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

Eliglustat tartrate is an investigational oral substrate reduction therapy for Gaucher disease type 1 that is pharmacologically distinct from intravenous enzyme replacement therapy. Eliglustat tartrate improved clinical manifestations in patients who received 50 or 100 mg twice daily for 1 year duri...

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Κύριοι συγγραφείς: Lukina, Elena, Watman, Nora, Arreguin, Elsa Avila, Dragosky, Marta, Iastrebner, Marcelo, Rosenbaum, Hanna, Phillips, Mici, Pastores, Gregory M., Kamath, Ravi S., Rosenthal, Daniel I., Kaper, Mathilde, Singh, Tejdip, Puga, Ana Cristina, Peterschmitt, M. Judith
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: American Society of Hematology 2010
Θέματα:
Clinical Trials and Observations
Διαθέσιμο Online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2993616/
https://ncbi.nlm.nih.gov/pubmed/20713962
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2010-06-293902
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