Article
A new severity score index for phenotypic classification and evaluation of responses to treatment in type I Gaucher disease.
Haematologica - 1 Aug 2008
Di Rocco Maja, Giona Fiorina, Carubbi Francesca, Linari Silvia, Minichilli Fabrizio, Brady Roscoe O, Mariani Giuliano, Cappellini Maria Domenica
Abstract excerpt
BACKGROUND: Gaucher disease is the first lysosomal storage disease for which specific therapy became available. Over 4800 patients have been treated with enzyme replacement therapy. Analysis of Gaucher disease registry data has outlined the clinical heterogeneity of the disease and the different responses to treatment from patient to patient, and for different organs. This variability in clinical response...
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