Treatments and outcomes for patients with spinal muscular atrophy (SMA) type 2: Findings from RESTORE registry

被引：0
|
作者：
Servais, L. [1 ]
Benguerba, K. [2 ]
De Vivo, D. [3 ]
Kirschner, J. [4 ]
Muntoni, F. [5 ]
Proud, C. [6 ]
Tizzano, E. [7 ]
Quijano-Roy, S. [8 ]
Desguerre, I. [9 ]
Saito, K. [10 ]
Raju, D. [11 ]
LaMarca, N. [11 ]
Sun, R. [11 ]
Anderson, F. [12 ]
Faulkner, E. [11 ]
Finkel, R. [13 ]
机构：
[1] Univ Oxford, Oxford, England
[2] Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland
[3] Columbia Univ, Irving Med Ctr, New York, NY USA
[4] Univ Med Ctr Freiburg, Freiberg, Germany
[5] UCL, London, England
[6] Childrens Hosp Kings Daughters, Norfolk, VA USA
[7] Hosp Valle De Hebron, Barcelona, Spain
[8] Raymond Poincare Univ Hosp, APHP, Garches, France
[9] Hop Necker Enfants Malad, Paris, France
[10] Tokyo Womens Med Univ, Tokyo, Japan
[11] Novartis Gene Therapies Inc, Bannockburn, IL USA
[12] Univ Massachusetts, Med Sch, Worcester, MA USA
[13] St Jude Childrens Res Hosp, Memphis, TN USA
来源：
NEUROMUSCULAR DISORDERS | 2022年 / 32卷
关键词：
D O I：
10.1016/j.nmd.2022.07.189
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
P.104
引用
收藏
页码：S87 / S87
页数：1
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