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 ...

Genethon is determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic ...

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 ...

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

Genethon gets UK MHRA nod to initiate pivotal phase 3 trial of GNT0004, a low-dose microdystrophin gene therapy for Duchenne muscular dystrophy: Paris, France Wednesday, July 30, ...

A recent FDA investigation has paused further distribution of Elevidys, following reports of at least three patient deaths.