Until I met Brian, I really did not know much about Duchenne Muscular Dystrophy. He was the first to tell me that one male child in 3,500 worldwide will be born with the disease and lose the ability to walk by age 10. He also told me about the terrible progression of the disease, how muscle deterioration in the back and chest begins to put pressure on the lungs, making it more and more difficult for a child to breathe.
What really caught my attention was the fact that the lifespan of children suffering from this disease has not been extended in any significant way in recent years. Current treatment options for boys like Matthew and Patrick are minimal and aimed simply at managing their symptoms in an effort to optimize their quality of life for the limited time they have with us. Last week was Duchenne Muscular Dystrophy Awareness Week, which came into being as a consequence of legislation I introduced at the request of Parent Project Muscular Dystrophy, a non-profit organization that helps those afflicted by Duchenne MD and their families. It also marked the two-year anniversary of the introduction of the Muscular Dystrophy Community Assistance Research and Education Act – or MD CARE Act – which I was pleased to introduce with the late Senator Paul Wellstone. Our legislation, which was signed into law in December 2001, will help raise awareness and expand federal support for medical research to find a cure for this devastating disease. Given our nation's wealth of scientific expertise coupled with the tremendous infusion of resources Congress has poured into the NIH in recent years, we can and should do more for families like the Dengers. That is why I worked so hard for the passage of the MD CARE Act, and why I will continue to work to make Americans aware of muscular dystrophy and its effects through efforts like Duchenne MD Awareness Week.
Since the passage of the MD CARE Act, the National Institutes of Health (NIH) have established grants for the creation of three Centers of Excellence in Muscular Dystrophy Research, which will provide focused research and development in all phases of the research spectrum: basic clinical, and transitional. In addition, the Centers for Disease Control and Prevention (CDC) have developed an in-depth surveillance and epidemiology study of Duchenne and Becker muscular dystrophy. A population-based epidemiological study of Duchenne and Becker muscular dystrophy will provide the extensive data necessary to inform research decisions, standards of care, physician training, and public health approaches to assist families living with Duchenne and Becker muscular dystrophy.
The NIH and the CDC are to be commended for the progress they are making in their research efforts related to muscular dystrophy. These efforts to improve the quality and length of life for thousands of children diagnosed with muscular dystrophy will someday make a real difference for the families struggling with this devastating disease.