Real‐world effectiveness of eliglustat in treatment‐naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry

Eliglustat is a first‐line oral therapy for adults with Gaucher disease type 1 (GD1) with extensive, intermediate, or poor CYP2D6‐metabolizer phenotypes (90% of patients). We report real‐world outcomes after 2 years of eliglustat therapy in the International Collaborative Gaucher Group Gaucher Regis...

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Detalles Bibliográficos
Publicado en:Am J Hematol
Main Authors: Mistry, Pramod K., Balwani, Manisha, Charrow, Joel, Kishnani, Priya, Niederau, Claus, Underhill, Lisa H., McClain, Monica R.
Formato: Artigo
Idioma:Inglês
Publicado: John Wiley & Sons, Inc. 2020
Assuntos:
Research Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7497238/
https://ncbi.nlm.nih.gov/pubmed/32438452
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajh.25875
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