Onasemnogene abeparvovec gene therapy in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update

被引：1

作者：

Strauss, K. ^{[1
]}

Farrar, M. ^{[2
]}

Swoboda, K. ^{[3
]}

Saito, K. ^{[4
]}

Chiriboga, C. ^{[5
]}

Finkel, R. ^{[6
]}

Iannaccone, S. ^{[7
]}

Krueger, J. ^{[8
]}

Kwon, J. ^{[9
]}

McMillan, H. ^{[10
]}

Servais, L. ^{[11
]}

Mendell, J. ^{[12
]}

Parsons, J. ^{[13
]}

Scoto, M. ^{[14
]}

Shieh, P. ^{[15
]}

Zaidman, C. ^{[16
]}

Schultz, M. ^{[17
]}

Ogrinc, F. ^{[17
]}

Group, S.

Muntoni, F. ^{[14
]}

机构：

[1] Childrens Hosp Pittsburgh, Strasburg, PA USA

[2] Sydney Childrens Hosp, Randwick, NSW, Australia

[3] MA Gen Hosp Children, Boston, MA USA

[4] TWMU, Tokyo, Japan

[5] Columbia Med Ctr, New York, NY USA

[6] St Jude Childrens Res Hosp, Memphis, TN USA

[7] Southwestern Med Ctr, Dallas, TX USA

[8] Helen DeVos Childrens Hosp, Grand Rapids, MI USA

[9] Univ Wisconsin, Madison, WI 53706 USA

[10] CHEO, Ottawa, ON, Canada

[11] MDUK Oxford Neuromuscular Ctr, Oxford, England

[12] Nationwide Childrens Hosp, Columbus, OH USA

[13] Univ Colorado, Aurora, CO USA

[14] Great Ormond St Hosp Sick Children, London, England

[15] Univ Calif Los Angeles, Los Angeles, CA USA

[16] Washington Univ, St Louis, MO 63110 USA

[17] AveXis Inc, Bannockburn, IL USA

来源：

NEUROMUSCULAR DISORDERS | 2020年 / 30卷

关键词：

D O I：

10.1016/j.nmd.2020.08.259

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P.260

引用

页码：S122 / S122

页数：1