The natural course of Gaucher disease in The Netherlands: Implications formonitoring of disease manifestations

This retrospective study in 20 untreated type I Gaucher disease patients sh ows that in Dutch patients clinical manifestations of Gaucher disease type I are progressive in the majority of patients, children as well as adults. This is in contrast with studies among Jewish patients. Our results emphasi ze the need for a regular follow-up to enable timely initiation of enzyme t herapy.