Duchenne Muscular Dystrophy (DMD)

Exon-Skipping Trial Results Are a 'Major Advance' in DMD Treatment

Editor's note (July 24, 2012): For a more in-depth discussion of the exon-skipping trial results, see A Closer Look: Extended Eteplirsen Treatment Benefits Walking in DMD.

DMD/BMD — Adam Engler, Ph.D.

Adam Engler, assistant professor at the University of California, San Diego, in La Jolla, was awarded an MDA research grant totaling $390,000 over three years to study cell-based therapies designed for Duchenne (DMD) and Becker (BMD) muscular dystrophies and other muscle diseases.

DMD/BMD — Dean Burkin, Ph.D.

Dean Burkin, associate professor of pharmacology at the University of Nevada School of Medicine in Reno, was awarded an MDA research grant totaling $308,028 over three years to study laminin-111 protein therapy for Duchenne (DMD) and Becker (BMD) muscular dystrophies.

DMD/BMD — Ryan Wuebbles, Ph.D.

Ryan Wuebbles, a postdoctoral fellow in pharmacology at the University of Nevada School of Medicine in Reno, was awarded an MDA development grant (DG) totaling $180,000 over three years to study the potential use of a protein called laminin-111 as the basis of therapies for Duchenne (DMD) and Becker (BMD) muscular dystrophies.

DMD/BMD — David Gokhin, Ph.D.

Research associate David Gokhin at the Scripps Research Institute in La Jolla, Calif., was awarded an MDA development grant totaling $180,000 over three years to study the role of a protein called gamma-actin in muscle degeneration and weakness in Duchenne (DMD) and Becker (BMD) muscular dystrophies.

DMD — Tathagata Chaudhuri, Ph.D.

Postdoctoral research fellow Tathagata Chaudhuri, at the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, was awarded an MDA development grant (DG) totaling $180,000 over three years to develop a stem cell therapy for muscular dystrophies, including Duchenne muscular dystrophy (DMD), that will lead to muscle regeneration.

MD Briefs: Corrected Stem Cells, Membrane Sealants

LGMD2D mice benefit from corrected human stem cells

A multinational team of scientists successfully transplanted genetically corrected muscle stem cells derived from people with type 2D limb-girdle muscular dystrophy (LGMD2D) into LGMD2D research mice and saw better muscle function in these mice than in similar mice that didn't receive the cells.

Heart Drug Being Tested in DMD

Eplerenone, a drug commonly used to prevent scarring after a heart attack, is being tested in a phase 2-3 clinical trial to determine whether it can stop or slow heart damage in people with Duchenne muscular dystrophy (DMD).  

Eplerenone blocks scarring that over time can turn heart muscle into nonfunctioning fatty tissue.

DMD/BMD — Zolt Arany

MDA awarded $352,188 to Zolt Arany, assistant professor in medicine at Beth Israel Deaconess Medical Center, part of Harvard Medical School in Boston, for research into the role of skeletal muscle metabolism in Duchenne muscular dystrophy (DMD).

DMD/BMD — Vihang Narkar

Vihang Narkar, assistant professor at the University of Texas Health Science Center at Houston, was awarded $302,326 to study the potential therapeutic value of increasing the overall amount of a specific type of muscle called "aerobic muscle" in Duchenne muscular dystrophy (DMD).

Loss of the structural protein dystrophin, the underlying cause of DMD, causes muscle instability, damage and progressive weakness, accompanied by a decrease in the ability to produce energy, along with severe fatigue.

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