The need for a better understanding of Duchenne muscular dystrophy (DMD) natural history and the role of novel outcome measures and their impact in current and future clinical trials is well recognized. We aimed to assess the natural history of ambulant and non-ambulant DMD patients in an international multicentre study across 5 centres (London, Newcastle, Paris, Leiden, Nijmegen) with patients being assessed 6-monthly according to a shared protocol. Assessments included 6-minute walk distance (6MWD), North Star Ambulatory Assessment (NSAA), Performance of Upper Limb (PUL 1.2), MyoSet, respiratory function (forced vital capacity – FVC, maximum inspiratory and expiratory pressures – MEP and MIP, peak expiratory flow – PEF, peak cough flow – PCF).

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