Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)
出版年份 2017 全文链接
标题
Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)
作者
关键词
Hunter syndrome, Lysosomal storage disease, Idursulfase, Efficacy, Disease registry
出版物
Orphanet Journal of Rare Diseases
Volume 12, Issue 1, Pages -
出版商
Springer Nature
发表日期
2017-10-04
DOI
10.1186/s13023-017-0712-3
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS)
- (2017) Barbara K. Burton et al. JOURNAL OF INHERITED METABOLIC DISEASE
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- Diagnosis, quality of life, and treatment of patients with Hunter syndrome in the French healthcare system: a retrospective observational study
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- Clinical efficacy of Enzyme Replacement Therapy in paediatric Hunter patients, an independent study of 3.5 years
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- A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4–7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy
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- Clinical and Ultrasonographic Measurement of Liver Size in Normal Children
- (2013) Puja Amatya et al. INDIAN JOURNAL OF PEDIATRICS
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- The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5years and older treated with intravenous idursulfase
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- (2011) Annerose Keilmann et al. JOURNAL OF INHERITED METABOLIC DISEASE
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