Onasemnogene Abeparvovec Gene Therapy in Presymptomatic spinal muscular atrophy (SMA): SPR1NT study update in children with 3 Copies of SMN2

被引：2

作者：

Strauss, K. ^{[1
]}

Muntoni, F. ^{[2
]}

Farrar, M. ^{[3
]}

Saito, K. ^{[4
]}

Mendell, J. ^{[5
]}

Servais, L. ^{[6
]}

McMillan, H. ^{[7
]}

Swoboda, K. ^{[8
]}

Kwon, J. ^{[9
]}

Zaidman, C. ^{[10
]}

Chiriboga, C. ^{[11
]}

Iannaccone, S. ^{[12
]}

Krueger, J. ^{[13
]}

Parsons, J. ^{[14
]}

Shieh, P. ^{[15
]}

Kavanagh, S. ^{[16
]}

Chand, D. ^{[16
]}

Tauscher-Wisniewski, S. ^{[16
]}

Macek, T. ^{[16
]}

机构：

[1] Clin Special Children, Strasburg, PA USA

[2] UCL, London, England

[3] Sydney Childrens Hosp Network, Randwick, NSW, Australia

[4] Tokyo Womens Med Univ, Tokyo, Japan

[5] Nationwide Childrens Hosp, Columbus, OH USA

[6] MDUK Oxford Neuromuscular Ctr, Oxford, England

[7] Childrens Hosp Eastern Ontario, Ottawa, ON, Canada

[8] Massachusetts Gen Hosp, Boston, MA 02114 USA

[9] Univ WI, Sch Med & Publ Hlth, Madison, WI USA

[10] Washington Univ, Sch Med, St Louis, MO USA

[11] Columbia Univ, Med Ctr, New York, NY USA

[12] Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA

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

[14] Univ Colorado, Sch Med, Aurora, CO USA

[15] Univ Calif Los Angeles, David Geffen Sch Med, Los Angeles, CA 90095 USA

[16] Novartis Gene Therapies, Bannockburn, IL USA

来源：

NEUROMUSCULAR DISORDERS | 2021年 / 31卷

关键词：

D O I：

10.1016/j.nmd.2021.07.299

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

EP.274

引用

页码：S133 / S133

页数：1

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