Patient and physician perspectives inform clinical trial design for a single intravenous dose of HMI-203, a gene therapy candidate for adults with mucopolysaccharidosis type II (MPS II, Hunter syndrome)

被引：0
|
作者：
Haroldson, Jeffrey [1 ]
Witalisz, Cj [1 ]
Mayfield, Cara [1 ]
Ellinwood, N. Matthew [2 ]
Urdaneta, Leslie [2 ]
Martin, Rick [1 ]
Cohn, Gabriel [1 ]
机构：
[1] Homol Med, Bedford, MA USA
[2] Natl MPS Soc, Durham, NC USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2022年 / 135卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2021.11.133
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
118
引用
收藏
页码：S54 / S55
页数：2
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