Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

Roche has announced positive results from the Phase III EMBARK clinical trial of Elevidys (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy (DMD). Following two years of ...

This test is used to diagnose myotonic muscular dystrophy, which causes muscles to stay contracted for an abnormally long time. Facioscapulohumeral muscular dystrophy (FSH MD) begins in adolescence or ...

There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...

The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1) in a clinical trial ... DM1 is a form of muscular dystrophy ...

Dystrophy is any condition in which a part of the body weakens or wastes away. In muscular dystrophy, the weakness is in the muscles. An inherited genetic mistake prevents the body from making a ...

Muscular dystrophy can appear in infancy up to middle age or later, and its form and severity are determined in part by the age at which it occurs. Some types of muscular dystrophy typically ...

I made it to the garage with no more than the problems I usually have because of my facioscapulohumeral muscular dystrophy (FSHD). Once I got to the garage, I sat down, put my shoes on, and struggled ...

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic condition that affects muscle function, often making everyday tasks — like smiling or lifting one’s arms — difficult. For individuals ...

Our researchers are also exploring strategies to block deleterious gene expression in facioscapulohumeral muscular dystrophy. Cell-based therapies — including stem cell and bone marrow transplants — ...