Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications

@inproceedings{Dussen2014ModellingGD,
  title={Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications},
  author={Laura van Dussen and Marieke Biegstraaten and Marcel G. W. Dijkgraaf and Carla E. M. Hollak},
  booktitle={Orphanet journal of rare diseases},
  year={2014}
}
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. 
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