Duchenne Muscular Dystrophy (DMD)

DMD — Eric Hoffman, Ph.D.

Eric Hoffman, director of the Research Center for Genetic Medicine at Children's National Medical Center in Washington, D.C., was awarded an MDA research grant totaling $321,659 over a period of three years to study whether a process called asynchronous repair contributes to muscle degeneration in Duchenne muscular dystrophy (DMD).

DMD — Kay Davies, M.A., Ph.D.

Dame Kay Davies, Dr. Lee’s Professor of Anatomy in the department of physiology, anatomy and genetics at the University of Oxford in the United Kingdom, was awarded an MDA research grant totaling $207,566 over a period of two years to conduct a drug screen for molecules that can increase levels of a protein called utrophin, which can be developed for clinical trials in Duchenne muscular dystrophy (DMD).

DMD, General MD — Radbod Darabi, M.D., Ph.D.

Radbod Darabi, assistant professor of medicine at the University of Minnesota in Minneapolis, was awarded an MDA research grant totaling $380,049 over a period of three years to develop methods to use cells derived from skin to regenerate muscle tissue in a model of Duchenne muscular dystrophy (DMD).

DMD, BMD — Joseph Beavo, Ph.D.

Joseph Beavo, professor of pharmacology at the University of Washington in Seattle was awarded an MDA research grant totaling $412,500 over a period of three years to study how the drug sildenafil acts to improve the function of heart muscle in animal models of Duchenne muscular dystrophy (DMD).

BMD, DMD — Linda Baum, M.D., Ph.D.

Linda Baum, professor and vice chair of pathology and laboratory medicine at the Geffen School of Medicine at the University of California, Los Angeles, was awarded an MDA research grant totaling $405,000 over a period of three years to study molecules on the muscle surface that regulate important aspects of cellular communication and survival.

DMD — Matthew Alexander, Ph.D.

Matthew Alexander, a postdoctoral research fellow in genetics and pediatrics at Harvard Medical School in Boston, Mass., was awarded an MDA development grant totaling $180,000 over a period of three years to study the role of microRNAs in Duchenne muscular dystrophy (DMD).

DMD: Dystrophin-Deficient Dogs Benefit from Gene Therapy

For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne muscular dystrophy.

DMD, BMD: Nationwide Children's Podcast Explores Dystrophin's Interactions

In a December 2012 podcast from Nationwide Children's Hospital, cell biologist Federica Montanaro discusses her team's recent progress in understanding how various proteins interact with dystrophin and how these interactions differ in the heart versus the skeletal muscles.

MD Briefs: Gene Therapy, Exon Skipping, Stem Cells

Update (Jan. 23, 2013):The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.

Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.

Lee Breidenbach

2012
Full name: 
Mr. Lee Breidenbach
Artist: 
Lee Breidenbach
Disease: 
Duchenne Muscular Dystrophy (DMD)
Medium: 
Watercolor

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