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First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD

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Fig. 1: Data collection and analysis using SV95C.

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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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Authors and Affiliations

  1. Department of Paediatrics, MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK

    Laurent Servais

  2. Division of Child Neurology, Department of Pediatrics, Centre de Référence des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium

    Laurent Servais

  3. SYSNAV, Paris, France

    Damien Eggenspieler, Marc Grelet & Mélanie Annoussamy

  4. Department of Neurology, Centre de Référence des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium

    Margaux Poleur

  5. Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, London, UK

    Francesco Muntoni

  6. F. Hoffmann-La Roche Ltd, Basel, Switzerland

    Paul Strijbos

Authors
  1. Laurent Servais
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  2. Damien Eggenspieler
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  3. Margaux Poleur
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  4. Marc Grelet
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  5. Francesco Muntoni
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  6. Paul Strijbos
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  7. Mélanie Annoussamy
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Corresponding author

Correspondence to Laurent Servais.

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Competing interests

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

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