Enzyme replacement therapy with investigational rhGUS in an infant with non-immune hydrops fetalis and mucopolysaccharidosis type VII

Lau Heather A.; Parmar Swapnil; Kazachkov Mikhail; Shah Rasikial; Wells John; Yachelevich Naomi; Chopra Arun; Haller Christine; Kakkis Emil

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Enzyme replacement therapy with investigational rhGUS in an infant with non-immune hydrops fetalis and mucopolysaccharidosis type VII

机译：用研究性rhGUS对非免疫性胎儿积水和VII型黏多糖贮积症的婴儿进行酶替代治疗

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来源
《Molecular genetics and metabolism 》 |2016年第2期| 共1页
作者
Lau Heather A.; Parmar Swapnil; Kazachkov Mikhail; Shah Rasikial; Wells John; Yachelevich Naomi; Chopra Arun; Haller Christine; Kakkis Emil;
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正文语种 eng
中图分类内分泌腺疾病及代谢病 ;
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1. Enzyme replacement therapy with investigational rhGUS in an infant with non-immune hydrops fetalis and mucopolysaccharidosis type VII [J] . Lau Heather A., Parmar Swapnil, Kazachkov Mikhail, Molecular genetics and metabolism . 2016 ,第2期

机译：用研究性rhGUS对非免疫性胎儿积水和VII型黏多糖贮积症的婴儿进行酶替代治疗
2. Long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis type VII [J] . Molecular genetics and metabolism . 2020 ,第2期

机译：VESTRIDASE ALFA，RHGUS酶替代治疗的长期安全性和疗效，粘性多型型α
3. First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient [J] . Fox Joyce E., Volpe Linda, Bullaro Josephine, Molecular genetics and metabolism . 2015 ,第2期

机译：在MPS VII晚期患者中首次采用人类研究用rhGUS酶替代疗法治疗
4. RETROSPECTIVE IMPLEMENTATION OF QUALITY BY DESIGN FOR LEGACY COMMERCIALIZED ENZYME REPLACEMENT THERAPIES [C] . Anup Agarwal, Eric Hayduk Cell culture engineering XV . 2016

机译：通过设计对传统商业化酶替代疗法进行质量的追溯实施
5. Human hematopoietic progenitor cell-directed gene therapy in a murine xenotransplantation model of mucopolysaccharidosis type VII. [D] . Hofling, August Alexander. 2005

机译：人类造血祖细胞指导的基因治疗在粘多糖贮积症类型VII的小鼠异种移植模型中。
6. First Human Treatment With Investigational rhGUS Enzyme Replacement Therapy in an Advanced Stage MPS VII Patient [O] . Joyce E Fox, Linda Volpe, Josephine Bullaro, -1

机译：在晚期MPS VII患者中首次使用rhGUS酶替代疗法进行人为治疗
7. Correction to: Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials [O] . Yulan Qi, Kathleen McKeever, Julie Taylor, 2018

机译：校正：药代动力学和药效学建模以优化veryrenidase Alfa的剂量，一种酶替代疗法治疗粘性糖尿病患者VII：结果来自三次试验

Enzyme replacement therapy with investigational rhGUS in an infant with non-immune hydrops fetalis and mucopolysaccharidosis type VII

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