Development of high sustained anti-drug antibody titers and corresponding clinical decline in a late-onset Pompe disease patient after 11+ years on enzyme replacement therapy
A late-onset Pompe disease patient developed high sustained antibody titers (HSAT) of ≥51,200 after 11+ years on alglucosidase alfa and previous tolerance. There was a corresponding worsening of motor function and rise in urinary glucose tetrasaccharide (Glc4). Following immunomodulation therapy, HS...
| Main Authors: | , , , , , |
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| 格式: | 文件 |
| 语言: | English |
| 出版: |
Elsevier
2023-09-01
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| 丛编: | Molecular Genetics and Metabolism Reports |
| 主题: | |
| 在线阅读: | http://www.sciencedirect.com/science/article/pii/S2214426923000277 |