The National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) funds the University of Massachusetts Medical School Senator Paul D, Wellstone Muscular Dystrophy Cooperative Research Center Cooperative Center for Facioscapulohumeral Muscular Dystrophy (FSHD) Research. The type of funding mechanism utilized to fund the center is known as a U54 or a NIH Cooperative Agreement Specialized Centers (U54) funding mechanism. This differs from many NIH funding mechanisms is that NIH program and scientific staff are actively involved with ongoing activities and research in the center.
"The NICHD was initially established to investigate the broad aspects of human development as a means of understanding developmental disabilities, including mental retardation, and the events that occur during pregnancy. Today, the Institute conducts and supports research on all stages of human development, from preconception to adulthood, to better understand the health of children, adults, families, and communities.
The mission of the NICHD is to ensure that every person is born healthy and wanted, that women suffer no harmful effects from reproductive processes, and that all children have the chance to achieve their full potential for healthy and productive lives, free from disease or disability, and to ensure the health, productivity, independence, and well-being of all people through optimal rehabilitation."
"The NICHD has made revolutionary progress toward achieving its goals. Since the Institute was founded:
"The MD-CARE Act authorized the U.S. National Institutes of Health (NIH) to establish centers of excellence for muscular dystrophy research, and NIH currently funds six Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers" at the University of North Carolina, the University of Rochester, the University of Massachusetts Medical School, the University of Iowa, Children's National Medical Center, Washington DC, and the University of Pennsylvania/Johns Hopkins University. "These Centers are supported by cooperative agreements with three of the NIH institutes; the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Child Health & Human Development and the National Institute of Neurological Disorders and Stroke. The Wellstone Centers promote side-by-side basic, translational, and clinical research and provide resources that can be used by the national muscular dystrophy research community. They also serve as focal points for research collaborations, training and communication. Collectively the centers are engaged in research on Duchenne/Becker, myotonic dystrophy, facioscapulohumeral, limb-girdle, congenital and other forms of muscular dystrophy. They are exploring promising approaches for treatment of these diseases including gene, stem cell, pharmacological and molecular therapies. The Wellstone External Advisory Committee provides guidance to the NIH on issues involving the centers."
"The Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 ("MD-CARE Act"; P.L. 107-84) authorized the establishment of the Muscular Dystrophy Coordinating Committee (MDCC) to coordinate activities across NIH and with other Federal health programs and activities relevant to the various forms of muscular dystrophy. The MD-CARE Act directed the Committee to develop a plan for conducting and supporting research and education on muscular dystrophy through the national research institutes, and to submit this plan to Congress within the first year of the establishment of the MDCC. The MDCC has conducted two stages of planning. The first stage led to the Muscular Dystrophy Research and Education Plan for NIH, which was submitted to Congress in August 2004. This formed the basis for a subsequent, more intensive planning process that produced the MDCC Action Plan for the Muscular Dystrophies (approved by the MDCC in December 2005). The Action Plan contains specific research objectives that are appropriate to the missions of all MDCC member agencies and organizations and thus serves as a central focus for coordination of research in muscular dystrophy."
The Muscular Dystrophy Coordinating Committee (Committee) shall coordinate research activities across the National Institutes of Health (NIH) and with other Federal health programs and activities relating to the various forms of muscular dystrophy, including Duchenne, myotonic, facioscapulohumeral muscular dystrophy and other forms of muscular dystrophy.
42 U.S.C. 283g, section 404E of the Public Health Service Act, as amended. The Committee is governed by the provisions of the Federal Advisory Committee Act, as amended (5 U.S.C Appendix 2), which sets forth standards for the formation and use of advisory committees.
The Committee shall develop a plan for conducting and supporting research and education on muscular dystrophy through the national research institutes, and shall periodically review and revise the plan. The plan shall (a) provide for a broad range of research and education activities relating to biomedical, epidemiological, psychosocial, and rehabilitative issues, including studies of the impact of these diseases in rural and underserved communities; (b) identify priorities among the programs and activities of the National Institutes of Health regarding these diseases; and (c) reflect input from a broad range of scientists, patients, and advocacy groups.
The Committee shall consist of not more than 15 members, including the Chair, appointed by the Secretary of Health and Human Services (Secretary). Two-thirds of the members shall represent governmental agencies, including the directors or their designees of each of the national research institutes involved in research with respect to muscular dystrophy and representatives of all other Federal departments and agencies whose programs involve health functions or responsibilities relevant to these diseases, including the Centers for Disease Control and Prevention, the Health Resources and Services Administration, the Food and Drug Administration, and representatives of other governmental agencies that serve children with muscular dystrophy, such as the Department of Education. One-third of the members shall be members of the public, including a broad cross section of persons affected with muscular dystrophies, including parents or legal guardians, affected individuals, researchers, and clinicians. None of these members serve as Representatives. A quorum for the conduct of business by the full Committee shall consist of a majority of currently appointed members. A quorum for each subcommittee shall be three members.
Public members shall be invited to serve for a term of three years, and may serve for an unlimited number of terms if reappointed. Terms of more than two years are contingent upon the renewal of the Committee by appropriate action prior to its termination. Members may serve after the expiration of their terms until their successors have taken office.
The Chair of the Committee shall serve as the principal advisor to the Secretary, the Assistant Secretary for Health, and the Director of NIH, and shall provide advice to the Director of the Centers for Disease Control and Prevention, the Commissioner of the Food and Drug Administration, and to the heads of other relevant agencies. The Committee shall select the Chair for a term not to exceed two years. The Chair of the Committee shall be appointed by, and be directly responsible to, the Secretary.
As necessary, subcommittees may be established to perform functions within the Committee's jurisdiction. The advice/recommendations of a subcommittee must be deliberated by the parent advisory committee. A subcommittee may not report directly to a Federal official unless there is statutory authority to do so.
Subcommittee membership may be drawn in whole or in part from the parent advisory committee. All subcommittee members may vote on subcommittee actions and all subcommittee members count towards the quorum for a subcommittee meeting. Ad hoc consultants do not count towards the quorum and may not vote. Subcommittee members who are not members of the parent committee may attend closed sessions of the parent committee meeting but they may not count towards the quorum of the parent committee and they cannot vote on committee actions.
The Department Committee Management Officer will be notified upon establishment of each standing subcommittee and will be provided information on its name, membership, function, and estimated frequency of meetings.
The Committee may call upon special consultants, assemble ad hoc working groups, and convene conferences and workshops as necessary to assist in the work of the Committee.
Administrative support shall be provided by the Department of Health and Human Services (Department).
The Committee shall meet as appropriate as determined by the Secretary in consultation with the Chair. All meetings will be held at the call of the Chair with the advance approval of a Government official, who shall also approve the agenda. A Government official shall be present at all meetings.
Meetings shall be open to the public, except as determined otherwise by the Secretary or other designated official to whom the authority has been delegated; notice of all meetings shall be given to the public.
Meetings shall be conducted and records of the proceedings kept, as required by applicable laws and Departmental regulations."
The Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 (the MD-CARE Act, Public Law 107-84) specified a number of provisions for expanding and intensifying research on muscular dystrophy. One important provision of the Act was the establishment of the Muscular Dystrophy Coordinating Committee (MDCC), an interagency committee consisting of representatives from federal agencies with an interest in muscular dystrophy as well as members of the public including patient advocates and representatives from patient voluntary groups. Since its formation, the MDCC has met four times and has been instrumental in coordinating and facilitating muscular dystrophy research activities. Most importantly, the MDCC, with input from scientific experts in the field, developed the Muscular Dystrophy Research and Education Plan for NIH, and recently revised this Plan and created the more comprehensive Action Plan for the Muscular Dystrophies. This Action Plan identifies research objectives that should be considered by the entire muscular dystrophy community to help achieve the effective detection, diagnosis, treatment and prevention of all types of muscular dystrophy.
Action Plan for the Muscular Dystrophies (January 2006)
http://www.ninds.nih.gov/about_ninds/groups/mdcc/MDCC_Action_Plan.doc [MS Word]
The Department of Health and Human Services (HHS) has also been actively implementing the provisions of the MD-CARE Act. The National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC) have initiated and expanded a number of activities to advance basic, translational, and clinical research on the muscular dystrophies and to address issues related to epidemiology, diagnosis, and screening for muscular dystrophy.
The MD-CARE Act requires that the MDCC biennially submit a report to Congress describing these HHS activities and that the report also include any revisions to the Research and Education Plan. This report is submitted in response to this request.
Report on Implementation of the MD-CARE Act: July 2007
Report on Implementation of the MD-CARE Act: May 2006
Annual Report on Implementation of the MD-CARE Act: March 2005