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 ...

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

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

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

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

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

Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys ...

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 ...

The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to pause dosing in another study.

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

Roche announces positive results from EMBARK phase III study of Elevidys in ambulatory individuals with DMD: Basel Tuesday, January 28, 2025, 11:00 Hrs [IST] Roche announced posit ...