Duchenne Muscular Dystrophy

Floor Speech

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Ms. COLLINS. Mr. President, I wish to raise awareness about Duchenne muscular dystrophy and the boys and young men who suffer from this devastating disease.

Duchenne muscular dystrophy was first brought to my attention 15 years ago, when I met Brian and Alice Denger of Biddeford, ME. The Dengers had two wonderful sons, Matthew and Patrick, who were both born with Duchenne muscular dystrophy. Patrick, now 19, is a student at the University of New England. He recently received his driver's license and enjoys driving in Maine. His brother Matthew was a 20-year-old student at UNE when he died from the disease about 3 years ago. The Dengers also have a daughter, Rachel, with juvenile diabetes. They are a loving and courageous family whose strength and spirit directly inspired me to become involved in the fight for research funding to combat muscular dystrophy.

Brian Denger was the first to tell me of the terrible progression of this type of muscular dystrophy. Symptoms begin in early childhood, and boys quickly experience severe and rapidly progressing muscle degeneration, which often results in their losing the ability to walk. Tragically, most die prematurely as a result of muscle-related cardiac and respiratory problems.

In 2001, what really caught my attention was that the treatment options for boys with Duchenne muscular dystrophy were incredibly limited and aimed at managing symptoms in an attempt to optimize quality of life for the limited time that these children would have to share with us. Research had not yielded any meaningful way to extend the lifespan of children suffering from the disease. That is why I joined with the late Senator Paul Wellstone in introducing the MD CARE Act, to raise awareness and expand Federal support for research into this debilitating disease. It was signed into law and last reauthorized in 2014 and has resulted in dramatically improved and standardized clinical care for those with the disease. I have also fought diligently for increased funding for the Duchenne programs at the National Institutes of Health and the Centers for Disease Control and Prevention.

Today there is some good news for the boys--and now--young men with Duchenne muscular dystrophy and their families. A number of therapeutic strategies are currently under development, and we have made dramatic progress to improve the quality and length of life for those who suffer from the disease. In fact, the average lifespan of Duchenne patients has increased by about a decade since the MD CARE Act became law.

Given our Nation's wealth of scientific expertise, however, we can and should do more for families like the Dengers. We are making progress, but this is no time to take our foot off the accelerator. The $2 billion increase in funding for NIH that was included in the fiscal year 2016 funding bill will pay dividends for patients and their families. I urge my colleagues to continue to work collaboratively to sustain this commitment to biomedical research, which holds tremendous promise for finding better treatments and, ultimately, a cure for devastating diseases like Duchenne muscular dystrophy.

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