Saier, Christina
Sansen, Stefaan
Berghout, Joanne
Freyler, Kathrin
Einhorn, Moshe
Einhorn, Yaron
Matalonga, Leslie
Beltran, Sergi
Novelli, Antonio
Selvatici, Rita
Fortunato, Fernanda
Montanari, Silvia
Martinez-Fresno, Maria
Gumus, Gulcin
Agolini, Emanuele
Garnier, Nicolas
Ferlini, Alessandra
Bertini, Enrico
Kirschner, Janbernd https://orcid.org/0000-0003-1618-7386
Funding for this research was provided by:
Universitätsklinikum Freiburg
Article History
Received: 14 November 2024
Accepted: 20 March 2025
First Online: 15 May 2025
Declarations
:
: The disease selection process described in this manuscript did not involve any experiments with human subjects. The planed pilot project for genetic newborn screening with the TREAT-panel gene list has been approved by the Freiburg University Ethics Committee in Germany (24-1084_1-S1), the Italian Ethics Committee of the University of Ferrara (758/2023/Sper/AOUFe) and the French Ethics Committee (24.01886.000348).
: Not applicable.
: EB has received funds for Advisory Board from Roche, Biogen, PTC, Pfizer. JK has received funds for Advisory Boards from Biogen, Novartis, Pfizer, Roche, Santhera and research funding from Biogen, Novartis and Roche. SS is an employee of Sanofi. JB is an employee of Pfizer. ME and YE are employees of Genoox. All the other authors declare that they have no competing interests.
: EB has received funds for Advisory Board from Roche, Biogen, PTC, Pfizer. JK has received funds for Advisory Boards from Biogen, Novartis, Pfizer, Roche, Santhera and research funding from Biogen, Novartis and Roche. SS is an employee of Sanofi. JB is an employee of Pfizer. ME and YE are employees of Genoox. NG was a full-time employee of Pfizer Inc. at the beginning of this work and is now a full-time employee of Servier Affaires Médicales. All the other authors declare that they have no competing interests.