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Publication Information
Affiliation
Division of Nephrology, Tufts Medical Center, Tufts University School of Medicine, Boston, Massachusetts.; Eloxx Pharmaceuticals, Watertown, Massachusetts.; Sanofi, Chilly Mazarin, France.; Department of Internal Medicine, Seoul National University, Seoul, Republic of Korea.; Department of Medicine, University of Chicago, Chicago, Illinois.; Department of Urology, Juntendo University Graduate School of Medicine, Tokyo, Japan.; Université de Paris, AP-HP, Service de Néphrologie, Hôpital Necker-Enfants Malades, Paris, France.; Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama and Department of Veterans Affairs Medical Center, Birmingham, Alabama.; Academic Nephrology Unit, Department of Infection Immunity & Cardiovascular Disease, University of Sheffield Medical School, Sheffield, United Kingdom.; Division of Nephrology, University of Toronto, Toronto, Ontario, Canada.; Division of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota.; Eloxx Pharmaceuticals, Watertown, Massachusetts.; Department of Nephrology, University Medical Center Groningen, The Netherlands.
Authors
Perrone Ronald D, Hariri Ali, Minini Pascal, Ahn Curie, Chapman Arlene B, Horie Shigeo, Knebelmann Bertrand, Mrug Michal, Ong Albert C M, Pei York P C, Torres Vicente E, Modur Vijay, Gansevoort Ronald T
Studies
Abstract
Venglustat, a glucosylceramide synthase inhibitor, inhibits cyst growth and reduces kidney failure in mouse models of autosomal dominant polycystic kidney disease (ADPKD). STAGED-PKD aims to determine the safety and efficacy of venglustat and was designed using patient enrichment for progression to end-stage kidney disease and modeling from prior ADPKD trials.