WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.

A second patient receiving its experimental gene therapy Elevidys has died from liver failure, raising serious questions on ...

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

The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...

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

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

Sarepta Therapeutics has temporarily suspended use of the gene therapy Elevidys in non-ambulatory patients with Duchenne muscular dystrophy (DMD) after a second patient taking the drug died from ...

Solid Bio’s gene therapy candidate, SGT-003, was designed to improve both safety and efficacy. Using a lower dose, this therapy is able to achieve greater levels of microdystrophin expression.

Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. The most common adverse reactions reported were vomiting, nausea, increased liver function tests ...

The gene therapy is administered as a single intravenous infusion. Sarepta evaluated Elevidys in open-label studies as well as a randomized, placebo-controlled Phase 2 clinical trial .

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings ...