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How A "Superbaby" Is Helping To Find Muscular Dystrophy Treatments
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."
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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Reminds me of the movie Unbreakable.
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what effect does it have on your heart or your tongue?
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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?i
Picture of the baby: link
I'm a leaf on the wind. Watch how I soar.
What is really scary is you wanna know who was really interested in myostatin? Victor Conte...the BALCO guy who was involved in the whole Barry Bonds steroid scandal and did jail time for it. He actually published papers on the topic. Not saying that they ever came up with anything useful, but these were the people who came up with "The Clear" which was undetectable by steroid screens.
http://en.wikipedia.org/wiki/Tetrahydrogestrinone
Marylin Monroe was 118 pounds. Even accounting for size inflation, she was NEVER a size 16. In fact, according to standardized clothing sizes (which are completely ignored anymore, but were developed around the time Marylin was around), she would have worn at most a size 12 (based on her hips) and probably something closer to a 6 (based on her waist).