Muscular Dystrophies

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."

Podcast: Howard Worman Discusses Research Related to Heart Disease in EDMD and LGMD1B

Recently, MDA grantee Howard Worman at Columbia University, and colleagues, published encouraging results about the effects of a drug called selumetinib on the hearts of mice with a genetic mutation that's found in a form of Emery-Dreifuss muscular dystrophy (EDMD)

MDA Urges Legislators to Speed Up Approval of Rare Disease Drugs

Update (July 10, 2012) —Reauthorization of the Prescription Drug User Fee Act (PDUFA) was signed by President Obama on July 9, 2012, complete with a provision that creates an accelerated approval pathway for drugs for rare diseases.

Selumetinib Helps Heart in EDMD Mice

Update (June 1, 2012): This story was updated to include the availability of a podcast on this topic.

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.

2012 AAN Meeting: Limb-Girdle MD, Distal MD Briefs

The 2012 annual meeting of the American Academy of Neurology (AAN), held in New Orleans April 21-28, included findings related to limb-girdle muscular dystrophy (LGMD) and distal muscular dystrophy.

'Turning Off the Switch' May Provide New FSHD Strategy

Researchers in Italy and Japan, supported in part by MDA, have identified what they believe is a molecular "switch" that may be inappropriately activating several genes in facioscapulohumeral muscular dystrophy (FSHD).

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