Sarepta Therapeutics has rejected a call by the Food and Drug Administration to stop shipments of its muscular dystrophy drug ...

FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due to safety concerns.

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 ...

Sarepta Therapeutics Inc. shares plunged after European regulators rejected its gene therapy Elevidys, intensifying scrutiny on the drugmaker after it was pressured to halt shipments of its treatment ...

The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment continues.

With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is frustrated by the delay of approved treatments in the rare disease ...

A common drug used for inflammatory skin conditions may hold the key to treating both alcohol addiction and pain sensitivity. That’s what scientists at a leading research institute have found.

Family shares frustrations after FDA rejects drug for ultra-rare Barth Syndrome By CNN Newsource Published June 28, 2025 12:54 pm ...

SAN FRANCISCO (KPIX) — With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is frustrated by the delay of approved ...

Capricor Therapeutics said the U.S. Food and Drug Administration has rejected its application seeking approval of its lead cell-therapy candidate Deramiocel in the neuromuscular genetic disease ...