Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)

被引：0
|
作者：
Hughes, Derralynn [1 ]
Wilcox, William [2 ]
Hopkin, Robert J. [3 ]
Ganesh, Jaya [4 ]
Bernat, John [5 ]
Goker-Alpan, Ozlem [6 ]
Nicholls, Kathy [7 ]
Deegan, Patrick [8 ]
Pahl, Madeleine [9 ]
Whitley, Chester B. [10 ]
Chen, Michael
Cao, Liching
Schreeb, Katharina H.
机构：
[1] Royal Free London Hosp, London, England
[2] Emory Univ, Sch Med, Atlanta, GA USA
[3] Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA
[4] Icahn Sch Med Mt Sinai, New York, NY USA
[5] Univ Iowa, Iowa City, IA USA
[6] Lysosomal & Rare Disorders Res & Treatment Ctr, Fairfax, VA USA
[7] Addenbrookes Hosp, Cambridge, England
[8] Univ Calif Irvine, Irvine, CA USA
[9] Univ Minnesota, Minneapolis, MN USA
[10] Sangamo Therapeut Inc, Richmond, CA USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2025年 / 144卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2024.108773
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
146
引用
收藏
页数：2
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