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

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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Bibliographic Details
Main Authors: Ankit K. Desai, Carolyn H. Baloh, John W. Sleasman, Amy S. Rosenberg, Priya S. Kishnani
Format: Article
Language:English
Published: Frontiers Media S.A. 2020-08-01
Series:Frontiers in Immunology
Subjects:
anti-drug antibodies
enzyme replacement therapy
immune tolerance induction
anti-rhGAA IgG antibody
alglucosidase alfa
immunogenicity
Online Access:https://www.frontiersin.org/article/10.3389/fimmu.2020.01727/full

Internet

https://www.frontiersin.org/article/10.3389/fimmu.2020.01727/full

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