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Article History

Received: 14 March 2023

Accepted: 14 April 2023

First Online: 24 April 2023

Declarations

:

: This study was approved by the Medical Ethics Committee of the University Medical Center Utrecht (No. 20–143 (Version 3) /NL72562.041.20) and registered in the Dutch registry for clinical studies and trials (ExternalRef removed). The trial is monitored by an internal party of the University Medical Center Utrecht. The study is conducted according to the principles of the Declaration of Helsinki, adapted 19–10-2013, and in accordance with the Medical Research Involving Human Subjects Act (WMO). Informed consent to participate will be obtained from all participants and/or their parents or legal representative in case of minors. Minors are participants aged 12 through 16 years old as defined by the regulation of the Dutch Central Committee on Research Involving Human Subjects.

: Not applicable.

: HG has received research grants from Prinses Beatrix spierfonds, travel grants from Shire/Takeda and speaker fees from Takeda paid to the institution. BB receives research support from the Prinses Beatrix Spierfonds, Stichting Spieren for Spieren and Health Holland, all non-profit foundations. His employer receives fees for SMA-related consultancy activities for Biogen, Novartis, Scholar Rock and Roche. IC provides ad hoc consultancy for AveXis (Novartis). BS and RvE receive research support from the Netherlands ALS foundation. RW receives research support from the Prinses Beatrix Spierfonds and Stichting Spieren voor Spieren. WP serves on the scientific advisory board for SMA Europe, is a member of the Branaplam data monitoring committee for Novartis, provides ad hoc consultancy for Biogen and AveXis (Novartis), and receives research support from the Prinses Beatrix Spierfonds, Vriendenloterij and Stichting Spieren voor Spieren. All other authors have no competing interests.