An updated management approach of Pompe disease patients with high-sustained anti-rhGAA IgG antibody titers: experience with bortezomib-based immunomodulation

An updated management approach of Pompe disease patients with high-sustained anti-rhGAA IgG antibody titers: experience with bortezomib-based immunomodulation

IntroductionHigh sustained anti-rhGAA antibody titers (HSAT; ≥12,800) are directly linked to reduced efficacy of enzyme replacement therapy (ERT) and subsequent clinical deterioration in infantile-onset Pompe disease (IOPD). We have previously demonstrated the safety and effectiveness of a bortezomi...

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Bibliographic Details
Main Authors: Ankit K. Desai, Garima Shrivastava, Christina L. Grant, Raymond Y. Wang, Trevor D. Burt, Priya S. Kishnani
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-03-01
Series:Frontiers in Immunology
Subjects:
alglucosidase alfa
anti-drug antibodies
entrenched immune tolerance induction
bortezomib
Pompe disease
anti-rhGAA IgG antibodies
Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2024.1360369/full

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https://www.frontiersin.org/articles/10.3389/fimmu.2024.1360369/full

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