Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1

BACKGROUND: Eliglustat is a first-line oral treatment for adults with Gaucher disease type 1 who have an extensive, intermediate or poor CYP2D6 metabolizer phenotype (> 90% of patients). Whereas enzyme replacement therapy for Gaucher disease has been widely used for more than two decades, eliglus...

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Detalles Bibliográficos
Publicado en:Orphanet J Rare Dis
Main Authors: Peterschmitt, M. Judith, Freisens, Selena, Underhill, Lisa H., Foster, Meredith C., Lewis, Grace, Gaemers, Sebastiaan J. M.
Formato: Artigo
Idioma:Inglês
Publicado: BioMed Central 2019
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Research
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6555985/
https://ncbi.nlm.nih.gov/pubmed/31174576
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-019-1085-6
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