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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Bibliographic Details
Published in:Genet Med
Main Authors: Berrier, Kathryn L., Kazi, Zoheb B., Prater, Sean N., Bali, Deeksha S., Goldstein, Jennifer, Stefanescu, Mihaela C., Rehder, Catherine W., Botha, Eleanor G., Ellaway, Carolyn, Bhattacharya, Kaustuv, Tylki-Szymanska, Anna, Karabul, Nesrin, Rosenburg, Amy S., Kishnani, Priya S.
Format: Artigo
Language:Inglês
Published: 2015
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Online Access: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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