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...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
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.
Format: Artigo
Sprog:Inglês
Udgivet: 2012
Fag:
Article
Online adgang: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: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker