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 ...

People with myotonic dystrophy type 1 (DM1), the most common adult-onset form of muscular dystrophy, progressively lose ...

Myotonic muscular dystrophy, which is sometimes called ... It has been described as congenital onset, juvenile onset, and adult onset, based on the age at which the symptoms begin.

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 ...

What Is Duchenne Muscular Dystrophy? Muscular dystrophies are ... The disease almost always affects boys, and symptoms usually begin early in childhood. Children with DMD have a hard time standing ...

Duchenne muscular dystrophy (DMD) is a genetic condition ... Typically, a carrier of DMD will not show symptoms. However, some individuals may show mild symptoms, such as muscle weakness.

The FDA has granted fast track designation to DYNE-101, a therapy for myotonic dystrophy type 1 (DM1) now in testing in a ...

Baylor Medicine’s General Adult Genetics Clinic sees patients 18 years or older ... Loeys-Dietz syndrome, Duchenne Muscular Dystrophy, Williams syndrome and velocardiofacial syndrome. The clinic is ...

Although often considered to be diseases of childhood, adult forms of muscular dystrophy occur and represent ... occur in the presence or absence of symptoms of congestive heart failure.

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings ...