title: The natural course of Gaucher disease in The Netherlands: implications for monitoring of disease manifestations.
abstract: This retrospective study in 20 untreated type I Gaucher disease patients shows 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 emphasize the need for a regular follow-up to enable timely initiation of enzyme therapy.
authors.affiliation: Department of Pediatrics, Leiden University Medical Centre, The Netherlands.
authors.forename: P
authors.initials: P
authors.lastname: Maaswinkel-Mooij
authors.forename: C
authors.initials: C
authors.lastname: Hollak
authors.forename: M
authors.initials: M
authors.lastname: van Eysden-Plaisier
authors.forename: M
authors.initials: M
authors.lastname: Prins
authors.forename: H
authors.initials: H
authors.lastname: Aerts
authors.forename: R
authors.initials: R
authors.lastname: Pöll
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D005776:Gaucher Disease / Q000150:complications*; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies