Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial

Eliglustat is a first‐line oral therapy for adults with Gaucher disease type 1 (GD1) and poor, intermediate or extensive CYP2D6‐metabolizer phenotypes (>90% of patients). We report the final results of a Phase 2 trial and extension (NCT00358150) in previously untreated adult GD1 patients who had...

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Detalhes bibliográficos
Publicado no:Am J Hematol
Main Authors: Lukina, Elena, Watman, Nora, Dragosky, Marta, Lau, Heather, Avila Arreguin, Elsa, Rosenbaum, Hanna, Zimran, Ari, Foster, Meredith C., Gaemers, Sebastiaan J. M., Peterschmitt, M. Judith
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons, Inc. 2018
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6587500/
https://ncbi.nlm.nih.gov/pubmed/30264864
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajh.25300
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