{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,6]],"date-time":"2026-06-06T00:06:29Z","timestamp":1780704389222,"version":"3.54.1"},"reference-count":41,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2021,8,28]],"date-time":"2021-08-28T00:00:00Z","timestamp":1630108800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100007393","name":"Association Fran\u00e7aise contre les Myopathies","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007393","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2021,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To understand the natural disease upper limb progression over 3\u00a0years of ambulatory and non\u2010ambulatory patients with Duchenne muscular dystrophy (DMD) using functional assessments and quantitative magnetic resonance imaging (MRI) and to exploratively identify prognostic factors.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Forty boys with DMD (22 non\u2010ambulatory and 18 ambulatory) with deletions in dystrophin that make them eligible for exon 53\u2010skipping therapy were included. Clinical assessments, including Brooke score, motor function measure (MFM), hand grip and key pinch strength, and upper limb distal coordination and endurance (MoviPlate), were performed every 6\u00a0months and quantitative MRI of fat fraction (FF) and lean muscle cross sectional area (flexor and extensor muscles) were performed yearly.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In the whole population, there were strong nonlinear correlations between outcome measures. In non\u2010ambulatory patients, annual changes over the course of 3\u00a0years were detected with high sensitivity standard response mean (|SRM| \u22650.8) for quantitative MRI\u2010based FF, hand grip and key pinch, and MFM. Boys who presented with a FF&lt;20% and a grip strength &gt;27% were able to bring a glass to their mouth and retained this ability in the following 3\u00a0years. Ambulatory patients with grip strength &gt;35% of predicted value and FF &lt;10% retained ambulation 3\u00a0years later.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>We demonstrate that continuous decline in upper limb strength, function, and MRI measured muscle structure can be reliably measured in ambulatory and non\u2010ambulatory boys with DMD with high SRM and strong correlations between outcomes. Our results suggest that a combination of grip strength and FF can be used to predict important motor milestones.<\/jats:p><\/jats:sec>","DOI":"10.1002\/acn3.51417","type":"journal-article","created":{"date-parts":[[2021,8,28]],"date-time":"2021-08-28T09:45:09Z","timestamp":1630143909000},"page":"1938-1950","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy"],"prefix":"10.1002","volume":"8","author":[{"given":"Charlotte","family":"Lilien","sequence":"first","affiliation":[{"name":"Institut de Myologie Paris France"},{"name":"Department of Paediatrics MDUK Oxford Neuromuscular Centre University of Oxford Oxford United 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