Muscular Dystrophies

Immune Response Must Be Considered in DMD Gene Therapy

Editor's note: This story was updated April 27, 2012.

Immune-system rejection of newly synthesized dystrophin protein occurred in at least some of the boys with Duchenne muscular dystrophy (DMD) who participated in a safety trial of dystrophin gene therapy.

The finding was a partial surprise to researchers and demonstrates the value of small, phase 1 clinical trials.

The Future of Antisense: FDA, NIH Talk It Out

This story was updated Oct. 6, 2010.

Researchers, clinicians, pharmaceutical industry executives, and representatives from advocacy groups, including MDA, met in Washington Sept. 27-28, 2010, to discuss moving forward with antisense-based therapies for neuromuscular disease.

NIH Continues Funding for MD Research

The U.S. National Institutes of Health (NIH) announced Sept. 29, 2010, that it will allocate more than $4.5 million for the first year of a five-year commitment to explore new treatment strategies for various forms of muscular dystrophy.

Support will go to three U.S. institutions: Nationwide Children's Hospital in Columbus, Ohio; the University of Pennsylvania in Philadelphia; and the University of Iowa in Iowa City.

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

Duchenne muscular dystrophy

Advocacy a Focus for New Ms. Wheelchair America with LGMD

McArthur was crowned Ms. Wheelchair American at the pageant in Grand Rapids, Mich., this August. (Glen Lipton Photography)

Deshae Lott Scholarships Awarded

Three accomplished scholars determinedly pursuing their educations while living with neuromuscular disease are the first winners of the CMMS Deshae Lott Ministries outreach program scholarships.

“We were so impressed,” with the winning candidates, said Deshae Lott, 39, a teacher and minster from Bossier City, La., who founded the nonprofit that bears her name. “These three show great strength of mind and character. Each of them strives to maximize within their limitations.”

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.

Michigan Man with CMD Vies for US Rowing Team Spot

Come the 2012 Olympics in London, Michael Lehmann stands a reasonable chance of representing the United States as a vital member of the U.S. rowing team.

Lehmann got close to making the team this year as a coxswain (“KOK-sun”), taking second place in individual tryouts. His biggest handicaps were his age (23) and level of experience (five years) – not his congenital muscular dystrophy (CMD).

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