Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients

We retrospectively compared biochemical responses in type 1 Gaucher disease patients to treatment with glycosphingolipid synthesis inhibitors miglustat and eliglustat and ERT.

Source: Orphanet Journal of Rare Diseases - March 24, 2016 Category: Internal Medicine Authors: Bouwien E. Smid, Maria J. Ferraz, Marri Verhoek, Mina Mirzaian, Patrick Wisse, Herman S. Overkleeft, Carla E. Hollak and Johannes M. Aerts Source Type: research