Article
Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe.
European journal of internal medicine - 1 Jan 2017
Belmatoug Nadia, Di Rocco Maja, Fraga Cristina, Giraldo Pilar, Hughes Derralynn, Lukina Elena, Maison-Blanche Pierre, Merkel Martin, Niederau Claus, Plӧckinger Ursula, Richter Johan, Stulnig Thomas M, Vom Dahl Stephan, Cox Timothy M
Abstract excerpt
PURPOSE: In Gaucher disease, diminished activity of the lysosomal enzyme, acid β-glucosidase, leads to accumulation of glucosylceramides and related substrates, primarily in the spleen, liver, and bone marrow. Eliglustat is an oral substrate reduction therapy approved in the European Union and the United States as a first-line treatment for adults with type 1 Gaucher disease who have compatible CYP2D6 metabolism...
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