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.

MJ, Peterschmitt; H, Saiki; T, Hatano; T, Gasser; SH, Isaacson; SJM, Gaemers; P, Minini; S, Saubadu; J, Sharma; S, Walbillic; RN, Alcalay; G, Cutter; N, Hattori; GU, Höglinger; K, Marek; AHV, Schapira; CR, Scherzer; T, Simuni; N, Giladi; SP, Sardi; TZ, Fischer

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.

Shared by NobleBlocks on Jan 1, 2022 • 12:00 AM UTC

Authors:

Peterschmitt MJ

Saiki H

Hatano T

Abstract

<h4>Background</h4>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.<h4>Objective</h4>Ev...

Subject

Parkinson’s Disease

Glucosylceramide (Gl-1)

Glucosylceramide Synthase (Gcs) Inhibition

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