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Glucosylceramide synthase-IN-2 (compound T-690) is a potent, blood-brain barrier-permeable, orally active inhibitor of glucosylceramide synthase (GCS) that acts on human GCS (IC50: 15 nM) and mouse GCS (IC50: 190 nM). Glucosylceramide synthase-IN-2 is capable of non-competitive inhibition of C8 ceramide and UDP glucose.