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Article History

Received: 28 October 2022

Revised: 24 November 2022

Accepted: 27 November 2022

First Online: 8 December 2022

Declarations

:

: J. van den Dorpel, M. Dremmen, D. Rizopoulos, R Muetzel and P. van Doorn report no competing interests. A. van der Ploeg received funding for research, clinical trials and as advisor from Sanofi-Genzyme, Amicus Therapeutics, Biomarin, Ultragenix, Sarepta, Audentes and Spark Therapeutics 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. N. van der Beek received funding for research, clinical trials and as advisor from Sanofi-Genzyme, Amicus Therapeutics, Biomarin and Spark Therapeutics on enzyme replacement therapy or next-generation therapies in the field of Pompe disease or neuromuscular disorders, under agreements with Erasmus MC University Medical Center. J. van den Hout received funding for research, clinical trials and as advisor from Sanofi Genzyme, Amicus Therapeutics, Biomarin, Sarepta, Takeda and Chiesi working on enzyme replacement therapy or next-generation therapies in the field of Pompe disease, other lysosomal storage diseases, under agreements with Erasmus MC University Medical Center.

: All procedures followed were in accordance with the ethical standards of the responsible committee on human experimentation (institutional and national) and with the Helsinki Declaration of 1975, as revised in 2000. This study was approved by the Institutional Review Board at the Erasmus MC University Medical Center (MEC-2007-103; amendment 11).

: Informed consent was obtained from all patients for being included in the study. All forms are on file and available in case of request.