FIREFISH Part 1: 16-month safety and exploratory outcomes of risdiplam (RG7916) treatment in infants with type 1 spinal muscular atrophy

被引：13

作者：

Baranello, G. ^{[1
]}

Servais, L. ^{[2
]}

Day, J. ^{[3
]}

Deconinck, N. ^{[4
]}

Mercuri, E. ^{[5
]}

Klein, A. ^{[6
]}

Darras, B. ^{[7
]}

Masson, R. ^{[8
]}

Kletzl, H. ^{[9
]}

Cleary, Y. ^{[9
]}

El-Khairi, M. ^{[10
]}

Seabrook, T. ^{[9
]}

Czech, C. ^{[11
]}

Gerber, M. ^{[9
]}

Nguyen, C. ^{[9
]}

Gelblin, K. ^{[9
]}

Gorni, K. ^{[9
]}

机构：

[1] UCL Great Ormond St, London, England

[2] Inst Myol, Paris, France

[3] Stanford Univ, Palo Alto, CA 94304 USA

[4] Univ Libre Bruxelles, Brussels, Belgium

[5] Catholic Univ, Rome, Italy

[6] Univ Childrens Hosp, Basel, Switzerland

[7] Harvard Med Sch, Boston, MA 02115 USA

[8] Carlo Besta Neuro Inst, Milan, Italy

[9] Roche Innovat Ctr Basel, Basel, Switzerland

[10] Roche Prod Ltd, Welwyn Garden City, Herts, England

[11] Therachon AG, Basel, Switzerland

来源：

关键词：

D O I：

10.1016/j.nmd.2019.06.515

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P.353

引用

页码：S184 / S184

页数：1