DMD, BMD: Trial of Ataluren in Nonwalkers

Update (March 3, 2010): An update to the article Ataluren Results Disappointing was posted.

New DMD Care Recommendations Released

A comprehensive set of clinical care recommendations for the diagnosis and management of Duchenne muscular dystrophy (DMD) is now available to medical professionals and families. The guidelines pull together recommendations previously released by other groups, such as the American Academy of Neurology, and clarify some areas in which consensus has been lacking.

New DMD Care Recommendations

A comprehensive set of clinical care recommendations for the diagnosis and management of Duchenne muscular dystrophy (DMD) is now available to medical professionals and families.

The complete recommendations,  in printable PDF format, can be read here:

DMD Research: US Exon Skipping Trial

The first human trial in the United States of a treatment strategy known as "exon skipping" for Duchenne muscular dystrophy (DMD) is scheduled to begin in March 2010 at Nationwide Children's Hospital in Columbus, Ohio, one of five elite centers comprising MDA's DMD Clinical Research Network.

First Human Exon Skipping Trial in US Planned for 2010

A doctor exams a young boy with DMD.
Exon skipping is an experimental strategy that could help some boys and young men with Duchenne muscular dystrophy produce dystrophin, the muscle protein that's missing in this disease.

Genetics: You, Me and PGD

Update 5/21/12:This study is closed, and results are available at Decision Making About PGD Is Complex, Study Finds. Information about future studies will be posted at PGD: Couples' Decision Making when available.

Preimplantation genetic diagnosis (PGD) is a way for couples to avoid passing along a hereditary disease to their offspring by pre-screening embryos.

MMD Research: 'Bright' Prospect

Researchers at the University of Rochester (N.Y.) Wellstone Muscular Dystrophy Cooperative Research Center have identified a compound that has the potential to be developed into a treatment for type 1 myotonic dystrophy (MMD1, or DM1).

The compound, dubbed CAG25, is an "antisense oligonucleotide," a type of construct that's been used to block disease-causing genetic instructions in laboratory experiments and human trials in other diseases.

Scientists Continue to Explore Stem Cells

Stem cells — immature cells with the potential to develop into different tissue types — have been heralded as a major advance for developing treatments for a variety of diseases. That’s true for diseases of the nerves and muscles, where such cells could potentially be transplanted into the body and either support or replace a patient’s ailing cells.

Plugs and Patches Improve Muscle Health

Much attention has been paid to gene therapy and stem cell strategies for treating muscle diseases, but several less dramatic strategies also appear to hold potential, especially if used in conjunction with more definitive therapies to enhance their effectiveness.

Research Updates Summer 2009

Story includes research about: Becker muscular dystrophy, Duchenne muscular dystrophy and oculopharyngeal muscular dystrophy.

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