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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Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Hematology 2010
Soggetti:
Clinical Trials and Observations
Accesso online: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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