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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
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...
Autores principales: | 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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
IOS Press
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8925113/ https://www.ncbi.nlm.nih.gov/pubmed/34897099 http://dx.doi.org/10.3233/JPD-212714 |
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