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.

MDA Mailbag Spring 2009

Letters to Quest: Camp is so important ** Moms on Wheels ** All camps not the same ** No guarantees (life expectancy) ** FMLA and sibling caregivers ** Pay for family caregivers.

UK Trial: 'Robust Response' to Exon-Skipping Compound Seen

On Jan. 21, AVI BioPharma of Portland, Ore., announced its experimental compound AVI4658 for the treatment of Duchenne muscular dystrophy (DMD) yielded promising results in a phase 1 clinical trial in the United Kingdom.

Speeding the Course of Clinical Trials

Research Administration Winter 2009

Updated MD-CARE Act is now law

The Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education (MD-CARE) Amendments of 2008 received final Congressional approval on Sept. 27 and were signed into law by President Bush Oct. 8.

Drug Development Progress: How Are They Doing?

In the era of molecular biology, the drug development process has moved from a “let’s try it and see what happens” approach to a scientifically based process of discovery and application.

For many of the diseases in MDA’s program, drug discovery begins with gene discovery — identifying a gene that, when flawed, causes a disease.

European Team Combines Stem Cells, Gene Correction to Treat DMD-Affected Mice

A European group of researchers successfully used a combination of genetic correction and stem cells to treat Duchenne muscular dystrophy (DMD) in mice with the disease. The compound they used for the genetic correction was developed by Judith van Deutekom, then at Leiden (Netherlands) University Medical Center, with MDA support.

DMD results from the loss of the muscle protein dystrophin because of any of a number of genetic errors in the dystrophin gene.

Moving Away from the Blasts of the Past

In the early 1990s, amid great excitement, six trials took place of transplants of immature muscle cells from healthy relatives into boys with Duchenne muscular dystrophy. (Five were funded by MDA.)

Stars & Stripes Grab World Cup in Overtime

What a magnificent tribute to the gutsiness of eight incredible athletes!

On Oct. 14, after days of intense competition, Team USA emerged victorious in the Power Soccer World Cup in Tokyo.

On their way to the top they beat world-class power soccer teams from Belgium, Denmark, England, France, Japan and Portugal. Their final win came in a sudden-death overtime match against France, which had never before been beaten in international competition.

Debatable Destinies

When Rena Szymanski turned 40, she expected to slow down a bit. She never had been athletic, and it sometimes seemed to her that climbing stairs was harder for her than it was for other people, but she thought her strength was “normal” in general.

Soon after her birthday, though, she noticed increasing weakness. “I didn’t know what was wrong with me,” says Szymanski, who until recently was working at a stressful job an hour away from her home in Stony Point, N.Y. To her knowledge, there is no history of muscle disease in her family.

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