On September 18, an important battle was won in the war against muscular dystrophy when the Senate enacted the “MD CARE Act Amendments of 2014.” This legislation, which I introduced with Sen. Amy Klobuchar (D-Minn.), passed the House of Representatives this summer, and the President signed it into law on September 26.

We should be encouraged by the success of federal efforts to promote muscular dystrophy research. In 2001, I authored the original “Muscular Dystrophy Community Assistance, Research, and Education (MD CARE) Act,” giving impetus to federal efforts to combat this devastating disease. The law established the first research programs for Duchenne muscular dystrophy at the National Institutes of Health (NIH). Its reauthorization was unanimously passed by Congress in 2008.

Prior to 2001, no legislation had specifically addressed Duchenne muscular dystrophy – the most common fatal genetic disorder diagnosed in childhood. Duchenne affects 1 in 3,500 boys worldwide. Despite progress made by scientists, it remains both incurable and terminal. Since MD CARE, federal research has paved the way for groundbreaking therapies, extending the lifespan of muscular dystrophy patients by an average of 12 years and significantly improving their quality of life. Now that children with muscular dystrophy are reaching young adulthood, an updated law was needed to advance research for older patients.

Building on Our Progress

The latest update to MD CARE removes restrictions on researchers to focus only on pediatric patients. Researchers and medical professionals will be able to move forward with treatments based on current needs and challenges, building on the incredible progress that has been made. The legislation also now covers multiple forms of muscular dystrophy and adds accountability measures to the Muscular Dystrophy Coordinating Committee, which establishes objectives for research and education.

It is remarkable that children who once faced a bleak prognosis are now enjoying longer, more active lives. Muscular dystrophies, which affect more than 100,000 Americans, involve the progressive debilitation of muscle strength. Although there are varying types of the disorder, severe forms lead to serious complications in the heart and lungs. The law now recognizes the need to focus research on these complications as patients live longer.

Encouraging Collaboration for Pediatric Research

We are coming closer to a cure for muscular dystrophy, and bipartisan support in Congress for targeted and effective research is helping make a crucial difference. The updated MD CARE Act follows last year's passage of the “National Pediatric Research Network Act” (NPRNA), another innovative achievement.

NPRNA, which I sponsored with Sen. Sherrod Brown (D-Ohio), facilitates the coordination of findings among NIH researchers on childhood diseases, encouraging the most efficient use of federal resources. The legislation, which became law last November, allows pediatric research institutions across the nation to join forces to combat horrible childhood diseases like Duchenne muscular dystrophy. For example, the newly created National Pediatric Research Network will allow children to participate in some of the most revolutionary clinical trials, even if they live thousands of miles from the primary institution. Additionally, because of the law, rare pediatric diseases will receive increased attention by NIH.

I intend to press forward in the fight against muscular dystrophy. I joined this battle 13 years ago when a fellow Mississippian told me about his son’s diagnosis with Duchenne. Daunting challenges remain; however, the gains we have made hold the promise of even greater breakthroughs to come and, one day, a cure.