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- 2016
Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patientsDOI: 10.1186/s13023-016-0413-3 Keywords: Gaucher disease, Eliglustat, Miglustat, Chitotriosidase, Glucosylsphingosine, Glucosylceramide, Enzyme replacement therapy Abstract: We retrospectively compared biochemical responses in type 1 Gaucher disease patients to treatment with glycosphingolipid synthesis inhibitors miglustat and eliglustat and ERT
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