Elgün, Saskia
Waibel, Jakob
Kehrer, Christiane
van Rappard, Diane
Böhringer, Judith
Beck-Wödl, Stefanie
Just, Jennifer
Schöls, Ludger
Wolf, Nicole
Krägeloh-Mann, Ingeborg
Groeschel, Samuel http://orcid.org/0000-0002-2706-7163
Article History
Received: 1 March 2019
Accepted: 4 June 2019
First Online: 11 June 2019
Ethics approval and consent to participate
: The study was approved by the local ethics committees (401/2005 and 2012/098). Written informed consent was given by at least one caregiver.
: We have only used patient data from whom we have a consent to participation and publication.
: S.G. reports an institutional research grant from Shire plc, outside of the submitted work, and serves as co-investigator and advisor in a multicentre enzyme replacement trial sponsored by Shire plc, but receives no personal payment related to this role.