Panagiotakaki, Eleni
Tiziano, Francesco D.
Mikati, Mohamad A.
Vijfhuizen, Lisanne S.
Nicole, Sophie
Lesca, Gaetan http://orcid.org/0000-0001-7691-9492
Abiusi, Emanuela http://orcid.org/0000-0001-9028-012X
Novelli, Agnese
Di Pietro, Lorena
Harder, Aster V. E. http://orcid.org/0000-0002-5477-7232
Walley, Nicole M.
De Grandis, Elisa
Poulat, Anne-Lise
Portes, Vincent Des
Lépine, Anne
Nassogne, Marie-Cecile http://orcid.org/0000-0001-6936-1611
Arzimanoglou, Alexis http://orcid.org/0000-0002-7233-2771
Vavassori, Rosaria http://orcid.org/0000-0002-1598-0692
Koenderink, Jan
Thompson, Christopher H.
George, Alfred L. Jr.
Gurrieri, Fiorella
van den Maagdenberg, Arn M. J. M. http://orcid.org/0000-0002-9310-5535
Heinzen, Erin L. http://orcid.org/0000-0002-7268-8559
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Funding for this research was provided by:
Cure AHC
AISEA
AFM-Telethon, AFHA
U.S. Department of Health & Human Services | National Institutes of Health (NS108874)
Article History
Received: 1 August 2023
Revised: 25 September 2023
Accepted: 18 October 2023
First Online: 14 December 2023
Competing interests
: ALG serves on a scientific advisory board for Tevard Biosciences, and consults for Praxis Precision Medicines. ALG receives grant funding from Tevard Biosciences, Praxis Precision Medicines, and Neurocrine Biosciences for unrelated work. AMJMvdM received funding from Schedule 1 Therapeutics and Praxis Precision Medicines for unrelated work. The other authors declare no conflicts of interest.
: All subjects were consented to participate in this research study through protocols approved by local ethics boards.