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

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 ...

Regenxbio (RGNX) stock gains and Solid Biosciences (SLDB) stock falls after Sarepta Therapeutics' (SRPT) safety update on ...

Ed Kaye, former CEO of Sarepta Therapeutics, is stepping down from his current role as chief executive of a Boston-area ...

Gypsy Rose Blanchard has opened up about how one specific detail of the day her boyfriend murdered her mother has stuck with ...

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 ...

Sarepta Therapeutics shares dropped 20% after a patient's death raises Elevidys safety concerns. Read why I am downgrading ...