Chow, Andrea J.
Pugliese, Michael
Tessier, Laure A.
Chakraborty, Pranesh
Iverson, Ryan
Coyle, Doug
Kronick, Jonathan B.
Wilson, Kumanan
Hayeems, Robin
Al-Hertani, Walla
Inbar-Feigenberg, Michal
Jain-Ghai, Shailly
Laberge, Anne-Marie
Little, Julian
Mitchell, John J.
Prasad, Chitra
Siriwardena, Komudi
Sparkes, Rebecca
Speechley, Kathy N.
Stockler, Sylvia
Trakadis, Yannis
Walia, Jagdeep S.
Wilson, Brenda J.
Potter, Beth K. http://orcid.org/0000-0001-8374-6542
Funding for this research was provided by:
Canadian Institutes of Health Research (TR3–119195)
Article History
Accepted: 29 June 2021
First Online: 20 July 2021
Declarations
:
: This study was funded by the Canadian Institutes of Health Research Emerging Grant: Rare Diseases (Emerging team in rare diseases: Achieving the ‘triple aim’ for inborn errors of metabolism), # TR3–119195. The open access fee is supported by funding from the Canadian Insitutes for Health Research.
: Kumanan Wilson is the CEO of CANImmunize Inc. He is a paid member of the data safety board for the Medicago vaccine trial and a paid external advisor for the Auditor General report. In the past 3 years, Michal Inbar-Feigenberg has had the following relationships, none of which is connected directly to the material covered in this article: served on advisory boards for Audentes Therapeutics, Sanofi Genzyme, Shire/Takeda, and Horizon Therapeutics; received consulting fees from Shire/Takeda; performed contracted research for Shire/Takeda and Sanofi Genzyme; received research support from Shire/Takeda, Sanofi Genzyme, and Vtess Inc/Mallinckrodt; received speaker fees from Horizon Therapeutics (May 2019); and had travel expenses paid by Sanofi Genzyme and Shire/Takeda. John J. Mitchell has worked with pharmaceutical companies that market products for the treatment of inborn errors mentioned in this article. This relationship did not have any impact on the design or review of this paper. Andrea J. Chow<b>,</b> Michael Pugliese, Laure A. Tessier, Pranesh Chakraborty, Ryan Iverson, Doug Coyle, Jonathan B. Kronick, Robin Hayeems, Walla Al-Hertani, Shailly Jain-Ghai, Anne-Marie Laberge, Julian Little, Chitra Prasad, Komudi Siriwardena, Rebecca Sparkes, Kathy N. Speechley, Sylvia Stockler, Yannis Trakadis, Jagdeep S. Walia, Brenda J. Wilson, and Beth K. Potter have no conflicts of interest that are directly relevant to the content of this article.
: This study was approved by the Children’s Hospital of Eastern Ontario (Reference 16/101X) and the Ottawa Health Science Network Research Ethics Board (Reference 20160608-01H), as well as the research ethics boards of all participating hospital centers.
: Informed consent was explained on the cover page of the questionnaire. Completion and return of the questionnaire constituted informed consent.
: The Research Ethics Board did not require us to obtain consent specifically for publication of the results but participants received information about the survey and were instructed that completion and return of the questionnaire constituted consent to participate in the study.
: The study questionnaire is available from the corresponding author upon reasonable request.
: The code is available from the corresponding author upon reasonable request.
: BKP, PC, DC, JBK, KW, RH, WA, MIF, SJG, AML, JL, JJM, CP, KS, RS, KNS, SS, YT, JSW, and BJW conceptualized, designed, and planned the study. MP, LAT, PC, JBK, RH, SJG, AML, JJM, CP, KS, RS, KNS, SS, YT, JSW, and BKP acquired the data. AJC, MP, RI, and BKP analyzed and interpreted the data. DC, JBK, KW, RH, WA, MIF, AML, JL, JJM, CP, and SS interpreted the data. AJC, MP, and BKP drafted the manuscript. All authors critically revised the manuscript and approved the final manuscript.