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One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

机译：酸性鞘磷脂酶缺乏症患者橄榄酶ALFA酶替代疗法的临床试验的一年结果

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George A. Diaz; Simon A. Jones; Maurizio Scarpa; Karl Eugen Mengel; Roberto Giugliani; Nathalie Guffon; Isabela Batsu; Patricia A. Fraser; Jing Li; Qi Zhang; Catherine Ortemann-Renon;
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1. Preliminary data from first clinical trial of enzyme replacement therapy with olipudase alfa in pediatric patients with chronic visceral and neurovisceral acid sphingomyelinase deficiency [J] . Molecular genetics and metabolism . 2020,第2期

机译：酶替代治疗第一临床试验的初步数据与慢性内脏和神经阳离子酸鞘氨基氨基酶缺乏的儿科蛋白酶ALFA
2. PK/PD model of plasma ceramide in patients with acid sphingomyelinase deficiency following enzyme replacement therapy with olipudase alfa [J] . Li Jing, Kanamalaru Vanaja, Culm-Merdek Kerry, Journal of pharmacokinetics and pharmacodynamics . 2016,第Suppla期

机译：阿尔法寡糖酶替代治疗后酸性鞘磷脂酶缺乏症患者血浆神经酰胺的PK / PD模型
3. Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology and Pharmacology [J] . Chanchala D. Kaddi, Bradley Niesner, Rena Baek, CPT: Pharmacometrics & Systems Pharmacology . 2018,第7期

机译：酸性鞘氨醇酶缺乏症和酶替代疗法Olipudase Alfa的定量系统药理建模是一种将病理生理学和药理学联系起来的创新工具
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6. Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology and Pharmacology [O] . Chanchala D. Kaddi, Bradley Niesner, Rena Baek, 2018

机译：酸性鞘磷脂酶缺乏症和酶替代疗法Olipudase Alfa的定量系统药理建模是一种将病理生理学和药理学联系起来的创新工具
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One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

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