Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study

Schulz Angela; Specchio Nicola; Gissen Paul; de los Reyes Emily; Cahan Heather; Slasor Peter; Ajayi Temitayo; Jacoby David

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Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study

机译：患有Cln2疾病儿童Cerliponase Alfa的长期安全性和疗效替代治疗：持续多中心，跨国推广研究中的临时结果

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来源
《Molecular genetics and metabolism》 |2017年第2期|共1页
作者
Schulz Angela; Specchio Nicola; Gissen Paul; de los Reyes Emily; Cahan Heather; Slasor Peter; Ajayi Temitayo; Jacoby David;
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作者单位

Univ Med Ctr Hamburg Eppendorf Hamburg Germany;

Bambino Gesu Childrens Hosp IRCCS Rome Italy;

Great Ormond St Hosp Sick Children London England;

Ohio State Univ Nationwide Childrens Hosp Columbus OH 43210 USA;

BioMarin Pharmaceut Inc Novato CA USA;

BioMarin Pharmaceut Inc Novato CA USA;

BioMarin Pharmaceut Inc Novato CA USA;

BioMarin Pharmaceut Inc Novato CA 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;
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1. Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study [J] . Schulz Angela, Specchio Nicola, Gissen Paul, Molecular genetics and metabolism . 2017,第1a2期

机译：患有Cln2疾病儿童Cerliponase Alfa的长期安全性和疗效替代治疗：持续多中心，跨国推广研究中的临时结果
2. Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study [J] . Schulz Angela, Specchio Nicola, Gissen Paul, Molecular genetics and metabolism . 2017,第1a2期

机译：患有Cln2疾病儿童Cerliponase Alfa的长期安全性和疗效替代治疗：持续多中心，跨国推广研究中的临时结果
3. Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: Two year results from an ongoing multicenter extension study [J] . Schulz Angela, Specchio Nicola, Gissen Paul, Molecular genetics and metabolism . 2018,第2期

机译：在CLN2疾病儿童中脑内酶酶替代疗法替代治疗的长期安全性和疗效：持续多中心扩展研究的两年结果
4. 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
5. Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration [O] . Aryun Kim, Anita Grover, Kevin Hammon, 2021

机译：Cerliponase Alfa的临床药代动力学和药效学Intracebrinallary施用Crn2疾病酶替代疗法
6. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study [O] . Loren D.M. Pena, Richard J. Barohn, Barry J. Byrne, 2019

机译：新型酶替代治疗的安全性，可耐药性，药代动力学，药效学和探索性疗效脂肪溶酶酶Alfa（Neogaa）治疗 - 幼稚和alglucosidase Alfa治疗的晚期疾病患者：1期，开放标签，多中心，跨国，上升剂量学习

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study

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