Article
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.
Journal of Parkinson's disease - 1 Jan 2022
Peterschmitt M Judith, Saiki Hidemoto, Hatano Taku, Gasser Thomas, Isaacson Stuart H, Gaemers Sebastiaan J M, Minini Pascal, Saubadu Stéphane, Sharma Jyoti, Walbillic Samantha, Alcalay Roy N, Cutter Gary, Hattori Nobutaka, Höglinger Günter U, Marek Kenneth, Schapira Anthony H V, Scherzer Clemens R, Simuni Tanya, Giladi Nir, Sardi Sergio Pablo, Fischer Tanya Z
Abstract excerpt
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 venglustat pharmacology, safety, and tolerability in patients with PD and GBA mutations (GBA-PD). METHODS: Part 1 of the...
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