In a previous trial (MOVES-PD, NCT02906020), the glucosylceramide-synthase inhibitor venglustat was investigated in PD patients with a heterozygous GBA-mutation with the aim to lower the substrate for the formation of complex sphingolipids and thereby prevent impairment of lysosomal function and accumulation of toxic αSyn species. The gene discussed is GBA1; the disease is Parkinson disease.