Nusinersen in infants who initiate treatment in a presymptomatic stage of spinal muscular atrophy: interim results from the phase 2 NURTURE study

被引：0

作者：

Ryan, M. ^{[1
]}

De Vivo, D. ^{[2
]}

Bertini, E. ^{[3
]}

Hwu, W. ^{[4
]}

Crawford, T. ^{[5
]}

Swoboda, K. ^{[6
]}

Finkel, R. ^{[7
]}

Kirschner, J. ^{[8
]}

Kuntz, N. ^{[9
]}

Parsons, J. ^{[10
]}

Butterfield, R. ^{[11
]}

Topaloglu, H. ^{[12
]}

Ben Omran, T. ^{[13
]}

Sansone, V. ^{[14
]}

Jong, Y. ^{[15
]}

Shu, F. ^{[16
]}

Foster, R. ^{[17
]}

Bhan, I. ^{[18
]}

Fradette, S. ^{[18
]}

Farwell, W. ^{[18
]}

机构：

[1] Royal Childrens Hosp, Melbourne, Vic, Australia

[2] Columbia Univ, New York, NY USA

[3] Postgrad Bambino Gesu, Rome, Italy

[4] Natl Taiwan Univ Hosp, Taipei, Taiwan

[5] Johns Hopkins Univ, Baltimore, MD USA

[6] Ctr Genom Med, Boston, MA USA

[7] Nemours Childrens Hosp, Orlando, FL USA

[8] Univ Hosp Bonn, Bonn, Germany

[9] Ann & Robert H Lurie, Chicago, IL USA

[10] Childrens Hosp Colorado, Aurora, CO USA

[11] Univ Utah, Salt Lake City, UT USA

[12] Hacettepe Univ, Ankara, Turkey

[13] Weill Cornell Med Coll, Doha, Qatar

[14] Neuromuscular Omniserv, Milan, Italy

[15] Inst Clin Med, Kaohsiung, Taiwan

[16] David Geffen Sch Med, Los Angeles, CA USA

[17] Biogen, Maidenhead, Berks, England

[18] Biogen, Cambridge, MA USA

来源：

NEUROMUSCULAR DISORDERS | 2019年 / 29卷

关键词：

D O I：

10.1016/j.nmd.2019.06.518

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P.356

引用

页码：S185 / S185

页数：1