RGX-111 GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSACCHARIDOSIS TYPE I (MPS I): INTERIM ANALYSIS OF DATA FROM A PHASE 1/2 STUDY

被引：0
|
作者：
Wang, Raymond [1 ]
Ficicioglu, Can [2 ]
Giugliani, Roberto [3 ]
Nevoret, Marie-Laure [4 ]
Fiscella, Michele [4 ]
Yang, Lin [4 ]
Cho, Yoonjin [4 ]
Phillips, Dawn [4 ]
Falabella, Paulo [4 ]
机构：
[1] Univ Calif Irvine, CHOC Childrens Hosp, Dept Pediat, Div Metab Disorders, Orange, CA 92668 USA
[2] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[3] Univ Fed Rio Grande do Sul, HCPA, Med Genet Serv, Dept Genet, Porto Alegre, RS, Brazil
[4] REGENXBIO, Rockville, MD USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2022年 / 135卷 / 04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：
92
引用
收藏
页码：305 / 305
页数：1
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