The symptoms of myotonic dystrophy may be obvious from birth or they can develop later -- during the teenage or adult years. Like other forms of muscular dystrophy, myotonic dystrophy leads to ...

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness and wasting. It is an X-linked recessive disorder that occurs due to changes in the DMD gene ...

making it the most common adult-onset muscular dystrophy. We believe that the market opportunity for a new DM1 therapy is very attractive, given the important unmet medical need and the lack of an ...