Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson’s Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial

Peterschmitt, Michel; behalf of the MOVES-PD Investigators, on; Saiki, Hidemoto; Hatano, Taku; Gasser, Thomas; Isaacson, Stuart; J.M. Gaemers, Sebastiaan; Minini, Pascal; Saubadu, Stéphane; Sharma, Jyoti; Walbillic, Samantha; N. Alcalay, Roy; Cutter, Gary; Hattori, Nobutaka; U. Höglinger, Günter; Marek, Kenneth; H.V. Schapira, Anthony; R. Scherzer, Clemens; Simuni, Tanya; Giladi, Nir; Pablo Sardi, S.; Fischer, Tanya

doi:10.3233/jpd-212714

Public

Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson’s Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial

Published in Journal of Parkinson s Disease • Dec 10, 2021

Authors:

Michel Peterschmitt

on behalf of the MOVES-PD Investigators

Hidemoto Saiki

Abstract

BACKGROUND: Glucocerebrosidase gene (GBA) mutations influence risk and prognosis of Parkinson's disease (PD), possibly through accumulation of glycosphingolipids, including glucosylceramide (GL-1). Venglustat is a novel, brain penetrant glucosylceramide synthase inhibitor. OBJECTIVE: Evaluate venglu...

Subject

Parkinson's disease

Pharmacodynamics

Medicine

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