REGENXBIO is a longtime player in the gene therapy space, but one that’s yet to bring its own therapy to market (in the early ...

"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 ...