Sarepta has issued a safety update regarding Elevidys (delandistrogene moxeparvovec-rokl) following a second reported case of acute liver failure resulting in death.

The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from sufficient to maintain momentum in the rare disease space.

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 ...

In another tragic incident, Sarepta Therapeutics (NASDAQ: SRPT) saw a second death in connection with its Duchenne muscular ...

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

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in ...

Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...

How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene ...

Sarepta Therapeutics Inc. plummeted after reporting a second patient died of acute liver failure while being treated with its gene therapy for a rare muscle disorder.