Three patient deaths linked to gene therapies developed by Sarepta Therapeutics (SRPT) have drawn FDA scrutiny on the viral ...

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

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 targets.

Sarepta and Roche face a regulatory setback as the EMA's CHMP issues a negative opinion on Elevidys for Duchenne muscular ...

Sarepta said it accepts the CHMP decision on Elevidys, while partner Roche said it will continue working with the EMA.

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

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

A timeline of key developments surrounding Sarepta Therapeutics' Duchenne muscular dystrophy drug Elevidys, and the potential ...

Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the ...

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

Company CEO Doug Ingram said the pause was necessary for Sarepta to maintain a "productive and positive working relationship" with the regulator.