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

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

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

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

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

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

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

Spinraza remains a big product for Biogen, but has seen its sales go into reverse as competition in SMA treatment has emerged ...

A pilot study in 19 LGMD patients and one with Becker MD found low-dose prednisone helped lower markers of muscle damage and ...

Benitec has a market cap of $250mn, $67.8mn in cash, and a cash runway of over 10 quarters, but faces risks due to its small ...