Text and Data Mining valid from 2017-10-03
Received: 24 April 2017
Accepted: 24 September 2017
First Online: 3 October 2017
Ethics approval and consent to participate
: Institutional Review Board/Ethics Committee approval was obtained for all participating centres. HOS is conducted in accordance with Good Pharmacoepidemiology Practices, 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 HOS.
: Each patient, their parents or a legal representative provided informed consent for publication of data.
: JM is a consultant to BioMarin, Eloxx, Sanofi Genzyme, Green Cross, Janssen Pharmaceuticals, PTC Therapeutics and Shire. He also serves on advisory boards for BioMarin, Sanofi Genzyme and Shire. He is principal investigator for phase 1/2 and phase 2/3 trials, sponsored by Shire, that investigate intrathecal ERT for patients with the severe form of Hunter syndrome.RG has received travel grants from BioMarin, Sanofi Genzyme and Shire, research grants from Actelion Pharmaceuticals, Alexion, Amicus, BioMarin, Sanofi Genzyme and Shire, and honoraria for speaking engagements from Actelion Pharmaceuticals, Alexion, Amicus, BioMarin, Sanofi Genzyme, PTC Therapeutics and Shire.MS has received grants, travel support and honoraria from Actelion Pharmaceuticals, Alexion, BioMarin, Sanofi Genzyme, Shire and PTC Therapeutics.AT-S has received travel grants and speaker fees from BioMarin, Sanofi Genzyme, Shire and Synageva BioPharma.Ms. Jego is an employee of Cytel, Inc. and has received consulting fees from Shire.MB has received honoraria, travel support and unrestricted grants from Actelion Pharmaceuticals, BioMarin, Sanofi Genzyme and Shire.
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