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

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

The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...

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

US regulators are investigating the death of an 8-year-old boy in Brazil who received Sarepta Therapeutics Inc.’s Elevidys.

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

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

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

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

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

The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne ...

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