potential BIC oral GCS inhibitor

Ph. III in genetic diseases (15 mg QD)

brain-penetrant, allosteric inhibitor

Scientific Reports


Chemical structure of molecule Venglustat Sanofi glucosylceramide synthase (GCS) inhibitor

The Sanofi glucosylceramide synthase (GCS) inhibitor, venglustat, is an oral, brain-penetrant clinical candidate for GBA-mutant Parkinson’s disease and other diseases where GBA-mutations are relevant. Though the molecule did not demonstrate efficacy within…

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