A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Indiana FFA Association organizes Unified Horticulture Career Development Event for students with intellectual disabilities.

Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

Sarepta Therapeutics said on Tuesday that a 16-year-old boy died from acute liver failure months after receiving the company's U.S-approved gene therapy for a rare muscular dystrophy. The patient ...

Sarepta stock crashed Tuesday after a patient who received its approved gene therapy, Elevidys, died due to acute liver failure.

Cambridge-based Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for ...

Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...

Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

The FDA expanded the indication of eculizumab (Soliris) to include pediatric patients ages 6 and older with generalized ...

Chronic inflammatory demyelinating polyneuropathy (CIPD) can have a substantial impact on patients everyday life and, ...