CAMPSIITET Phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II)

被引：0
|
作者：
Pisani, Laura [1 ]
Ficicioglu, Can [2 ]
Harmatz, Paul [3 ]
Giugliani, Roberto [4 ]
Rajan, Deepa [5 ]
Hagood, Joseph [1 ]
Fiscella, Michele [1 ]
Yang, Lin [1 ]
Gilmor, Michelle [1 ]
Cho, Yoonjin [1 ]
Mulatya, Caroline [1 ]
Phillips, Dawn [1 ]
Boulos, Nidal [1 ]
Falabella, Paulo [1 ]
机构：
[1] REGENXBIO Inc, Rockville, MD USA
[2] Childrens Hosp Philadelphia, Philadelphia, MD USA
[3] UCSF, Benioff Childrens Hosp Oakland, Oakland, CA USA
[4] Univ Fed Rio Grande do Sul, Dept Genet, Med Genet Serv, HCPA, Porto Alegre, RS, Brazil
[5] Univ Pittsburgh, Pittsburgh, PA USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 04期
关键词：
D O I：
10.1016/j.ymgme.2024.108245
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
069
引用
收藏
页数：1
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