JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naive patients with spinal muscular atrophy (SMA) receiving risdiplam

被引：3

作者：

Chiriboga, C. ^{[1
]}

Bruno, C. ^{[2
]}

Duong, T. ^{[3
]}

Fischer, D. ^{[4
]}

Kirschner, J. ^{[5
]}

Mercuri, E. ^{[6
,7
]}

Gerber, M. ^{[8
]}

Gorni, K. ^{[8
]}

Kletzl, H. ^{[9
]}

Carruthers, I. ^{[10
]}

Martin, C. ^{[10
]}

Warren, F. ^{[10
]}

Scoto, M. ^{[11
]}

机构：

[1] Columbia Univ, 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] Catholic Univ, Rome, Italy

[7] Nemo Pediat, 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

[11] UCL, London, England

来源：

关键词：

D O I：

10.1016/j.nmd.2021.07.304

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

EP.279

引用

页码：S134 / S135

页数：2