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Authors
van Kooten, Harmke A.
Ditters, Imke A. M.
Hoogeveen-Westerveld, Marianne
Jacobs, Edwin H.
van den Hout, Johanna M. P.
van Doorn, Pieter A.
Pijnappel, W. W. M. Pim
van der Ploeg, Ans T.
van der Beek, Nadine A. M. E. https://orcid.org/0000-0001-9161-3301
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Funding
Funding for this research was provided by:
ZonMw (09150161910230)
Sophia Children’s Hospital Foundation (S17-32)
Metakids (2016-063)
Ministry of Economic Affairs under TKI-Allowance under the TKI-program Life Sciences & Health (LSHM16008)
Sanofi Genzyme
Prinses Beatrix Spierfonds (W.F16-03)
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Article History
Received: 1 November 2021
Accepted: 16 January 2022
First Online: 2 February 2022
Declarations
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: This study was approved by the medical ethical committee of the Erasmus MC.
: We received informed consent forms from all participants and/or their caregivers in this study; all forms are on file and available in case of request. All individual patient data was de-identified.
: J.M.P. van den Hout, A.T. van der Ploeg, and N.A.M.E. van der Beek, 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. M. Hoogeveen-Westerveld and E.H. Jacobs performed diagnostic assays for Spark Therapeutics. H.A. van Kooten, I.A.M. Ditters, P.A. van Doorn and W.W.M. Pijnappel declare no potential conflict of interest.
