{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T16:30:47Z","timestamp":1789576247886,"version":"build-2803163510"},"reference-count":28,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2021,12,30]],"date-time":"2021-12-30T00:00:00Z","timestamp":1640822400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100014383","name":"Italfarmaco","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100014383","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100009882","name":"Regione Lombardia","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100009882","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2022,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>Becker muscular dystrophy (BMD) is characterized by variable disease severity and progression, prompting the identification of biomarkers for clinical trials. We used data from an ongoing phase II study to provide a comprehensive characterization of a cohort of patients with BMD, and to assess correlations between histological and magnetic resonance imaging (MRI) markers with muscle function and strength.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Eligible patients were ambulatory males with BMD, aged 18 to 65\u2009years (200 to 450\u2009meters on 6\u2010minute walk test). The following data were obtained: function test results, strength, fat\u2010fraction quantification using chemical shift\u2010encoded MRI (whole thigh and quadriceps), and fibrosis and muscle fiber area (MFA) of the brachial biceps.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Of 70 patients screened, 51 entered the study. There was substantial heterogeneity between patients in muscle morphology (histology and MRI), with high fat replacement. Total fibrosis correlated significantly and mostly moderately with all functional endpoints, including both upper arm strength assessments (left and right elbow flexion rho \u2212.574 and\u2009\u2212.588, respectively [both <jats:italic>P<\/jats:italic>\u00a0&lt;\u2009.0001]), as did MRI fat fraction (whole thigh and quadriceps), for example, with four\u2010stair\u2010climb velocity\u2009\u2212.554 and\u2009\u2212.550, respectively (both <jats:italic>P<\/jats:italic>\u00a0&lt;\u2009.0001). Total fibrosis correlated significantly and moderately with both MRI fat fraction assessments (.500 [<jats:italic>P<\/jats:italic>\u00a0=\u2009.0003] and .423 [.0024], respectively).<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>In this BMD cohort, micro\u2010 and macroscopic morphological muscle parameters correlated moderately with each other and with functional parameters, potentially supporting the use of MRI fat fraction and histology as surrogate outcome measures in patients with BMD, although additional research is required to validate this.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27475","type":"journal-article","created":{"date-parts":[[2021,12,17]],"date-time":"2021-12-17T06:54:53Z","timestamp":1639724093000},"page":"326-333","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["Characterization of patients with Becker muscular dystrophy by histology, magnetic resonance imaging, function, and strength assessments"],"prefix":"10.1002","volume":"65","author":[{"given":"Giacomo P.","family":"Comi","sequence":"first","affiliation":[{"name":"Neuromuscular and Rare Diseases Unit, Department of Neuroscience Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico  Milan Italy"},{"name":"Dino Ferrari Center, Department of Pathophysiology and Transplantation University of Milan  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik H.","family":"Niks","sequence":"additional","affiliation":[{"name":"Department of Neurology Leiden University Medical Center  Leiden The Netherlands"},{"name":"Duchenne Center Netherlands  The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia M.","family":"Cinnante","sequence":"additional","affiliation":[{"name":"Neuroradiology Unit Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hermien E.","family":"Kan","sequence":"additional","affiliation":[{"name":"Duchenne Center Netherlands  The Netherlands"},{"name":"C.J. Gorter Center for High Field MRI, Department of Radiology Leiden University Medical Center  Leiden The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Krista","family":"Vandenborne","sequence":"additional","affiliation":[{"name":"ImagingDMD and Department of Physical Therapy University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca J.","family":"Willcocks","sequence":"additional","affiliation":[{"name":"ImagingDMD and Department of Physical Therapy University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniele","family":"Velardo","sequence":"additional","affiliation":[{"name":"Neuromuscular and Rare Diseases Unit, Department of Neuroscience Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico  Milan Italy"},{"name":"Dino Ferrari Center, Department of Pathophysiology and Transplantation University of Milan  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Ripolone","sequence":"additional","affiliation":[{"name":"Neuromuscular and Rare Diseases Unit, Department of Neuroscience Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jules J.","family":"van Benthem","sequence":"additional","affiliation":[{"name":"Department of Orthopedics, Rehabilitation and Physiotherapy Leiden University Medical Center  Leiden The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nienke M.","family":"van de Velde","sequence":"additional","affiliation":[{"name":"Department of Neurology Leiden University Medical Center  Leiden The Netherlands"},{"name":"Duchenne Center Netherlands  The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simone","family":"Nava","sequence":"additional","affiliation":[{"name":"Neuroradiology Unit Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Ambrosoli","sequence":"additional","affiliation":[{"name":"OPIS srl  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Cazzaniga","sequence":"additional","affiliation":[{"name":"Italfarmaco SpA  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paolo U.","family":"Bettica","sequence":"additional","affiliation":[{"name":"Italfarmaco SpA  Milan 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