Outcomes in patients with spinal muscular atrophy and four or more SMN2 copies treated with onasemnogene abeparvovec: Findings from RESTORE

被引：0
|
作者：
Finkel, R. [1 ]
Benguerba, K. [2 ]
Reid, A. [3 ]
Raju, D. [4 ]
Faulkner, E. [4 ]
LaMarca, N. [4 ]
Servais, L. [5 ]
机构：
[1] St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USA
[2] Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland
[3] Novartis Global RWE Gene Therapies, London, England
[4] Novartis Gene Therapies Inc, Bannockburn, IL USA
[5] Univ Oxford, Oxford, England
来源：
NEUROMUSCULAR DISORDERS | 2022年 / 32卷
关键词：
D O I：
10.1016/j.nmd.2022.07.073
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
P.37
引用
收藏
页码：S59 / S59
页数：1
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