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

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

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

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

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

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 ...

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

Sarepta Therapeutics suffered another major setback on Friday as Europe's drug regulator decided not to recommend the ...

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

Friday said that the European Medicines Agency has issued a negative opinion on the conditional marketing authorization for Elevidys, intended for ambulatory patients aged three to seven with Duchenne ...

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

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