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

Affiliations

• ¹ Department of Paediatrics, MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK. laurent.servais@paediatrics.ox.ac.uk.
• 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@paediatrics.ox.ac.uk.
• ³ SYSNAV, Paris, France.
• ⁴ Department of Neurology, Centre de Référence des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium.
• ⁵ Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, London, UK.
• ⁶ F. Hoffmann-La Roche Ltd, Basel, Switzerland.