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Title: Property-based design of a glucosylceramide synthase inhibitor that reduces glucosylceramide in the brain.
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Title: Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
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Title: Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study.
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Title: Eliglustat tartrate, an orally active glucocerebroside synthase inhibitor for the potential treatment of Gaucher disease and other lysosomal storage diseases.
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Title: A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.
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Title: Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy.
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Title: Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease.
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Title: A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease.
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