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Advocates in Muscular Dystrophy Community Press Congress for Fast Action on Muscle Disease Research and Treatment

TUCSON, Ariz., Feb. 14, 2013 — The Muscular Dystrophy Association, Parent Project Muscular Dystrophy and the Foundation to Eradicate Duchenne today called on the U. S. Senate and the U. S. House of Representatives to reauthorize the MD CARE Act of 2001, and to continue federal support for the accelerated pace of research and treatment development for muscular dystrophy. The nonprofits also are urging the public to contact their U.S. Senators and ask them to vote for reauthorization of The Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education (MD CARE) Amendments of 2013. The Senate introduced the bill last night (S. 315), and the House introduced the bill (H.R. 594) Feb. 8.

Since 2001, federal funding and coordination of muscular dystrophy research has dramatically enhanced discovery and development of potential treatments for the muscular dystrophies, including: Duchenne and Becker muscular dystrophies (DMD/BMD, or also known as DBMD); congenital muscular dystrophy (CMD); facioscapulohumeral muscular dystrophy (FSHD); limb-girdle muscular dystrophy (LGMD); and myotonic muscular dystrophy (MMD).

"Progress in muscle disease research since the MD CARE Act was passed has been extraordinary," said MDA President and Chief Executive Officer Steven M. Derks. "Sixty-seven clinical trials for drugs or therapies have been conducted since 2001, with 37 clinical trials currently under way. Additionally, new clinical care guidelines will be valuable tools to improve standards of care for those living with muscle diseases. We are counting on the unparalleled strength of our nationwide muscular dystrophy community to ensure that Congress understands the importance of this reauthorization. We must build on the success of the MD CARE Act and deliver effective treatments to those affected."

Senator Roger Wicker of Mississippi, author of the MD CARE Act of 2001, and Senator Amy Klobuchar of Minnesota, sponsor of the 2008 reauthorization, are lead Senate sponsors of the bill. In the House, Eliot Engel of New York and Dr. Michael Burgess of Texas are leading the effort for reauthorization of the MD CARE Act as they did in 2008.

"In 2001, as we celebrated the signing of the MD CARE Act, we could not imagine its impact," said Parent Project Muscular Dystrophy President and CEO Pat Furlong. "Over the years, we have seen scientific breakthroughs across the muscular dystrophies, which have led to the expansion and intensification of muscular dystrophy research, including the leveraging of significant non-federal sources of funding. Today, people with muscular dystrophy are living longer, more clinical trials are in progress, and the hope of treatments is palpable within our community."

As a result of the accelerated pace of discovery and development of therapeutic treatments, more young people with muscle disease are living longer and making the transition into adulthood. This year’s Amendment addresses that progress by requiring studies to develop optimal clinical care interventions for young adults with Duchenne and Becker muscular dystrophies (DMD/BMD).

"We are extremely grateful to members of Congress that have recognized the vital importance of this legislation to advance Duchenne muscular dystrophy research and produce real therapeutic approaches that are extending the lives of young men living with the devastating impact of Duchenne muscular dystrophy every day," said Joel Wood, president of the Foundation to Eradicate Duchenne. "This legislation and the research and therapeutics it produces are bringing us closer to our goal of finding a cure for this generation of Duchenne men."

The proposed legislation also mandates studies to demonstrate the cost effectiveness of providing independent living resources and support services for young adults with all forms of muscular dystrophy.

"When I was first diagnosed with Becker muscular dystrophy 36 years ago, I’m not sure that anyone would have envisioned I would be a 44-year-old man with a passion for the Mets, Bruce Springsteen and serving as the University Dean for Student Affairs at City University of New York," said Chris Rosa, Ph.D. "I’ve had to overcome many obstacles along the way, and all too often the resources I needed to overcome those obstacles did not exist. It’s very encouraging to see this included in the Amendment so that we can create smarter policies and clear away some of the hurdles."

The 2013 MD CARE Amendment also:

  • directs the Muscular Dystrophy Coordinating Committee to consider a plan to expedite approval of emerging therapies and personalized medicines with the potential to treat people with muscular dystrophy;
  • expands areas of research focus within the NIH-funded Paul D. Wellstone Muscular Dystrophy Centers of Excellence to include heart and lung function;
  • directs the CDC to develop and disseminate care considerations for adults with Duchenne and Becker muscular dystrophies (DMD/BMD, or DBMD); and
  • directs the CDC to develop and disseminate acute care considerations for adults with all muscular dystrophies.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy — our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, D.C., and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do — and everything we have done since our founding in 1994 — helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to ParentProjectMD.org for more information, or to learn how you can support our efforts and help families affected by Duchenne.

About the Foundation to Eradicate Duchenne

The Foundation to Eradicate Duchenne is a 501c(3) organization established in 2002 with the goal of finding treatments and an ultimate cure for Duchenne muscular dystrophy, the world’s leading lethal childhood genetic disease.

The Foundation to Eradicate Duchenne was established by Dana and Joel Wood of Alexandria, Va. Their son James Wood, now 15, was diagnosed in May 2000 with Duchenne muscular dystrophy. The Woods are both lobbyists in Washington, D.C., and have devoted much of their time and energies to this cause, working with others to attain millions of dollars in federal earmarks for Duchenne muscular dystrophy research and a significant increase in the attention devoted to DMD at the National Institutes of Health. Additionally, through the FED and other fundraising efforts, they have raised approximately $10 million in private donations since James was diagnosed.

About MDA

MDA is the nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.

In addition to funding more than 250 research projects worldwide, MDA maintains a national network of 200 medical clinics; facilitates hundreds of support groups for families affected by neuromuscular diseases; and provides local summer camp opportunities for thousands of youngsters living with progressive muscle diseases.

For more information, visit mda.org and follow MDA on Facebook (facebook.com/MDAorg) and Twitter (@MDAorg).

Roxan Olivas
MDA — Vice President Public Relations
(520) 529-5317
Will Nolan
Dir. Communications, PPMD
(201) 250-8440
Bill Quirk
Vice President, FED
(202) 471-4803