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

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

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

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

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

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

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

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

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

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 ...

of its gene therapy Elevidys approved for the treatment of Duchenne muscular dystrophy. Results from the study showed that treatment with Elevidys brought sustained benefit and disease ...