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CRIM-negative infantile Pompe disease: Characterization of immune responses in patients treated with ERT monotherapy
PURPOSE: Enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA) prolongs survival in infantile Pompe disease (IPD). However, the majority of cross reactive immunologic material (CRIM)-negative (CN) patients have immune responses with significant clinical decline despite continued ERT. W...
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| 出版年: | Genet Med |
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| 主要な著者: | , , , , , , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
2015
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| 主題: | |
| オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4561024/ https://ncbi.nlm.nih.gov/pubmed/25741864 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gim.2015.6 |
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