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

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

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 ...

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

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...

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

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

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

Sarepta Therapeutics shares snapped a three-day losing streak on Tuesday, as analysts said resumption of U.S. shipments for ...

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 ...