RGX-121: AN INVESTIGATIONAL GENE THERAPY FOR THE TREATMENT OF NEURONOPATHIC MUCOPOLYSACCHARIDOSIS TYPE II (MPS II): INTERIM ANALYSIS OF DATA FROM THE FIRST-IN-HUMAN STUDY

被引：0
|
作者：
Blair, D. R. [1 ,2 ]
Harmatz, P. [2 ]
Ficicioglu, C. [3 ]
Giugliani, R. [4 ]
Rajan, D. S. [5 ]
Hagood, J. [6 ]
Fiscella, M. [6 ]
Yang, L. [6 ]
Gilmor, M. [6 ]
Cho, Y. [6 ]
Mulatya, C. [6 ]
Phillips, D. [6 ]
Falabella, P. [6 ]
Pisani, L. [6 ]
机构：
[1] Univ Calif San Francisco, San Francisco, CA 94143 USA
[2] UCSF Benioff Childrens Hosp Oakland, Oakland, CA USA
[3] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[4] HCPA, Porto Alegre, RS, Brazil
[5] Univ Pittsburgh, Pittsburgh, PA USA
[6] REGENXBIO Inc, Rockville, MD USA
来源：
JOURNAL OF INVESTIGATIVE MEDICINE | 2024年 / 72卷 / 01期
关键词：
D O I：
暂无
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
566
引用
收藏
页码：638 / 638
页数：1
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