H.R. 5265/S. 2618 -- PAUL D. WELLSTONE MUSCULAR DYSTROPHY
COMMUNITY ASSISTANCE, RESEARCH AND EDUCATION
AMENDMENTS OF 2008

Muscular dystrophy (MD) is characterized by progressive weakness and loss of voluntary muscles that control movement. MD affects hundreds of thousands of children and adults throughout the United States and worldwide. Of the nine types of MD, Duchenne muscular dystrophy is the most common lethal genetic disorder of children worldwide, affecting 1 in every 3,500 boys.

Congress enacted the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 (MD-CARE Act) to coordinate and focus federal research on muscular dystrophy, to develop therapies, and to develop epidemiologic data. The House of Representatives approved the MD-CARE Act without opposition; the Senate approved the legislation by unanimous consent; and President George W. Bush signed the bill into law (Public Law 107-84) on December 18, 2001.

The 2001 MD CARE Act has provided critical authority and direction for MD research:

  • Six cooperative MD research Centers of Excellence were established by the National Institutes of Health (NIH). The centers work individually and collaboratively. Each has both basic and clinical research projects, and one or more core facilities to support them. Centers must also make core resources or services available to the national muscular dystrophy research community. The Muscular Dystrophy Association (MDA) provided $1.5 million to each of the first three centers, to ensure that their critical research could begin as quickly as possible.

  • The public-private Muscular Dystrophy Coordinating Committee, established under the bill, has worked to expand, intensify, and coordinate research activities related to muscular dystrophy.

  • The MD STARnet (the Muscular Dystrophy Surveillance Tracking and Research Network) is a data collection and surveillance mechanism aimed at epidemiological research and is overseen by the Centers for Disease Control (CDC).

To build upon the progress made by the MD-CARE Act, reauthorization bills were introduced on February 8, 2008 by Congressmen Eliot Engel (D-NY) and Michael Burgess (R-TX) [H.R. 5265] and by Senators Amy Klobuchar (D-MN) and Johnny Isakson (R-GA) [S. 2618]. These reauthorization bills would:

  • Expand the number of NIH institutes involved to include the National Heart, Lung, Blood Institute.

  • Officially name the Centers of Excellence, the Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers.

  • Emphasize the need for an enhanced clinical and translational research infrastructure, as promising therapies emerge.

  • Ensure MD STARnet data is regularly updated to reflect patients’ changing conditions over time and includes tracking of health outcomes. CDC is required to report annually to Congress on the status of data collection.

  • Direct the CDC to disseminate care considerations for the muscular dystrophies.

  • Direct the Agency for Healthcare Research and Quality (AHRQ) to work with medical and patient organizations to develop standards of care for Duchenne and Becker muscular dystrophy, and to replicate the methods used in developing these care considerations to include other muscular dystrophies.