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

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 ...