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TP-060 is an inhibitor of UDP-glucose ceramide glucosyltransferase (IC50 = 0.031 µM).1 It is selective for UDP-glucose ceramide glucosyltransferase over human ether-a-go-go (hERG) at 10 µM but also inhibits serotonin receptors (5-HT; IC50 = 0.31 µM). In vivo, TP-060 (30 mg/kg per day) decreases the cerebral cortex and serum levels of glucosylceramide and glucosylsphingosine in a Gba D409V transgenic mouse model of Gaucher disease.2
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