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How A "Superbaby" Is Helping To Find Muscular Dystrophy Treatments

Posted by ScuttleMonkey on from the hercules-hercules dept.

An anonymous reader writes to tell us that a baby boy with unusually big muscles — caused by a gene mutation — is helping scientists to discover new muscular dystrophy drugs. "Myostatin was discovered in mice in 1992 in Lee's Johns Hopkins lab. In 1996 he proved its importance by showing that mice without the myostatin-producing gene got twice as big. The next year he discovered that the bulging Belgian Blue cow was a myostatin mutant, the first of eight prized cattle breeds later found to have the mutation. The company he had co-founded, MetaMorphix, is working on manipulating myostatin to beef up livestock. Wyeth picked up the rights to develop a drug for humans. Its experimental antibody drug produced bulked-up mice in 2002, and results of a trial in adults with muscular dystrophy are expected as early as March."

4 of 93 comments (clear)

  1. Cool, but no silver bullet. by Short+Circuit · · Score: 5, Informative

    This is actually pretty cool. I have a step-mother and a half-brother with muscular distrophy.

    It won't be a silver bullet, though. His disability is so bad that he's never been able to talk, much less walk on his own. Some of his joints have essentially locked up due to disuse. Even if the treatment were available today, he'd still have to learn how to talk. He might even need knee-replacement surgery before he could start learning to walk.

    At least he'd be able to feed himself, though.

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  2. Unbreakable by FredDC · · Score: 2, Informative

    Reminds me of the movie Unbreakable.

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  3. Re:Some concerns by eli+pabst · · Score: 3, Informative

    what effect does it have on your heart or your tongue?
    It's my understanding that myostatin is only expressed in skeletal muscle, so cardiac muscle would be unaffected by a myostatin inhibitor.

    I'm not an expert on it, but my lab has done a lot of research on myostatin and has identified some of the mutations in humans (including some cool papers looking at mutation prevalence in world class body builders).

    This might be informative reading for you: http://www.ncbi.nlm.nih.gov/entrez/dispomim.cgi?id =601788

  4. Baby pic! by bgarcia · · Score: 3, Informative

    Picture of the baby: link

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