WASHINGTON, Feb. 25, 2014 — At a Congressional hearing today in Washington, the Muscular Dystrophy Association’s (MDA) chief executive briefed legislators on the enormous impact of the MD-CARE Act passed in 2001 and implored lawmakers to reauthorize this crucial legislation on behalf of the 100,000 Americans with muscular dystrophies.
MDA co-hosted the briefing with Parent Project Muscular Dystrophy (PPMD) and the Foundation to Eradicate Duchenne (FED), nonprofit groups that fight certain muscular dystrophies. Each group also spoke in support of the reauthorization at the briefing today.
"The passage of the MD-CARE Act in 2001 was a game-changer for the nation's muscular dystrophy community," said Steven M. Derks, MDA president and chief executive officer. "A premier example of private/public partnership, this landmark legislation leveraged the passion of our heroic Congressional champions behind our well-organized and long-established national clinical and research infrastructures and has yielded life-altering research breakthroughs.”
The original law, called the Muscular Dystrophy Community Assistance, Research and Education Amendments, established university-based centers of excellence in muscular dystrophy research and set up the Muscular Dystrophy Coordinating Committee (MDCC), an advisory board to help the National Institutes of Health (NIH) work with other federal health agencies on MD research. The MDCC comprises experts in the field as well as patient advocates.
The law also aims to standardize clinical care for muscular dystrophy throughout the U.S. by mandating the development of care standards for each form of the disorder. The goal is to ensure that every MD patient receives optimal care whether that care is delivered in a major medical center or a small community practice.
Among the strides that this landmark bill has yielded are therapeutic advances in three major forms of MD — Duchenne, myotonic and facioscapulohumeral (FSH). Today’s Hill briefing highlighted progress in these three MD types from the vantage point of a world-renowned clinician-researcher and a family affected by each disease.
Derks argued that while the muscular dystrophy community has moved the needle in clinical care and therapy development, the urgency of our community is no less dire than it was 13 years ago.
“We have made great strides, but the muscular dystrophies continue to be among the nation's most devastating health conditions,” he said. “Today, on behalf of the infants, children, teens, adults and their families we serve, I implore you to keep the life-changing momentum of the MD-CARE Act moving forward by reauthorizing this lifesaving legislation."
The one-hour briefing started at noon in room 106 of the Dirksen Senate Office Building.
In addition to Derks' presentation, it included excerpts from the original congressional hearing on the MD-CARE Act from 2001; comments from U.S. Senators Roger Wicker (R-Miss.) and Amy Klobuchar (D-Minn.); comments from Pat Furlong, president and CEO of Parent Project Muscular Dystrophy (PPMD); and an overview of the involvement of NIH in muscular dystrophy research given by John Porter, Ph.D., program director at NIH's National Institute of Neurological Disorders and Stroke (NINDS).
A key element of the briefing was a panel discussion with families affected by muscular dystrophy and directors of some of the Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers, the centers of excellence in MD research that were established through the MD-CARE Act.
Panelists representing affected families included Anessa Fehsenfield, a woman from Michigan who has a son with Duchenne MD; Lisa Harvey, from California, who has myotonic dystrophy, as does her daughter; and Lilleen Walters, from Virginia, who is affected by facioscapulohumeral MD, as is her son.
Panelists who direct Wellstone MD centers include Eric Hoffman, Ph.D., from Children's National Medical Center in Washington; Stan Nelson, M.D., from the University of California, Los Angeles (who also has a son with Duchenne); Charles Thornton, M.D., from the University of Rochester Medical Center in New York; and Kathryn Wagner, M.D., Ph.D., Kennedy Krieger Institute/Johns Hopkins University in Baltimore.
MDA today contacted more than 50,000 of its families and supporters and encouraged them to contact their elected officials and thank them for supporting the reauthorization of the MD-CARE. To read more about the MD-CARE Act and how it has "moved the needle" in muscular dystrophy research and treatment, go to Summary of Key Programs Made Possible by the MD-CARE Act Since 2001.
The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement. Visit mda.org and follow us at facebook.com/MDAnational and @MDAnews.