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

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

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

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

Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the ...

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

According to Dr Ann Agnes Mathew, Consultant Pediatric Neurologist at Bangalore Baptist Hospital, DMD is life-threatening and ...

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

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

The Muscular Dystrophy Association (MDA) is honoring a longtime researcher and an LGMD patient advocate with its 2025 Legacy ...

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

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