Funding for this research was provided by:
Prinses Beatrix Spierfonds (W.OR15-10)
Received: 5 July 2020
Accepted: 27 November 2020
First Online: 7 January 2021
Ethics approval and consent to participate
: The protocol was approved by the Medical Ethical Committee at our hospital (MEC-2007-103, amendment 7). All participants provided written informed consent.
: Not applicable.
: NAMEB received funding for research and as advisor or speaker from Sanofi-Genzyme, under agreements with Erasmus MC University Medical Center. ATP received funding for research and/or clinical trials and as advisor or speaker from Amicus, Sanofi-Genzyme, Ultragenix, Spark Therapeutics, Audentes, Biomarin, Shire, Ciesi, Sarepta under agreements with Erasmus MC University Medical Center and the relevant industry. HAWMT received unconditional research grants outside the submitted work from Roche, Novartis, CFF, Vertex, Chiesi, Vectura, and Gilead and participated in the last 5 years in expert panels for Vertex and Gilead. All financial aspects for the grants are handled by Sophia Research BV. All other authors report no relevant disclosures.