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

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

Abstract 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,...

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Bibliographic Details
Main Authors: M. Judith Peterschmitt, Selena Freisens, Lisa H. Underhill, Meredith C. Foster, Grace Lewis, Sebastiaan J. M. Gaemers
Format: Article
Language:English
Published: BMC 2019-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Gaucher disease type 1
Acid β-glucosidase deficiency
Eliglustat
Cerdelga
Substrate reduction therapy
Safety
Online Access:http://link.springer.com/article/10.1186/s13023-019-1085-6

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http://link.springer.com/article/10.1186/s13023-019-1085-6

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