Real-World outcomes following onasemnogene abeparvovec in patients with SMA and One SMN2 gene copy: findings from the RESTORE Registry

被引：0

作者：

Servais, L. ^{[1
,2
,3
]}

Mathews, K. ^{[4
]}

Bernes, S. ^{[5
]}

Lakhotia, A. ^{[6
]}

Tizzano, E. ^{[7
]}

Raju, D. ^{[8
]}

Benguerba, K. ^{[9
]}

Reyna, S. ^{[8
]}

Dabbous, O. ^{[8
]}

Finkel, R. ^{[10
]}

机构：

[1] Univ Oxford, Oxford, England

[2] Univ Liege, Liege, Belgium

[3] Univ Hosp Liege, Liege, Belgium

[4] Univ Iowa, Carver Coll Med, Iowa City, IA USA

[5] Univ Arizona, Coll Med, Phoenix, AZ USA

[6] Univ Louisville, Norton Childrens Med Grp, Louisville, KY USA

[7] Hosp Valle De Hebron, Barcelona, Spain

[8] Novartis Gene Therapies Inc, Bannockburn, IL USA

[9] Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland

[10] St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USA

来源：

关键词：

D O I：

10.1016/j.nmd.2024.07.607

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

163P

引用

页数：2