Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease

Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease

Abstract Background Enzyme replacement therapy (ERT) with recombinant human alpha-glucosidase (rhGAA, alglucosidase alfa) has improved survival, motor outcomes, daily life activity and quality of life in Pompe patients. However, ERT in Pompe disease often induces formation of antibodies, which may r...

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Bibliographic Details
Main Authors: Harmke A. van Kooten, Imke A. M. Ditters, Marianne Hoogeveen-Westerveld, Edwin H. Jacobs, Johanna M. P. van den Hout, Pieter A. van Doorn, W. W. M. Pim Pijnappel, Ans T. van der Ploeg, Nadine A. M. E. van der Beek
Format: Article
Language:English
Published: BMC 2022-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Antibodies
Enzyme replacement therapy
Pompe disease
Recombinant human alpha-glucosidase
Online Access:https://doi.org/10.1186/s13023-022-02175-2

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