Muscular Dystrophies

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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Heat Shock Proteins May Be New Approach to DMD Treatment

New research suggests that a potential strategy for treating Duchenne muscular dystrophy (DMD) may be to increase levels of heat shock proteins inside muscle fibers.

Heat shock proteins exist naturally inside cells, where they assist in the folding and unfolding of other proteins. Their levels are increased when cells are exposed to various stresses, such as high temperatures.

MDA 2012 Conference Report: Targeted Therapies

The progress of several experimental therapies currently in development for neuromuscular diseases was discussed at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7.

MDA Funds Testing of Anti-Inflammatory Compounds for DMD

The Muscular Dystrophy Association has awarded $120,000 to Cambridge, Mass.-based Catabasis Pharmaceuticals as part of a strategic partnership under which the pharmaceuticals company will test two compounds called CAT-1004 and CAT-1040 in the mdx research mouse model of Duchenne muscular dystrophy (DMD).

MMD1: 'Invasive' Approach to Cardiac Management Improved Survival

New evidence suggests that relatively aggressive management of seemingly minor cardiac conduction defects in adults with type 1 myotonic dystrophy (MMD1, or DM1) can prolong survival.

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