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Article History

Received: 27 June 2024

Accepted: 15 November 2024

First Online: 1 July 2025

Declarations

:

: Independent Review Board/Ethics Committee approval was obtained for all participating centers. The Hunter Outcome Survey is conducted in accordance with Good Pharmacoepidemiology Practice (GPP), Good Research for Comparative Effectiveness principles, and the relevant principles of the International Conference on Harmonisation (ICH) Good Clinical Practice guidelines (ICH E6). Each patient, their parents, or a legal representative provided signed and dated written informed consent for participation in the Hunter Outcome Survey. All patient information is managed in accordance with national data protection standards.

: Not applicable.

: 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 program 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). H. Amartino has received consulting fees, fees for service, and/or participated in advisory boards for Amicus Therapeutics, BioMarin Pharmaceutical, Janssen Pharmaceuticals, PTC Therapeutics, Sanofi Genzyme, Ultragenyx, and Takeda. He has performed contracted research for Allievex, Amicus Therapeutics, bluebird bio, JCR Pharmaceuticals, Minoryx Therapeutics, PTC Therapeutics, Sanofi Genzyme, and Takeda. R. Giugliani has received consulting fees, fees for service, speaker fees, and/or travel expenses from, and/or participated in advisory boards for Abeona Therapeutics, Alnylam Pharmaceuticals, Amicus Therapeutics, BioMarin Pharmaceutical, Chiesi Farmaceutici, 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, Janssen Pharmaceuticals, JCR Pharmaceuticals, Lysogene, Sanofi Genzyme, Takeda, and Ultragenyx. P. Harmatz has received consulting fees/other remuneration from Aeglea, Alexion Pharmaceuticals, ArmaGen, Audentes, AVROBIO, BioMarin Pharmaceutical, Capsida Biotherapeutics, Chiesi, Denali Therapeutics, EdiGene, Enzyvant, Fondazione Telethon, Grace Science, Inventiva Pharma, JCR Pharmaceuticals, Neurogene, Novel Pharma, Orchard Therapeutics, Orphazyme, Paradigm Biopharma, PTC Therapeutics, Rallybio, REGENXBIO, Renovion, Sangamo Therapeutics, SalioGen, Sanofi Genzyme, Takeda, and Ultragenyx Pharmaceutical; and has received research support from Alexion Pharmaceuticals, Adrenas, ArmaGen, Amicus, Ascendis, ASPA, Azafaros, BioMarin Pharmaceutical, Calcilytics, Denali, Enzyvant, Homology, Inventiva Pharma, JCR, Orphazyme, Prevail, QED, RegenXbio, Sangamo Therapeutics, Swedish Orphan Biovitrum, Takeda, and Ultragenyx Pharmaceutical. S.-P. Lin has received fees for service from BioMarin Pharmaceutical, Sanofi Genzyme, and Takeda. B. Link has received fees for service and/or travel expenses from BioMarin Pharmaceutical, Chiesi Farmaceutici, Sanofi Genzyme, and Takeda. D. Molter has received fees for service and/or travel expenses from BioMarin Pharmaceutical and Takeda. U. Ramaswami has participated in advisory boards for Amicus Therapeutics, JCR Pharmaceuticals, Sanofi, and Takeda. She has received research grants from Amicus Therapeutics, IntraBio, and Takeda. M. Scarpa has received consulting fees, fees for service, and/or participated in advisory boards for Actelion Pharmaceuticals, Alexion Pharmaceuticals, BioMarin Pharmaceuticals, Chiesi Farmaceutici, Orchard Therapeutics, Orphazyme, Paradigm Biopharma, 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. J. Botha is a full-time employee of Takeda and stockholder of Takeda Pharmaceuticals Company Limited. J. Audi was an employee of Takeda and stockholder of Takeda Pharmaceuticals Company Limited at the time of this analysis (current affiliation is Ultragenyx Europe GmbH, Allschwil, Basel, Switzerland). B.K. Burton has received consulting fees and/or participated in advisory boards for Agios Pharmaceuticals, Alexion, Alltrna, Amgen, Aro, Applied Therapeutics, BioMarin Pharmaceutical, Chiesi Farmaceutici, JCR Pharmaceuticals, Jnana Therapeutics, Moderna, Orchard Therapeutics, Passage Bio, Sanofi Genzyme, Takeda, Travere, and Ultragenyx. She has performed contracted research for Takeda and has been involved in company-sponsored clinical trials with BioMarin Pharmaceutical, Denali Therapeutics, Homology Medicines, JCR Pharmaceuticals, Jnana Therapeutics, Sangamo Therapeutics, Synlogic, Takeda, and Ultragenyx.