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Article History

Received: 1 November 2024

Accepted: 25 March 2026

First Online: 6 June 2026

Declarations

:

: The study was conducted in accordance with the Guidelines for Good Pharmacoepidemiology Practice published by the International Society for Pharmacoepidemiology and the laws and regulations of the countries in which the research was conducted. Ethical approval for the study was obtained from Salus Institutional Review Board in all countries except Canada. Ethical approval in Canada was obtained from Veritas. All participants were required to provide written informed consent.

: Not applicable.

: K. Stepien has received consulting fees and/or participated in advisory boards for BioMarin Pharmaceutical, Chiesi, Sanofi Genzyme, Sigilon Therapeutics and Takeda. O. Ayodele is a full-time employee of Takeda Development Center Americas, Inc. and stockholder of Takeda Pharmaceutical Company Limited. J. Muenzer has received consulting fees and/or participated in advisory boards for Denali Therapeutics, JCR Pharmaceuticals, REGENXBIO, Sanofi Genzyme and Takeda. He is a Principal Investigator for a post-trial access programme for intrathecal ERT for the neuronopathic form of MPS II (sponsored by Takeda), a phase 1/2 gene editing trial for adults with MPS II (sponsored by Sangamo Therapeutics), and phase 1/2 and phase 2/3 trials of IV ERT for MPS II (sponsored by Denali Therapeutics). R. Giugliani has received consulting fees, fees for service, speaker fees and/or travel expenses from and/or has participated in advisory boards for Abeona Therapeutics, Alnylam Pharmaceuticals, Amicus Therapeutics, BioMarin Pharmaceutical, Chiesi, Inventiva Pharma, Janssen Pharmaceuticals, JCR Pharmaceuticals, Novartis, Orphan Disease Center, Praxis Precision Medicines, PTC Therapeutics, REGENXBIO, Sanofi Genzyme, Sigilon Therapeutics, Swedish Orphan Biovitrum, Takeda and Ultragenyx. He has performed contracted research for or received research grants from Allievex, Amicus Therapeutics, BioMarin Pharmaceutical, GC Pharma, Idorsia Pharmaceuticals, Janssen Pharmaceuticals, JCR Pharmaceuticals, Lysogene, Sanofi Genzyme, Takeda and Ultragenyx. M. Scarpa has received consulting fees, fees for service and/or participated in advisory boards for Actelion Pharmaceuticals, Alexion Pharmaceuticals, BioMarin Pharmaceuticals, Chiesi, Orchard Therapeutics, Orphazyme, Paradigm Biopharmaceuticals, PTC Therapeutics, Sanofi Genzyme, Takeda and Ultragenyx. He has performed contracted research for Alexion Pharmaceuticals, BioMarin Pharmaceutical, CTD, Orchard Therapeutics, Orphazyme, Paradigm Biopharmaceuticals, PTC Therapeutics, Sanofi Genzyme and Takeda. C. Lampe has received advisory board and speaker fees, travel expenses and consultancy fees from Alexion Pharmaceuticals, Amicus Therapeutics, BioMarin Pharmaceutical, Chiesi, REGENXBIO, Sanofi and Takeda. J. Audi was an employee of Takeda Pharmaceuticals International AG and a stockholder of Takeda Pharmaceuticals Company Limited at the time of this analysis (current affiliation is Ultragenyx Europe GmbH, Basel, Switzerland). S. Jain is a full-time employee of Takeda Development Center Americas Inc. and a stockholder of Takeda Pharmaceutical Company Limited. C. Jackson was an employee of Sprout Health Solutions, which provides research and other consulting services to multiple pharmaceutical and biotechnology companies, at the time the study was performed. Sprout Health Solutions was contracted by Takeda Pharmaceutical Company Limited to perform this study. L. Meade is an employee of Sprout Health Solutions, which provides research and other consulting services to multiple pharmaceutical and biotech companies. Sprout Health Solutions was contracted by Takeda Pharmaceutical Company Limited to perform this study. M. del Toro has received consulting fees and speaker honoraria, travel expenses and congress fees from BioMarin, Orchard, Sanofi Genzyme and Takeda, and is an investigator for industrial trials (Takeda, Ultragenyx, Denali, Chiesi).