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Article History

Received: 29 September 2022

Accepted: 22 January 2023

First Online: 3 March 2023

Declarations

:

: LMW has participated in clinical trials with ReveraGen, Ascendis, PTC, Catabasis, Novartis, Ultragenyx and Amgen, received unrestricted educational grants from Alexion, Ipsen and Ultragenyx, and received consulting fees from Santhera, Ipsen, Ultragenyx, PTC, Novartis, and Amgen (with funds to LMW's institution). MER has participated in clinical trials with Amgen (with funds to MER’s institution). SC has received research grant funding from Zimmer Biomet and ConMed Linvatec, consulting fees for assisting with surgical training from Stryker and Smith & Nephew, and has participated in a clinical trial with Ascendis Biopharma (with Funds to SC’s institution). KS has consulted for Medtronic and has received research grants from Meditronics, Ascendis and SpinoModulation (all funds to KS’s institution). FR has been a consultant to Sanofi and Ultragenyx, received research grants from MITACS and Mereo BioPharma and a study grant from Ultragenyx, Catabasis, PreciThera (all funds to FR’s institution). The other co-authors have no competing interests to declare.

: All procedures performed in this study that involved human participant were in accordance with the 1964 Helsinki Declaration and its later amendments. Written consent was obtained from the patient’s caregiver in line with the Children’s Hospital of Eastern Ontario Research Institute ethical requirements. Written consent was obtained from the patient’s caregiver in line with the Children’s Hospital of Eastern Ontario Research Institute ethical requirements.