Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

PURPOSE: Infantile Pompe disease resulting from a deficiency of lysosomal acid α-glucosidase (GAA) requires enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA). Cross-reactive immunologic material negative (CRIM-negative) Pompe patients develop high-titer antibody to the rhGAA and do...

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Main Authors: Messinger, Yoav H., Mendelsohn, Nancy J., Rhead, William, Dimmock, David, Hershkovitz, Eli, Champion, Michael, Jones, Simon A., Olson, Rebecca, White, Amy, Wells, Cara, Bali, Deeksha, Case, Laura E., Young, Sarah P., Rosenberg, Amy S., Kishnani, Priya S.
Formáid: Artigo
Teanga:Inglês
Foilsithe: 2012
Ábhair:
Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3711224/
https://ncbi.nlm.nih.gov/pubmed/22237443
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gim.2011.4
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