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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Dettagli Bibliografici
Pubblicato in:Orphanet J Rare Dis
Autori principali: van Dussen, Laura, Biegstraaten, Marieke, Dijkgraaf, Marcel GW, Hollak, Carla EM
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2014
Soggetti:
Research
Accesso online: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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