Second patient death from liver failure after Sarepta's Elevidys gene therapy triggers FDA investigation. Stock crashes 41% ...

Shortly after the U.S. FDA announced an investigation into the death of a patient who had received Elevidys, a gene therapy ...

The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...

Elevidys, approved by the FDA in 2024 for ambulatory Duchenne muscular dystrophy patients aged four and older, is the only gene therapy available for the disease. It carries a known risk of liver ...

In June, 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 ...

In June, 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 ...