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

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

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

Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines ...

Cumberland Pharmaceuticals’ Duchenne muscular dystrophy (DMD) drug has improved the amount of blood pumped by the heart, ...

Some of the market’s biggest winners start off as the smallest players. While trillion-dollar tech giants like the ...

Cumberland Pharmaceuticals Inc. (NASDAQ:CPIX) released topline results from its Phase 2 FIGHT DMD trial. The study evaluated ...

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

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

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.