The drug Givinostat has been brought into parts of Britain. We want that too, but we need the government to pay for it,' said Una Ennis ...

U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s ...

One visit to Virginia Peninsula Community College’s Historic Triangle Campus last summer eased any concerns they might have had. Soon after entering the building, Nicholas and his mother, Liz, were ...

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

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 ...

Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...

The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North ...

The initial signs of Duchenne muscular dystrophy in a 3 or 4-year-old child may be subtle, explains Dr. Alexandra Bonner of the Center for Pediatric Neurosciences at Cleveland Clinic.

He was diagnosed with Duchenne muscular dystrophy at age 5. "I can remember the day he got diagnosed," said his older sister, ...

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 ...