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Article History

Received: 10 April 2026

Accepted: 6 July 2026

First Online: 22 July 2026

Declarations

Ethics approval and consent to participate: 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 for publication: Consent was obtained from participants for the use of quotes and publication.

Competing interests: JBH: Principal Investigator for SPARKLE registry. Received honoraria and/or travel expenses from Chiesi Farmaceutici S.p.A., Takeda, Sanofi and Immedica. NG: Principal Investigator for SPARKLE registry. Received hospital grants as well as consulting fees and/or honoraria/travel support from BioMarin, Chiesi Farmaceutici S.p.A., Immedica, JCR pharmaceuticals, Lucane Pharma, Sanofi winthrop, Ultragenyx, and Takeda. ST: The MPS Society has received money from Chiesi for its patient support services, Alpha Mannosidosis online meeting, review of patient resources and lay materials, Alpha Mannosidosis awareness campaign, participation in advisory boards, presenting at meetings, attendance and travel to meetings. KMS: Principal Investigator for SPARKLE registry, speaker’s and consultancy fees from Chiesi, Biomarin. CL: Principal Investigator for SPARKLE registry, speaker’s and consultancy fees from Chiesi. NMM: Principal Investigator for sparkle registry, received travel support, consulting fees and/or speaker’s honoraria from Amicus, Biomarin, Chiesi, GC Biopharma, JCR Pharmaceuticals, Sanofi Genzyme, Spruce Biosciences, Takeda and Ultragenyx. MJBM: No competing interests to declare. JC: The Spanish MPS Society has received money from Chiesi for its patient support services and participation in advisory boards, presenting at meetings, attendance and travel to meetings. MLR: Has received research grants, speakers’ honoraria and/or consultation fees from Chiesi, Amicus, Sanofi, and Takeda. ABar: Has received consulting fees and/or honoraria/travel support from BioMarin, Chiesi Farmaceutici S.p.A., Sanofi Genzyme, and Takeda. AML: Received consulting fees and/or meeting/travel cover from Chiesi Farmaceutici S.p.A., Immedica, Moderna, Sanofi Genzyme, Takeda and Ultragenyx. VP: Principal Investigator for SPARKLE registry, speaker’s and consultancy fees from Chiesi. CD: Employee of Chiesi Farmaceutici S.p.A. HMM: Employee of Chiesi USA. ABal: Former employee of Chiesi Farmaceutici S.p.A. at the time of this study. MM: received lecture honoraria and travel expenditures from Chiesi Farmaceutici S.p.A., BioMarin and Takeda.