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

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

Sarepta and Roche face a regulatory setback as the EMA's CHMP issues a negative opinion on Elevidys for Duchenne muscular ...

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

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

Sarepta has suffered another regulatory setback after the European Medicines Agency’s (EMA’s) Committee for Medicinal ...

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 ...

The U.S. Food and Drug Administration said on Friday that it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics ( NASDAQ: SRPT) gene therapy for Duchenne ...

After a tough few weeks for Sarepta Therapeutics Inc., the EMA dealt another blow on July 25, announcing it will not be ...