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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...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Genet Med |
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| Κύριοι συγγραφείς: | , , , , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
2015
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| Θέματα: | |
| Διαθέσιμο Online: | 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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