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Dec. 10, 2021

Venglustat: A Brain-Penetrant GCS Inhibitor for GBA-Mutant Parkinson’s Disease


potential BIC oral GCS inhibitor Ph. III in genetic diseases (15 mg QD) brain-penetrant, allosteric inhibitor Scientific Reports Sanofi

Drug Hunter Team

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 one year in early GBA -PD patients and was discontinued in this indication [...]



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