Funding for this research was provided by:
Sanofi Genzyme (n/a)
Received: 6 January 2020
Accepted: 17 March 2020
First Online: 15 April 2020
Ethics approval and consent to participate
: All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. Ethics approval for this study was granted by Schulman IRB and Advarra IRB. Informed consent was obtained from all individual participants included in the study.
: Written informed consent for the publication of the study results was obtained from patients and caregivers. De-identified information is presented to ensure anonymity.
: NL, RPJ, CR, JK, TF, and AH are employees of Sanofi Genzyme. RK and NS were employees of IQVIA, Inc. at the time the study was conducted and were paid consultants of Sanofi Genzyme. CG is an employee of Gwaltney Consulting and was a paid consultant of Sanofi Genzyme. GFC is an employee of Gerald Cox Rare Care Consulting, LLC and was an employee of Sanofi Genzyme at the time this study was conducted.