The natural course of Gaucher disease in The Netherlands:: Implications for monitoring of disease manifestations
The natural course of Gaucher disease in The Netherlands:: Implications for monitoring of disease manifestations
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DOI：
10.1023/a:1005655031239
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发表时间：
2000-02-01
影响因子：
4.2
通讯作者：
Pöll, R
中科院分区：
文献类型：
--
作者：
Maaswinkel-Mooij, P;Hollak, C;Pöll, R
本研究对20例未经治疗的I型戈谢病患者进行回顾性研究，结果表明，在荷兰患者中，无论是儿童还是成人，大多数患者的I型戈谢病临床表现都是进行性的。这与对犹太病人的研究形成对比。我们的结果强调需要定期随访，以便及时启动酶治疗。
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.