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Article History

Received: 30 October 2017

Accepted: 25 January 2018

First Online: 8 February 2018

Ethics approval and consent to participate

: This study follows the Helsinki Declaration’s principles and was carried out from retrospective review of routine clinical data: formal ethics review was therefore not requested.

: The patients or their parents/guardians gave a written consent to publication of their anonymized clinical data.

: • RP received travel grants for scientific meetings and honoraria for speaking engagements or consultations from Shire International, Sanofi Genzyme, BioMarin and SOBI. She is involved in Advisory Boards on Lysosomal diseases upon request of BioMarin, Shire International and Sanofi Genzyme. She is or has recently been involved as PI in clinical trials with BioMarin, Shire Inernational and Synageva.• BB received honoraria for speaking engagement and post-doc grants from Sanofi Genzyme, Actelion and Shire international.• AB received honoraria for speaking engagement from Sanofi Genzyme and Actelion• DC received travel grants for scientific meetings from Shire International and Sanofi Genzyme, and honoraria for speaking engagements from Shire.• MAD received travel grants for scientific meetings and honoraria for speaking engagements from Shire International, Sanofi Genzyme and BioMarin.• AF received travel grants for scientific meetings by Sanofi Genzyme, Bio Marin, Shire International and Actelion. She is involved in Advisory Boards on Lysosomal diseases upon request of BioMarin and Sanofi Genzyme.• SG received travel grants for scientific meetings and honoraria for speaking engagement from Sanofi Genzyme, SOBI and Shire international. She is involved as PI in clinical studies with Shire and Alexion and as SI in clinical studies with BioMarin, Shire International and Sanofi Genzyme.• MaS received travel grants for scientific meetings and honoraria for speaking engagement from Sanofi Genzyme, and Shire International.• MDR received travel grants for scientific meetings and honoraria for speaking engagements or consultation from Sanofi Genzyme• FM, RDC, FD, VP and RT received travel grants for scientific meetings from Sanofi Genzyme and Shire International.• PDL, AC, PC, MiS, MGV declare competing interest.

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