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

Satellos said that it will be advancing its DMD small molecule to a Phase II trial, following the Phase I data.

Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for ...

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

Sarepta Therapeutics (NASDAQ:SRPT) and its partner Roche (OTCQX:RHHBY) received a 'go ahead' from the regulatory body in U.K.

Researchers are working to redefine diagnosis and treatment with data-driven tools aimed at identifying patients earlier and ...

New data demonstrated the benefits of Elevidys, a gene therapy for multiple Duchenne muscular dystrophy, in different age ...

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.

Multiple first-in-human trials showed functional and biomarker improvements in early data reported at ASGCT's annual meeting.

In cohort 6, subjects aged two years old at the time of treatment showed a mean expression of 93.87% of normal dystrophin ...

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