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 a male teenage patient who had received its gene therapy, Elevidys ...

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 Therapeutics Inc. misled investors about the safety and prospects for one of its Duchenne muscular dystrophy ...

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

The first death — a teenage boy who also died from acute liver failure after receiving Elevidys — was reported by the company in March. Both deaths occurred in non-ambulatory individuals with DMD.

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

According to Sarepta, the patient was the second to succumb to acute liver failure (ALF) after being treated with Elevidys, which is the only gene therapy that has won FDA approval as a treatment ...

Pompe's disease is a lysosomal storage disorder caused by a genetic deficiency of GAA. The disease is associated with severe ...

Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new ...