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Authors
Ditters, Imke A. M. https://orcid.org/0000-0002-2377-3108
Huidekoper, Hidde H. https://orcid.org/0000-0003-1276-0931
Hardon, Jacqueline F.
Kruijshaar, Michelle E. https://orcid.org/0009-0009-8700-0591
van der Ploeg, Ans T. https://orcid.org/0000-0002-3359-1324
van den Hout, Johanna M. P. https://orcid.org/0000-0001-8091-263X
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Funding
Funding for this research was provided by:
ZonMw
Prinses Beatrix Spierfonds
TKI Life Sciences & Health
Sanofi
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Article History
Accepted: 3 March 2026
First Online: 20 July 2026
Declarations
Funding: This study was partly supported by ZonMw, Prinses Beatrix Spierfonds, TKI Life Sciences & Health, and Sanofi.
Competing interest statement: I.A.M. Ditters, J.F. Hardon, M.E. Kruijshaar declare no competing interests. H.H. Huidekoper received funding for research and advisory fees from Biomarin under agreements with Erasmus MC University Medical Center and the relevant industry. J.M.P. van den Hout and A.T. van der Ploeg received funding for research, clinical trials and as advisor from various industries working on enzyme-replacement therapy or next-generation therapies in the field of Pompe disease, other lysosomal storage diseases or neuromuscular disorders, under agreements with Erasmus MC University Medical Center and the relevant industry.
Ethics declaration: This study was approved by the medical ethical committee of the Erasmus MC (2007-103).
Consent to participate: Written informed consent was obtained from all patients and/or their parents/legal guardians.
Consent for publication: Our paper does not contain any identifiable photos or patient data.
Data availability: Deidentified data will be shared upon request to the corresponding author from any qualified investigator for the sole purpose of replicating procedures and results presented in the Article, in agreement with EU legislation on the General Data Protection Regulation.
Author contributions: Conceptualization: ID, JvdH, HH, MK, AvdP. Data curation: ID. Formal analysis: ID. Funding acquisition: JvdH, HH, AvdP. Investigation: ID, HH, JvdH. Methodology: ID, JvdH, HH, AvdP. Project administration: ID, JvdH, AvdP. Resources: ID, JvdH, HH, JH Supervision: JvdH, HH, AvdP. Validation: ID, JvdH. Visualization: ID. Writing—original draft: ID. Writing—review and editing: ID, JvdH, HH, MK, AvdP.
