Safety and efficacy of intravenous onasemnogene abeparvovec in patients with spinal muscular atrophy: interim findings from the phase 3 SMART study

被引：0

作者：

McMillan, H. ^{[1
]}

Baranello, G. ^{[2
,3
,4
]}

Farrar, M. ^{[5
,6
]}

Zaidman, C. ^{[7
]}

Seibert, J. ^{[8
]}

Bernardo, R. ^{[9
]}

Alecu, I. ^{[8
]}

Freischlager, F. ^{[10
]}

Muntoni, F. ^{[2
,3
,4
]}

机构：

[1] Childrens Hosp Eastern Ontario, Ottawa, ON, Canada

[2] UCL, Dev Neurosci Res & Teaching Dept, Dubowitz Neuromuscular Ctr, Gt Ormond St Inst Child Hlth, London, England

[3] NIHR Gt Ormond St Hosp Biomed Res Ctr, London, England

[4] Gt Ormond St Hosp NHS Fdn Trust, London, England

[5] UNSW Sydney, UNSW Med & Hlth, Sch Clin Med, Sydney, Australia

[6] Sydney Childrens Hosp Network, Dept Neurol, Randwick, NSW, Australia

[7] Washington Univ, Div Pediat Neurol, Dept Neurol, Sch Med, St Louis, MO USA

[8] Novartis Pharmaceut, Basel, Switzerland

[9] Novartis Global Drug Dev Neurosci, E Hanover, NJ USA

[10] Freischlager Consulting, Bad Nauheim, Germany

来源：

关键词：

D O I：

10.1016/j.nmd.2023.07.489

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

O20

引用

页码：S191 / S191

页数：1