Saffari, Afshin
Brechmann, Barbara
Böger, Cedric
Saber, Wardiya Afshar http://orcid.org/0000-0001-9488-7893
Jumo, Hellen http://orcid.org/0000-0003-0495-5317
Whye, Dosh http://orcid.org/0000-0002-6895-9627
Wood, Delaney
Wahlster, Lara
Alecu, Julian E.
Ziegler, Marvin http://orcid.org/0000-0002-0504-7815
Scheffold, Marlene
Winden, Kellen
Hubbs, Jed http://orcid.org/0000-0002-3923-3392
Buttermore, Elizabeth D. http://orcid.org/0000-0003-3194-5413
Barrett, Lee
Borner, Georg H. H. http://orcid.org/0000-0002-3166-3435
Davies, Alexandra K. http://orcid.org/0000-0002-1594-8780
Ebrahimi-Fakhari, Darius http://orcid.org/0000-0002-0026-4714
Sahin, Mustafa http://orcid.org/0000-0001-7044-2953
Funding for this research was provided by:
BCH | Manton Center for Orphan Disease Research, Boston Children’s Hospital
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (1K08NS123552-01)
Deutsche Forschungsgemeinschaft (448402208)
Studienstiftung des Deutschen Volkes
Deutscher Akademischer Austauschdienst
U.S. Department of Health & Human Services | National Institutes of Health (1U54HD090255)
Article History
Received: 7 June 2023
Accepted: 6 December 2023
First Online: 17 January 2024
Competing interests
: This work was supported by a joint research agreement between Boston Children’s Hospital and Mitobridge Inc., now owned by Astellas Pharmaceuticals Inc. D.E.F. has served as a consultant to Health Advances LLC, has received speaker honoraria from the Movement Disorders Society and publishing royalties from Cambridge University Press. M.Sa. reports grant support from Novartis, Biogen, Astellas, Aeovian, Bridgebio, and Aucta unrelated to this project. He has served on Scientific Advisory Boards for Novartis, Roche, Regenxbio, SpringWorks Therapeutics, Jaguar Therapeutics, and Alkermes. The remaining authors declare no competing interests.