When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...

The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying the agency was investigating the three deaths due to acute liver failure ...

Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

Gene therapy approach developed offers new hope for those with Duchenne muscular dystrophy by restoring the full-length dystrophin protein. Systemic delivery of full-length dystrophin in ...

As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...

The death of a 51-year-old man in the study follows two other deaths of Duchenne patients treated with Sarepta’s marketed ...