一项收集唯铭赞在中国IVA型黏多糖贮积症患者中安全性和有效性数据的多中心、非干预性研究
[Translation] A multicenter, non-interventional study to collect safety and efficacy data of Weimingzan in Chinese patients with mucopolysaccharidosis type IVA
表征在真实世界环境中观察到的MPS IVA患者使用唯铭赞治疗的安全性;表征唯铭赞在MPS IVA患者中的有效性,特别是在真实世界环境中观察到的对耐力,呼吸功能的影响。
[Translation] To characterize the safety observed in real-world settings in patients with MPS IVA using Vimezan treatment; to characterize the efficacy of Vimezam in MPS IVA patients, especially the observed effects on endurance, respiratory function in real-world settings Influence.
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