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Sustained immune tolerance induction in enzyme replacement therapy–treated CRIM-negative patients with infantile Pompe disease
BACKGROUND. Cross-reactive immunological material–negative (CRIM-negative) infantile Pompe disease (IPD) patients develop an immune response against enzyme replacement therapy (ERT) with alglucosidase alfa that nullifies ERT efficacy. Prophylactic immune tolerance induction (ITI) with rituximab, met...
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| Pubblicato in: | JCI Insight |
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| Autori principali: | , , , , , , , , , , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
American Society for Clinical Investigation
2017
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5621909/ https://ncbi.nlm.nih.gov/pubmed/28814660 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.94328 |
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