Muscular dystrophy
Muscular dystrophy is more than one disease. It describes a group of neuromuscular diseases with different causes and progressions. Muscular dystrophy has become synonymous with Duchenne muscular dystrophy, the most severe and, unfortunately, most common form. But though all muscular dystrophies lead to progressive weakness and degeneration of skeletal muscles, they differ in age of onset, severity, and rate of progression. There is no cure.
Duchenne muscular dystrophy appears primarily in boys, is early onset, progresses at a terribly rapid rate and is fatal. Other forms, including myotonic dystrophy and facioscapulohumeral muscular dystrophy, occur in teens and adults as well as children, and progression is relatively slow. The genetic cause has been found for many forms of the disease, including Duchenne, but effective treatments remain elusive. A great deal of work and support have gone into research—think comedian Jerry Lewis and his annual telethon—and more understanding about how muscles and nerves work together holds the hope for a better prognosis.
Muscular dystrophy research at The Jackson Laboratory
Research into muscular dystrophy at The Jackson Laboratory focuses on identifying how degeneration of skeletal muscles actually occurs, in a variety of different model systems. Learning the fundamental processes of the disease and how it progresses has the potential to provide more effective therapies and better targets for clinical treatment.
