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 ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its ...

When the FDA unexpectedly uploaded around 200 drug rejection letters this week, the regulator provided an in-depth view | Although many of the complete response letters uploaded by the FDA this ...

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 ...

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

Ryan Wach shared the “hard part” of his son Zane Wach’s recovery has been drug withdrawal following his a 120-foot fall from California’s Mount Whitney in June.

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 treatments ...

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