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PO1593 : Glucosylceramide Synthase Inhibition with Venglustat in Classic Fabry Disease Patients Leads to Progressive Reduction of Endothelial Cell Globotriaosylceramide Inclusion Volume

Researchers

Presenter

  • Najafian Behzad

Principal Investigators

  • Wilcox William

  • Hopkin Robert

  • Lukina Elena

  • Deegan PatrickB

  • Sensinger Charlotte

  • Hailman Eric

  • Modur Vijay

  • Mauer Michael

Medical Centers

  • Department of Pathology University of Washington Seattle Washington United States

  • Mercer University School of Medicine, Macon, Georgia, United States

  • Division of Rheumatology, Cincinnati Children's Hospital Medical Center and The University of Cincinnati College of Medicine, Cincinnati, OH

  • National Research Center for Hematology, Moscow, Russia

  • Lysosomal Disorders Unit Addenbrooke's Hospital Cambridge United Kingdom

  • Sanofi Genzyme, Massachusetts, United States

  • Department of Internal Medicine, University of Minnesota, Minneapolis, Minnesota, United States

Locations

  • United States

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

Disease

  • Fabry disease

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A


Study Design

  • Double Blind/Blinded

Phase

  • II

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A