Methods to treat .alpha.1-antitrypsin deficiency
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United States of America Patent
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Dec 2, 2003
Grant Date -
Jan 17, 2002
app pub date -
Jan 22, 2001
filing date -
Jan 20, 2000
priority date (Note) -
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Abstract
Inhibitors of glucosidase, especially those related to castanospermine, are effective in preventing or ameliorating conditions such as liver damage and emphysema that are present in individuals who produce a mutant form of antitrypsin, .alpha.1-ATZ. Also effective in the method of the invention are imino sugars and their reduced forms in general as well as phenylbutyric acid. These compounds enhance the secretion of the mutant form, which retains substantial biological activity, and do not impair its degradation in the endoplasmic reticulum.
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