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An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naiuml;ve to enzyme replacement therapy

机译：用于法布里疾病的儿童AgAlsidase Alfa酶替代疗法的开放标签临床试验，该临床疾病是幼稚疾病患者酶替代疗法

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Ozlem Goker-Alpan; Nicola Longo; Marie McDonald; Suma Shankar; Raphael Schiffmann; Peter Chang; Yinghua Shen; Arian Pano;
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1. An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are na?ve to enzyme replacement therapy [J] . Ozlem Goker-Alpan, Nicola Longo, Marie McDonald, Drug Design, Development and Therapy . 2016,第5期

机译：阿糖苷酶α酶替代治疗对不愿意进行酶替代治疗的法布里儿童的开放标签临床试验
2. Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa [J] . D A Hughes, P M Elliott, J Shah, Heart . 2008,第2期

机译：酶替代疗法对安德森-法布里病心肌病的影响：阿加糖酶α的随机，双盲，安慰剂对照临床试验
3. Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1‐year Phase 1/2 clinical trial [J] . Schiffmann Raphael, Goker‐Alpan Ozlem, Holida Myrl, Journal of inherited metabolic disease . 2019,第3期

机译：Pegunigalsidase Alfa，一种用于法布里疾病的新型聚乙二醇化酶替代疗法，提供了持续的血浆浓度和有利的药效学：1年期1/2临床试验
4. Endothelial Targeted Enzyme Replacement Therapy for Fabry Disease [C] . D. Serrano, J. Hsu, K.V.N. Kumar, Annual meeting exposition of the Controlled Release Society . 2009

机译：Fabry病的内皮靶向酶替代治疗
5. Concurrent validity and responsiveness of the Peabody Developmental Motor Scales-2 in infants and children with Pompe disease undergoing enzyme replacement therapy [D] . Phillips, Dawn. 2012

机译：皮博迪发育运动量表2在接受酶替代疗法的庞贝病婴儿和儿童中的并发有效性和反应性
6. An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy [O] . Ozlem Goker-Alpan, Nicola Longo, Marie McDonald, 2016

机译：阿糖苷酶α酶替代疗法在未接受酶替代疗法的法布里儿童中的开放标签临床试验
7. Clinical observations on enzyme replacement therapy in patients with Fabry disease and the switch from agalsidase beta to agalsidase alfa [O] . Hsiang-Yu Lin, Yu-Hsiu Huang, Hsuan-Chieh Liao, 2014

机译：法布里病患者酶替代疗法及从阿加糖酶β转换为阿加糖酶α的临床观察
8. Pilot Scale Isolation of Ceramide Trihexosidase from Human Plasma and Clinical Evaluation of Enzyme Replacement Therapy in Fabry's Disease. [R] . sweeley,charles c. 1975

机译：人体血浆中神经酰胺三己糖苷酶的中试分离及法布里氏病酶替代疗法的临床评价。

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naiuml;ve to enzyme replacement therapy

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