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Authors
D’Amore, Simona
Page, Kathleen
Donald, Aimée
Taiyari, Khadijeh
Tom, Brian
Deegan, Patrick
Tan, Chong Y.
Poole, Kenneth
Jones, Simon A.
Mehta, Atul
Hughes, Derralynn
Sharma, Reena
Lachmann, Robin H.
Chakrapani, Anupam
Geberhiwot, Tarekegn
Santra, Saikat
Banka, Siddarth
Cox, Timothy M. https://orcid.org/0000-0002-4951-9941
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Cox, T. M.
Platt, F. M.
Banka, S.
Chakrapani, A.
Deegan, P. B.
Geberhiwot, T.
Hughes, D. A.
Jones, S.
Lachmann, R. H.
Santra, S.
Sharma, R.
Vellodi, A.
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Funding
Funding for this research was provided by:
Medical Research Council (MR/K015338/1)
National Institute for Health Research (BRC-1215-20014)
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Article History
Received: 22 May 2021
Accepted: 19 September 2021
First Online: 14 October 2021
Declarations
:
: This study was conducted according to the principles of the Declaration of Helsinki (2013) []. 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 centres. 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.
: No restrictions.
: SHIRE Biopharmaceuticals UK (now part of Takeda) supported the licence fee for the SF-36v2 questionnaire (< $6000). The authors confirm that neither SHIRE nor any other external agency played any part in the study design, data collection, analysis—nor interpretation. The named authors are solely responsible for the manuscript and the decision to submit it for publication.
