Understanding Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy pic
Duchenne Muscular Dystrophy
Image: WebMD.com

A philanthropist and CEO in the healthcare management sector, Jeffrey Goffman serves as chairman of the Board of Directors with CureDuchenne. In his leadership role, Jeffrey Goffman helps the nonprofit organization in its efforts to find life-saving treatments for Duchenne muscular dystrophy.

Muscular dystrophy represents not a single disease, rather it functions as a category encompassing several genetic conditions with progressive symptoms related to muscle atrophy. Some muscular dystrophies begin in adulthood, while others begin in childhood. Duchenne muscular dystrophy is a childhood disease that almost always affects boys.

Symptoms of Duchenne muscular dystrophy can start appearing in infancy and often get worse with time. Muscle weakness, a hallmark of the disease, usually begins in the lower extremities. Eventually, children with the condition lose their ability to walk and may develop breathing as well as cardiovascular problems by early adulthood. Moreover, Duchenne muscular dystrophy may cause intellectual disabilities.

Though there is no cure for the disease, researchers are working on gene therapies, some of which are in early human clinical trials.


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