Muscular Dystrophy Community Assistance Research and Education Amendments of 2001
Encyclopedia
The Muscular Dystrophy Community Assistance Research and Education Amendments of 2001 ("MD CARE Act") amended the Public Health Service Act
to provide for research with respect to various forms of muscular dystrophy
, including Duchenne
, Becker
, limb girdle
,
congenital
, facioscapulohumeral
, myotonic, oculopharyngeal
, distal
, and Emery-Dreifuss
muscular dystrophies.
called the Muscular Dystrophy Coordinating Committee (MDCC), which is composed of no more than 15 members to coordinate the activities across the National Institutes and with other Federal health programs and activities relating to the various forms of muscular dystrophy. By law the committee's composition is 2/3s governmental agencies and 1/3 public membership, including a broad cross section of persons affected with muscular dystrophies including parents or legal guardians, affected individuals, researchers, and clinicians. Public members appointed serve for a term of 3 years, and may serve for an unlimited number of terms if reappointed.
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 January 2006 MDCC Action Plan for the Muscular Dystrophies. 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.
Public Health Service Act
The Public Health Service Act is a United States federal law enacted in 1944. The full act is captured under Title 42 of the United States Code "The Public Health and Welfare", Chapter 6A "Public Health Service"....
to provide for research with respect to various forms of muscular dystrophy
Muscular dystrophy
Muscular dystrophy is a group of muscle diseases that weaken the musculoskeletal system and hamper locomotion. Muscular dystrophies are characterized by progressive skeletal muscle weakness, defects in muscle proteins, and the death of muscle cells and tissue.In the 1860s, descriptions of boys who...
, including Duchenne
Duchenne muscular dystrophy
Duchenne muscular dystrophy is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking, breathing, and death. The incidence is 1 in 3,000 boys. Females and males are affected, though females are rarely affected and are more often carriers...
, Becker
Becker's muscular dystrophy
Becker muscular dystrophy is an X-linked recessive inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis....
, limb girdle
Limb-girdle muscular dystrophy
Limb-girdle muscular dystrophy or Erb's muscular dystrophy is an autosomal class of muscular dystrophy that is similar but distinct from Duchenne muscular dystrophy and Becker's muscular dystrophy...
,
congenital
Congenital muscular dystrophy
Congenital muscular dystrophy is the term used to describe muscular dystrophy that is present at birth. CMD describes a number of autosomal recessive diseases of muscle weakness and possible joint deformities, present at birth and slowly progressing...
, facioscapulohumeral
Facioscapulohumeral muscular dystrophy
Facioscapulohumeral muscular dystrophy , which is also known as Landouzy-Dejerine, is a usually autosomal dominant inherited form of muscular dystrophy that initially affects the skeletal muscles of the face , scapula and upper arms...
, myotonic, oculopharyngeal
Oculopharyngeal muscular dystrophy
Oculopharyngeal muscular dystrophy is an autosomal dominant neuromuscular disease which appears in early middle age . OPMD is an example of a trinucleotide repeat disorder caused by expanding 10 to 11-17 at the 5' end of the coding region for PABPN1...
, distal
Distal muscular dystrophy
Distal muscular dystrophy is a group of disorders characterized by onset in the hands or feet.Many types involve dysferlin, but it has been suggested that not all cases do.Types include:...
, and Emery-Dreifuss
Emery-Dreifuss muscular dystrophy
Emery–Dreifuss muscular dystrophy is a condition that chiefly affects muscles used for movement and heart muscle.It is named after Alan Eglin H. Emery and Fritz E. Dreifuss.-Presentation:...
muscular dystrophies.
MDCC
The act established a Federal Advisory CommitteeFederal Advisory Committee Act
The Federal Advisory Committee Act is a United States federal law , which governs the behavior of federal advisory committees. There are now approximately 1,000 such committees...
called the Muscular Dystrophy Coordinating Committee (MDCC), which is composed of no more than 15 members to coordinate the activities across the National Institutes and with other Federal health programs and activities relating to the various forms of muscular dystrophy. By law the committee's composition is 2/3s governmental agencies and 1/3 public membership, including a broad cross section of persons affected with muscular dystrophies including parents or legal guardians, affected individuals, researchers, and clinicians. Public members appointed serve for a term of 3 years, and may serve for an unlimited number of terms if reappointed.
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 January 2006 MDCC Action Plan for the Muscular Dystrophies. 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.