Dataset from A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638

The primary objective of this study was to evaluate the efficacy and safety of once daily
(QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants
with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of
eliglustat tartrate (Genz-112638). The secondary objective was to evaluate the
pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) was administered
QD and BID in participants with Gaucher disease type 1 who had demonstrated clinical
stability on BID dosing of eliglustat tartrate (Genz-112638).