A second child has died after receiving Elevidys, an experimental gene therapy for Duchenne muscular dystrophy—raising serious concerns about regulatory shortcuts, patient safety, and the urgent need ...

The agency said it will evaluate whether further regulatory action is needed after investigating the deaths of two patients ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...

Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company that discovers and develops ...

The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from ...

Wall Street’s major market averages seesawed back and forth on Friday and finished mixed as Federal Reserve Governor ...

The S&P500 (SP500) closed in the red, with investors keeping a close eye on the escalating tensions in the Middle East. For ...

Sarepta shares face pressure after a second Elevidys-linked death and analyst downgrade over safety concerns and lowered revenue prospects.

Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...

In this week’s edition of InnovationRx, we look at the dangers of RFK Jr.’s vaccine advisory committee, how Trump’s visa ban ...

Another patient has died from acute liver failure after receiving Sarepta’s gene therapy for DMD ; After a quiet start to the ...