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

Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of ...

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

The death of a boy with Duchenne muscular dystrophy, tied to a gene therapy, is above all a tragedy. But it is also ...

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...

Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for ...

Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for ...

Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain ...

Shares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.

Sarepta’s shares crashed 41% in premarket trading Monday morning to $21.01 after the biotech reported a second death from ...