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Authors
Bolton, Shaun C. https://orcid.org/0000-0003-0160-1505
Soran, Vina
Marfa, Mercedes Pineda
Imrie, Jackie
Gissen, Paul
Jahnova, Helena
Sharma, Reena
Jones, Simon
Santra, Saikat
Crushell, Ellen
Stampfer, Miriam
Coll, Maria Jose
Dawson, Charlotte
Mathieson, Toni
Green, James
Dardis, Andrea
Bembi, Bruno
Patterson, Marc C.
Vanier, Marie T.
Geberhiwot, Tarekegn
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Funding
Funding for this research was provided by:
Consumers, Health and Food Executive Agency (2012 1201)
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Article History
Received: 27 September 2021
Accepted: 30 January 2022
First Online: 14 February 2022
Declarations
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: This study was conducted in accordance with the Declaration of Helsinki and the International Conference for Harmonisation—Good Clinical Practice. This study was approved by the research ethics committees and institutional review boards of the participating centres. Written informed consent was obtained from study participants, parents or legal representatives.
: AD, BB, TH, SJ, PG and MTV has received travel grant from Actelion. MP has received travel grant from Actelion, Biomarin, Orphazyme, Esteve, Janssen and Chiesi. SCB has received travel grant from Amicus Therapeutics and Sanofi Genzyme. Takeda has provided funding to TH, SJ, MTV, AD, BB and MP. Genzyme has provided funding to TH, YN, SJ, SS, JI, MH and MP. Orphazyme has provided funding to JI, AD and BB. Alexion has provided funding to MP. Vtesse has provided travel grants to MTV and MP. MP has received funding from Agio, Amicus and Novartis. SJ has received funding from Biomarin, Ultragenyx, PTC and Orchard Theraputics.
