Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy

Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy

IntroductionPompe disease, a lysosomal storage disorder, is characterized by acid α-glucosidase (GAA) deficiency and categorized into two main subtypes: infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD). The primary treatment, enzyme replacement therapy (ERT) with recombinant...

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Bibliographic Details
Main Authors: Hui-An Chen, Rai-Hseng Hsu, Ching-Ya Fang, Ankit K. Desai, Ni-Chung Lee, Wuh-Liang Hwu, Fuu-Jen Tsai, Priya S. Kishnani, Yin-Hsiu Chien
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-04-01
Series:Frontiers in Immunology
Subjects:
Pompe disease
enzyme replacement therapy
alglucosidase alfa
immunomodulation therapy
anti-drug antibody
Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2024.1336599/full

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https://www.frontiersin.org/articles/10.3389/fimmu.2024.1336599/full

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