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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Autores principales: 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
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Hematology 2010
Materias:
Clinical Trials and Observations
Acceso en línea: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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