Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview

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作者
David Stevens
Shadi Milani-Nejad
Tahseen Mozaffar
机构
[1] Departments of Neurology,Pathology & Laboratory Medicine, School of Medicine
[2] University of California,The Institute for Immunology, School of Medicine
[3] University of California,undefined
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关键词
Pompe disease; Glycogen storage disease II (GSDII); Acid maltase deficiency; Alpha-glucosidase deficiency; Enzyme replacement therapy; Gene therapy;
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页码:573 / 588
页数:15
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