Document is current

Article History

Received: 2 January 2019

Accepted: 25 April 2019

First Online: 29 May 2019

Ethics approval and consent to participate

: Not applicable.

: Not applicable.

: P Harmatz has conducted consultancy work and/or has received grant support from Alexion, Armagen, BioMarin Pharmaceutical Inc., Chiesi, Denali, Genzyme, Enzyvant, Inventiva, JCR Pharmaceuticals, Orphazyme, Pfizer, PTC Therapeutics, RegenXbio, Sangamo, Shire, SOBI and Ultragenyx. C Hendriksz; is Owner and Director of FYMCA Medical Ltd.; has conducted consultancy work for Actelion, Amicus, Alexion, Audentes, BioMarin Pharmaceutical Inc., Chiesi, Evidera, Inventiva, GSK, SOBI, Health Care at Home, Sanofi Genzyme, Shire. R Giugliani has received consultancy fees, and/or investigator fees, and/or speaker honoraria and/or travel grants to attend scientific meetings from Actelion, Amicus, Armagen, BioMarin, Chiesi, GC Pharma, Inventiva, JCR Pharmaceuticals, Lysogene, RegenxBio, Sanofi Genzyme, Shire, Sobi, and Ultragenyx. M Scarpa has received honoraria, research and travel grants from Alexion, BioMarin Pharmaceutical Inc., Chiesi, Sanofi Genzyme, Shire, Ultragenix and Sangamo. MU Akyol has received speaker honoraria and travel grants from Shire. J Ashworth has received honoraria, travel expenses and a research grant from BioMarin Pharmaceutical Inc. and has received consultancy fees from AbbVie and Inventiva. T Alden has received consultant and honoraria payments from BioMarin Pharmaceutical Inc. and Shire. K Belani was a co-investigator on several studies funded by grants from Lysogene and Shire and has received a travel grant from BioMarin Pharmaceutical Inc. for attendance at an advisory meeting. H Amartino has conducted investigator, speaker and consultant work for Amicus, BioMarin Pharmaceutical Inc., Bluebird Bio, Sanofi Genzyme and Shire. K Berger has received consultancy and honoraria payments from BioMarin Pharmaceutical Inc. and Sanofi Genzyme. A Borgo has received travel grants and honoraria from BioMarin Pharmaceutical Inc., and honoraria from Brains for Brains and Shire. E Braunlin has received speaker and travel grants from BioMarin Pharmaceutical Inc. and Sanofi Genzyme, a research funding from BioMarin Pharmaceutical Inc. and Bluebird Bio and has conducted consultancy work for Ultragenyx. Y Eto has received research grants and honoraria from BioMarin Pharmaceutical Inc., Alexion Pharmaceutical Inc., Sanofi Genzym and Actelion Pharmaceuticals Ltd., and honoraria from Dainippon Sumitomo Inc., Japan Chemical Research and Shire Japan. JI Gold has received honoraria and travel grants from BioMarin Pharmaceutical Inc. A Jester has received honoraria, travel grants, and research funding from BioMarin Pharmaceutical Inc. and funding from Shire for equipment to conduct outcomes assessment. S Jones has conducted investigator, speaker and consultancy work for Alexion, BioMarin Pharmaceutical Inc., Orchard therapeutics, Sanofi Genzyme, Shire and Ultragenyx. C Karsli has nothing to disclose. W MacKenzie has received travel grants and honoraria from BioMarin Pharmaceutical Inc. and has conducted consultancy work for Johnson & Johnson. A McFadyen has received unrestricted educational grants from Alexion, BioMarin Pharmaceutical Inc., Sanofi Genzyme, Shire, and Johnson and Johnson. J McGill has conducted speaker, consultancy, board member work and received travel grants from Actelion, BioMarin Pharmaceutical Inc., Sanofi Genzyme and Shire. He has been an investigator for BioMarin Pharmaceutical Inc. J Mitchell has received research support, advisory board fees, and travel grants from BioMarin Pharmaceutical Inc. and has been involved in the conduct of BioMarin clinical trials. He has also received research funding from Sanofi Genzyme and Shire. He has received consultancy fees from Ultragenyx, BioMarin Pharmaceutical Inc., Sanofi Genzyme and Shire. J Muenzer has conducted consultancy work and/or has received grant support from BioMarin Pharmaceutical Inc., Denali, Green Cross, Eloxx, PTC Therapeutics, RegenXbio, Sangamo, Sanofi Genzyme, Shire and Sobi. T Okuyama has received research grant from BioMarin Pharmaceutical Inc., Green Cross, Sanofi Genzyme, Shire, Orchard Therapeutics, and JCR, and has attended advisory boards for Sanofi Genzyme. He is Principal Investigator for ERT clinical trials for MPS II sponsored by Green Cross and JCR. P Orchard has received honoraria, travel and research support from Sanofi Genzyme, and has received research support from BioMarin Pharmaceutical Inc., Bluebird Bio and Horizon Pharma. B Stevens has received grants for the MPS Society from Sanofi Genzyme, Shire, Sangamo, Regenxbio, BioMarin Pharmaceuticals Inc. and Ultragenyx for direct advocacy, expert meetings, literature and travel. S Thomas has received grants for the MPS Society from Sanofi Genzyme, Shire, Sangamo, Regenxbio, BioMarin Pharmaceuticals Inc., Chiesi and Ultragenyx for direct advocacy, expert meetings, honoraria, literature and travel. DR Marinho has received honoraria and travel grants from BioMarin Pharmaceutical Inc. R Walker has received travel grants, honorarium and payments for consulting work from BioMarin Pharmaceutical Inc., Sanofi Genzyme and Shire. R Wynn has conducted consultancy work for Chimerix, Bluebird Bio and Orchard Therapeutics.

: Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.