Padidela, Raja http://orcid.org/0000-0002-3350-9896
Whyte, Michael P. http://orcid.org/0000-0002-8755-8490
Glorieux, Francis H. http://orcid.org/0000-0001-6616-3484
Munns, Craig F. http://orcid.org/0000-0001-5898-5808
Ward, Leanne M. http://orcid.org/0000-0003-1557-9185
Nilsson, Ola http://orcid.org/0000-0002-9986-8138
Portale, Anthony A. http://orcid.org/0000-0003-2054-6898
Simmons, Jill H. http://orcid.org/0000-0002-4189-6718
Namba, Noriyuki http://orcid.org/0000-0002-3803-4500
Cheong, Hae Il http://orcid.org/0000-0001-7556-1265
Pitukcheewanont, Pisit
Sochett, Etienne http://orcid.org/0000-0002-7027-7123
Högler, Wolfgang http://orcid.org/0000-0003-4328-6304
Muroya, Koji http://orcid.org/0000-0002-1810-9894
Tanaka, Hiroyuki http://orcid.org/0000-0002-9117-3534
Gottesman, Gary S. http://orcid.org/0000-0002-0302-3852
Biggin, Andrew http://orcid.org/0000-0002-3443-9359
Perwad, Farzana http://orcid.org/0000-0001-7983-0127
Williams, Angela
Nixon, Annabel http://orcid.org/0000-0003-3454-9491
Sun, Wei
Chen, Angel
Skrinar, Alison
Imel, Erik A. http://orcid.org/0000-0002-7284-3467
Funding for this research was provided by:
Kyowa Kirin International
Article History
Received: 1 October 2020
Accepted: 14 December 2020
First Online: 23 January 2021
Compliance with Ethical Standards
:
: The following authors served as clinical investigators for one or more studies, including this trial, sponsored by Ultragenyx Pharmaceutical Inc. in partnership with Kyowa Kirin International plc: RP, MPW, FHG, CFM, LMW, ON, AAP, JHS, NN, HIC, PP, ES, WH, KM, HT, GSG, AB, FP, and EAI. AAP, NN, FP, and EAI have also received honoraria for serving as advisory board members or for lectures from Ultragenyx Pharmaceutical Inc. RP has received personal fees from Ultragenyx Pharmaceutical Inc. and Kyowa Kirin International plc, a research grant, consultation fees, honoraria, and travel grants from Kyowa Kirin International plc and Alexion UK, and non-financial support from Kyowa Kirin International plc. MPW has received research grant support, honoraria, and travel from Alexion Pharmaceutical Inc. FHG has received personal fees from Kyowa Kirin International plc and research funding from Amgen and Mereo BioPharma. LMW has served as a consultant to Ultragenyx, with funds to LMW’s institution. NN has received personal fees and non-financial support from Kyowa Kirin International plc and Ultragenyx Pharmaceutical Inc. during the conduct of the study, and personal fees and non-financial support (honoraria, consulting fees, and travel support) from Alexion UK outside the submitted work. PP has received research funding from Ultragenyx Pharmaceutical Inc. and is currently an employee of Ascendis Pharma Inc. WH has received honoraria, consulting fees, and travel support from Ultragenyx Pharmaceutical Inc. and research funding, honoraria, and travel support from Kyowa Kirin. GSG has received consulting fees from Ultragenyx Pharmaceutical Inc. AW and WS are employees of Kyowa Kirin International plc. AN is an employee of Chilli Consultancy and has received consultancy fees from Kyowa Kirin International plc to support the development of this manuscript. AC and AS are employees and stockholders of Ultragenyx Pharmaceutical Inc.
: The study was conducted in accordance with the Declaration of Helsinki and the Good Clinical Practice guidelines developed at the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use. Parents or guardians provided written informed consent for their children to participate, and children gave written assent according to local guidelines.