JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment-naive patients with spinal muscular atrophy (SMA)

被引：0
|
作者：
Chiriboga, C. [1 ]
Bruno, C. [2 ]
Duong, T. [3 ]
Fischer, D. [4 ]
Kirschner, J. [5 ]
Scoto, M. [6 ]
Mercuri, E. [7 ]
Gerber, M. [8 ]
Gorni, K. [8 ]
Kletzl, H. [9 ]
Carruthers, I. [10 ]
Martin, C. [10 ]
Gidaro, T. [8 ]
Muntoni, F. [6 ]
机构：
[1] Columbia Irving Med Ctr, New York, NY USA
[2] Ist Giannina Gaslini, Genoa, Italy
[3] Stanford Univ, Palo Alto, CA 94304 USA
[4] Univ Childrens Hosp, Basel, Switzerland
[5] Univ Freiburg, Freiburg, Germany
[6] UCL, London, England
[7] Catholic Univ, Rome, Italy
[8] F Hoffmann La Roche Ltd, Basel, Switzerland
[9] Roche Innovat Ctr Basel, Basel, Switzerland
[10] Roche Prod Ltd, Welwyn Garden City, Herts, England
来源：
NEUROMUSCULAR DISORDERS | 2022年 / 32卷
关键词：
D O I：
10.1016/j.nmd.2022.07.195
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
P.110
引用
收藏
页码：S88 / S88
页数：1
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