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Article History

Received: 9 April 2021

Accepted: 14 September 2022

First Online: 19 January 2023

Competing interests

: V.R. was a Solid Biosciences employee when the study was conducted. V.R. is cofounder and stockholder of DiNAQOR AG, the products of which are not related to this study. T.V. was a consultant for Antisense Therapeutics, Biophytis, Catabasis, German Centre for Cardiovascular Research, Italfarmaco, Santhera, Sarepta, Servier, Syneos Health and Solid Biosciences. T.V. is a scientific advisory board member and stockholder of Constant Pharmaceuticals and a cofounder and stockholder of DiNAQOR AG. A.A.F. has consulted in the domain of AI for Airbus and Celestial Group. He received research funding paid to his institution from Fresenius Kabi, Shell and Airbus. He received donations given to his institution from Facebook (now Meta), NVIDIA and Microsoft. R.F. has consulted for Pfizer and has received funding from Pfizer but not related to this study. E.M. has received funding as a member of advisory boards for spinal muscular atrophy (SMA) studies for AveXis, Biogen, Ionis Pharmaceuticals, Novartis, Scholar Rock and Roche. E.M. has served as principal investigator for Ionis Pharmaceuticals, Biogen, AveXis and Roche clinical trials and has received funding from Famiglie SMA Italy, Italian Telethon, Biogen and SMA Europe. A.F., R.F., V.R. and T.V. are coinventors of an International Patent Application no. PCT/EP2020/077703, ‘Systems and methods for monitoring the state of a disease using a biomarker’. B.K. and V.S. declare no competing interests.