Methods for Treating Pompe Disease
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United States of America Patent
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Issued Date -
Apr 19, 2012
app pub date -
Oct 7, 2011
filing date -
Nov 13, 2006
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Abandoned
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Abstract
The present invention provides methods for treating Pompe disease in a subject by administering to the subject a therapeutically effective amount of a fusion protein which includes human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain. The lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
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