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...

Description complète

Enregistré dans:
Détails bibliographiques
Auteurs principaux: 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.
Format: Artigo
Langue:Inglês
Publié: 2012
Sujets:
Article
Accès en ligne: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
Tags: Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!

Documents similaires