Yee, Karen S. http://orcid.org/0000-0002-6026-1435
Lewis, Sandy
Evans, Emily
Romano, Carla
Alexanderian, David
Funding for this research was provided by:
Shire, a Takeda company
Article History
Received: 12 June 2023
Accepted: 19 January 2024
First Online: 10 March 2024
Declarations
:
: The Phase 2/3 study was approved by the relevant institutional review boards/institutional ethics committees and was conducted in compliance with the International Conference on Harmonisation Good Clinical Practice guidelines and the Declaration of Helsinki. For all patients, written informed consent was obtained from the parent(s) or legally authorized guardian(s); assent from the patient was also acquired, if applicable.
: Informed consent to participate and for the interviews to be audio-recorded was obtained from each caregiver prior to interview.
: Caregivers provided written consent for transcripts to be made available to the sponsor and included in associated publications.
: KSY was an employee of Takeda Development Center Americas, Inc., and stockholder of Takeda Pharmaceuticals Company Limited at the time of study and of the writing of the manuscript (now at Alexion Pharmaceuticals, Inc., AstraZeneca Rare Disease). SL and CR are employees of RTI Health Solutions and were contracted by Shire, a Takeda company, to perform this analysis. EE was an employee of RTI Health Solutions at the time of the study (now at OPEN Health). DA was an employee of Takeda Development Center Americas, Inc., and stockholder of Takeda Pharmaceuticals Company Limited at the time of this study and of the writing of the manuscript (now at Merck).