Updates are available Correction dated 2025-12-15

Article History

Accepted: 9 November 2025

First Online: 24 November 2025

Change Date: 14 December 2025

Change Type: Update

Change Details: The original online version of this article was revised: The author’s name W. W. M. Pim Pijnappel was incorrectly written as Pim W. W. M. Pijnappel.

Change Date: 15 December 2025

Change Type: Correction

Change Details: A Correction to this paper has been published:

Change Details: https://doi.org/10.1007/s40259-025-00760-y

Declarations

:

: Ans T. van der Ploeg and Johanna M.P. van den Hout received funding for research, clinical trials, and/or as an advisor from various industries working on ERT or next-generation therapies in the field of Pompe disease under agreements with Erasmus MC University Medical Center and the relevant industry. Pim W.W.M. Pijnappel is a co-founder and unpaid Chief Scientific Officer of LentiCure, a company developing gene therapy for lysosomal disorders including Pompe disease. Nadine A.M.E van der Beek has received consulting fees for advisory boards or speaker honoraria from Sanofi, Amicus Therapeutics, Shionogi, and Bayer under agreements with Erasmus MC University Medical Center and the relevant industry. Ed H. Jacobs and Marianne Hoogeveen-Westerveld received funding for clinical trial and contract work from Spark Therapeutics and Amicus Therapeutics. Martha C. Faraguna, Daan Lambregts, Ina Barzel, Serena Gasperini, and Tim Preijers have no conflicts of interest that are directly relevant to the content of this article.

: Patients with Pompe disease followed at the Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center in Rotterdam, the Netherlands are enrolled in the prospective protocol MEC-2007-103-NL, approved by the Medical Ethical Committee of Erasmus Medical Center.

: Patients or their parents/legal guardians provided written consent to participate in the study.

: Patients or their parents/legal guardians consented to publication of this study.

: All original data are present in the article. Further enquiries can be addressed to the corresponding author.

: Enquiries concerning the code can be addressed to the corresponding author.

: MCF participated in planning the project and design of experiments, collected data, analyzed and interpreted data. and wrote the draft of the manuscript. ATP and JMPH conceived the project, designed experiments, interpreted data, participated in discussions, and edited several versions of this manuscript. IB and TP contributed to the PK analysis, results interpretation, improvement of figure quality, and reviewed several versions of the paper. DL, SG, PWWMP, and NAME participated in data interpretation and reviewed several drafts of the manuscript. EHJ and MHW supervised enzyme activity measurement and the enzyme-linked immunosorbent assay, analyzed and interpreted data, and contributed to the manuscript preparation.