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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2018
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6587500/
https://www.ncbi.nlm.nih.gov/pubmed/30264864
http://dx.doi.org/10.1002/ajh.25300

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