Antibody formation and efficacy of enzyme replacement therapy in adults with Pompe disease: Unlocking long-term insights.
Theunissen MTM, Ditters IAM, van Kooten HA, van Doorn PA, van den Hout JMP, Hoogeveen-Westerveld M, Jacobs EH, van der Ploeg AT, van der Beek NAME
Genetics in medicine : official journal of the American College of Medical Genetics, 2026 Apr 13
Abstract
Anti-recombinant human acid α-glucosidase (rhGAA) antibodies can develop in adult Pompe patients receiving enzyme replacement therapy (ERT), but their long-term impact on clinical treatment outcomes is currently unclear.