Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II.

"Declarations Ethics approval and consent to participateThe 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 consentInformed consent to participate and for the interviews to be audio-recorded was obtained from each caregiver prior to interview. Consent for publicationCaregivers provided written consent for transcripts to be made available to the sponsor and included in associated publications. Competing interestsKSY 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). Competing interests 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)."

"Funding This study was funded by Shire, a Takeda company. Under the direction of the authors, medical writing support was provided by Helen Bremner PhD of Oxford PharmaGenesis, Oxford, UK, and was funded by Takeda Development Center Americas, Inc."