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Authors
Mole, Sara E.
Gissen, Paul
Nordstrom, Shannon https://orcid.org/0009-0007-6553-9647
Wait, Suzanne
Allen, Louise
Antonini, Mathilda
Brownnutt, Liz
Brown, Richard
Cole, Barbara
Gibbon, Frances
Henderson, Robert H.
Kenrick, Sarah
Sisic, Zlatko
Thompson, Bob
Nightingale, Joanna
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Funding
Funding for this research was provided by:
BioMarin Pharmaceutical (BioMarin Pharmaceutical)
Amicus Therapeutics (Amicus Therapeutics)
REGENXBIO (REGENXBIO)
Robert Luff Foundation (Robert Luff Foundation)
The Frognal Trust (The Frognal Trust)
The Catherine Cookson Charitable Trust (The Catherine Cookson Charitable Trust)
The Sir Samuel Scott of Yews Trust (The Sir Samuel Scott of Yews Trust)
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Article History
Received: 20 June 2024
Accepted: 18 April 2025
First Online: 12 May 2025
Declarations
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: This research was conducted in accordance with the British Healthcare Business Intelligence Association’s Legal & Ethical Guidelines for Market Research. The study was non-interventional, with subjects recruited via the BDFA. All participants provided written consent before participating in the study.
: Not applicable.
: S.W. is an employee and S.N. is a consultant at The Health Policy Partnership Ltd, the organisation which received funding to conduct this research and draft the manuscript from the BDFA. P.G. has received payments to his institution as a study site according to clinical trial budget and honoraria and travel support from BioMarin Pharmaceutical. F.G. has received fees for advisory boards and travel grants from BioMarin Pharmaceutical.
