Cleary, Maureen http://orcid.org/0000-0002-1282-6315
Davison, James
Gould, Rachel
Geberhiwot, Tarekegn
Hughes, Derralynn
Mercer, Jean
Morrison, Alexandra
Murphy, Elaine
Santra, Saikat
Jarrett, James
Mukherjee, Swati
Stepien, Karolina M.
Funding for this research was provided by:
BioMarin Pharmaceutical
Article History
Received: 22 June 2020
Accepted: 5 January 2021
First Online: 21 January 2021
Ethics approval and consent to participate
: All patients or their parents provided written, signed informed consent to participate in the programme. Institutional review board/ethics committee oversight was not applicable; patients receiving elosulfase alfa under the managed access agreement received care from their treating physician in accordance with all local standards of care.
: Not applicable.
: Maureen Cleary received personal fees from BioMarin for attending an advisory board related to this study, and personal fees for advisory boards and a travel grant to attend a metabolic meeting from BioMarin not related to his work. James Davison received an honorarium from BioMarin for attending an advisory board related to this study and personal fees for travel, accommodation and conference registration costs to attend a conference not related to the submitted work. Rachel Gould has received a non-restricted travel grant and participated in BioMarin advisory boards. Tarekegn Geberhiwot received personal fees from BioMarin for consultancy not related to this study. Derralynn Hughes received personal fees for advisory boards from BioMarin related to this study. Jean Mercer has nothing to disclose. Alexandra Morrison declares that MPS Commercial is a Private Limited Company trading as Rare Disease Research Partners and is a wholly owned, not-for-profit subsidiary of the Society for Mucopolysaccharide Diseases, UK. Rare Disease Research Partners provides professional PRO data collection services for the elosulfase alfa MAA under contract to BioMarin. Elaine Murphy has worked as a clinical trial investigator for BioMarin, related to this work. Saikat Santra has received non-restricted travel and educational grants, participated as an investigator in clinical research studies, and served on advisory boards for BioMarin. James Jarrett is an employee and stockholder of BioMarin. Swati Mukherjee is an employee and stockholder of BioMarin. Karolina Stepien has received speaker fees and a non-restricted travel and educational grant and participated in BioMarin advisory boards.