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
in 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.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Bone Diseases
/
Enzyme Replacement Therapy
/
Gaucher Disease
Type of study:
Incidence_studies
/
Risk_factors_studies
Limits:
Adult
/
Humans
Country/Region as subject:
Europa
Language:
En
Journal:
Orphanet J Rare Dis
Journal subject:
MEDICINA
Year:
2014
Document type:
Article