Funding for this research was provided by:
Deutsche Forschungsgemeinschaft (GR 4688/2-1)
Medizinischen Fakultät, Eberhard Karls Universität Tübingen (443-0-0)
Received: 4 March 2020
Accepted: 9 August 2020
First Online: 3 September 2020
Ethics approval and consent to participate
: The study was approved by the local Ethical Committee of the University of Tübingen (no. 401/2005). Written informed consent was given by at least one caregiver.
: Not applicable.
: S.G. reports an institutional research grant from Shire plc, outside of the submitted work, and serves as co-investigator in a multicenter enzyme replacement trial sponsored by Shire plc, but receives no personal payment related to this role.