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Authors
Kilavuz, Sebile
Wallom, Kerri-Lee
Caçote, Ana Catarina Gomes Almeida Augusto
Donald, Aimée
D’Amore, Simona
Page, Kathy
Tan, Chong Yew
Vruchte, Danielle te
Sturchio, Andrea
Tsitsi, Panagiota
Hertz, Ellen
Andréasson, Mattias
Markak, Ioanna
Svenningsson, Per
Cox, Timothy M.
Platt, Frances M. https://orcid.org/0000-0001-7614-0403
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Article History
Received: 18 November 2024
Accepted: 16 September 2025
First Online: 28 October 2025
Declarations
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: Ethical approval was given by the national ethics review committee (14/EE/1168) under the purview of the NHS Research and Development (R&D) in the United Kingdom and from the local R&D boards at each of the eight participating specialist clinical centers. Informed consent was obtained from all subjects at the time of enrolment into GAUCHERITE. The research cohort is registered with ClinicalTrials.gov, NCT code, 03240653. Ethical approval for the Parkinson’s disease cohort (BioPark cohort) was obtained from the local ethics review board at the Karolinska Institute, Sweden.
: No restrictions.
: We acknowledge some limitations to our study. Plasma gpNMB concentrations were analysed in patients who were already on treatment. Since analyses were performed at a specific time for all patients, long-term gpNMB alterations during the disease process were not captured in this study. Additionally, the relatively small number of patients receiving SRT may limit the statistical power to detect subtle differences between treatment modalities.
: The authors declare that they have no competing interests.
