Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study

Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study

Abstract Background Pompe disease is a lysosomal storage disease treated with life-long enzyme replacement therapy (ERT). Home-based ERT has been provided in the Netherlands since 2008 because it diminishes the burden of treatment, increases patient flexibility and autonomy, and is thus a more patie...

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Bibliographic Details
Main Authors: Imke A. M. Ditters, Nadine A. M. E. van der Beek, Esther Brusse, Ans T. van der Ploeg, Johanna M. P. van den Hout, Hidde H. Huidekoper
Format: Article
Language:English
Published: BMC 2023-05-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Pompe disease
Glycogen storage disease type II
Enzyme replacement therapy (ERT)
Alglucosidase alfa
Home treatment
Infusion associated reactions (IARs)
Online Access:https://doi.org/10.1186/s13023-023-02715-4

Internet

https://doi.org/10.1186/s13023-023-02715-4

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