期刊
JOURNAL OF INHERITED METABOLIC DISEASE
卷 23, 期 1, 页码 77-82出版社
KLUWER ACADEMIC PUBL
DOI: 10.1023/A:1005655031239
关键词
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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.
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