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Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model

Mutations in GBA, the gene encoding the lysosomal enzyme glucocerebrosidase (GCase), represent the greatest genetic risk factor for developing synucleinopathies including Parkinson’s disease (PD). Additionally, PD patients harboring a mutant GBA allele present with an earlier disease onset and an ac...

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Detalles Bibliográficos
Autores principales:	Viel, Catherine, Clarke, Jennifer, Kayatekin, Can, Richards, Amy M., Chiang, Ming Sum R., Park, Hyejung, Wang, Bing, Shihabuddin, Lamya S., Sardi, S. Pablo
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8536659/ https://www.ncbi.nlm.nih.gov/pubmed/34686711 http://dx.doi.org/10.1038/s41598-021-00404-5

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