Keynote Speakers
Steven Greenberg, MD
Brigham and Women’s Hospital
Experiences in Academic Translational Medicine
Francesco Muntoni, MD
University College London
Clinical Trials in DMD 2016: Where are we?
Session I: DMD as a Model of Neuromuscular Disease Across the Lifespan
Anne Connolly, MD
Washington University School of Medicine
Outcomes Measures in infants and young boys with DMD
Valeria Sansone, MD, PhD
University of Milan, NEMO Center
Trial Design in DMD: Lessons Learned from Natural History
Robert B. Weiss, PhD
University of Utah
Genetic Modifiers in DMD
Session II: Extracellular Matrix Dysfunction as a Disease Mechanism
Dean Burkin, MD
University of Nevada School of Medicine
The Myomatrix Reloaded: Integrin and ECM therapies for the muscular dystrophies
Katherine Mathews, MD
University of Iowa Children’s Hospital
Dystroglycanopathy Cohort
Carsten Bonnemann, MD
National Institute of Neurological Disorders and Stroke/NIH
Collagen VI-related dystrophies: A translational update
Session III: Non-coding Repeates as a Disease Mechanism
Thomas A. Cooper, MD
Baylor College of Medicine
Myotonic dystrophy as a paradigm for RNA gain function disease mechanisms
Charles A. Thornton, MD
University of Rochester
Update on myotonic dystrophy trial readiness
Nicholas Johnson, MD
University of Utah
Disease Progression and pathogenesis in congenital myotonic dystrophy
Platform Poster Presentations
David Arnold, MD
Open Label Trial of Ranolazine in Myotonia Congenita
Chad Heatwole, MD
PRISM-SMA: Patient Reported Impact of Symptoms in Adult Spinal Muscular Atrophy
Summer Karafiath, MD
The Clinical Phenotype and Neuropathic Outcomes of Paclitaxel-Acute Pain Syndrome
Pedro Machado, MD
Baseline characteristics of a prospective natural history study of sporadic inclusion body myositis including MRI assessment
Mary Pautler, MPH
Limits of Postural Stability in Myotonic Dystrophy Compared to Normative Values
Mario Saporta, MD
Axonal RNA Profiling of Human Motor Neurons from Patients with Charcot-Marie-Tooth Disease as a Novel Approach to Study Axon Degeneration
Gita Ramdharry, PhD
Evaluating the benefits of community based aerobic training on the physical health and well-being of people with Neuromuscular Disease
Don McCorquodale, MD, PhD
RNA-Seq gene expression profiling identifies up-regulation of novel muscle-specific gene musclin in adult and congenital myotonic dystrophy
Session IV: Mechanisms of Chaperone Dysfunction
Chris Weihl, MD
Washington University
Chaperone Function and Dysfunction in Neuromuscular Disease
Rudolf Kley, MD
Heimer Institute for Muscle Research
Proteomic Identification of Aggregates and Chaperones in Disease Muscle
Zohar Argov, MD
Hadassah Hospital and BioBlast Pharma
Phase 2 Trial Results of Autographic Manipulation with Trehalose in Oculopharyngeal Muscular Dystrophy
Session V: Autoimmunity
Mark Anderson, MD
UCSF- Diabetes Center
Immune Regulation, Tolerance, and Autoimmunity
Gil Wolfe, MD
University of Buffalo
MGTX: Results from the Thymectomy trial in non-thymomatous myasthenia gravis
Renato Mantegazza, MD
Instituto Neurologico C. Besta
Outcome Measures in Autoimmune NMJ diseases
Jan L. Hillson, DM Momenta Pharmaceuticals
Targeting FcRN in Neuromuscular Disease
Jon Lindstrom, PhD
University of Pennsylvania
AChR-Specific Immunosuppressive Therapy for MG
Hot Topics
Osvaldo Nascimento, MD
Universidade Federal Fluminense, Niteroi
Zika Virus Update saliva
James Howard, MD
UNC Chapel Hill
REGAIN: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy if Eculizumab in Subjects with Refractory Generalized Myasthenia Gravis
Session VI: Mentoring for Young and Underrepresented NM Investigators to Promote Increased Engagement in Neuromuscular Research
Glen Nuckolls, PhD
NINDS/NIH
NIH Programs for Training, Career Development and New Investigators
Richard J. Barohn, MD
University of Kansas Medical Center
Advice for Young Investigators
Jeffrey Guptill, MD
Duke University
Research Support Resources for Junior Investigators