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Article History

Received: 24 February 2021

Accepted: 20 September 2021

First Online: 30 October 2021

Declarations

:

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

: Each patient, their parents or a legal representative provided informed consent for publication of data.

: JM has received consulting fees from and/or participated in advisory boards for Bluebird Bio, Denali Therapeutics, Homology Medicines, JCR Pharmaceuticals, REGENXBIO, Sanofi Genzyme and Shire (a Takeda company). He is a Principal Investigator for phase 1/2 and phase 2/3 intrathecal enzyme replacement trials for the neuronopathic form of MPS II (sponsored by Shire, a Takeda company), a phase 1/2 gene editing trial for adults with MPS II (sponsored by Sangamo Therapeutics) and a phase 1/2 intravenous enzyme replacement therapy trial for MPS IIIA (sponsored by Swedish Orphan Biovitrum). JB is a full-time employee of Takeda and stockholder of Takeda Pharmaceuticals Company Limited. PH has received consulting fees and/or fees for service from Aeglea BioTherapeutics, Alexion Pharmaceuticals, ArmaGen, Audentes Therapeutics, BioMarin Pharmaceutical, Chiesi Farmaceutici, Denali Therapeutics, Enzyvant, Homology Medicines, Inventiva Pharma, JCR Pharmaceuticals, Orphazyme, Paradigm Biopharma, Pfizer, PTC Therapeutics, REGENXBIO, Sangamo Therapeutics, Sanofi Genzyme, Shire (a Takeda company), Swedish Orphan Biovitrum and Ultragenyx Pharmaceutical. RG 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, Denali Therapeutics, Inventiva Pharma, Janssen Pharmaceuticals, JCR Pharmaceuticals, Novartis, Orphan Disease Center, REGENXBIO, Sanofi Genzyme, Shire (a Takeda company), Sigilon Therapeutics, Swedish Orphan Biovitrum and Ultragenyx Pharmaceutical. He has performed contracted research for Allievex, Amicus Therapeutics, Idorsia Pharmaceuticals, JCR Pharmaceuticals, Lysogene, Sanofi Genzyme, Shire (a Takeda company) and Ultragenyx Pharmaceutical. CK has no competing interests to declare. BKB has received consulting and/or speaker fees from and/or participated in advisory boards for Aeglea, Agios, Alexion Pharmaceuticals, BioMarin Pharmaceutical, Chiesi Farmaceutici, Denali Therapeutics, Horizon Therapeutics, Inventiva Pharma, JCR Pharmaceuticals, Moderna, Pfizer, REGENXBIO, Reneo Pharmaceuticals, Sanofi Genzyme and Shire (a Takeda company). She has performed contracted research for Shire (a Takeda company) and has been involved in company-sponsored clinical trials with BioMarin Pharmaceutical, Homology Medicines, Sangamo Therapeutics, Shire (a Takeda company) and Ultragenyx Pharmaceutical.