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

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

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

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.

The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the ...

The patient with Duchenne muscular dystrophy, who died in Brazil, was treated with Elevidys but was not a participant in a ...

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

We recently published 10 Shares Explode as Earnings Impress. Avidity Biosciences, Inc. (NASDAQ:RNA) is one of the ...

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 ...

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

Nucleic acid therapies offer targeted treatment for genetic diseases, but delivery remains a key hurdle. Explore how advances ...