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- W2775080814 abstract "Background The need for deeper understanding of Duchenne muscular dystrophy (DMD) natural history is supported by the rapid advances in translational research, the increase in clinical trials and outcome of recent studies. Aim Assess the natural history of DMD through a composite assessment tool encompassing ambulant and non-ambulant phases of the disease. Methods Multicentre study (5 centres: London, Newcastle, Paris, Leiden, Nijmegen) with a recruitment target of 80-DMD patients assessed 6-monthly according to a shared protocol. Assessments include 6 min walk distance (6 MWD), NorthStar Ambulatory Assessment (NSAA), Performance of Upper Limb (PUL), MyoSet, FVC and whole body DEXA scan (subgroup). Results We analysed longitudinal data for 79 patients, 39 with >24 months follow-up. Ambulant boys>7 years old declined 2.33 points/year in the NSAA (p 45% total lean body mass were ambulant. Conclusion This on-going study adds to current knowledge on DMD natural history including expected mean changes per year and the relationship between different outcome measures that will contribute to future trial design and interpretation." @default.
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- W2775080814 date "2017-12-01" @default.
- W2775080814 modified "2023-09-24" @default.
- W2775080814 title "PO195 Outcome measures for duchenne muscular dystrophy: implications for trials" @default.
- W2775080814 doi "https://doi.org/10.1136/jnnp-2017-abn.216" @default.
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