Duchenne’s Muscular Dystrophy Advance – Healthy Kids and …

Drug May Partially Correct Deadly Form of Muscular Dystrophy

By Daniel J. DeNoon WebMD Health News

Reviewed By Louise Chang, MD

Dec. 27, 2007 -- An "antisense" compound may partially correct the genetic defect that causes deadly Duchenne's muscular dystrophy.

Researchers are not calling the finding a breakthrough, but they are cautiously optimistic that the approach may one day lead to a treatment that extends the lives of people afflicted with the always-fatal disease.

Duchenne's muscular dystrophy is a genetic disease seen in one in 3,500 baby boys. The boy's muscles get weaker and weaker, landing them in wheelchairs by their twelfth birthday. Their hearts and lungs also weaken, and the brain may be affected.

Most of these boys die in early childhood; with steroid treatment and breathing aids, their life span can be as long as 25 to 35 years. Nothing, however, prevents eventual death.

Now there's a small glimmer of hope. Scientists now know the specific genetic mutation that causes Duchenne's muscular dystrophy. That mutation causes a "misreading" of the gene that encodes dystrophin, a substance crucial for muscle cell survival.

In the test tube, a compound called an antisense oligonucleotide partially corrects this genetic misreading. It seems to work in animals. Might it work in humans?

Judith C. van Deutekom, PhD, of Netherlands' Leiden University, and colleagues tested the treatment, called PRO051, in four 10- to 13-year-old boys with Duchenne's muscular dystrophy. They'd all been confined to wheelchairs since ages 7 to 11.

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Duchenne's Muscular Dystrophy Advance - Healthy Kids and ...