Percheron Therapeutics says it will abandon its lead drug candidate altogether, following December's Duchenne muscular dystrophy trial flop.

Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for ...

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

The Muscular Dystrophy Association (MDA) proudly announces the launch of its 75th anniversary campaign, marking a legacy of progress in research, care, and advocacy for people living with any of over ...

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

(IN BRIEF) Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for Duchenne muscular ...

The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.

The 33-year-old took to Instagram on Friday (January 24) to address a slew of false rumors. First, she hit back at a claim ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

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

The former followed a man born with muscular dystrophy, who finds a beautiful social life in the most unexpected way—while damn near reinventing the genre at the same time. Meanwhile, Will Ferrell and ...