First Drug OKd to Treat Rare Pompe Disease
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From Times Staff and Wire Reports
The Food and Drug Administration has approved the first drug to treat Pompe disease, a rare genetic disorder in which patients cannot metabolize the complex sugar glycogen into simple glucose.
In the absence of a key enzyme, victims develop lethal thickening of the heart muscle and respiratory problems.
The new drug, Myozyme, is produced by genetic engineering and replaces the missing enzyme.
About one in every 10,000 people are born with Pompe.
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