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Genetic mutation directs search for DMD drug

Posted on February 16, 2007 in Spinal Muscular Atrophy Science and Research

The February 22nd issue of Forbes magazine has an article about an infant’s genetic mutation and how scientists are now seeing this mutation as a possible help for muscular dystrophy patients.

In 2000, a child was born with larger than normal muscles. Doctors confirmed the the boy had a genetic mutation discovered by researchers at Johns Hopkins University a few years earlier. The mutation blocks the gene responsible for myostatin which is a protein that slows muscle growth. Less myostatin equals larger muscles.

Researchers are now working to design drugs that will block myostatins and hope that such drugs would help patients such as those with Duchenne muscular dystrophy. Pharmaceutical company Wyeth developed an experimental antibody drug that increased the muscles of mice in 2002 and a trial involving adults with muscular dystrophy is expected to produce results soon.

But researchers warn that there is still lots of room for failure. The blocking drugs could produce adverse side effects. There are a variety of these proteins and finding the correct one or combination to manipulate will take time. And, the effects of myostatin manipulation may be the greatest only in fetal development.

To read the full article, click here and login. (Membership to Forbes.com is free)


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