The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...

Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely affects multiple organs including skeletal muscle, heart, brain and the ...

Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...

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

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.

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 ...

The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...

The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.