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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...
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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2021
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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 |
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