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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Hauptverfasser: 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
Sprache:Inglês
Veröffentlicht: American Society of Hematology 2010
Schlagworte:
Clinical Trials and Observations
Online Zugang: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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