%0 Journal Article
%T Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients
%A Bouwien E. Smid
%A Carla E. Hollak
%A Herman S. Overkleeft
%A Johannes M. Aerts
%A Maria J. Ferraz
%A Marri Verhoek
%A Mina Mirzaian
%A Patrick Wisse
%J Archive of "Orphanet Journal of Rare Diseases".
%D 2016
%R 10.1186/s13023-016-0413-3
%X We retrospectively compared biochemical responses in type 1 Gaucher disease patients to treatment with glycosphingolipid synthesis inhibitors miglustat and eliglustat and ERT
%K Gaucher disease
%K Eliglustat
%K Miglustat
%K Chitotriosidase
%K Glucosylsphingosine
%K Glucosylceramide
%K Enzyme replacement therapy
%U https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4806476/