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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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Detalles Bibliográficos
Autores principales: De Groot, Anne S., Desai, Ankit K., Lelias, Sandra, Miah, S. M. Shahjahan, Terry, Frances E., Khan, Sundos, Li, Cindy, Yi, John S., Ardito, Matt, Martin, William D., Kishnani, Priya S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8242953/
https://www.ncbi.nlm.nih.gov/pubmed/34220802
http://dx.doi.org/10.3389/fimmu.2021.636731