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Welcome from NDF CEO/Executive Director Lale Welsh

Lale' Welsh -- CEO/Executive Director, Neuromuscular Disease Foundation


Effects of ManNAc in GNE Myopathy

Dr. Marjan Huizing -- Clinical Project Manager of GNE Myopathy Clinical Studies, NIH

Leadiant Biosciences

Nancy Parsons -- VP Commercial Operations, Pharmaceutical Products

Update on NIH Clinical Trials of ManNAc for GNE Myopathy

Kennan Bradley -- Protocol Coordinator, NIH

GNE Myopathy Panel of Experts

•Ralph Loren -- Biotech Attorney and NDF Board Member

•Dr. Tahseen Mozaffar -- UC Irvine, NDF Board Member and Scientific Advisory Committee Member

•Dr. Wayne Grody -- Professor in the Department of Pathology &  Laboratory Medicine, Pediatrics and Human Genetics, UCLA  School of Medicine and NDF Scientific Advisory Committee Member

•Dr. Gidon Akler -- Board Certified Physician in genetics/genomics and its application to medicine and health

•Michela Onali -- Cultural mediator specialized in EU-Med policies and cooperation. GNEM patient and advocate trained in patient engagement, medicines development and regulations in  Europe.

Whole Genome Sequencing for GNE Myopathy - The NDF Initiative

Dr. Madhuri Hegde -- V.P. and Chief Scientific Officer, Perkin-Elmer and Adjunct Professor, Emory University

Scientific Directions in Preparation for Gene Therapy

Dr. Monkol Lek -- NDF's Scientific Director and an Assistant Professor and Research Group Leader at The Yale School of Medicine's Department of Genetics & Dr. Angela Lek NDF's Associate Scientific Director and an Associate Research Scientist at Yale University with extensive experience in elucidating the molecular mechanisms of neuromuscular diseases, joining via live video.