Duchenne Muscular Dystrophy (DMD)

DMD/BMD - Jasmin

MDA awarded Professor Bernard Jasmin, vice-dean of research at the University of Ottawa in Canada, a grant totaling $360,000 to study the impact of small molecules called "exercise mimetics" in Duchenne muscular dystrophy (DMD).

DMD/BMD - Forbes

MDA awarded $179,327 to scientific researcher Sean Forbes at the University of Florida in Gainesville for research into impaired blood flow to muscles lacking the dystrophin protein in Duchenne muscular dystrophy (DMD).

DMD/BMD - Cornelison

MDA awarded a grant totaling $160,000 to Dawn Cornelison, assistant professor in the division of biology at the University of Missouri-Columbia, for research into the role of a protein called syndecan-4 in muscle tissue repair after damage.

DMD/BMD - Calos

Michele Calos, professor in the department of genetics at Stanford University School of Medicine in Stanford, Calif., received a $200,000 MDA research grant to develop a new stem cell-based therapy for Duchenne muscular dystrophy (DMD).

"Funding from MDA is critical for us to develop these new ideas that could lead to novel strategies to combat DMD," Calos said. "A prior MDA grant has allowed us to make progress in the studies, and we are now poised to perform the critical experiments."

DMD/BMD - Bertoni

MDA awarded $408,915 to Carmen Bertoni, assistant professor of neurology at the University of California, Los Angeles, to continue research into DNA repair strategies in Duchenne muscular dystrophy (DMD).

DMD/BMD - 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).

Research Briefs: BMD, DMD, Pompe disease

PTC begins non-US study of ataluren in DMD/BMD

Stop Codon Read-Through Drug Performs Well in DMD Mice

An experimental drug called RTC13, designed to treat Duchenne muscular dystrophy (DMD) by restoring production of the muscle protein dystrophin, has shown promise in experiments in dystrophin-deficient mice that have a DMD-like disease.

RTC13's MDA-supported developers say they're optimistic about the compound but that refinement of its chemistry and further testing will be needed before it can be taken into clinical trials in people with DMD.

MDA Symposium Looks at Partnerships Between Industry and Academia

Enhancing collaboration and transfer of technology between academia and industry was the subject of MDA's recent Translational Research Symposium, one of a series of four MDA-sponsored research symposia scheduled in 2012.

The symposium was held June 27 in New Orleans, in conjunction with the 2012 New Directions in Biology and Disease of Skeletal Muscle Conference.

Stem Cell Briefs: Contributions of PAX7, S1P, MCAM

Update (July 10, 2012) — This story was updated to reflect the availability of a podcast with MDA research grantee Emanuela Gussoni, who discusses the development of stem-cell-based treatments for muscular dystrophies.

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