The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...

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

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

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

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

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

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

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

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

Shares of drugmaker Sarepta Therapeutics surged in afterhours trading Monday after the company said it would resume shipping ...