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 randomiz... von Gansevoort, R.T, Hariri, A, Minini, P, Ahn, C, Chapman, A.B, Horie, S, Knebelmann, B, Mrug, M, Ong, A.C.M, Pei, Y.P.C, Torres, V.E, Modur, V, Antonshchuk, I, Perrone, R.D

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The STAGED-PKD 2-stage adaptive study with a patient enrichment strategy and treatment effect modeling for improved study design efficiency in patients with ADPKD von Perrone, R.D, Hariri, A, Minini, P, Ahn, C, Chapman, A.B, Horie, S, Knebelmann, B, Mrug, M, Ong, A.C.M, Pei, Y.P.C, Torres, V.E, Modur, V, Gansevoort, R.T

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