Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update

被引：0

作者：

Day, John W. ^{[1
]}

Chiriboga, Claudia A. ^{[2
]}

Crawford, Thomas O. ^{[3
]}

Darras, Basil T. ^{[4
]}

Finkel, Richard S. ^{[5
]}

Connolly, Anne M. ^{[6
,7
,8
,9
]}

Iannaccone, Susan T. ^{[10
]}

Kuntz, Nancy L. ^{[11
]}

Pena, Loren D. M. ^{[12
]}

Shieh, Perry B. ^{[13
]}

Smith, Edward C. ^{[14
]}

Schultz, Meredith ^{[15
]}

Feltner, Douglas E. ^{[15
]}

Tauscher-Wisniewski, Sitra ^{[15
]}

Ogrinc, Francis G. ^{[15
]}

Shah, Ankita ^{[15
]}

Ouyang, Haojun ^{[15
]}

Macek, Thomas ^{[15
]}

Kernbauer, Elaine ^{[15
]}

Sproule, Douglas M. ^{[15
]}

Mendell, Jerry R. ^{[6
,7
]}

机构：

[1] Stanford Univ, Med Ctr, Dept Neurol, Stanford, CA 94305 USA

[2] Columbia Univ, Med Ctr, Div Pediat Neurol, New York, NY 10027 USA

[3] Johns Hopkins Med, Dept Neurol, Baltimore, MD USA

[4] Boston Childrens Hosp, Dept Neurol, Boston, MA USA

[5] Nemours Childrens Hosp, Dept Pediat, Orlando, FL USA

[6] Ohio Univ, Dept Pediat, Athens, OH 45701 USA

[7] Ohio Univ, Dept Neurol, Athens, OH 45701 USA

[8] Nationwide Childrens Hosp, Ctr Gene Therapy, Orlando, FL USA

[9] Nationwide Childrens Hosp, Dept Neurol, Orlando, FL USA

[10] Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, Dallas, TX USA

[11] Ann & Robert H Lurie Childrens Hosp Chicago, Div Neurol, Chicago, IL 60611 USA

[12] Cincinnati Childrens Hosp, Div Human Genet, Cincinnati, OH USA

[13] Univ Calif Los Angeles, David Geffen Sch Med, Dept Neurol, Los Angeles, CA 90024 USA

[14] Duke Univ, Sch Med, Dept Pediat, Durham, NC 27706 USA

[15] AveXis Inc, Bannockburn, IL USA

来源：

NEUROLOGY | 2020年 / 94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：

1828

引用

页数：5

共 50 条

[21] Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial
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[22] Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2a Clinical Trial
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[23] Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) in Pre-symptomatic Spinal Muscular Atrophy (SMA)
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[25] Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)
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[26] Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG)
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[28] Onasemnogene abeparvovec gene therapy in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update
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