Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...

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

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

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

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

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

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

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.