many multicentric clinical trials for FAP. He is now involved in research programmes of early diagnosis in sporadic cases and genetic carriers. Professor Adams was an Advisory Board member for the International Symposium on FAP, and on Liver Transplantation in FAP since 2007. Professor Adams also served on the local organising committee for Peripheral Nerve Society (PNS) in Saint-Malo (France) in July 2013. He organized the first European Congress for ATTR Amyloidosis the 2-3 november 2015 in Paris. He was elected in the board of PNS since 2015 and as member of EAN Scientific Panel Neuropathies since 2015. Professor Adams has published 100 articles in national and international journals, including Annals of Neurology, Archives of Neurology, Brain, Current Opinions in Neurology, JACC, JNNP, Nature Reviews Drug Discovery, Neurology, New England Journal of Medicine, and PNAS.


Keynote Speaker: Dongsheng Duan

University of Missouri

PL 3.1 Gene Therapy for Muscular Dystrophy

  • Friday, July 8, 8:00 – 3:30

About Dongsheng Duan

Dongsheng Duan is the Margaret Proctor Mulligan Professor in Medical Research, in the Department of Molecular Microbiology and Immunology, and Department of Neurology at the University of Missouri ( School of Medicine. Dr. Duan received his medical training in China from the West China University of Medical Sciences in 1987 and his Ph.D. from the University of Pennsylvania in 1997. His major research interest is to develop adeno-associated virus (AAV)-based gene therapy for Duchenne muscular dystrophy. He was the first to demonstrate that AAV is an episomal vector, a critical safety feature of the AAV vector. He has invented a number of dual vector technologies to double AAV packaging capacity for large genes (such as the dystrophin gene). Dr. Duan’s lab pioneered Duchenne cardiomyopathy gene therapy and bodywide AAV gene delivery in large mammals. His lab also identified the pivotal nNOS-binding site in dystrophin and developed new mini-/micro-dystrophin vectors with improved therapeutic efficacy. Current research efforts are focused on scaling-up AAV gene therapy in the symptomatic canine DMD model in the hope of bridging the translational gap between mice and human patients. Dr. Duan has received a number of awards including Spurgeon Distinguished Medical Research Award in 2004, Outstanding New Investigator Award from the American Society of Gene Therapy in 2006, and Chancellor’s Award for Outstanding Research and Creative Activity from the University of Missouri in 2009. He has published 120 peer-reviewed manuscripts and has also edited two books entitled “Muscle Gene Therapy” and “Muscle Gene Therapy: Methods and Protocols”.


Keynote Speaker: Dana Martin

Sarepta Therapeutics

PL 3.1 RNA Therapeutics for Duchenne Muscular Dystrophy

  • Friday, July 8, 8:30 – 9:00

About Dana Martin

Dana R. Martin, PharmD is Vice President of Medical Affairs and Patient Advocacy at Sarepta Therapeutics, a company based in Cambridge, Massachusetts and focused on developing RNA-targeted therapeutics to treat Duchenne muscular dystrophy and other neuromuscular disorders.  He is a clinical pharmacist by training and holds Bachelor of Science and Doctor of Pharmacy degrees from the Massachusetts College of Pharmacy and Health Sciences – Boston.   Prior to joining Sarepta, Dr. Martin has contributed to several successful rare disease clinical development programs at Genzyme Corporation (Fabry, Pompe) and Synageva BioPharma (LAL Deficiency).  He is adjunct faculty and preceptor of students from the University of North Carolina’s Eshelman College of Pharmacy and the University of New England’s College of Pharmacy (UNE-COP) and is an industry representative on the Dean’s Advisory Council of UNE-COP.


Charles Thornton
University of Rochester Medical Center, School of Medicine and Dentistry

PL 3.2 Antisense Therapy for Myotonic Dystrophy

  • Friday, July 8, 9:00 – 9:30

Myotonic dystrophy stands at the crossroads of human genetics, neuromuscular medicine, RNA biology, and antisense therapeutics. There is still much to learn about how an expanded CTG repeat in the DMPK gene causes a remarkably diverse set of clinic findings. However, the major thrust of research over the past 15 years indicates that RNA toxicity underlies most of the symptoms. The RNA acquires a toxic property because it too contains an expanded repeat, and one of its major effects is to bind extensively to RNA binding proteins, to such an extent that the proteins cannot perform their normal function. This presentation will focus on connections between repetitive RNA and symptoms of myotonic dystrophy, and current efforts to harness gene silencing technologies, such as, antisense oligonucleotides, to develop targeted treatments for this disease.

About Charles Thornton

Dr Thornton is the Saunders Family Distinguished Professor of Neuromuscular Research at the University of Rochester. He received his medical training at the University of Iowa and his specialty training in neuromuscular disease at the University of Rochester. He is co-director of the Wellstone Muscular Dystrophy Cooperative Research Center in Rochester. Recently his work has focused on disease mechanisms and targeted therapies for myotonic dystrophy.


Ronald D. Cohn
The Hospital for Sick Children

PL 3.3 CRISPR Based Gene Editing for Muscular Dystrophy

  • Friday, July 8, 9:30 – 10:00

The session will discuss how the CRISPR/Cas9 technology can be employed for treatment strategies for genetic disorder with a special emphasis on muscular dystrophies. Emphasis will also be given to the regulatory and ethical issues associated with this technologies.

About Ronald Cohn

Ronald Cohn joined The Hospital for Sick Children as the Chief of the Division of Clinical and Metabolic Genetics, Co-Director of the Centre for Genetic Medicine and Senior Scientist in September 2012. He also became the Inaugural Women’s Auxiliary Chair in Clinical and Metabolic Genetics in April of 2013, as well as joining the department of Molecular Genetics at the University of Toronto. Research in Cohn’s laboratory focuses on the biology of muscle regeneration as it relates to various inherited and acquired myopathic states with a particular interest in muscular dystrophies. More recently, he used a novel approach to understand the mechanisms of maintaining muscle mass and regeneration, by studying hibernating ground squirrels. Hibernating mammals have evolved mechanisms to survive prolonged immobility without pathologic loss/atrophy of muscle mass. The molecular mechanisms underlying this fascinating phenomenon are largely unknown. His laboratory is currently for the first time applying knowledge of normal mechanisms of muscle protection in the hibernating mammal to the disease process of disuse muscle atrophy in non-hibernating mammals. This will provide unique insights into the fundamental cellular and molecular pathways underlying skeletal muscle atrophy and the protection against it. Cohn plans to continue his research activities and ensure that novel genetic technologies will be translated into clinical medicine with the goal of one day making individualized treatment a standard of care for all children.


Keynote Speaker: Michael Benatar
University of Miami Leonard M. Miller School of Medicine

PL 4.1 ALS Therapy Development: Challenges and Opportunities

  • Saturday, July 9, 8:00 – 9:00

About Michael Benatar

Dr. Michael Benatar is Professor of Neurology and of Public Health Sciences, and Chief of Neuromuscular Division at the University of Miami where he also holds the Walter Bradley Chair in ALS Research. He obtained his medical degree at the University of Cape Town in South Africa and is also trained in both basic neuroscience (DPhil, Oxford) and clinical research methods (Masters in the Science of Clinical Research Emory). As the Executive Director of the Kessenich Family ALS Center at the University of Miami, he oversees the activities of the multi-disciplinary ALS clinic. He also directs an active clinical and translational research program focused on ALS biomarker and therapy development funded by the NIH, FDA, DOD, MDA and the ALS Association. He is the principal investigator on an investigator-initiated randomized controlled trial of arimoclomol in people with SOD1+ familial ALS; the Pre-symptomatic familial ALS (Pre-fALS), a systematic and longitudinal biomarker study of people at genetic risk for developing ALS; as well as the NCATS/NINDS funded CReATe Consortium (part of the NIH’s Rare Diseases Clinical Research Network), that is focused on biomarker development and the relationship between genotype and phenotype relationships in ALS and related disorders.


Leonard Petrucelli 
Mayo Clinic

PL 4.2 Biology of C9orf72 Disease

  • Saturday, July 9, 9:00 – 9:30

About Leonard Petrucelli

Leonard Petrucelli, Ph.D., is Chair of the Department of Neuroscience at Mayo Clinic Florida and is recognized as the Ralph B. and Ruth K. Abrams Professor with full faculty privileges at Mayo Graduate School. In expanding upon his commitment to understanding the causes of neurodegeneration in Alzheimer’s disease, frontotemporal dementia and amyotrophic lateral sclerosis, he is now emphasizing translational research geared toward identifying and developing therapies. His research, funded by several NIH grants, has been published in peer-reviewed journals, including Nature Medicine, PNAS, Journal of Clinical Investigation and Neuron. Dr. Petrucelli is also an invited member of the NIH Cellular and Molecular Biology of Neurodegeneration Study Section.


John Kissel
The Ohio State University Wexner Medical Center

PL 4.3 Antisense Therapy for Spinal Muscular Atrophy

  • Saturday, July 9, 9:30 – 10:00

About John Kissel

Dr. John Kissel is currently Professor of Neurology, Pediatrics and Neuroscience and The Gilbert and Kathryn Mitchell Chair in the Department of Neurology at The Ohio State University Wexner Medical Center. He serves as co-director of the both the Muscular Dystrophy Association Clinic at OSU and the Spinal Muscular Atrophy Clinic at Nationwide Children’s Hospital. He did his neurology residency at Washington University in St. Louis, followed by a one-year clinical neuromuscular fellowship and two years of laboratory research fellowship at Ohio State. He has been on the staff at Wexner Medical Center and Nationwide Children’s since 1985 and was named Neurology Department Chair in 2014. Dr. Kissel has published extensively in a wide range of peripheral nerve, muscle, and anterior horn cell disorders. His current interests include SMA and ALS, and he has also participated in clinical trials involving the inflammatory neuropathies, anterior horn cell diseases, and myasthenia gravis. He is the co-author of a popular textbook of peripheral nerve disease, “Diagnosis and Management of Peripheral Nerve Disorders”, and has written chapters for many neuromuscular textbooks. He is a Past-President of the Neuromuscular Section and former member of the Science Committee of the American Academy of Neurology, a Fellow in the American Academy of Neurology, and a member of the American Neurological Association. He serves on the Venture Capital Committee and Clinic Committee of the Muscular Dystrophy Association and the Advisory Board for Cure SMA, as well as the Executive Committee for the NIH-funded NeuroNEXT clinical trial network.

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