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An unstable triplet repeat in a gene related to myotonic muscular dystrophy. For years, neurologists have noticed a strange phenomenon: Patients afflicted by myotonic muscular dystrophy, a muscle-stiffening disorder, often have children with a more severe form of the disease. The drug, which was launched in the second quarter of 2006, is also being investigated for various other indications with unmet medical needs, including severe insulin resistance, myotonic muscular dystrophy and HIV-Associated Adipose Redistribution Syndrome (HARS). |
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