Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in Patients at Risk of Rapidly Progressing ADPKD: Primary Results of a Double-Blind, Placebo-Controlled, Phase 2/3 Randomized Clinical Trial
Ronald T. Gansevoort, Ali Hariri, Pascal Minini, Curie Ahn, Arlene B. Chapman, Shigeo Horie, Bertrand Knebelmann, Michal Mrug, Albert Ong, York Pei, Vicente E. Torres, Vijay Modur, Igor Antonshchuk, Ronald D. Perrone
Topics & Concepts
MedicineAutosomal dominant polycystic kidney diseasePlaceboUrologyTolerabilityRenal functionInternal medicineInterim analysisKidney diseaseStage (stratigraphy)Polycystic kidney diseaseRandomized controlled trialKidneyAdverse effectPathologyPaleontologyAlternative medicineBiologyGenetic and Kidney Cyst DiseasesSphingolipid Metabolism and SignalingRenal Diseases and Glomerulopathies