RGX-121 gene therapy for severe Mucopolysaccharidosis Type II (MPS II): Interim neurodevelopmental outcomes and biomarker results

被引：0
作者：
Harmatz, Paul [1 ]
Escolar, Maria [2 ]
Ficicioglu, Can [3 ]
Giugliani, Roberto [4 ]
Cho, Yoonjin [5 ]
Phillips, Dawn [5 ]
Nevoret, Marie-Laure [5 ]
Falabella, Paulo [5 ]
机构：
[1] UCSF Benioff Childrens Hosp Oakland, Gastroenterol, Oakland, CA USA
[2] UPMC Childrens Hosp Pittsburgh, Pediat, Pittsburgh, PA USA
[3] Childrens Hosp Philadelphia, Pediat, Philadelphia, PA 19104 USA
[4] Univ Fed Rio Grande do Sul, Med Genet, Porto Alegre, RS, Brazil
[5] REGENXBIO, Rockville, MD USA
来源：
NEUROLOGY | 2021年 / 96卷 / 15期
关键词：
D O I：
暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
4164
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收藏
页数：4
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