Johnny Quintana’s life changed at the age of 19 when he was diagnosed with a rare form of muscular dystrophy called ...

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 ...

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

MDA launches 75th Anniversary campaign and premieres new public service announcement (PSA) to mark 75 years of impact in the ...

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 ...

Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approv ...

The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National ...

Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...