On the recordJune 10, 2015
Mr. Chairman, I rise today to offer an amendment to add an additional $1 million for research and development for Duchenne muscular dystrophy. Duchenne muscular dystrophy is the most common lethal genetic disorder affecting American children today. {time} 1800 It is a progressive neuromuscular disorder that affects approximately 1 in every 3,500 boys or 200,000 babies born each year worldwide. Over time, patients experience severe loss of muscle strength and control. Most boys diagnosed with Duchenne lose their ability to walk by the time they become teenagers. There is no known cure for Duchenne, and life expectancies for individuals with this disease are significantly shortened. Many do not live past their 21st birthday. Like many of my colleagues, I have met with many Duchenne patients and their families and have seen the impact this disease has and what it imparts on their daily lives. There have been very promising advances in recent years, including development of a new drug which has achieved success in early clinical trials. I have had one child in my district confined to a wheelchair who, under this clinical trial, is able to walk by himself currently. However, much more work needs to be done to find a cure for this disease and to better understand what causes Duchenne in the first place. This amendment will directly benefit the thousands of Duchenne patients throughout the United States, as well as their countless loved ones who care for them every day.…





