Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...

Although multiple sclerosis (MS) and muscular dystrophy (MD) are both debilitating ... that affects the central nervous system (CNS)—the brain, spinal cord, and optic nerves.

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...

Medcare’s approach goes beyond clinical excellence. Patients benefit from a multidisciplinary team that includes experts in ...

Liver damage is a well-recognised complication of gene therapies based on adeno-associated virus (AAV) vectors, and other ...

Intrathecal onasemnogene abeparvovec for patients with spinal muscular atrophy: Phase 3 randomized, sham-controlled, double-blind STEER study. Presented at: Muscular Dystrophy Association Clinical ...

drawing participants to raise awareness and support for those living with spinal muscular atrophy, a form of muscular dystrophy. Attendees enjoyed a variety of activities including games ...