Study design of STR1VE-EU, a phase 3 trial of AVXS-101 gene-replacement therapy (GRT) in patients with spinal muscular atrophy type 1 (SMA1) in Europe

被引：0

作者：

Muntoni, F. ^{[1
]}

Baranello, G. ^{[2
]}

Bruno, C. ^{[3
]}

Corti, S. ^{[4
]}

Masson, R. ^{[2
]}

Straub, V. ^{[5
]}

Vita, G. ^{[6
]}

Kernbauer, E. ^{[7
]}

Williamson, S. ^{[7
]}

Ouyang, H. ^{[7
]}

Feltner, D. E. ^{[7
]}

Meriggioli, M. ^{[7
]}

Lavrov, A. ^{[7
]}

L'italien, J. ^{[7
]}

Sproule, D. M. ^{[7
]}

Mercuri, E. ^{[8
,9
]}

机构：

[1] UCL, Dept Dev Neurosci, London, England

[2] Fdn IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy

[3] Ist Giannina Gaslini, Translat & Expt Myol Ctr, Genoa, Italy

[4] Univ Milan, IRCCS Fdn Ca Granda Osped Maggiore Policlin, Dept Pathophysiol & Transplantat DEPT, Neurosci Sect,Dino Ferrari Ctr,Neurol Unit, Milan, Italy

[5] Univ Newcastle, MRC Ctr Neuromuscular Dis Inst Genet Med, Newcastle Upon Tyne, Tyne & Wear, England

[6] Messina Univ Hosp, Unit Neurol & Neuromuscular Dis, Messina, Italy

[7] AveXis Inc, Bannockburn, IL USA

[8] Catholic Univ, Dept Paediat Neurol, Rome, Italy

[9] Catholic Univ, Nemo Clin Ctr, Rome, Italy

来源：

EUROPEAN JOURNAL OF NEUROLOGY | 2019年 / 26卷

关键词：

D O I：

暂无

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

EPO3149

引用

页码：830 / 830

页数：1

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