Sarepta Therapeutics said early Tuesday that a patient had died after receiving its gene therapy Elevidys, which treats the rare genetic condition Duchenne muscular dystrophy. A young man with ...

Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of a male teenage patient who had received its gene therapy, Elevidys ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure after being treated with Elevidys, the only gene therapy that has won ...

After an initial accelerated approval decision in 2023, delandistrogene moxeparvovec received full FDA approval in June 2024 to treat ambulatory or non-ambulatory patients ages 4 years and older ...

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular dystrophy. Accessibility statement Skip to main content Democracy Dies ...

Rocket Pharmaceuticals shares plummeted 60% Tuesday after the firm said it halted a study of a gene-therapy drug for Danon disease because a patient had a "Serious Adverse Event (SAE)" and later died.

Patient Dies in Gene Therapy Trial, ... The clinical trial participant who was hospitalized for severe complications after receiving the high dose of Neurogene’s experimental Rett syndrome gene ...

The FDA has slapped a clinical hold on Rocket Pharmaceuticals’ pivotal gene therapy trial in response to a death. Rocket, which was aiming to report data in mid-2026, dropped its targeted trial ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...

Outside the United States, rights to Elevidys are held by Roche, which generated $4 million in royalty revenue during Q1, and $16.8 million in 2024 on sales of the gene therapy.

Sarepta Therapeutics said Tuesday that a 16-year-old boy died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy, the first known death linked to the treatment.