Highlighting a brief history of muscular dystrophy advocacy

I recently penned a column that took a surface look at the history of the disability movement in the United States. As many readers pointed out, my list of world changers and their accomplishments was far from exhaustive.

I’d like to share another small part of the movement that has been particularly significant to the muscular dystrophy (MD) community: the passage of the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 — more commonly known as the MD-CARE Act.

This legislation was drafted chiefly by Roger Wicker, then a member of the U.S. House of Representatives and an early advocate for people living with MD.

The seeds of the MD-Care Act were planted by patient advocacy groups such as Parent Project Muscular Dystrophy and the Muscular Dystrophy Association. It is important to note here that people living with different forms of MD, including Duchenne, Becker, limb-girdle, and others, had a hand in early drafts of this legislation and spoke to members of Congress before it was signed into law.

Recommended Reading

September 18, 2024 Columns by Shalom Lim

Why patient advocacy is important for those of us with Duchenne

Those champions include Pat Furlong, Chris Rosa, Donavon Decker, Jerry Lewis, Lee Sweeney, and Ben Cumbo. The transcript of the congressional hearing is available online for anyone who may wish to read it. The testimonies are moving and still relevant today, especially since more people with MD are leading longer lives.

The MD-Care Act established centers of excellence with the aim of creating the standards of care that we have today. It also created the framework for the Muscular Dystrophy Coordinating Committee (MDCC), a federal advisory committee that includes several institutes of the National Institutes of Health, other federal agencies, and patient advocates. The National Institute of Neurological Disorders and Stroke website explains:

“The MDCC identifies the many challenges confronting people with muscular dystrophy and their families and coordinates efforts to address them. In 2005, the MDCC collected and integrated the recommendations of muscular dystrophy researchers, physicians, patients, family members, and other stakeholders into the Action Plan for the Muscular Dystrophies. The Action Plan was revised and updated in 2015, and outlines priority needs to improve treatments and reduce disease burden for all forms of muscular dystrophy.”

The Action Plan also sheds a light on employment issues, housing challenges, and laws that don’t allow for savings if one is to qualify for programs such as Medicaid. It provides a decent blueprint for the kinds of changes our MD community needs.

Another federal program that aims to benefit people living with MD is currently funded by the Department of Defense through its Congressionally Directed Medical Research Programs. Chiefly focused on Duchenne, the Duchenne Muscular Dystrophy Research Program (DMDRP) considers “a broad range of unanswered research questions that are potentially critical to treating Duchenne patients, improving their quality of life, and developing a cure.” Its efforts are focused on the following areas:

• “Support discovery and development of therapeutics, related devices, and tools”
• “Accelerate promising therapeutic ideas into clinical applications”
• “Advance basic research on the effect of Duchenne on the heart, bone, [central nervous system], and gastrointestinal system”
• “Expand the Duchenne researcher community by supporting early career investigators.”

That last point is important, as the DMDRP provides critical funding to young researchers and pharmaceutical companies that are doing important work to help people living with MD.

In looking at some of the advances from recent decades, it is clear that ordinary people are changing the world. That means all of us can and should be involved. Raise your voice. The future is what we build today.

Note: Muscular Dystrophy News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.