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

• Richie Khanna ,

  Contributed equally to this work with: Richie Khanna, Allan C. Powe Jr

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Allan C. Powe Jr ,

  Contributed equally to this work with: Richie Khanna, Allan C. Powe Jr

  Current address: Vertex Pharmaceuticals, San Diego, California, United States of America

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Yi Lun,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Rebecca Soska,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Jessie Feng,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Rohini Dhulipala,

  Current address: Life Technologies, Austin, Texas, United States of America

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Michelle Frascella,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Anadina Garcia,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Lee J. Pellegrino,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Su Xu,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Nastry Brignol,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Matthew J. Toth,

  Current address: Barth Syndrome Foundation, Larchmont, New York, United States of America

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Hung V. Do,

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• David J. Lockhart,

  Current address: OrphiTherapeutics, Carlsbad, California, United States of America

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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• Brandon A. Wustman,

  Current address: OrphiTherapeutics, Carlsbad, California, United States of America

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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•  [ ... ],
• Kenneth J. Valenzano

  * E-mail: kvalenzano@amicusrx.com

  Affiliation Amicus Therapeutics Inc., Cranbury, New Jersey, United States of America

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