Muscular Dystrophies

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.

Eteplirsen Led to Dystrophin Production in US DMD Trial

Editor's note: This story was updated on April 27, 2012, and revised April 3, 2012.

MDA 2012 Conference Report: Genetics and Immunology Update

More than 500 physicians, allied health care professionals and MDA staff attended the MDA's 2012 Clinical Conference in Las Vegas, March 4-7.

The program emphasized:

Podcast Describes Results of LGMD2C Gene Therapy Trial

A March 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, presents the results of a phase 1 trial of gene therapy for gamma-sarcoglycan-deficientlimb-girdle muscular dystrophy (LGMD), also known as LGMD2C.

Podcast: Matthew Disney Discusses Drug Development for MMD

In type 1 myotonic dystrophy (MMD1, or DM1), expansions of DNA on chromosome 19 known as CTG repeats are converted to expansions in RNA called CUG repeats, which are toxic to nerve and muscles cells in a variety of ways.

Several MDA-supported research teams are targeting the toxic CUG repeats, with the goal of either blocking their interaction with other cellular substances or destroying them entirely.

Efficient System Developed for DMD Newborn Screening

Update 5/21/12: A podcast on this topic is now available; see Podcast Explores Newborn Screening for DMD.

'Gapmer Antisense' Targets MMD1 Defect for Destruction

Researchers at Baylor College of Medicine in Houston and Isis Pharmaceuticals in Carlsbad, Calif., have announced encouraging results for their antisense-based strategy in development for the treatment of type 1 myotonic muscular dystrophy (DM1, or MMD1).

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