Description of Collaborative Activity: |
This Muscular Dystrophy Association (MDA) and National Institute of Neurological Disorders and Stroke (NINDS) co-sponsored workshop will bring together experts from academia, industry, and regulatory agencies to address core challenges in the development of gene therapy for neuromuscular diseases and develop recommendations to help guide specific efforts in neuromuscular disease, as well as in the broader field of gene therapy in neurological disorders. The focus of the workshop will be on three neuromuscular diseases, spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS), where unmet medical need and the state of the science are compelling. |
NIH Participating Institutes/Centers/Office of the Director: |
NCATS, NHLBI, NIAMS, NICHD, NINDS |
