Duchenne Muscular Dystrophy (DMD)

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.

DMD Research: New Funding for Imaging Studies

The use of magnetic resonance imaging (MRI) as an assessment tool in boys with Duchenne muscular dystrophy (DMD) is being studied by former MDA grantee Krista Vandenborne, through a $7.5 million grant from the National Institutes of Health (NIH).

The goal of the study is to assess whether MRI technology can be used as a precise, noninvasive measure of muscle tissue, gauging both disease progression and the effectiveness of therapies tested in children with DMD.

Texas Achiever with DMD Turns 50

January 17 was a particularly special day for Tom Mecke. It was his 50th birthday, and he had beaten a pretty long set of odds to get there.

Mecke, a San Antonio, Texas, resident all his life, has Duchenne muscular dystrophy (DMD). He and his parents got the news that he has the disease when he was 6 years old. They also were told he’d probably die before he turned 20.

Caution: Immune Response Seen in DMD Gene Therapy

Unwanted responses by the immune system to dystrophin have been seen in a small, MDA-supported clinical trial of gene therapy for Duchenne muscular dystrophy (DMD)— an unexpected finding, investigators say.

Rather than a setback, the finding is “the beginning of a new way of thinking” about gene therapy, said Jerry Mendell, director of the Center for Gene Therapy at Nationwide Children's and a long-time MDA research grantee and MDA-associated clinician. Mendell was the neurologist on this trial.

DMD/BMD: Taking a Closer Look at Ataluren

PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, has announced findings that reflect the company's closer look at a large-scale trial of its experimental drug ataluren.

The additional results, presented April 16 at the American Academy of Neurology  meeting in Toronto, show that trial participants who took the lower dose of ataluren did better on a six-minute walking test than did participants who took the placebo or higher dose.

Luring Away Myostatin Can Boost Muscle Size

ACE031, a laboratory-modified protein developed by Acceleron Pharma of Cambridge, Mass., has shown promise as a therapy to increase muscle mass, based on results of a trial in healthy volunteers. The company will now test it in Duchenne muscular dystrophy (DMD).

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