Home infusion with recombinant human α-glucosidase in classic infantile and other children with Pompe disease is safe: The Dutch experience over 20 years with enzyme replacement therapy

Molecular Genetics and Metabolism 2026

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Authors: Imke A. M. Ditters, Hidde H. Huidekoper, Michelle Elisabeth Kruijshaar, Ans T. van der Ploeg, Hannerieke J.P. van den Hout

DOI: 10.1016/j.ymgme.2025.109388

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Metabolism and Genetic Disorders