The Muscular Dystrophy Association (MDA) is honoring a longtime researcher and an LGMD patient advocate with its 2025 Legacy ...

"Connections with Evan Dawson" brings you special rebroadcasts on 1/30/25. First, AI expert Landon Gray on the future of ...

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...

Sarepta Therapeutics (NASDAQ:SRPT), a biotechnology company specializing in precision genetic medicines for rare diseases with a market capitalization of $11.2 billion, stands at a critical juncture ...

In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did ...

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...