{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,8]],"date-time":"2026-02-08T10:05:44Z","timestamp":1770545144060,"version":"3.49.0"},"reference-count":45,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2025,11,26]],"date-time":"2025-11-26T00:00:00Z","timestamp":1764115200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2025,11,26]],"date-time":"2025-11-26T00:00:00Z","timestamp":1764115200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100000069","name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000069","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005202","name":"Muscular Dystrophy Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005202","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008091","name":"University of Rochester","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008091","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":[[2026,2]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>To optimize therapeutic trials involving individuals with myotonic dystrophy type 2 (DM2), it is useful to have a fully validated, regulatory\u2010grade, disease\u2010specific patient\u2010reported outcome (PRO) measure that is capable of detecting clinically relevant changes in response to therapeutic interventions and designed in accordance with U.S. Food and Drug Administration guidelines. The purpose of this research was to develop and validate a DM2\u2010specific instrument that is relevant to the patient population and capable of quantifying multifactorial disease.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We conducted semi\u2010structured qualitative interviews and a cross\u2010sectional study to identify the most important and prevalent symptoms for individuals with DM2. Symptom items were selected for inclusion in the Myotonic Dystrophy Type 2 Health Index (MD2HI) based on their high prevalence, relative impact, and potential ability to respond to therapeutic intervention. Further validation and optimization of the MD2HI were performed through beta interviews, test\u2013retest reliability assessments, factor analysis, and subgroup analysis.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Seventy\u2010four individuals with DM2 participated in a cross\u2010sectional study to identify the most common and important symptoms to include in the MD2HI. Beta testing with 20 participants with DM2 demonstrated that the MD2HI is highly relevant, easy to use, and comprehensive. During a short longitudinal study of 24 DM2 participants, the MD2HI depicted high reliability (ICC\u2009=\u20090.97) and high internal consistency (Cronbach's\n                      <jats:italic>\u03b1<\/jats:italic>\n                      \u2009=\u20090.98), resulting in a 17\u2010subscale instrument.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Initial assessment of the MD2HI provides evidence that it is a valid and reliable PRO capable of quantifying a patient's perception of their disease burden to better detect clinically relevant changes in response to therapeutic intervention.<\/jats:p>\n                  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School of Medicine  Salt Lake City Utah USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer","family":"Weinstein","sequence":"additional","affiliation":[{"name":"University of Rochester, Center for Health + Technology  Rochester New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anika","family":"Varma","sequence":"additional","affiliation":[{"name":"University of Rochester, Center for Health + Technology  Rochester New York USA"},{"name":"University of Rochester, School of Medicine and Dentistry  Rochester New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jamison","family":"Seabury","sequence":"additional","affiliation":[{"name":"University of Rochester, Center for Health + Technology  Rochester New York USA"},{"name":"University of Rochester, School of Medicine and Dentistry  Rochester New York 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of Rochester, Center for Health + Technology  Rochester New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nuran","family":"Dilek","sequence":"additional","affiliation":[{"name":"Department of Biostatistics and Neurology University of Rochester  Rochester New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2900-6612","authenticated-orcid":false,"given":"Chad","family":"Heatwole","sequence":"additional","affiliation":[{"name":"University of Rochester, Center for Health + Technology  Rochester New York USA"},{"name":"Department of Neurology University of Rochester  Rochester New York 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