Home Infusion With Recombinant Human α-Glucosidase in Children With Pompe Disease: The Dutch Experience Over 20 Years Across the Spectrum From Classic Infantile to Late-Onset Phenotypes.

doi:10.1007/s40259-026-00773-1

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Home Infusion With Recombinant Human α-Glucosidase in Children With Pompe Disease: The Dutch Experience Over 20 Years Across the Spectrum From Classic Infantile to Late-Onset Phenotypes.

BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy - 1 Sept 2026

Ditters Imke A M, Huidekoper Hidde H, Hardon Jacqueline F, Kruijshaar Michelle E, van der Ploeg Ans T, van den Hout Johanna M P

Abstract excerpt

INTRODUCTION: Enzyme replacement therapy (ERT) is the standard treatment for patients with Pompe disease, a hereditary metabolic myopathy. While ERT in the home situation is increasingly common in adults with Pompe disease, experience in children remains limited due to higher dosing requirements and increased risk of infusion-associated reactions (IARs). We analysed the results of the in-hospital and home-based...

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Topics

Humans
Glycogen Storage Disease Type II
alpha-Glucosidases
Netherlands
Enzyme Replacement Therapy

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Child

Female

Infant

Male

Child, Preschool

Phenotype

Recombinant Proteins

Adolescent

Home Infusion Therapy

Infusions, Intravenous

Age of Onset

Source

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Identifiers

PMID: 42477279
DOI: 10.1007/s40259-026-00773-1
PMCID: PMC13570904

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