{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T18:54:48Z","timestamp":1787684088642,"version":"build-2784847793"},"reference-count":29,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2024,3,20]],"date-time":"2024-03-20T00:00:00Z","timestamp":1710892800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004040","name":"KU Leuven","doi-asserted-by":"publisher","award":["C24\/18\/047"],"award-info":[{"award-number":["C24\/18\/047"]}],"id":[{"id":"10.13039\/501100004040","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003130","name":"Fonds Wetenschappelijk Onderzoek","doi-asserted-by":"publisher","award":["1159121N"],"award-info":[{"award-number":["1159121N"]}],"id":[{"id":"10.13039\/501100003130","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100010648","name":"Universitaire Ziekenhuizen Leuven, KU Leuven","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100010648","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2024,7]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background and purpose<\/jats:title>\n                    <jats:p>Because Becker muscular dystrophy (BMD) is a heterogeneous disease and only few studies have evaluated adult patients, it is currently still unclear which outcome measures should be used in future clinical trials.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Muscle magnetic resonance imaging, patient\u2010reported outcome measures and a wide range of clinical outcome measures, including motor function, muscle strength and timed\u2010function tests, were evaluated in 21 adults with BMD at baseline and at 9 and 18\u2009months of follow\u2010up.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Proton density fat fraction increased significantly in 10\/17 thigh muscles after 9\u2009months, and in all thigh and lower leg muscles after 18\u2009months. The 32\u2010item Motor Function Measurement (MFM\u201032) scale (\u22121.3%,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.017), North Star Ambulatory Assessment (\u22121.3 points,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.010) and patient\u2010reported activity limitations scale (\u22120.3 logits,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.018) deteriorated significantly after 9\u2009months. The 6\u2010min walk distance (\u221228.7\u2009m,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.042), 10\u2010m walking test (\u22120.1\u2009m\/s,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.032), time to climb four stairs test (\u22120.03\u2009m\/s,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.028) and Biodex peak torque measurements of quadriceps (\u22124.6\u2009N\u2009m,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.014) and hamstrings (\u22125.0\u2009N\u2009m,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.019) additionally deteriorated significantly after 18\u2009months. At this timepoint, domain 1 of the MFM\u201032 was the only clinical outcome measure with a large sensitivity to change (standardized response mean 1.15).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>It is concluded that proton density fat fraction imaging of entire thigh muscles is a sensitive outcome measure to track progressive muscle fat replacement in patients with BMD, already after 9\u2009months of follow\u2010up. Finally, significant changes are reported in a wide range of clinical and patient\u2010reported outcome measures, of which the MFM\u201032 appeared to be the most sensitive to change in adults with BMD.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ene.16282","type":"journal-article","created":{"date-parts":[[2024,3,20]],"date-time":"2024-03-20T03:48:29Z","timestamp":1710906509000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Lessons for future clinical trials in adults with Becker muscular dystrophy: Disease progression detected by muscle magnetic resonance imaging, clinical and patient\u2010reported outcome measures"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1742-1744","authenticated-orcid":false,"given":"Bram","family":"De\u00a0Wel","sequence":"first","affiliation":[{"name":"Department of Neurology University Hospitals Leuven  Leuven 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