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...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
मुख्य लेखकों: Kazi, Zoheb B., Desai, Ankit K., Berrier, Kathryn L., Troxler, R. Bradley, Wang, Raymond Y., Abdul-Rahman, Omar A., Tanpaiboon, Pranoot, Mendelsohn, Nancy J., Herskovitz, Eli, Kronn, David, Inbar-Feigenberg, Michal, Ward-Melver, Catherine, Polan, Michelle, Gupta, Punita, Rosenberg, Amy S., Kishnani, Priya S.
स्वरूप: मूलपाठ
भाषा:अंग्रेज़ी
प्रकाशित: American Society for Clinical Investigation 2017
विषय:
ऑनलाइन पहुंच:https://pmc.ncbi.nlm.nih.gov/articles/PMC5621909/
https://pubmed.ncbi.nlm.nih.gov/28814660
http://dx.doi.org/10.1172/jci.insight.94328
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