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
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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
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页数：1
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