[Enzyme replacement therapy of Fabry's disease: the French experience].

Abstract excerpt

This study describes the experience of one to five years of follow-up of 43 patients (35 men, 8 women, 40% have less than 18 years to the moment of the diagnostic) after treatment of Fabry's disease by Fabrazyme. A simple self-report questionnaire was developed in order to measure the effectiveness of the treatment on pain (present in the vast majority of patients since many years), activity, heat tolerance,...

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