Wicker Recounts Efforts to Combat Rare Disease

The first quarter of this century is coming to a close, prompting me to look back at the work Congress has done in the past 25 years. Among the most important achievements I see are our efforts to combat rare diseases, especially those afflicting children.

Identifying a Need

Many people become involved in the fight against rare diseases because they have a personal connection with someone battling one of these afflictions. That was certainly true for me. In 2001, I was serving in the U.S. House of Representatives. A friend came to my office and told me the story of his young son, who had recently been diagnosed with a rare disease called Duchenne Muscular Dystrophy (DMD).

In our conversation, the father explained the impacts of this illness. DMD is a progressive genetic disorder primarily affecting boys-roughly one in 3,600 each year. The disease gradually weakens muscle strength, which can lead to mobility loss and create problems for a patient's heart and lungs. In 2001, many DMD patients lived only into their late teens. Most health care providers at that time were focusing treatments on symptoms, but this boy's father and I believed we could build support for the search for a cure.

Kickstarting the Search for a Cure

Within a few days, we had a bill, called the Muscular Dystrophy Community Assistance Research and Education (MD CARE) Act. The text directed federal research funding toward muscular dystrophies, including DMD. Crucially, it focused research activities on finding cures.

I began walking up and down the aisles of the House of Representatives, getting hundreds of Republicans and Democrats to support the legislation. Eventually, over 300 of my House colleagues signed onto the MD CARE Act, and the Senate passed companion legislation. By the end of the year, President Bush had signed it into law.

Expanding to More Rare Diseases

Passing the MD CARE Act was a big step forward, but it was just the beginning. In the years since, we have seen more research investment, trials, and even follow-up legislation. In 2013, I authored and helped pass a law called the "National Pediatric Research Network Act." That law was meant to streamline research activity for childhood illnesses and give more children access to clinical trials and treatments-especially for those with rare diseases. Unfortunately, the law faced a number of implementation challenges. I was able to help solve some of those issues in the 21st Century Cures Act, a 2016 law that accelerated medical treatment and cures.

Expanding to More Rare Diseases

Best of all, as research has increased, so has the lifespan of DMD patients. That positive development led to a wonderful problem: The MD CARE Act needed to be updated so DMD research could be expanded to adults, not just children. In 2014, I worked across the aisle to author and pass updates to the original MD CARE Act. We empowered scientists to expand muscular dystrophy research beyond pediatric cases, allowing them to tackle the most pressing needs they identified.

In the past 25 years, these legislative and research efforts have shone a spotlight on DMD and other rare diseases, and I am encouraged by the successes we have seen so far. That young boy I learned about in 2001 is now nearly 30 years old. He has attended college. As we enter the next quarter century, I will continue championing the cause of patients like him, eager to see what other breakthroughs we can achieve on their behalf.