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Article History

Received: 18 December 2024

Accepted: 21 March 2025

First Online: 7 May 2025

Declarations

:

: RDRP conducted this market research in accordance with the British Healthcare Business Intelligence Association’s (BHBIA) Legal & Ethical Guidelines for Market Research. In line with these standards, this market research did not require review of a Research Ethics Committee because it falls outside the remit of the UK Research Governance Framework. The confidentiality of information gathered during the study is protected in accordance with applicable data protection legislation and BHBIA’s Legal & Ethical Guidelines. Informed consent was obtained online from all participants in their local language at the start of the online survey. Respondents to the survey had to confirm they were 18 years old or over. Those not providing consent were screened out and were not able to continue with the survey.

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

: KMS: PI for SPARKLE registry, consulting fees and/or honoraria/travel support from Chiesi, Sanofi Genzyme, BioMarin, and Takeda, Immedica. ST: The MPS Society has received money from Chiesi for participation and chairing of advisory boards, satellite symposiums (participation, time, travel & accommodation), Fabry matters conference, patient support services, mental health services, website re-development, consultation fees for review of lay materials. JBH: Received consulting fees and/or honoraria from Chiesi, Sanofi, Takeda, Amicus, and Immedica as well as travel support from Chiesi and Amicus. CL: Has received research grants, speakers’ honoraria and/or consultation fees from Chiesi, Amicus, Alexion, BioMarin, Sanofi, and Takeda. NMM: Has received research grants, speakers’ honoraria and/or consultation fees from Chiesi, Amicus, BioMarin, JCR, Lysogene, Sanofi, and Takeda. MJBM: PI for SPARKLE registry, no competing interests. JC: No competing interests. MLR: Has received research grants, speakers’ honoraria and/or consultation fees from Chiesi, Amicus, Sanofi, and Takeda. AB: Has received consulting fees and/or honoraria/travel support from BioMarin, Chiesi Farmaceutici S.p.A., Sanofi Genzyme, and Takeda. MM: PI for SPARKLE registry, consulting fees and/or honoraria/travel support from Chiesi, BioMarin, and Takeda. AML: Has received hospital grants as a PI for studies from Chiesi Farmaceutici S.p.A. as well as consulting fees and/or honoraria/travel support from Alexion, BioMarin, Chiesi Farmaceutici S.p.A., Sanofi Genzyme, and Shire. VP: PI for SPARKLE registry, speakers’ honoraria/travel support and/or consulting fees from BioMarin, Chiesi, Sanofi Genzyme, Shire/Takeda. AB: Former employee of Chiesi Farmaceutici S.p.A. during the time of this study. FD: Employee of Chiesi Farmaceutici S.p.A. HMM: Employee of Chiesi USA Inc. NG: Has received hospital grants as well as consulting fees and/or honoraria/travel support from BioMarin, Chiesi Farmaceutici S.p.A., JCR pharmaceuticals, Sanofi winthrop, Ultragenyx, and Takeda.