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elosulfasealfa(商品名:Vimizim)是2014年2月由美国食品药品监督管理局(FDA)正式批准上市的首个治疗儿童黏多糖贮积症IVA型(mucopolysaccharidosis IVA,MPS IVA)的药物,作为内源性N-乙酰半乳糖胺-6-硫酸酯酶的补充,它能显著改善患者的肌肉强度、关节功能和心肺功能等,提高患者6 min内行走距离,改善患者的生存质量。现对其作用机制、药效学、药动学、临床研究及安全性等作一综述。
Elosulfasealfa (trade name: Vimizim) was the first drug to treat mucopolysaccharidosis IVA (MPS IVA) in the United States Food and Drug Administration (FDA) officially approved in February 2014 as endogenous N-acetylgalactosamine-6-sulfatase supplementation, it can significantly improve the patient’s muscle strength, joint function and heart and lung function, improve patient walking distance within 6 min, improve patient quality of life. Now its mechanism of action, pharmacodynamics, pharmacokinetics, clinical research and safety are reviewed.