Eliglustat

Eliglustat is a potent and specific inhibitor of glucosylceramide synthase. Eliglustat is a new promising substrate reduction therapy (SRT) for Gaucher disease (GD). Eliglustat works by inhibiting UDP-glucosylceramide synthase, the first enzyme that catalyzes the biosynthesis of glycosphingolipids, thus reducing the load of glucosylceramide influx into the lysosome.