Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications.
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ABSTRACT:
SUBMITTER: van Dussen L
PROVIDER: S-EPMC4226965 | biostudies-literature | 2014
REPOSITORIES: biostudies-literature
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Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications.
van Dussen Laura L Biegstraaten Marieke M Dijkgraaf Marcel Gw MG Hollak Carla Em CE
Orphanet journal of rare diseases 20140724
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. ...[more]
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