Stevens, Bob
Kenny, Tom
Thomas, Sophie
Morrison, Alexandra
Jarrett, James
Jain, Mohit
Funding for this research was provided by:
BioMarin Pharmaceutical Inc.
Article History
Received: 28 August 2020
Accepted: 20 May 2021
First Online: 25 September 2021
Declarations
:
: 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. Participating patients were required to sign a Managed Access Patient Agreement, agreeing to the terms of the managed access agreement and providing consent for data to be collected and shared with the manufacturer, NHS England and NICE.
: Not applicable.
: BS, ST: Employees of MPS Society; The MPS Society has received grants and honorariums for attendance at meetings, international travel and meetings, direct advocacy, awareness campaigns, conference & expert meetings, literature and digital communications; TK, AM: Employees of Rare Disease Research Partners; JJ, MJ: Employees of BioMarin Europe Ltd.