The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

Three patient deaths linked to gene therapies developed by Sarepta Therapeutics (SRPT) have drawn FDA scrutiny on the viral ...

Sarepta Therapeutics (SRPT) in focus as the biotech says the death of a patient who had received its gene therapy Elevidys ...

A biopharmaceutical research company with ties in Andover has laid off a third of its workforce as one of the leaders in gene therapy goes through a “strategic ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.

Sarepta maintains it reported the incident to the FDA in June, and the boy's doctor had deemed his death was unrelated to treatment.