title: Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications.
abstract: Long-term complications and associated conditions of type 1 Gaucher Disease (GD) can include splenectomy, bone complications, pulmonary hypertension, Parkinson disease and malignancies. Enzyme replacement therapy (ERT) reverses cytopenia and reduces organomegaly. To study the effects of ERT on long-term complications and associated conditions, the course of Gaucher disease was modelled.
authors: Laura
authors: L
authors: van Dussen
authors: Marieke
authors: M
authors: Biegstraaten
authors: Marcel Gw
authors: MG
authors: Dijkgraaf
authors: Carla Em
authors: CE
authors: Hollak
mesh_terms: D000328:Adult; D001847:Bone Diseases / Q000150:complications*; D018450:Disease Progression; D056947:Enzyme Replacement Therapy; D005776:Gaucher Disease / Q000150:complications / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D009426:Netherlands; D013156:Splenectomy