Effects of immunomodulation in classic infantile Pompe patients with high antibody titers

Effects of immunomodulation in classic infantile Pompe patients with high antibody titers

Abstract Purpose To evaluate whether immunomodulation can eliminate high sustained antibody levels, and thereby improve clinical outcome in classic infantile Pompe patients receiving enzyme replacement therapy (ERT) with recombinant human alpha-glucosidase (rhGAA). Methods Three patients (two cross-...

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Bibliographic Details
Main Authors: E. Poelman, M. Hoogeveen-Westerveld, J. M. P. van den Hout, R. G. M. Bredius, A. C. Lankester, G. J. A. Driessen, S. S. M. Kamphuis, W. W. M. Pijnappel, A. T. van der Ploeg
Format: Article
Language:English
Published: BMC 2019-03-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Pompe disease
ERT
Antibodies
Immunomodulation
Bortezomib
Cross-reactive immunologic material (CRIM)
Online Access:http://link.springer.com/article/10.1186/s13023-019-1039-z

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http://link.springer.com/article/10.1186/s13023-019-1039-z

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