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Children's Health

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New Drug May Treat Muscular Dystrophy

Drug Targets Gene Glitch Seen in Genetic Disorders Including Duchenne Muscular Dystrophy
WebMD Health News
Reviewed by Louise Chang, MD

April 23, 2007 -- An experimental drug called PTC124 may override a gene glitch seen in Duchenne muscular dystrophy and various other genetic disorders.

Duchenne muscular dystrophy is one of nine major forms of muscular dystrophy. It's the most common type of muscular dystrophy in children and affects males.

In Duchenne muscular dystrophy, the muscles decrease in size and grow weaker over time yet may appear larger. Disease progression varies, but many patients need a wheelchair by the time they're 12 years old.

The new drug, called PTC124, isn't available yet, but clinical trials are currently under way.

Today, researchers posted the results from lab tests in mice with a genetic glitch like one seen in a large group of genetic disorders, including Duchenne muscular dystrophy.

The gene glitch hampers the production of dystrophin, a protein needed for muscle development. PTC124 is designed to override that gene glitch, putting dystrophin production back on track.

In lab tests, mice with muscular dystrophy (MD) got PTC124 orally and/or by injection for two to eight weeks.

"Enough dystrophin accumulated in the muscles of the MD mice so that we could no longer find defects in the muscle when we examined them," researcher H. Lee Sweeney, PhD, says in a news release.

Sweeney chairs the University of Pennsylvania's physiology department.

"For all intents and purposes, the disease was corrected by treatment with PTC124" in the mice, Sweeney says.

If PTC124 succeeds in human tests, it may help treat Duchenne muscular dystrophy and similar genetic disorders, note the researchers, who included scientists from PTC Therapeutics, the company that makes PTC124.

The study appears in the advance online edition of Nature.

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