Theunissen, Maudy T. M.
van den Elsen, Renee M.
House, Tiffany L.
Crittenden, Brad
van Doorn, Pieter A.
van der Ploeg, Ans T.
Kruijshaar, Michelle E.
van der Beek, Nadine A. M. E. http://orcid.org/0000-0001-9161-3301
Funding for this research was provided by:
Helen Walker Research Grant
Article History
Received: 28 July 2023
Revised: 11 September 2023
Accepted: 13 September 2023
First Online: 20 November 2023
Declarations
:
: A.T.v.d.P. has received funding for research, clinical trials and advisory fees from Sanofi, Amicus Therapeutics, Spark Therapeutics, Denali Therapeutics and Takeda working on ERT 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. N.A.M.E.v.d.B has received speaker honorarium and research funding from Sanofi, under agreements with Erasmus MC University Medical Center and the relevant industry. The other authors have no relevant financial or non-financial interests to disclose.
: This is an observational study. This study design was submitted for approval by the Medical Ethics Committee of the Erasmus MC University Medical Center. It was labeled as not subject to Medical Research Involving Human Subjects Act (WMO) on the June 28, 2022 (MEC-2022-0411).
: Informed consent was obtained from all individual participants included in the study. This was done by providing online informed consent by checking the consent question in the online questionnaire.
: Anonymized data are available from the corresponding author on request by a qualified academic investigator. Data transfer will be in agreement with EU legislation on the general data protection regulation and decisions by the Ethical Review Board of the ErasmusĀ MC University Medical Center.