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MDA

About the MDA

MDA is the largest nongovernmental sponsor of neuromuscular disease research. Neuromuscular diseases affect more than 1 million Americans. About 250,000 have some form of muscular dystrophy.

Included in the more than 40 neuromuscular diseases covered by MDA are nine forms of muscular dystrophy, amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), myasthenia gravis, spinal muscular atrophy, Charcot-Marie-Tooth disease and others.

MDA is the first nonprofit agency to be recognized by the American Medical Association with a Lifetime Achievement Award for "significant and lasting contributions to the health and welfare of humanity." MDA was founded in 1950 and awarded its first research grant to Dr. Ade Milhorat, a pioneer of modern muscular dystrophy research.

  • Financial

    MDA is funded almost entirely by individual, private contributions. MDA seeks no fees from those it serves. MDA dedicates 77 cents of every dollar it spends directly to services.

  • Services

    MDA sent more than 4,000 kids with neuromuscular diseases to MDA summer camps in 2005, at a cost to MDA of $600 per camper. There’s no charge to campers’ families.

    MDA In the St. Louis metropolitan area, camp is held each June at the Babler State Park for more than 65 children.

    Tens of thousands of people affected by neuromuscular diseases visit MDA’s 235 hospital-affiliated clinics and 37 ALS centers/MDA every year.

    In the St. Louis area, more than 2000 children and adults are seen, free-of-charge, at MDA clinics at the Washington University Medical Center (Barnes-Jewish Hospital and St. Louis Children’s Hospital).

    The Association also:

  • Helps buy and repair wheelchairs, leg braces and augmentative communication devices;

    MDA

  • Facilitates meetings of some 290 MDA support groups;
  • Provides thousands of free flu vaccines to those affected by neuromuscular diseases;
  • Conducts an ongoing public education program through its Web sites, publications, videos, seminars and media placements; and

  • Sponsors professional education programs for MDA clinicians.

    Research

  • MDA awards research grants to some 400 teams of scientists and physicians worldwide. This year, MDA has allocated $34.3 million for research, funding some 350 projects worldwide including many projects at the Washington University School of Medicine and the St. Louis University School of Medicine.

    MDA-funded scientists are making progress in decoding disease mechanisms and in identifying and testing promising treatments. In April, the U.S. Food and Drug Administration approved Myozyme, a replacement enzyme for people with Pompe’s disease (acid maltase deficiency), which was tested in MDA clinics and developed by the biopharmaceutical industry with the help of MDA-supported research. The treatment prolongs the lives of children born with the disease.

    Since last year’s Telethon, researchers also have:

  • With a biotechnology company, launched an MDA-funded human gene therapy trial in children with Duchenne muscular dystrophy, a disorder that proves fatal in young adulthood

  • Demonstrated that systemically delivered genes for the muscle protein dystrophin, missing in children with Duchenne MD, can reach all muscles and improve life span and health in mice lacking dystrophin and another protein

  • Restored significant production of missing dystrophin in mice with a disease resembling Duchenne muscular dystrophy, by administering weekly intravenous injections of “antisense” molecules that cause existing genetic information to be processed in a new way

  • Identified variations in PON genes, which normally allow the body to detoxify pesticides and other poisons, that occur more often in people with the deadly adult disease amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease)

  • Begun comparing DNA from 1,000 people with amyotrophic lateral sclerosis with DNA from 1,000 unaffected subjects to search all genes for differences that may contribute to ALS

  • Developed strategies to cause cells lacking SMN, the protein needed by people with spinal muscular atrophy, to make the protein

  • Identified an overabundance of a protein called FRG1 as a likely factor in facioscapulohumeral muscular dystrophy
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