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Structure and site of action of eliglustat tartrate. Eliglustat binds to glucosylceramide synthase inhibiting the formation of glucosylceramide, the glycolipid that accumulates in Gaucher disease due to low or absent activity of β-glucocerebrosidase. Eliglustat also inhibits the formation of globotriaosylceramide the glycolipid that accumulates in Fabry disease in addition to several gangliosides.
