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Article History

Received: 19 March 2020

Accepted: 21 July 2020

First Online: 3 August 2020

Ethics approval and consent to participate

: The EFACTS study was approved by the local committee of each participating center. Written informed consent was given from each subject and legal guardian before inclusion in the study.

: Consent for publication of anonymized data was given by each participant or legal guardian at the time of inclusion in the study.

: PG is supported by the National Institute for Health Research University College London Hospitals Biomedical Research Centre. CM reports grant from REATA Pharmaceuticals for phase 2 study in the treatment of Friedreich ataxia. TK discloses research funding in the last 12 months from the Deutsche Forschungsgemeinschaft (DFG, German Research Foundation) for an investigator-initiated trial of nicotinamide in Friedreich ataxia, as well as (unrelated to this manuscript) research funding from the Bundesministerium für Bildung und Forschung (BMBF, Federal Ministry of Education and Research), and research funding, travel support and speaker/consultancy honoraria from Santhera Pharmaceuticals, Retrophin Inc. and ApoPharma Inc.. LS discloses research funding unrelated to this study from CureVac AG (Tübingen, Germany). MP reports grants from the Friedreich Ataxia Research Alliance, grants from FNRS (Belgium), grants and personal fees from Biomarin, grants and personal fees from Voyager Therapeutics, personal fees from Apopharma, personal fees from Vertex, personal fees from Pfizer, and grants from Euroataxia outside the submitted work. JBS serves on scientific advisory boards for Lundbeck Inc., TEVA, Novartis, ForwardPharma, and Lilly, received funding for travel and speaker honoraria from GlaxoSmithKline, Merz Pharmaceuticals, Medical Tribune, Lundbeck Inc., Pfizer Inc., Boehringer, Bayer, serves as Editor-in-Chief of the Journal of Neurochemistry, and is Associate Editor for eNeuro. SB reports funding from E-Rare-3 “Clinical research for new therapeutic uses of already existing molecules (repurposing) in rares diseases” (E_Rare-3JTC2016).