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Acknowledgements
The authors thank the collaborators, investigators, patients and family members who have contributed to previous and ongoing SV95C studies. This Correspondence article is dedicated to M. Annoussamy, who could not approve the final version, as she sadly passed away shortly before the final draft; the authors are immensely grateful for her leadership and invaluable efforts during the EMA qualification process. Medical writing support was provided by J. Ciarochi of Nucleus Global, in accordance with Good Publication Practice (GPP) 2022 guidelines (https://www.ismpp.org/gpp-2022) and was funded by F. Hoffmann-La Roche Ltd.
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L.S. has conducted consultancy and given lectures for F. Hoffmann-La Roche Ltd, Sarepta Therapeutics, Santhera Pharmaceuticals, Pfizer, Dyne, RegenexBio and SYSNAV. D.E. is an employee of SYSNAV. M.G. is a former employee of SYSNAV. F.M. has conducted consultancies and given lectures for F. Hoffmann-La Roche Ltd, Sarepta Therapeutics, Santhera Pharmaceuticals, Eli Lilly and Company, Pfizer, Wave Life Sciences Ltd, PTC Therapeutics and Dyne Therapeutics. P.S. is an employee of and holds stock in F. Hoffmann-La Roche Ltd. M.A. is an employee of SYSNAV. M.P. declares no conflicts of interest.
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Servais, L., Eggenspieler, D., Poleur, M. et al. First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD. Nat Med 29, 2391–2392 (2023). https://doi.org/10.1038/s41591-023-02459-5
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DOI: https://doi.org/10.1038/s41591-023-02459-5