{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T14:41:59Z","timestamp":1786632119057,"version":"build-2736575974"},"reference-count":42,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2026,6,17]],"date-time":"2026-06-17T00:00:00Z","timestamp":1781654400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,6,17]],"date-time":"2026-06-17T00:00:00Z","timestamp":1781654400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2026,9]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>The patient experience of Becker muscular dystrophy (BMD) is not well understood, making it difficult to evaluate the conceptual relevance of proposed patient\u2010reported outcome (PRO) measures. This study aimed to conceptualize the patient experience of BMD and evaluate content validity and perceptions of meaningful changes of PRO measures in this population.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Semi\u2010structured interviews were conducted with ambulatory adult and adolescent US\u2010based participants with BMD. Transcripts were analyzed using content and framework analysis. Participant quotes were coded to identify concepts and themes, illustrated in a conceptual model of BMD experience. Cognitive debriefing methods assessed patient understanding of the brief pain inventory\u2010short form (BPI\u2010SF), quality of life in neurological disorders (Neuro\u2010QoL), and three patient\u2010reported outcomes measurement information system (PROMIS\u201057) domains: Physical Function, Fatigue, and Pain Interference.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Twenty\u2010nine patients (mean age: 27.0\u2009years; mean age at BMD diagnosis: 15.1\u2009years) and two caregivers described a wide range of signs, symptoms, and impacts. Physical fatigue, pain, weakness, and impacts on physical mobility and emotional wellbeing were considered most bothersome. The BPI\u2010SF Item 3 and PROMIS\u201057 domains were well understood and relevant; however, the Neuro\u2010QoL Upper Extremity questionnaire demonstrated low conceptual relevance.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>The conceptual model illustrated the burden of living with BMD. The BPI\u2010SF Item 3 and evaluated PROMIS\u201057 domains are considered content\u2010valid PRO measures in BMD, with most participants associating score improvements with meaningful changes to their health\u2010related quality of life.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70302","type":"journal-article","created":{"date-parts":[[2026,6,18]],"date-time":"2026-06-18T06:45:07Z","timestamp":1781765107000},"page":"559-569","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Exploring the Content Validity of Patient\u2010Reported Outcome Measures to Capture the Patient Experience of Becker Muscular Dystrophy"],"prefix":"10.1002","volume":"74","author":[{"given":"Abby","family":"Bronson","sequence":"first","affiliation":[{"name":"Edgewise Therapeutics  Boulder Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4054-5975","authenticated-orcid":false,"given":"Katie","family":"Mellor","sequence":"additional","affiliation":[{"name":"Clinical Outcomes Assessment, Clarivate  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicole","family":"Kilburn","sequence":"additional","affiliation":[{"name":"Edgewise Therapeutics  Boulder Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ryan","family":"Fischer","sequence":"additional","affiliation":[{"name":"Foundation for Angelman Syndrome Therapeutics, Formerly Parent Project Muscular Dystrophy  Austin Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6573-7431","authenticated-orcid":false,"given":"Natalie","family":"Aldhouse","sequence":"additional","affiliation":[{"name":"Clinical Outcomes Assessment, Clarivate  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"Marshall","sequence":"additional","affiliation":[{"name":"Clinical Outcomes Assessment, Clarivate  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tamara","family":"Al\u2010zubeidi","sequence":"additional","affiliation":[{"name":"Clinical Outcomes Assessment, Clarivate  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,6,17]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/srep32439"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51925"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1212\/NXG.0000000000200285"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-025-13126-9"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-023-11837-5"},{"key":"e_1_2_13_7_1","unstructured":"Orphanet \u201cBecker Muscular Dystrophy. 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