Metabolic Diseases of the Muscle

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"Lumizyme has stopped the progression of my disease," says one of first to receive the drug since the FDA granted commercial approval

posted on June 21, 2010 - 4:25pm
 Monique Griffin of Orlando, Fla., was one of the first in the nation to receive the commercially available treatment Lumizyme for acid maltase deficiency (AMD, or Pompe disease).
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A new study seeks to investigate the possible relationship between religion/spirituality and disability acceptance

posted on November 10, 2010 - 5:01pm
With approximately 54 million Americans living with disabilities, exploring and understanding factors that might facilitate or hinder acceptance of one’s disability may be an important area of research. But what exactly is the relationship between religious/spiritual attitudes and acceptance or lack of acceptance of disability for people with neuromuscular disorders?
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MDA has awarded 38 grants totaling more than $12 million to support research into general muscle health and more than 15 neuromuscular diseases in its program

posted on February 1, 2012 - 10:00am
The Muscular Dystrophy Association has awarded 38 new grants totaling more than $12 million to fund research projects focused on its continuing mission to uncover the causes of, and develop therapies for, the more than 40 neuromuscular diseases in its program. MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical...
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Another trial of idebenone shows that, although safe and well tolerated, the substance doesn't lead to significant improvement in Friedreich's ataxia

posted on May 28, 2010 - 12:09pm
Idebenone, a substance believed to act as an antioxidant and to aid in energy production in cellular structures called mitochondria, has failed to benefit people with Friedreich's ataxia (FA) in a phase 3 trial conducted at several European centers. The drug is similar to coenzyme Q10, a naturally occurring molecule.
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Thirty-eight new grants support research in ALS, central core disease, spinal muscular atrophy, muscular dystrophy, and other diseases in MDA's program   

posted on August 17, 2010 - 6:39pm
MDA has awarded 38 new research grants totaling more than $14 million and covering more than a dozen neuromuscular diseases.  MDA's Board of Directors met in Los Angeles July 16, where it reviewed and approved the new grants based on recommendations from the MDA Scientific and Medical Advisory Committees. Grants were scored and recommended for approval based on the capabilities of the...
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News about research in Charcot-Marie-Tooth disease, congenital myasthenic syndromes, Duchenne/Becker MDs, Friedreich's ataxia, Pompe disease and spinal-bulbar muscular atrophy

posted on February 10, 2011 - 11:09am
Charcot-Marie-Tooth disease
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MDA has awarded 44 new grants aimed at uncovering the underlying molecular causes of, and developing therapies for, many of the diseases in its program

posted on February 4, 2011 - 1:22pm
The Muscular Dystrophy Association has awarded 44 grants totaling $13.5 million to support research efforts aimed at advancing understanding of disease processes and uncovering new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association’s program. The new grants were reviewed by MDA’s Scientific and Medical Advisory Committees, and approved...
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