The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease (Q28540925)

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scientific article (publication date: 2014)
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The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease
scientific article (publication date: 2014)

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    The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease (English)
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    Allan C Powe
    Rohini Dhulipala
    Michelle Frascella
    Anadina Garcia
    Lee J Pellegrino
    Nastry Brignol
    Matthew J Toth
    David J Lockhart
    Brandon A Wustman
    Kenneth J Valenzano
    2014
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    9
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    7
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    e102092
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