DMD/BMD — David Gokhin, Ph.D.

Research associate David Gokhin at the Scripps Research Institute in La Jolla, Calif., was awarded an MDA development grant totaling $180,000 over three years to study the role of a protein called gamma-actin in muscle degeneration and weakness in Duchenne (DMD) and Becker (BMD) muscular dystrophies.

MD Briefs: Corrected Stem Cells, Membrane Sealants

LGMD2D mice benefit from corrected human stem cells

A multinational team of scientists successfully transplanted genetically corrected muscle stem cells derived from people with type 2D limb-girdle muscular dystrophy (LGMD2D) into LGMD2D research mice and saw better muscle function in these mice than in similar mice that didn't receive the cells.

Research Briefs: BMD, DMD, Pompe disease

PTC begins non-US study of ataluren in DMD/BMD

MDA Hosts National Becker MD Conference in August

Update (Aug. 14, 2012): The Aug. 11 conference was attended by approximately 100 people with BMD and their families and friends, as well as some 40 clinicians and researchers. In addition, more than 70 individuals around the U.S. “attended” online. Videos of all presentations will be archived at Live Video Stream by early September 2012.

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: 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."

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.

This article looks at:

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 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:

The Breath of the Spirit

Artist: 
Carlos Serbia

Carlos began painting professionally in 2007.  During his teen years, he looked forward to playing baseball professionally.  However, weakness in his legs and pain while walking and running led to a diagnosis of BMD.  As a child Carlos enjoyed drawing, music and poetry.  This is his second donation to the Art Collection.  Carlos uses bright colors to represent positive energy, hope and the passion he puts into everything he does.

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