Systemic Delivery of a Glucosylceramide Synthase Inhibitor Reduces CNS Substrates and Increases Lifespan in a Mouse Model of Type 2 Gaucher Disease

• Mario A. Cabrera-Salazar ,

  * E-mail: Mario.Cabrera@genzyme.com

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• Matthew DeRiso,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• Scott D. Bercury,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• Lingyun Li,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• John T. Lydon,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• William Weber,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• Nilesh Pande,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• Mandy A. Cromwell,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• Diane Copeland,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• John Leonard,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• Seng H. Cheng,

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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• Ronald K. Scheule

  Affiliation Genzyme Corporation, Framingham, Massachusetts, United States of America

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