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

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...

Source: Orphanet Journal of Rare Diseases - June 7, 2019 Category: Internal Medicine Authors: M. Judith Peterschmitt, Selena Freisens, Lisa H. Underhill, Meredith C. Foster, Grace Lewis and Sebastiaan J. M. Gaemers Tags: Research Source Type: research