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自我给药酶替代疗法安全性分析:基于法布里病和戈谢病结局调查 ...
该研究旨在探究患者(或其伴侣 / 亲属)自我给药 agalsidase alfa 和 velaglucerase alfa 与医护支持给药相比的安全性概况。研究结果发表在《Orphanet Journal of Rare Diseases》上。 研究人员采用的主要关键技术方法为:从 FOS 和 GOS 登记处收集患者数据,这些数据涵盖了患者 ...
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