Duchenne Muscular Dystrophy (DMD)

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).

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:

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.

Self-Portrait - Christian Reynolds

Christian created this artwork at age 10 at an MDA-sponsored children's art workshop held June 6, 1992 in Houston.  He always enjoyed swimming and fishing and going to MDA summer camp.  Christian was interested in oceanliners and maritime history and went on 10 cruises.

Self-Portrait - Julian Dombrowski

Julian created this self-portrait at age 10 at an MDA-sponsored children's art workshop held Oct. 2, 1992, at the Tucson Museum of Art.  He is a former MDA goodwill ambassador for Southern Arizona.

MDA Genetic Counseling Webinar Answers Key Questions

Respondents to MDA’s Transitions Survey — in other words, people with a neuromuscular disease who are in their teens through late 30s — made it clear they had questions about the genetics of their disease, as well as questions about family planning and the value of diagnostic testing.

MDA’s Genetic Counseling Webinar, which occurred Feb. 22, 2012, answers many of those questions.

DMD: Eteplirsen Allows Production of Functional Dystrophin

Shortened versions of the muscle protein dystrophin— produced by skipping a section of genetic instructions called exon 51— appear to be functional, says a new report from the United Kingdom.

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