Understanding how the dystrophin and utrophin protein networks interact with other cellular components may reveal novel characteristics of cytoskeletal function and adaptability. We hypothesized ...

In DMD, genetic variations in the dystrophin gene affect the function of the dystrophin protein. These alterations mean that muscle damage occurs after they repeatedly contract and relax with use.

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

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

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

DMD is a rare genetic disorder caused by loss-of-function mutations in the dystrophin gene. An X-linked condition, the disease mostly affects boys, and usually manifests itself in the form of ...

indicating that the gene therapy did not provide a substantial motor function improvement over the placebo. Secondary ...

Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the belief the answer could come down to delivery.

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

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