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Benefits of Prophylactic Short-Course Immune Tolerance Induction in Patients With Infantile Pompe Disease: Demonstration of Long-Term Safety and Efficacy in an Expanded Cohort

Immune tolerance induction (ITI) with a short-course of rituximab, methotrexate, and/or IVIG in the enzyme replacement therapy (ERT)-naïve setting has prolonged survival and improved clinical outcomes in patients with infantile Pompe disease (IPD) lacking endogenous acid-alpha glucosidase (GAA), kno...

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Detalles Bibliográficos
Autores principales:	Desai, Ankit K., Baloh, Carolyn H., Sleasman, John W., Rosenberg, Amy S., Kishnani, Priya S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2020
Materias:	Immunology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7424004/ https://www.ncbi.nlm.nih.gov/pubmed/32849613 http://dx.doi.org/10.3389/fimmu.2020.01727

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