Silva, Patrick
Costa, Marina A.
Gaspar, Laetitia
Durães, João
Cunha, Inês
Ribeiro, Joana A
Januário, Cristina
Oliveiros, Bárbara
Hübener-Schmid, Jeannette
Faber, Jennifer
Raposo, Mafalda
Lima, Manuela
Garcia-Moreno, Hector
Giunti, Paola
Beichert, Lukas
Schöls, Ludger
van de Warrenburg, Bart P.
de Vries, Jeroen
Thieme, Andreas
Reetz, Kathrin
Jacobi, Heike
Infante, Jon
Klockgether, Thomas
,
Ferreira, Ana
Rosa, Ana
Gonzalez, Carlos
Gonzalez-Robles, Cristina
Timmann, Dagmar
Erdlenbruch, Friedrich
Lemos, João
Vasconcelos, João
Teves, Luís
Pires, Paula
Lopes, Pedro
Coelho, Pedro
Kay, Teresa
de Almeida, Luís Pereira
Santana, Magda M. https://orcid.org/0000-0003-4076-7516
Funding for this research was provided by:
EU Joint Programme – Neurodegenerative Disease Research (643417)
Article History
Received: 26 April 2025
Accepted: 24 September 2025
First Online: 17 October 2025
Declarations
:
: LPA has received funding from Servier and UCB. LS has served on advisory boards for VICO Therapeutics, VIGIL Neuroscience, and Novartis. BvdW has served on advisory boards or as a consultant for VICO Therapeutics, Biogen, and Biohaven Pharmaceuticals. PG has received grants and honoraria for advisory board participation from Vico Therapeutics, honoraria for an advisory board from Triplet Therapeutics, grants and personal fees from Reata Pharmaceutical, and grants from Wave Life Sciences. KR has received honoraria for presentations or advisory board participation from Biogen, Eisai, Lilly, and Roche. TK has received consulting fees from Biogen, UCB, and Vico Therapeutics. JF has received consultancy honoraria from Vico Therapeutics. PS, MAC, LG, JD, IC, JAR, CJ, BO, JHS, MR, ML, HGM, LB, JV, AT, HJ, JI, and MMS have no potential conflicts of interest to report.
: This study was conducted by ESMI with approval from local ethical committees at all centers, and in accordance with the Helsinki Declaration of 1964 and its later amendments, as well as local institutional ethics committee requirements. An exception to the Declaration was that the study was not prospectively or retrospectively registered, as it was a noninterventional observational analysis of existing registry data for which trial registration is not otherwise required.
: Written informed consent was obtained from all participants prior to enrollment. Participants were informed of the aims of the study, the voluntary nature of their participation, and their right to withdraw at any time without prejudice. Confidentiality of all personal and clinical data was strictly maintained.
: Not applicable.
: Most of the data that support the findings of this study are available in the Supplementary Material. Additional data are available from the corresponding author upon reasonable request.
: The code used for data processing and statistical analyses in this study is available from the corresponding author upon reasonable request.
: Design and conceptualization of the study: PS, MMS, and LPA. Subject recruitment/acquisition of participant data: PS, LG, JD, IC, JAR, CJ, JHS, JF, MR, ML, HGM, PG, LB, LS, BvdW, JdV, AT, KR, HJ, JI, TK, and MMS. Statistical analysis of data: PS, MAC, BO, and MMS. Drafting of the manuscript: PS and MMS. Revision of the manuscript: PS, LG, JD, JHS, MR, ML, HGM, LS, BvdW, JdV, AT, KR, HJ, JI, TK, and MMS. All authors read and approved the final submitted manuscript and agree to be accountable for the work.