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：

引用

页码：305 / 305

页数：1

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