As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm ...

Although Sarepta initially appeared to have avoided the worst possible outcome for Elevidys—a market withdrawal—thanks to the gene therapy’s new black box warning, the FDA is signaling stronger ...

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 ...

Sarepta will share the panel’s recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance. In the ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003.

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 ...

The rAAVrh74 viral vector is a key component in several of Sarepta’s gene therapy programs, including Elevidys, an already FDA-approved gene therapy for Duchenne muscular dystrophy. It is also ...

U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4 ...

FDA evaluating further regulatory actions after deaths Elevidys is the only gene therapy for Duchenne muscular dystrophy Sarepta suggested updating Elevidys' label June 24 (Reuters) - The U.S ...