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Defining Bayley Scales of Infant and Toddler Development Third Edition (BSITD-III) meaningful change and item relevance in children with neuronopathic MPS II: a caregiver interview-based study

Crossref DOI link: https://doi.org/10.1186/s13023-026-04205-9

Published Online: 2026-01-21

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Phillips, Dawn

Wilson, Catherine

Fernandez, Vivian
Funding

Funding for this research was provided by:

REGENXBIO (REGENXBIO)
License Information

Text and Data Mining valid from 2026-01-21

Version of Record valid from 2026-02-16
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Article History

Received: 29 July 2025

Accepted: 8 January 2026

First Online: 21 January 2026

Declarations

:

: Ethical approval was not required. Rare Disease Research Partners (RDRP) conducted the research in accordance with the British Healthcare Business Intelligence Association’s Legal and Ethical Guidelines. Online informed consent was obtained from all participants in their local language prior to the interviews. Proof that informed consent was obtained is available upon request for each of the pseudonymized participants. Optional consent for the use of direct quotes was obtained.

: Consent was obtained from participants for the use of quotes and publication.

: CW and VF are employees of REGENXBIO Inc. DP was an employee of REGEXNBIO at the time of the study.

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