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Authors
Ditters, Imke A. M.
van der Beek, Nadine A. M. E.
Brusse, Esther
van der Ploeg, Ans T.
van den Hout, Johanna M. P.
Huidekoper, Hidde H. https://orcid.org/0000-0003-1276-0931
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Funding
Funding for this research was provided by:
Prinses Beatrix Spierfonds
TKI Life Sciences & Health
ZonMw (09150161910230)
Sanofi
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Article History
Received: 13 February 2023
Accepted: 30 April 2023
First Online: 8 May 2023
Declarations
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: This study was approved by the medical ethical committee of the Erasmus MC (MEC-2019-0146). The institutional review board assessed the study not to be subject to the Medical Research Involving Human Subjects Act (WMO). We received informed consent from all participants and/or their caregivers in this study; all forms are on file at the Erasmus MC and are available in case of request. All individual patient data was de-identified.
: We received informed consent from all participants and/or their caregivers in this study; all forms are on file at the Erasmus MC and are available in case of request. All individual patient data was de-identified.
: ID, EB, and HH declare no competing interest. NvdB received funding for research, clinical trials, and advisory fees from Sanofi working on enzyme replacement therapy 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. AvdP received funding for research, clinical trials, and advisory fees from Sanofi, Amicus Therapeutics, Spark Therapeutics, Denali Therapeutics and Takeda working on enzyme replacement therapy 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. JvdH received funding for research, clinical trials, and advisory fees from Sanofi, Denali therapeutics, Amicus Therapeutics and Takeda working on enzyme replacement therapy 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.
