Companies in #Neuromuscular

Clinical-stage biopharmaceutical company developing novel small-molecule therapeutics for rare genetic neurodevelopmental, neuromuscular and central nervous system disorders.

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on precision genetic medicines for rare neuromuscular and central nervous system diseases.

Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Its lead product, apitegromab, is being developed for spinal muscular atrophy (SMA).