The GlcCer synthase inhibitor, venglustat (GZ/SAR402671), is safe and well-tolerated in GBA1-PD patients but did not meet its trial's primary endpoint in PD patients and development has been halted as of January 2021 (ClinicalTrials.gov identifier: NCT02906020) (45). The gene discussed is GBA1; the disease is Parkinson disease.