Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent

• Sushrusha Nayak ,

  * E-mail: sushrusha.nayak@ki.se (SN); bbyrne@ufl.edu (BJB)

  Affiliations Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, Florida, United States of America, Department of Medicine, Center for Infection Medicine, Karolinska Institute, Stockholm, Sweden

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• Phillip A. Doerfler,

  Affiliation Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, Florida, United States of America

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• Stacy L. Porvasnik,

  Affiliation Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, Florida, United States of America

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• Denise D. Cloutier,

  Affiliation Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, Florida, United States of America

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• Richie Khanna,

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

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• Ken J. Valenzano,

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

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• Roland W. Herzog,

  Affiliation Department of Pediatrics, Cellular and Molecular Therapy, University of Florida, Gainesville, Florida, United States of America

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• Barry J. Byrne

  * E-mail: sushrusha.nayak@ki.se (SN); bbyrne@ufl.edu (BJB)

  Affiliation Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, Florida, United States of America

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