Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synuclei...
Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model
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London: Nature Publishing Group UK
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Language
English
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Publisher
London: Nature Publishing Group UK
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Contents
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 accelerated disease progression of both...
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Full title
Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model
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TN_cdi_doaj_primary_oai_doaj_org_article_c4a35b81ff05400cb0b27881077b5aa3
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_c4a35b81ff05400cb0b27881077b5aa3
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ISSN
2045-2322
E-ISSN
2045-2322
DOI
10.1038/s41598-021-00404-5
