Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications.
Orphanet J Rare Dis
; 9: 112, 2014 Jul 24.
Article
em En
| MEDLINE
| ID: mdl-25056340
ABSTRACT
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doenças Ósseas
/
Terapia de Reposição de Enzimas
/
Doença de Gaucher
Tipo de estudo:
Incidence_studies
/
Risk_factors_studies
Limite:
Adult
/
Humans
País/Região como assunto:
Europa
Idioma:
En
Revista:
Orphanet J Rare Dis
Assunto da revista:
MEDICINA
Ano de publicação:
2014
Tipo de documento:
Article