The agency is considering further regulatory action after a second Duchenne patient died from acute liver failure less than two months after receiving the gene therapy.

The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...

SynaptixBio, the only company licensed to commercialise a treatment for a rare but deadly disease, welcomes the announcement but says it lacks a detailed plan The government has announced that every ...

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 ...

Precision BioSciences drew heightened retail investor chatter on Wednesday after the FDA awarded Rare Pediatric Disease ...

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

FDA is investigating Sarepta's Elevidys after two deaths from liver failure in non-ambulatory DMD patients, raising safety ...

Sarepta stock dropped again Wednesday after the FDA announced it's investigating the deaths of two patients who received Elevidys.