A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1

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). A multinational, open-label, single-arm phase 2 study of 26 GD1 patients (16 female, 10 male; mean age, 34 years) evalua...

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Main Authors: Lukina, Elena, Watman, Nora, Arreguin, Elsa Avila, Banikazemi, Maryam, Dragosky, Marta, Iastrebner, Marcelo, Rosenbaum, Hanna, Phillips, Mici, Pastores, Gregory M., Rosenthal, Daniel I., Kaper, Mathilde, Singh, Tejdip, Puga, Ana Cristina, Bonate, Peter L., Peterschmitt, M. Judith
Format: Artigo
Jezik:Inglês
Izdano: American Society of Hematology 2010
Teme:
Clinical Trials and Observations
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2924227/
https://ncbi.nlm.nih.gov/pubmed/20439622
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2010-03-273151
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