Immunophenotype associated with high sustained antibody titers against enzyme replacement therapy in infantile-onset Pompe disease

IntroductionThe efficacy of enzyme replacement therapy (ERT) with alglucosidase alfa for infantile-onset Pompe disease (IOPD) is limited in some patients due to the development of high and sustained antibody titers (HSAT; ≥12,800).MethodsWe carried out detailed immunophenotyping of IOPD patients (n=...

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Bibliographic Details
Main Authors: Ankit K. Desai, P. Brian Smith, John S. Yi, Amy S. Rosenberg, Trevor D. Burt, Priya S. Kishnani
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-01-01
Series:Frontiers in Immunology
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Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2023.1301912/full