Duchenne Muscular Dystrophy (DMD)

DMD — Hao Shi, Ph.D.

MDA has awarded a research development grant totaling $180,000 over three years to Hao Shi, associate research scientist at Yale University in New Haven, Conn. The new funds will help support Shi’s study of muscle repair and regeneration in Duchenne muscular dystrophy (DMD).

DMD — Pier Lorenzo Puri, M.D., Ph.D.

MDA has awarded a research grant totaling $309,336 over three years to Pier Lorenzo Puri, professor of pediatrics at the University of California, San Diego, and associate professor of muscle development and regeneration at the Sanford-Burnham Medical Research Institute, both in La Jolla, Calif. The funds will help support Puri's study of the molecular underpinnings of, and the identification of treatments for, Duchenne muscular dystrophy (DMD).

DMD — Grace Pavlath, Ph.D.

MDA has awarded a research grant totaling $295,269 over three years to Grace Pavlath, professor in the department of pharmacology at Emory University in Atlanta. The new funds will help support Pavlath’s study of abnormal muscle regeneration in the muscular dystrophies, particularly Duchenne muscular dystrophy (DMD).

DMD — Bradley Olwin, Ph.D.

MDA has awarded a research grant totaling $369,165 over three years to Bradley Olwin, professor of molecular, cellular & developmental biology at the University of Colorado in Boulder. The new funds will help support Olwin’s study of muscle regeneration in injured and diseased skeletal muscle — particularly in the muscular dystrophies, including Duchenne muscular dystrophy (DMD).

Olwin is a longtime MDA grantee, having received funding from the Association almost continuously since the early 1980s.

DMD — David Goldhamer, Ph.D.

MDA has awarded a research grant totaling $375,000 over three years to David Goldhamer, associate professor, director of the Center for Regenerative Biology, and associate director of the UConn Stem Cell Institute at the University of Connecticut in Storrs. The new funds will help support Goldhamer’s study of muscle stem cells and the repair of damaged muscle in Duchenne muscular dystrophy (DMD).

DMD — Dongsheng Duan, Ph.D.

Dongsheng Duan, professor in the department of molecular microbiology & immunology at the University of Missouri in Columbia, has received an MDA grant totaling $527,670 over three years. The funds will help support Duan's continued research into gene therapy in Duchenne muscular dystrophy DMD).

DMD is caused by a mutation in the dystrophin gene, which leads to the absence, or near absence, of dystrophin protein.

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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