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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."
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.