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

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...

In this week’s edition of InnovationRx, we look at Moderna’s use of quantum computers, a new top drug regulator at the FDA, ...

Sarepta has suffered another regulatory setback after the European Medicines Agency’s (EMA’s) Committee for Medicinal ...

While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency ...

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 ...

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.

Key Points Sarepta Therapeutics' most crucial product is turning out to have severe safety issues. The company's efforts to improve its situation were well received, but this victory didn't last long.

Wall Street analysts warn that if Sarepta Therapeutics doesn't resolve the Elevidys issues, it could jeopardize its ability to repay debt in 2027.

Shares of Sarepta Therapeutics fell close to 37% on Friday after reports emerged that the US Food and Drug Administration is ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli ...