Immunological challenges and approaches to immunomodulation in Pompe disease: a literature review

Annals of Translational Medicine
Ankit K DesaiPriya S Kishnani

Abstract

Pompe disease is an autosomal recessive disorder caused by a deficiency of acid alpha-glucosidase resulting in intralysosomal glycogen accumulation in multiple tissue types, especially cardiac, skeletal, and smooth muscle. Enzyme replacement therapy (ERT) with alglucosidase alfa has led to improved clinical outcomes and prolonged survival in patients with Pompe disease. While ERT has changed the natural course of Pompe disease, with many long-term survivors, several factors affect the response to ERT. Previous studies in Pompe disease have shown that IgG antibodies to ERT can lead to a decline in muscle strength, pulmonary function, and overall and ventilator-free survival. Additionally, antibody responses to ERT can also cause hypersensitivity reactions. Various strategies to prevent or eliminate the IgG antibody response have been attempted in patients with Pompe disease. A detailed literature search was performed to compile data regarding the consequences of IgG antibodies, clinical approaches to prevent or eliminate IgG antibodies in patients with Pompe disease, and to expand our understanding of new modalities being developed in non-clinical settings. All qualifying articles describing the impact of IgG antibodies on the r...Continue Reading

Citations

May 1, 2020·International Journal of Neonatal Screening·Laurie D SmithArindam Bhattacharjee
Jun 9, 2020·Frontiers in Immunology·Daniel C JulienPriya S Kishnani
Oct 20, 2020·International Journal of Neonatal Screening·Takaaki SawadaKimitoshi Nakamura
Oct 20, 2020·International Journal of Neonatal Screening·Raymond SaichSteven Krinks
Jan 19, 2021·EMBO Molecular Medicine·Giancarlo ParentiAndrea Ballabio
Feb 10, 2021·International Journal of Neonatal Screening·Raymond SaichSteven Krinks
Sep 22, 2021·Molecular Therapy : the Journal of the American Society of Gene Therapy·Jill A MorrisBeverly L Davidson

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