Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study
出版年份 2014 全文链接
标题
Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study
作者
关键词
Enzyme Replacement Therapy, Fabry Disease, Glycogen Storage Disease, Pompe Disease, Longitudinal Cohort Study
出版物
JOURNAL OF INHERITED METABOLIC DISEASE
Volume 37, Issue 6, Pages 945-952
出版商
Springer Nature
发表日期
2014-06-06
DOI
10.1007/s10545-014-9728-1
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。- The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders.
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