Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial

Harding Cary O.; Amato R. Stephen; Stuy Mary; Longo Nicola; Burton Barbara K.; Posner John; Weng Haoling H.; Merilainen Markus; Gu Zhonghua; Jiang Joy; Vockley Jerry

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Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial

机译：Pegvaliase用于治疗苯丙酮尿：枢轴，双盲随机停药期3临床试验

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Introduction: Pegvaliase is a recombinant Anabaena variabilis phenylalanine ammonia lyase (PAL) enzyme under investigation for treatment of adult phenylketonuria (PKU). This manuscript describes results of a randomized discontinuation trial (RDT) designed to evaluate the effects of pegvaliase treatment on blood phenylalanine (Phe) and neuropsychiatric outcomes in adults with PKU.

机译：介绍：PegvaliaSe是一种重组Anabaena Variabilis苯丙氨酸氨酶（PAL）酶，用于治疗成人苯醌（PKU）。该手稿描述了随机停止试验（RDT）的结果，旨在评估PEGValiaSe治疗对血苯丙氨酸（PHE）和PKU中的神经精神应结果的影响。

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来源
《Molecular genetics and metabolism》 |2018年第1期|共7页
作者
Harding Cary O.; Amato R. Stephen; Stuy Mary; Longo Nicola; Burton Barbara K.; Posner John; Weng Haoling H.; Merilainen Markus; Gu Zhonghua; Jiang Joy; Vockley Jerry;
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作者单位

Oregon Hlth &

Sci Univ Mol &

Med Genet 3181 SW Sam Jackson Pk Rd Portland OR 97239 USA;

Univ Kentucky Med Genet &

Metab 138 Leader Ave 119-130 Lexington KY 40506 USA;

Indiana Univ Dept Med &

Mol Genet 975 W Walnut St Res &

Lib Bldg IB 130 Indianapolis IN 46202;

Univ Utah Div Med Genet Dept Pediat 295 Chipeta Way Salt Lake City UT 84108 USA;

Ann &

Robert H Lurie Childrens Hosp Chicago Div Genet Birth Defects &

Metab Dept Pediat 225 E;

Kings Coll London Strand Dept Life Sci &

Med Franklin Wilkins Bldg Stamford St London England;

BioMarin Pharmaceut Inc 105 Digital Dr Novato CA 94949 USA;

BioMarin Pharmaceut Inc 105 Digital Dr Novato CA 94949 USA;

BioMarin Pharmaceut Inc 105 Digital Dr Novato CA 94949 USA;

BioMarin Pharmaceut Inc 105 Digital Dr Novato CA 94949 USA;

Univ Pittsburgh Div Med Genet Dept Pediat 4401 Penn Ave Pittsburgh PA 15224 USA;

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原文格式 PDF
正文语种 eng
中图分类内分泌腺疾病及代谢病;
关键词
Phenylketonuria; PKU; Recombinant Anabaena variabilis PEGylated phenylalanine ammonia lyase; Pegvaliase;

机译：phopyloNuria;pku;重组Anabaena Variabilis Pegymated苯丙氨酸氨裂解酶;PegvaliaSe;

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1. Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial [J] . Harding Cary O., Amato R. Stephen, Stuy Mary, Molecular genetics and metabolism . 2018,第1期

机译：Pegvaliase用于治疗苯丙酮尿：枢轴，双盲随机停药期3临床试验
2. PHASE 3 PRISM CLINICAL TRIALS: EVALUATING CHANGE IN DIET WITH PEGVALIASE TREATMENT IN ADULTS WITH PHENYLKETONURIA [J] . Rohr Fran, Burton Barbara K., Longo Nicola, Molecular genetics and metabolism . 2019,第3期

机译：第3阶段棱镜临床试验：在苯丙酮尿中的成人中评估饮食变化
3. PHASE 3 PRISM CLINICAL TRIALS: EVALUATING CHANGE IN DIET WITH PEGVALIASE TREATMENT IN ADULTS WITH PHENYLKETONURIA [J] . Rohr Fran, Burton Barbara K., Longo Nicola, Molecular genetics and metabolism . 2019,第3期

机译：第3阶段棱镜临床试验：在苯丙酮尿中的成人中评估饮食变化
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5. Phase II double-blinded randomized controlled clinical trial of chondroitinase ABC by intraspinal injection for treatment of severe chronic spinal cord injury in 60 pet dogs. [D] . Hu, Hilary. 2016

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6. Association of immune response with efficacy and safety outcomes in adults with phenylketonuria administered pegvaliase in phase 3 clinical trials [O] . Soumi Gupta, Kelly Lau, Cary O. Harding, 2018

机译：成人苯丙酮酸尿症患者使用pegvaliase的免疫应答与功效和安全性结果的3期临床试验相关性
7. Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial [O] . Cary O. Harding, R. Stephen Amato, Mary Stuy, 2018

机译：pegvaliase用于治疗苯酮尿剂：枢轴，双盲随机停药期3临床试验
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Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial

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