Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG)

被引：4

作者：

Finkel, R. ^{[1
]}

Day, J. ^{[2
]}

Darras, B. ^{[3
]}

Kuntz, N. ^{[4
]}

Connolly, A. ^{[5
]}

Crawford, T. ^{[6
]}

Butterfield, R. ^{[7
]}

Shieh, P. ^{[8
]}

Tennekoon, G. ^{[9
]}

Iannaccone, S. ^{[10
]}

Ogrinc, F. ^{[11
]}

Kavanagh, S. ^{[11
]}

Kernbauer, E. ^{[11
]}

Whittle, J. ^{[11
]}

L'Italien, J. ^{[11
]}

Kaspar, B. ^{[11
]}

Sproule, D. ^{[11
]}

Spector, S. ^{[11
]}

Feltner, D. ^{[11
]}

Mendell, J. ^{[12
]}

机构：

[1] Nemours Childrens Hosp, Orlando, FL USA

[2] Stanford Univ, Stanford, CA 94305 USA

[3] Boston Childrens Hosp, Boston, MA USA

[4] Lurie Childrens Hosp, Chicago, IL USA

[5] WashU St Louis, St Louis, MO USA

[6] Johns Hopkins Univ, Baltimore, MD USA

[7] Univ Utah Hlth, Salt Lake City, UT USA

[8] Univ Calif Los Angeles, Sch Med, Los Angeles, CA USA

[9] CHOP, Philadelphia, PA USA

[10] UT Southwestern Med Ctr, Dallas, TX USA

[11] AveXis Inc, Bannockburn, IL USA

[12] Nationwide Childrens Hosp, Columbus, OH USA

来源：

关键词：

D O I：

10.1016/j.nmd.2019.06.594

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

O.40

引用

页码：S207 / S207

页数：1