Speckmann, Carsten http://orcid.org/0000-0002-6217-1556
Nennstiel, Uta
Hönig, Manfred
Albert, Michael H.
Ghosh, Sujal
Schuetz, Catharina
Brockow, Inken
Hörster, Friederike
Niehues, Tim
Ehl, Stephan
Wahn, Volker
Borte, Stephan
Lehmberg, Kai
Baumann, Ulrich
Beier, Rita
Krüger, Renate
Bakhtiar, Shahrzad
Kuehl, Joern-Sven
Klemann, Christian
Kontny, Udo
Holzer, Ursula
Meinhardt, Andrea
Morbach, Henner
Naumann-Bartsch, Nora
Rothoeft, Tobias
Kreins, Alexandra Y.
Davies, E. Graham
Schneider, Dominik T.
Bernuth, Horst v.
Klingebiel, Thomas
Hoffmann, Georg F.
Schulz, Ansgar
Hauck, Fabian http://orcid.org/0000-0001-9644-2003
Funding for this research was provided by:
Else Kröner-Fresenius-Stiftung (EKFS, 2017_A110)
Bundesministerium für Bildung und Forschung (01GM1910C, 01GM1910C)
Wellcome Trust (222096/Z/20/Z)
Universitätsklinikum Freiburg
Article History
Received: 20 December 2022
Accepted: 8 February 2023
First Online: 27 February 2023
Declarations
:
: As regulated within the Pediatric Directive on NBS (§26), the legal guardians provided written informed consent for NBS and the transfer of data to the screening laboratory [CitationRef removed]. Evaluation of screening-related laboratory and clinical data was performed as part of the established plausibility assessment for the German NBS program [CitationRef removed]. Immunological investigation performed in patients from the University Medical Center Freiburg was approved by Ethics Committees of the University of Freiburg (No. 230/11–120179).
: Not applicable.
: MA reports activity as a consultant for CSL, Orchard Therapeutics, and FORGE. RB received honoria for invited lectures by Medac and for taking part in advisory board by Bluebirdbio.