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Article History

Accepted: 21 May 2023

First Online: 16 June 2023

Declarations

:

: This study was partly supported by ZonMw (project number 09150161910230), Prinses Beatrix Spierfonds, TKI Life Sciences and Health, and Sanofi.

: Imke A. M. Ditters, Harmke A. van Kooten, Jacqueline F. Hardon, Gamida Ismailova, Esther Brusse, Michelle E. Kruijshaar and Hidde H. Huidekoper declare no conflicts of interest. Ans T. van der Ploeg received funding for research, clinical trials, and advisory fees from Sanofi, Amicus Therapeutics, Spark Therapeutics, Denali Therapeutics and Takeda working on ERT or next­generation 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. Nadine A. M. E. van der Beek received funding for research, clinical trials, and advisory fees from Sanofi working on ERT or next­generation therapies in the field of Pompe disease, other lysosomal storage diseases or neuromuscular disorders, under an agreement between this company and Erasmus MC University Medical Center and the relevant industry. She also received a Veni grant from ZonMW (project no. 09150161910230). Johanna M. P. van den Hout received funding for research, clinical trials, and advisory fees from Sanofi, Denali Therapeutics and Takeda working on ERT or next­generation 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.

: Not applicable.

: We received consent to participate from all participants and/or their caregivers.

: We received consent for publication from all participants and/or their caregivers.

: De­identified data will be shared upon reasonable request to the corresponding author from any qualified investigator for the sole purpose of replicating procedures and results presented in this article, in agreement with EU legislation on the general data protection regulation.

: Not applicable.

: Conceptualization: IAM Ditters, NAME van der Beek, ME Kruijshaar, AT van der Ploeg, JMP van den Hout and HH Huidekoper. Data curation: IAM Ditters. Formal analysis: IAM Ditters, JMP van den Hout and HH Huidekoper. Investigation: IAM Ditters, JMP van den Hout and HH Huidekoper. Methodology: IAM Ditters, ME Kruijshaar, AT van der Ploeg, JMP van den Hout and HH Huidekoper. Project administration: IAM Ditters, JMP van den Hout and HH Huidekoper. Resources: IAM Ditters, HA van Kooten, NAME van der Beek, JF Hardon, G Ismailova, E Brusse, AT van der Ploeg, JMP van den Hout and HH Huidekoper. Supervision: AT van der Ploeg, JMP van den Hout and HH Huidekoper. Validation: IAM Ditters, JMP van den Hout and HH Huidekoper. Visualization: IAM Ditters, AT van der Ploeg, JMP van den Hout and HH Huidekoper. Writing original draft: IAM Ditters, AT van der Ploeg, JMP van den Hout and HH Huidekoper. Writing – review & editing: IAM Ditters, HA van Kooten, NAME van der Beek, JF Hardon, G Ismailova, E Brusse, ME Kruijshaar, AT van der Ploeg, JMP van den Hout and HH Huidekoper. All authors read and approved the final manuscript.