P. Maaswinkel-mooij et al., The natural course of Gaucher disease in The Netherlands: Implications formonitoring of disease manifestations, J INH MET D, 23(1), 2000, pp. 77-82
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.