Intravenous and intrathecal onasemnogene abeparvovec gene therapy in symptomatic and presymptomatic SMA: long-term follow-up study

被引：0

作者：

Darras, B. ^{[1
]}

Farrar, M. ^{[2
,3
]}

Mercuri, E. ^{[4
,5
]}

Strauss, K. ^{[6
,7
,8
]}

Day, J. ^{[9
]}

Chien, Y. ^{[10
]}

Masson, R. ^{[11
]}

Bernardo, R. ^{[12
]}

Alecu, I. ^{[13
]}

Dodd, N. ^{[14
]}

Mehl, L. ^{[15
]}

Connolly, A. ^{[16
,17
]}

机构：

[1] Harvard Med Sch, Boston Childrens Hosp, Boston, MA USA

[2] Sydney Childrens Hosp Network, Sydney, NSW, Australia

[3] UNSW Sydney, Sch Clin Med, UNSW Med & Hlth, Sydney, NSW, Australia

[4] Catholic Univ, Dept Paediat Neurol, Rome, Italy

[5] Catholic Univ, Nemo Clin Ctr, Rome, Italy

[6] Clin Special Children, Strasburg, PA USA

[7] Penn Med Lancaster Gen Hosp, Lancaster, PA USA

[8] Univ Massachusetts, Sch Med, Worcester, MA USA

[9] Stanford Univ, Med Ctr, Stanford, CA USA

[10] Natl Taiwan Univ Hosp, Taipei, Taiwan

[11] Fdn IRCCS Ist Neurol Carlo Besta, Milan, Italy

[12] Novartis Global Drug Dev Neurosci & Gene Therap, E Hanover, NJ USA

[13] Novartis Pharmaceut, Basel, Switzerland

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

[15] Novartis, BioMed Res, Cambridge, MA USA

[16] Nationwide Childrens Hosp, Ctr Gene Therapy, Columbus, OH USA

[17] Ohio State Univ, Coll Med, Columbus, OH USA

来源：

NEUROMUSCULAR DISORDERS | 2024年 / 43卷

关键词：

D O I：

暂无

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

179P

引用

页数：1

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