Muntau, Ania C.
Burlina, Alberto
Eyskens, François
Freisinger, Peter
Leuzzi, Vincenzo
Sivri, Hatice Serap
Gramer, Gwendolyn
Pazdírková, Renata
Cleary, Maureen
Lotz-Havla, Amelia S.
Lane, Paul
Alvarez, Ignacio http://orcid.org/0000-0002-2900-8378
Rutsch, Frank
Funding for this research was provided by:
BioMarin Pharmaceutical
Universitätsklinikum Hamburg-Eppendorf (UKE)
Article History
Received: 22 March 2021
Accepted: 19 July 2021
First Online: 3 August 2021
Declarations
:
: The study was performed in accordance with the protocol (and subsequent protocol amendments) and with the ethical principles laid down in the Declaration of Helsinki, in accordance with the International Conference on Harmonisation (ICH), Note for Guidance on Good Clinical Practice (ICH Topic E6, 1996) and applicable regulatory requirements. The local ethics committee/institutional review board at each of the participating centres approved the protocol.
: Not applicable.
: PL is an employee and shareholder of BioMarin. IA was an employee of BioMarin at the time of this study and during the initial preparation of this manuscript. All other authors had no competing interests.