Therapy for Gaucher disease: don't stop thinking about tomorrow.
Mol Genet Metab
; 90(2): 122-5, 2007 Feb.
Article
en En
| MEDLINE
| ID: mdl-17084653
ABSTRACT
While enzyme replacement therapy for Gaucher disease has been widely used and appears to be an efficacious and safe treatment, this success should not be a reason for complacency. Other treatment strategies currently under consideration for patients with Gaucher disease include gene therapy, substrate reduction therapy and chaperone therapy. Furthermore, improvements in enzyme therapy could also have a significant clinical impact. Individuals with Gaucher disease and other lysosomal disorders will greatly benefit from continual refinement and optimization of the current therapy, as well as from the development of new treatment modalities that offer improvements in efficacy, cost, safety and availability.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Enfermedad de Gaucher
Límite:
Humans
Idioma:
En
Revista:
Mol Genet Metab
Asunto de la revista:
BIOLOGIA MOLECULAR
/
BIOQUIMICA
/
METABOLISMO
Año:
2007
Tipo del documento:
Article
País de afiliación:
Estados Unidos