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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Bibliographic Details
Published in:JCI Insight
Main Authors: 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.
Format: Artigo
Language:Inglês
Published: American Society for Clinical Investigation 2017
Subjects:
Clinical Medicine
Online Access: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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