Duchenne Muscular Dystrophy (DMD)

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).

Podcast Explores Newborn Screening for DMD

An April 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores the implications of a recently developed strategy for newborn screening for Duchenne muscular dystrophy (DMD).

The podcast is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."

2012 AAN Meeting: DMD, BMD Briefs

Update (June 1, 2012)­  The brief Drugs help DMD-related cardiomyopathy has been updated to include a May 2012 podcast provided by Nationwide Children's Hospital's "This Month in Muscular Dystrophy."

Neuromuscular Disease Research Discussed at 2012 AAN Meeting

Top scientists and clinicians from around the world are discussing the latest research in neuroscience, and the care of individuals with nerve and muscle diseases, at the 2012 annual meeting of the American Academy of Neurology in New Orleans, April 21-28.

DMD Drug SMT C1100 Moves to Human Testing

Update (May 25, 2012) Summit announced today that the first group of healthy volunteers in the phase 1 trial of its newly formulated SMT C1100 has begun receiving the drug. The dose-escalating trial will evaluate whether the new formulation of SMT C1100 is safe and whether consistent blood levels of the drug can be achieved.Results are expected by the end of the year.

MDA 2012 Conference Report: Best Practices

Several experts presented their views of "best practices" for care of people with neuromuscular disorders at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7.

Many questions remain about optimal care in these disorders, but it's clear that attention to heart and respiratory function are of paramount importance.

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