Celebrating 25 Years of MD CARE Act
One of the most rewarding experiences of my congressional career has been joining the race to treat and eventually cure Duchenne Muscular Dystrophy (DMD). This year marks the 25th anniversary of the Muscular Dystrophy Community Assistance, Research, and Education, or MD CARE, Act. I wrote that 2001 legislation alongside the father of a boy diagnosed with Duchenne. After the law unlocked research funding, scientists quickly got to work.
25 Years of Progress
As experts studied this under-researched disease, they began to discover breakthrough treatments. It has been a joy to witness the progress we have seen so far. DMD impacts muscle strength, ultimately weakening patients’ hearts and lungs. But increased research has led to treatments and therapies to forestall that muscle decline. In 2001, children diagnosed with the disease had little reason to expect to live beyond their late teens. Today, many patients live into adulthood. That fact compelled my colleagues and I to update the MD CARE Act. The original had included funding only for pediatric research, but our 2014 additions paved the way for studies focused on adult patients.
These developments signify much more than positive statistics. Because of them, families are able to spend more time together. Young people get the chance to go to college, experience adulthood, and contribute even more to their communities. Their stories move us to continue fighting this disease.
Expanding Newborn Screenings
In the past year, I have worked to get more newborns tested for DMD—a powerful way to help children receive treatment quickly. The sooner a baby is screened, the better his or her outcomes. Many Duchenne patients receive testing only after the disease has progressed, making treatment less effective. But infant testing can detect DMD before symptoms develop. The advance notice buys time, allowing health care providers to administer treatment before the disease progresses.
The key to expanding newborn testing was changing the federal-level guidance, known as the Recommended Uniform Screening Panel, or RUSP. The Department of Health and Human Services (HHS) publishes this list, which calls states to test newborns for a variety of disorders, diseases, and other conditions. Many states follow these HHS recommendations. In other words, getting a test included on the RUSP was a surefire way to help more babies get screened for DMD.
Last December, we were successful. HHS updated the RUSP to include Duchenne, and states have begun adopting the test in their regimens. Tennessee’s Genetics Advisory Committee voted to add DMD to the state’s newborn screening panel. Arizona will soon require the test. Other states, including Mississippi, automatically update their screening lists to follow the RUSP.
More Breakthroughs Ahead
This quarter century of progress tells us that further breakthroughs are possible. As always, our ultimate ambition is to find a cure. A future without Duchenne would be a better world for everyone. In the meantime, we will continue combating the negative effects that follow a diagnosis.
I will continue advocating for research funding. Those grants give scientists certainty that they can afford to step into this crucial work and advance the frontiers of DMD studies. I will also keep working with the Food and Drug Administration and those developing novel medicines, helping facilitate innovative treatments. We have seen so much progress in 25 years, but there is much more work to do. Seeing more and more DMD patients able to live longer and fuller lives is the only encouragement we need.
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