Duchenne Muscular Dystrophy (DMD)

Research Briefs: DMD, FA, DM, PM, IBM, MG, LEMS

Duchenne muscular dystrophy

Race, Cardiomyopathy Shorten Life Span in MD

A new study reported by the Centers for Disease Control and Prevention (CDC) shows that survival time has significantly increased for certain categories of people with muscular dystrophy (MD) but that race and cardiac status have a large impact on survival.

DMD Clinical Research Network Studying Dystrophin-Deficient Heart

Decoy Receptor Lures Myostatin, Helps Muscles

Luring away myostatin, and possibly other proteins that inhibit muscle growth, using a gene for a "decoy receptor" increased muscle size and strength in mice with a muscle disease resembling human Duchenne muscular dystrophy (DMD), a new study has found.

Nationwide Children's Hospital Named MD Research Center

The National Institutes of Health (NIH) has awarded $7 million to Nationwide Children's Hospital in Columbus, Ohio, and designated the institution a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (Wellstone MDCRC).

Research Briefs: ALS, BMD, DMD, MMD, SMA, Muscle Regeneration

Amytrophic lateral sclerosis (ALS)

MDA Awards More Than $14 Million in Research Grants

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 applicant, the scientific merit of the project, and the proposal's relevance to developing treatments for the disease. The effective start date for all grants was July 1, 2010.

First US Exon-Skipping Trial Opens

Update (Nov. 5, 2012): Results announced in November 2012 showed the drug, now called drisapersen, reached blood levels approximately proportional to the injected dose at two of the three dosage levels tested and was not associated with serious adverse events. See Drisapersen Appears Safe in Non-Walking Boys With DMD.

DMD Trial: AVI4658 Increased Dystrophin Production

The biopharmaceutical company AVI BioPharma has announced additional encouraging results from its clinical trial of AVI4658, an experimental treatment for Duchenne muscular dystrophy (DMD).

The new results show that, at higher doses, AVI4658 can result in substantial production of the needed dystrophin protein in muscle fibers.

The company has not yet released results of any tests of muscle function in the 19 children in this trial.

FA Research: Idebenone Strikes Out Again

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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