The U.S. Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for ...
Tributes have been paid to an inspiring Duchenne muscular dystrophy sufferer who campaigned for easier access to wheelchairs. Karen Duckmanton, from Witham, was one of only a few women in the world ...
The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...
Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...