Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry

Eliglustat is a recently approved oral therapy in the United States and Europe for adults with Gaucher disease type 1 who are CYP2D6 extensive, intermediate, or poor metabolizers (> 90% of patients) that has been shown to decrease spleen and liver volume and increase hemoglobin concentrations and...

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Опубликовано в: :Mol Genet Metab Rep
Главные авторы: Ibrahim, Jennifer, Underhill, Lisa H., Taylor, John S., Angell, Jennifer, Peterschmitt, M. Judith
Формат: Artigo
Язык:Inglês
Опубликовано: Elsevier 2016
Предметы:
Short Communication
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4927653/
https://ncbi.nlm.nih.gov/pubmed/27408819
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2016.06.003
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