Duchenne Muscular Dystrophy (DMD)

DMD: Exon-Skipping Drug Drisapersen Appears to Increase Dystrophin Levels

Multinational pharmaceutical company GlaxoSmithKline (GSK) has released encouraging results about dystrophin protein production in a phase 2, non-U.S. trial of its exon-skipping compound drisapersen in boys with Duchenne muscular dystrophy (DMD).

MDA Funds Study of Prednisone in Very Young Boys with DMD

Pediatric neurologist Anne Connolly has received an MDA grant to study the effects of prednisone on Duchenne muscular dystrophy in very young children.

MDA Study Reveals ‘Cost of Illness’ for ALS, DMD, MMD

"We now have numbers to point to," said Annie Kennedy, MDA's senior vice president of advocacy. "It's not just a back-of-the-envelope calculation anymore. It's real data."

DMD — Christopher Penton, Ph.D.

Christopher Penton, who recently earned his doctorate in integrated biomedical sciences at Ohio State University in Columbus, was awarded an MDA research grant totaling $180,000 over three years to study ways to improve muscle regeneration and decrease muscle scar tissue formation in Duchenne muscular dystrophy (DMD).

Nationwide Children’s Podcast Explores Therapy Development for DMD

In a July 2013 podcast from Nationwide Children’s Hospital in Columbus, Ohio, longtime MDA grantee Jeffrey Chamberlain discusses recent advances in the development of gene therapy (gene transfer) and stem cell therapy (transplantation) for Duchenne muscular dystrophy (DMD), the results of which may apply to other types of muscular dy

DMD — Anne Connolly, M.D.

Anne Connolly, a pediatric neurologist at Washington University in St. Louis, was awarded an MDA human clinical trial grant totaling $343,787 over a period of nearly three years (through April 30, 2016) to conduct a trial of prednisone in boys with Duchenne muscular dystrophy (DMD) who are younger than 2½ years old.

DMD — Morayma Reyes, M.D., Ph.D.

Morayma Reyes, assistant professor of pathology at the University of Washington in Seattle, was awarded an MDA research grant totaling $300,000 over a period of three years to study a strategy for reducing heart muscle damage in Duchenne muscular dystrophy (DMD).

DMD: Multicenter Trial To Test Drug that Fights Muscle Scarring, Inflammation

A phase 1b/2a clinical trial to test the safety, tolerability and pharmacokinetics of single and multiple doses of HT-100 (also called delayed-release halofuginone) in boys with Duchenne muscular dystrophy (DMD) is now open at four sites in Maryland, Missouri and Ohio, with an additional site expected to open in California.

DMD/BMD — Terence Partridge, Ph.D.

Terence Partridge, professor of integrative systemic biology and pediatrics at George Washington University and associate director of the Children’s Research Institute in Washington, D.C., was awarded an MDA research grant totaling $300,000 over a period of three years to investigate differences in muscle repair mechanisms in mice and humans, in the context of Duchenne muscular dystrophy (DMD).

DMD — Rachelle Crosbie-Watson, Ph.D.

Rachelle Crosbie-Watson, professor of neurology at the University of California, Los Angeles, was awarded an MDA research grant totaling $300,000 over a period of three years to study whether increasing levels of the sarcospan protein can be therapeutic for Duchenne muscular dystrophy (DMD) and other muscle diseases.

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