Funding for this research was provided by:
Prinses Beatrix Spierfonds (W.OR16-07, W.F16-03)
Stichting Vrienden van het Sophia (S17-32)
Ministy of Economic Affairs, TKI-program Life Sciences and Health (LSHM16008)
Received: 16 April 2020
Accepted: 26 July 2020
First Online: 14 September 2020
Ethics approval and consent to participate
: 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. Informed consent was obtained from all parents of patients for being included in the study. The medical ethical committee of the Erasmus MC approved the study protocol.
: Not applicable.
: AvdP and HvdH have been advisor for Sanofi-Genzyme, Amicus Therapeutics, Biomarin, Ultragenix, Sarepta, Audentes, and Spark Therapeutics under agreements between these companies and Erasmus MC University Medical Center. NvdB has been advisor for Sanofi-Genzyme, and Amicus Therapeutics under agreements between these companies and Erasmus MC University Medical Center. The other authors declare that they have no conflict of interest. No funding sources had any influence on the content of this manuscript nor provided editorial support.