Duchenne Muscular Dystrophy (DMD)

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.

DMD: Phase 2 Trial of GSK Exon-Skipping Drug Opens in US

Update (April 9, 2013): The U.S.-based phase 2 trial of drisapersen stopped recruiting new participants in January 2013. Results are expected in early 2014.

Update (Aug. 15, 2012): GlaxoSmithKline's exon-skipping drug GSK2402968 has been given a new generic name, "drisapersen."

MDA Launches New Research Symposium Series

This month, MDA is launching a new U.S.-based symposium series to address important topics and ultimately to accelerate therapy development for neuromuscular diseases.

"MDA’s new symposium series is designed to address cutting-edge issues in neuromuscular disease research," said Sanjay Bidichandani, MDA's vice president of research. "Science is advancing at a rapid pace, and these small and focused meetings will allow us to be nimble in tackling multiple issues every year."

Decision Making About PGD Is Complex, Study Finds

Decision making about preimplantation genetic diagnosis (PGD) is a complex, multiphase process for couples, a new study has found. Understanding it, the investigators say, may be helpful to prospective parents who know they're at risk for transmitting a genetic disorder, and to the professionals who advise them.

MDA Funds Development of Gentler Glucocorticoid for DMD

The Muscular Dystrophy Association has awarded $1,549,725 to ReveraGen BioPharma, a Rockville, Md., biotechnology company, for development of a dissociative glucocorticoid to treat Duchenne muscular dystrophy (DMD).

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