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

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

Shares of Sarepta Therapeutics SRPT nosedived 42% in pre-market today after the company reported a second patient death following treatment with Elevidys, its one-shot gene therapy for Duchenne ...

Sarepta halts Elevidys shipments for non-ambulatory DMD patients after a second patient death in three months post gene therapy treatment.

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.

Sarepta reported a second death in a patient who had received its gene therapy, which raised concerns about the safety and future demand for the treatment. Both boys were non-ambulatory and their ...

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death of a second recipient.

Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for patients who can no longer walk, following the death of a second person who ...

KLTO‑202 is an experimental gene therapy that would deliver the instructions for a protein that may reduce inflammation and oxidative stress.

A single shot of gene therapy restored hearing in people born deaf, with most showing improvement within weeks and some ...

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...