Immune Tolerance-Adjusted Personalized Immunogenicity Prediction for Pompe Disease

Infantile-onset Pompe disease (IOPD) is a glycogen storage disease caused by a deficiency of acid alpha-glucosidase (GAA). Treatment with recombinant human GAA (rhGAA, alglucosidase alfa) enzyme replacement therapy (ERT) significantly improves clinical outcomes; however, many IOPD children treated w...

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Bibliographic Details
Main Authors: Anne S. De Groot, Ankit K. Desai, Sandra Lelias, S. M. Shahjahan Miah, Frances E. Terry, Sundos Khan, Cindy Li, John S. Yi, Matt Ardito, William D. Martin, Priya S. Kishnani
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-06-01
Series:Frontiers in Immunology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2021.636731/full