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Authors
Morsy, Adel Sabet
Mbua, Solomon
Mathieson, Toni
Hopkin, Justin
Bolton, Shaun https://orcid.org/0000-0003-0160-1505
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Funding
Funding for this research was provided by:
International Niemann-Pick Disease Registry (International Niemann-Pick Disease Registry)
Niemann-Pick UK (Niemann-Pick UK)
National Niemann-Pick Disease Foundation (National Niemann-Pick Disease Foundation)
Sanofi Genzyme (Sanofi Genzyme)
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Article History
Received: 2 May 2025
Accepted: 12 August 2025
First Online: 30 September 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. Informed consent was obtained from all participants during the survey stage and interview stage of the study.
: Not applicable.
: TM acted as an expert witness in the NICE evaluation process for olipudase alfa, alongside other patient and clinical experts. Additionally, NPUK has received grant funding from Sanofi in support of our educational activities for patients and healthcare professionals. As an advocacy leader, JH accepted reimbursement for travel from Sanofi to attend a Policy and Patient Advocacy Advisory Board in 2024, a patient Advocacy Council in 2025, and a Sanofi Registry Patient Council in 2019. Any honorarium for these events was deferred to either the INPDR or the NNPDF. SB has received travel grants from Amicus Therapeutics, Sangamo Therapeutics and Biomarin, and received travel grants and honorarium from Sanofi.
: Not applicable.
