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

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

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

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 ...

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today unveiled positive ...

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

Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The ...

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

He was diagnosed with Duchenne muscular dystrophy at age 5.Family remembers the day everything changed"I can remember the day ...

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...

Capricor's DMD therapy Deramiocel is under FDA Priority Review with no major issues flagged; PDUFA date set for August 31, 2025.

Wilson Lam, 36, and Hannah Lam, 31, from Northolt in North West London are taking on the 'Pedal Paddle Peak' challenge in the ...