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
收藏
页码：830 / 830
页数：1
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