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Pyrazole Ureas as Low Dose, CNS Penetrant Glucosylceramide Synthase Inhibitors for the Treatment of Parkinson’s Disease

12 Jan 2023

Abstract excerpt

Parkinson’s disease is the second most prevalent progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons in the substantia nigra. Loss-of-function mutations in GBA, the gene that encodes for the lysosomal enzyme glucosylcerebrosidase, are a major genetic risk factor for the development of Parkinson’s disease potentially through the accumulation of glucosylceramide and...

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PMID: 36793422
DOI: 10.1021/acsmedchemlett.2c00441

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