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

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-...

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Shranjeno v:
Bibliografske podrobnosti
izdano v:Orphanet J Rare Dis
Main Authors: van Dussen, Laura, Biegstraaten, Marieke, Dijkgraaf, Marcel GW, Hollak, Carla EM
Format: Artigo
Jezik:Inglês
Izdano: BioMed Central 2014
Teme:
Research
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4226965/
https://ncbi.nlm.nih.gov/pubmed/25056340
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-014-0112-x
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