As the Wood and Heil families have long been involved in political life in Washington, they are devoted to securing a commitment toward this disease commensurate with the toll of Duchenne Muscular Dystrophy compared to other afflictions. To this end, they are working cooperatively with the Parent Project Muscular Dystrophy, which is the only DMD grassroots organization that is working tirelessly on behalf of Duchenne Muscular Dystrophy sufferers in the halls of Congress, and in the institutions of government. Until the PPMD began its campaign of professional advocacy in 2000, there had never before been any significant effort expended on behalf of this disease group in Washington, despite the fact that the federal government is the number-one funder of basic scientific research in the world.
Unfortunately, the track record of the National Institutes of Health in funding Duchenne Muscular Dystrophy research is an unfortunately accurate reflection of that legacy of historic neglect – less than 1/2000th of the NIH budget being spent on research for the world’s number-one genetic child killer. Even to this day, none of the institutes of NIH have ever spent dollars to support translational, clinical research for Duchenne Muscular Dystrophy, or to support the infrastructure of a clinical trials network. (The Cooperative International Neuromuscular Research Group receives federal earmarks achieved through the Foundation to Eradicate Ducenne effort are administered through the Department of Defense’s Health Programs.)
Nonetheless, the Parent Project Muscular Dystrophy has made very significant strides toward improving this situation. The PPMD-authored “Muscular Dystrophy CARE Act” was the first comprehensive, disease-specific authorization bill signed into law (in December 2001) by President Bush. The Act forced NIH to erect a number of programs, including the development of multi-institution “Centers of Excellence” to ratchet up Muscular Dystrophy research. While we and the PPMD remain disappointed at the pace of implementation of the MD CARE Act, there is little doubt that it has had a galvanizing influence on the bureaucracy of NIH, which previously felt little if any pressure to expand the scope and nature of Duchenne Muscular Dystrophy research.
Along the way and as a result, the Duchenne Muscular Dystrophy community has built a significant network of champions in Congress: members of the House such as Rep. Denny Rehberg of Montana, and members of the Senate such as Sen. Thad Cochran of Mississippi, Sen. Roger Wicker of Mississippi and the late Sen. Paul Wellstone of Minnesota, who was a passionate and effective backer of the cause.
The Foundation to Eradicate Duchenne encourages every American family touched by Duchenne Muscular Dystrophy to join the Parent Project Muscular Dystrophy advocacy network – to participate in PPMD legislative conferences, to constructively build relationships with their home-state members of Congress, and to constantly be “on call” for opportunities to impact federal funding for Duchenne Muscular Dystrophy. Additionally, the PPMD has a portfolio of aggressive translational research (led by Dr. Lee Sweeney of the University of Pennsylvania) which we endorse.
Duchenne Muscular Dystrophy advocacy efforts grew in 2003 when the Children’s Research Institute at Children’s National Medical Center began devoting resources to lobbying Congress on these critical muscle-related research initiatives – a move supported and subsidized by the Foundation to Eradicate Duchenne.