Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.

Journal of Medical Genetics（2017）

引用 265|浏览17

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摘要

NCT00925301; Pre-results.

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关键词

Fabry disease,Pharmacological chaperone,enzyme replacement therapy,lyso-Gb3,lysosomal storage disorder

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