Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

Capricor Therapeutics is seeking FDA approval for CAP-1002, targeting heart function in Duchenne Muscular Dystrophy patients.

"I'm so happy because I thought he wasn't going to make it," Taevion Norris' mother said at the graduation ceremony.

Gene therapy could be the newest breakthrough for Duchenne muscular dystrophy. While it may not be a cure, it can give independence to boys diagnosed with DMD.

Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

Since Elevidys' accelerated approval in 2023, experts have been clamoring for more data, particularly in older and ...

Muscle weakness has the biggest impact on patients’ ability to walk, but other factors matter too, researchers found.

And for now, Logan and his family are reeling in the moments, one fish at a time.

Quince Therapeutics advances Ataxia-Telangiectasia treatment with innovative AIDE platform, insider support, and NEAT Phase 3 ...

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients ...

The National Institute of Allergy and Infectious Diseases (NIAID) plans to initiate phase 1 clinical trial of Capricor's ...