Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb₃) in Fabry Transgenic Mice and in the Plasma of Fabry Patients

• Brandy Young-Gqamana ,

  Contributed equally to this work with: Brandy Young-Gqamana, Nastry Brignol

  Affiliation University of Cape Town, Institute of Infectious Disease and Molecular Medicine, Division of Medical Biochemistry, Cape Town, South Africa

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• Nastry Brignol ,

  Contributed equally to this work with: Brandy Young-Gqamana, Nastry Brignol

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

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• Hui-Hwa Chang,

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

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• Richie Khanna,

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

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• Rebecca Soska,

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

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• Maria Fuller,

  Affiliation Lysosomal Diseases Research Unit, South Australia Pathology at Women’s and Children’s Hospital, Adelaide, South Australia, Australia

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• Sheela A. Sitaraman,

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

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• Dominique P. Germain,

  Affiliation Division of Medical Genetics, University of Versailles – Saint Quentin en Yvelines, Hôpital Raymond Poincaré (Assistance Publique - Hôpitaux de Paris), Garches, France

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• Roberto Giugliani,

  Affiliation Medical Genetics Service, Clinic Hospital of Porto Alegre Federal University of Rio Grande do Sul, Porto Alegre, Brazil

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• Derralynn A. Hughes,

  Affiliation Lysosomal Storage Disorders Unit, Department of Academic Haematology, Royal Free Hospital and University College Medical School, London, United Kingdom

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• Atul Mehta,

  Affiliation Lysosomal Storage Disorders Unit, Department of Academic Haematology, Royal Free Hospital and University College Medical School, London, United Kingdom

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• Kathy Nicholls,

  Affiliation Department of Nephrology, Royal Melbourne Hospital, Parkville, Victoria, Australia

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• Pol Boudes,

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

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• David J. Lockhart,

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

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• Kenneth J. Valenzano,

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

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•  [ ... ],
• Elfrida R. Benjamin

  * E-mail: ebenjamin@amicusrx.com

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

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