Article
Therapeutic goals in Gaucher disease.
La Revue de medecine interne - 1 Mar 2006
Mistry P, Germain D P
Abstract excerpt
Evidence-based therapeutic goals have been developed by several European and American experts in Gaucher disease in order to attempt to reverse the entire Type 1 Gaucher phenotype, prevent complications, improve quality of life and prevent life-threatening complications. Patients with Gaucher disease will benefit by maximum enzyme replacement treatment (ERT) efficiency if it is given at the optimal time i.e....
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