Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with ...

Pfizer's hemophilia therapy, Hympavzi, met the main goal of a late-stage study in patients with certain types of antibodies, ...

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

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 the death over the ...

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

Second patient death reported with gene therapy for muscular dystrophy BY Associated Press Washington, D.C. PUBLISHED 4:05 PM ET Jun. 16, 2025 PUBLISHED June 16, 2025 @4:05 PM ...

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 ...

Elevidys is the first gene therapy approved in the U.S. for the rare muscle-wasting condition, but it has faced scrutiny since its accelerated approval in 2023. Related Articles.