My name is Dr. Lee Sweeney, and I am the William Maul Measey Professor and Chairman of the Physiology Department at the University of Pennsylvania School of Medicine. Today, I am testifying at the request of the Parent Project MD, as an expert in muscle biology, regarding muscular dystrophy in general, but with a focus on Duchenne muscular dystrophy, and its weaker form, Becker muscular dystrophy. I appreciate the invitation to testify today and I want to especially thank Senator Specter and Senator Harkin for allowing me the opportunity to speak on behalf of the muscular dystrophy community. While Congress has been generous to NIH, research on muscular dystrophies needs special attention to attract new investigators into a historically under-funded area.
Muscular dystrophy is a broad term that refers to a number of primary diseases of muscle.
Many aspects of these diseases are not understood, and treatments are either unavailable or minimally effective. The age of onset and severity of these diseases is highly variable. Images of its victims, the young boys, don't easily leave your mind. DMD is the most severe of all of the muscular dystrophies. It is the world's most common and catastrophic form of genetic childhood disease.