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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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Publicado no: | Orphanet J Rare Dis |
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Main Authors: | , , , |
Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
BioMed Central
2014
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Assuntos: | |
Acesso em linha: | 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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