ALLOSTERIC CHAPERONES AND USES THEREOF
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United States of America Patent
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May 28, 2015
app pub date -
Jun 6, 2013
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Jun 6, 2012
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Abstract
The present invention relates to an allosteric non-inhibitory chaperone of the lysosomal acid alpha-glucosidase (GAA) for use in the treatment of a pathological condition characterized by a deficiency of the lysosomal acid alpha-glucosidase (GAA), to pharmaceutical composition thereof, to a method for increasing the activity of GAA in a subject and to a method for identifying an allosteric non-inhibitory chaperone for GAA.
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