Eliglustat tartrate (Genz-112638), a specific inhibitor of glucosylceramide synthase, is under development as an oral substrate reduction therapy for Gaucher disease type 1 (GD1
Key Points
Eliglustat treatment resulted in stable mean hemoglobin concentration, platelet count, and spleen and liver volumes for up to 4 years. Mean bone mineral density z scores also remained stable and were maintained in the healthy reference range for up to 4 years.
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