The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...

FDA has indicated that an Advisory Committee meeting is not required at this timeIn-person late-cycle review meeting ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...

Capricor Therapeutics updates on Deramiocel BLA for duchenne muscular dystrophy: San Diego Thursday, June 26, 2025, 15:30 Hrs [IST] Capricor Therapeutics, a biotechnology company ...

Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...

The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...

Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...

Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...

U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s focus in neuromuscular diseases Capricor remains on track for the ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in ...