Schaefers, J.
van der Giessen, L. J.
Klees, C.
Jacobs, E. H.
Sieverdink, S.
Dremmen, M. H. G.
Spoor, J. K. H.
van der Ploeg, A. T.
van den Hout, J. M. P.
Huidekoper, H. H. https://orcid.org/0000-0003-1276-0931
Funding for this research was provided by:
Stichting Stofwisselkracht
Article History
Received: 4 March 2021
Accepted: 6 May 2021
First Online: 14 May 2021
Declarations
:
: Parents provided written informed consent for the use of the anonymized data of their children. As these data were obtained within a standardized clinical follow-up protocol formal approval from a medical ethical committee was not required as the study did not fall under the scope of the Dutch Medical Research Involving Human Subjects Act.
: Parents provided written informed consent for the publication of the anonymized data of their children.
: HHH has received a speaker fee, a participation fee for an advisory board and reimbursement for the ongoing participation in the 190–504 registry study from BioMarin International Limited under an agreement between BioMarin International Limited and Erasmus MC University Medical Center. BioMarin International Limited was not involved at any stage of the study or during the preparation of the manuscript. All other authors declare no competing interests.