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

The Muscular Dystrophy Association, Parent Project Muscular Dystrophy and the Foundation to Eradicate Duchenne have 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. We 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. 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."

-- MDA President and Chief Executive Officer Steven M. Derks

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. 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."

-- Parent Project Muscular Dystrophy President and CEO Pat Furlong 

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. 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."

-- Joel Wood, president of the Foundation to Eradicate Duchenne

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. 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."

-- Chris Rosa, Ph.D., living with Becker muscular dystrophy

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.

MDA, PPMD and FED were instrumental in passage of the original act in 2001.

TUCSON, Ariz., Feb. 14, 2013 /PRNewswire-USNewswire

Read more PPMD Blogs

Views: 564


You need to be a member of PPMD Community to add comments!

Join PPMD Community

Need help using this community site? Visit Ning's Help Page.



© 2021   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service