Nusinersen in Infants Who Initiatte Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study

被引：0
|
作者：
De Vivo, Darryl C. [1 ,2 ]
Topaloglu, Haluk [3 ]
Swoboda, Kathryn J. [4 ,5 ]
Bertini, Enrico [6 ]
Hwu, Wuh-Liang [7 ]
Crawford, Thomas O. [8 ]
Foster, Richard [9 ]
Bhan, Ishir [10 ]
Fradette, Stephanie [10 ]
Farwell, Wildon [10 ]
Reyna, Sandra P. [10 ]
机构：
[1] Columbia Univ, Irving Med Ctr, Dept Neurol, New York, NY USA
[2] Columbia Univ, Dept Pediat, Irving Med Ctr, New York, NY 10027 USA
[3] Hacettepe Univ, Dept Pediat Neurol, Ankara, Turkey
[4] Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USA
[5] Massachusetts Gen Hosp, Dept Genet, Boston, MA 02114 USA
[6] Postgrad Bambino Gesu Childrens Res Hosp, Unit Neuromuscular & Neurodegenerat Disorders, Rome, Italy
[7] Natl Taiwan Univ Hosp, Dept Med Genet & Pediat, Taipei, Taiwan
[8] Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA
[9] Biogen, Maidenhead, Berks, England
[10] Biogen, Cambridge, MA USA
来源：
NEUROLOGY | 2019年 / 92卷 / 15期
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：
S25.001
引用
收藏
页数：3
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