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

Complete Response Letters recently issued by the FDA signal heightened scrutiny of trial design and reinforce the agency’s shifting regulatory expectations for sponsors and CROs.

This week's episode covers news about Sarepta, babies born following mitochondrial transfer, and AI identifying therapy ...

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

Morning. Today, we discuss how Duchenne patients and their families are responding to the shelving of Sarepta Therapeutics’ ...

Despite a new setback for Elevidys in Europe, Roche—which markets Sarepta’s gene therapy outside the U.S.—remains committed ...

Separately, Sareptea said last week that it would pause the development of most of its experimental gene therapies for a different type of muscular dystrophy. The stoppage came after one of the ...

Most Americans encounter the Federal Trade Commission only if they’ve been scammed: It handles identity theft, fraud, and ...

CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an ...

Salveen Richter, Goldman Sachs lead biotech analyst, joins 'Squawk Box' to discuss the troubles facing Sarepta and its ...

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

Two teenage boys who took the gene therapy died of acute liver failure, and a third liver failure death last month came after a 51-year-old patient took another Sarepta gene therapy to treat a ...