SUMMARY OF SCREENING DATA FROM 19 PATIENTS WITH LATE-ONSET POMPE DISEASE FOR A PHASE I CLINICAL TRIAL OF AAV VECTOR-MEDIATED GENE THERAPY

被引：0
|
作者：
Hannah, William B. [1 ]
Case, Laura E. [2 ]
Walters, Crista [1 ]
Kishnani, Priya S. [1 ]
Koeberl, Dwight D. [1 ]
机构：
[1] Duke Univ, Sch Med, Dept Pediat, Div Med Genet, Durham, NC USA
[2] Duke Univ, Sch Med, Dept Phys & Occupat Therapy, Durham, NC USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2019年 / 127卷 / 03期
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D O I：
暂无
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
67
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页码：274 / 274
页数：1
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