{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T14:42:24Z","timestamp":1786632144970,"version":"build-2736575974"},"reference-count":41,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2026,7,8]],"date-time":"2026-07-08T00:00:00Z","timestamp":1783468800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,7,8]],"date-time":"2026-07-08T00:00:00Z","timestamp":1783468800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100005202","name":"Muscular Dystrophy Association","doi-asserted-by":"publisher","award":["608054"],"award-info":[{"award-number":["608054"]}],"id":[{"id":"10.13039\/100005202","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005693","name":"Parent Project Muscular Dystrophy","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005693","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100009358","name":"Duchenne Research Fund","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100009358","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":[[2026,9]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>Regulatory\u2010grade outcome measures are essential for supporting future therapeutic trials. The goal of our study was to develop and validate a multifactorial, disease\u2010specific, caregiver\u2010reported outcome (CRO) measure for Duchenne muscular dystrophy (DMD).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We conducted qualitative interviews and a cross\u2010sectional study with caregivers of individuals with DMD to determine the most prevalent and impactful symptoms to individuals with DMD. Symptom items were selected for the Duchenne Muscular Dystrophy Caregiver Reported\u2010Health Index (DMDCR\u2010HI) based on their prevalence and impact to a national sample cohort of caregivers, along with their potential ability to respond to therapeutics. Subsequently, we conducted cognitive beta interviews, test\u2013retest reliability assessments, and internal consistency analyses to evaluate and optimize the DMDCR\u2010HI. Using known group analysis, we assessed the ability of the DMDCR\u2010HI to differentiate between patients with differing levels of disease burden.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The DMDCR\u2010HI is a regulatory\u2010grade CRO that comprehensively measures 16 subscales in DMD. The instrument has a high internal consistency, strong test\u2013retest reliability (intraclass correlation coefficient (ICC)\u2009=\u20090.96), and was identified by caregivers as easy to use, relevant, and comprehensive. Through known group analysis, DMDCR\u2010HI scores demonstrated an ability to differentiate between subgroups with varying levels of symptomatic burden.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>The DMDCR\u2010HI is a valid and reliable CRO designed to sensitively and reliably capture how individuals with DMD feel and function from the perspective of the caregiver. The DMDCR\u2010HI offers a robust mechanism to longitudinally track changes in disease burden in individuals with DMD, regardless of age, cognitive ability, or ambulation status.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70327","type":"journal-article","created":{"date-parts":[[2026,7,9]],"date-time":"2026-07-09T03:00:37Z","timestamp":1783566037000},"page":"623-635","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Development and Validation of a Novel, Disease\u2010Specific, Caregiver\u2010Reported Outcome Measure: The Duchenne Muscular Dystrophy Caregiver Reported\u2010Health Index (\n                    <scp>DMDCR<\/scp>\n                    \u2010\n                    <scp>HI<\/scp>\n                    )"],"prefix":"10.1002","volume":"74","author":[{"ORCID":"https:\/\/orcid.org\/0009-0002-2982-8197","authenticated-orcid":false,"given":"Charlotte","family":"Engebrecht","sequence":"first","affiliation":[{"name":"Center for Health + Technology (CHeT) University of Rochester Medicine  Rochester New York USA"},{"name":"University of Rochester School of Medicine and Dentistry  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Spencer","family":"Rosero","sequence":"additional","affiliation":[{"name":"Center for Health + Technology (CHeT) University of Rochester Medicine  Rochester New York USA"},{"name":"University of Utah Spencer Fox Eccles School of Medicine  Salt Lake City Utah USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Weinstein","sequence":"additional","affiliation":[{"name":"Center for Health + Technology (CHeT) University of Rochester Medicine  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jamison","family":"Seabury","sequence":"additional","affiliation":[{"name":"Center for Health + Technology (CHeT) University of Rochester Medicine  Rochester New York USA"},{"name":"University of Rochester School of Medicine and Dentistry  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anika","family":"Varma","sequence":"additional","affiliation":[{"name":"Center for Health + Technology (CHeT) University of Rochester Medicine  Rochester New York USA"},{"name":"University of Rochester School of Medicine and Dentistry  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine","family":"Zizzi","sequence":"additional","affiliation":[{"name":"Center for Health + Technology (CHeT) University of Rochester Medicine  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Heatwole","sequence":"additional","affiliation":[{"name":"Cornell University  Ithaca New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nuran","family":"Dilek","sequence":"additional","affiliation":[{"name":"Department of Biostatistics and Neurology University of Rochester Medicine  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron","family":"Kaat","sequence":"additional","affiliation":[{"name":"Department of Medical Social Sciences Northwestern University  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Debra","family":"Guntrum","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Rochester Medicine  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma","family":"Ciafaloni","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Rochester Medicine  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2900-6612","authenticated-orcid":false,"given":"Chad","family":"Heatwole","sequence":"additional","affiliation":[{"name":"Center for Health + Technology (CHeT) University of Rochester Medicine  Rochester New York USA"},{"name":"Department of Neurology University of Rochester Medicine  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,7,8]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136\u2010019\u201002320\u20108"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/d41573\u2010019\u201000088\u20107"},{"key":"e_1_2_12_4_1","unstructured":"Center for Drug Evaluation and Research \u201cPatient\u2010Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims \u201d(2020) accessed Jan 18 2024 https:\/\/www.fda.gov\/regulatory\u2010information\/search\u2010fda\u2010guidance\u2010documents\/patient\u2010reported\u2010outcome\u2010measures\u2010use\u2010medical\u2010product\u2010development\u2010support\u2010labeling\u2010claims."},{"key":"e_1_2_12_5_1","volume-title":"Patient\u2010Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Guidance for Industry","author":"Food and Drug Administration","year":"2009"},{"key":"e_1_2_12_6_1","volume-title":"Patient\u2010Focused Drug Development: Methods to Identify What Is Important to Patients","author":"Food and Drug Administration","year":"2025"},{"key":"e_1_2_12_7_1","volume-title":"Patient\u2010Focused Drug Development: Selecting, Developing, or Modifying Fit\u2010for\u2010Purpose Clinical Outcome Assessments","author":"Food and Drug Administration","year":"2025"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(18)30024\u20103"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(18)30026\u20107"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(18)30025\u20105"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26736"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000004570"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24555"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND\u2010210643"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2017.08.1918"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12955\u2010020\u201001511\u2010z"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24725"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26066"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.3233\/JHD\u2010210506"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(23)00127\u20108"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28102"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1152\/jn.00248.2016"},{"key":"e_1_2_12_23_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/20.7.1725"},{"key":"e_1_2_12_24_1","doi-asserted-by":"publisher","DOI":"10.1038\/348725a0"},{"key":"e_1_2_12_25_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awn253"},{"key":"e_1_2_12_26_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awf012"},{"key":"e_1_2_12_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/0960\u20108966(94)90072\u20108"},{"key":"e_1_2_12_28_1","doi-asserted-by":"publisher","DOI":"10.1097\/00004703\u2010200612000\u201000003"},{"key":"e_1_2_12_29_1","unstructured":"University of Rochester Neurology: Duchenne Muscular Dystrophy Clinic \u201cDuchenne Muscular Dystrophy Clinic\u2014Neurology\u2014University of Rochester Medical Center \u201dDuchenne Muscular Dystrophy Clinic Web Site accessed Jan 18 2024 https:\/\/www.urmc.rochester.edu\/neurology\/our\u2010divisions\/neuromuscular\/clinics\/duchenne\u2010muscular\u2010dystrophy\u2010clinic.aspx."},{"key":"e_1_2_12_30_1","unstructured":"Muscular Dystrophy Association \u201cMuscular Dystrophy Association Web Site \u201daccessed Jan 18 2024 https:\/\/www.mda.org\/."},{"key":"e_1_2_12_31_1","unstructured":"Parent Project Muscular Dystrophy \u201cParent Project Muscular Dystrophy Web Site \u201daccessed Jan 18 2024 https:\/\/www.parentprojectmd.org\/."},{"key":"e_1_2_12_32_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24097"},{"key":"e_1_2_12_33_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27223"},{"key":"e_1_2_12_34_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25282"},{"key":"e_1_2_12_35_1","doi-asserted-by":"publisher","DOI":"10.1212\/CPJ.0000000000200180"},{"key":"e_1_2_12_36_1","doi-asserted-by":"publisher","DOI":"10.1097\/MCG.0000000000001976"},{"key":"e_1_2_12_37_1","doi-asserted-by":"publisher","DOI":"10.1080\/21678421.2023.2204871"},{"key":"e_1_2_12_38_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0265879"},{"key":"e_1_2_12_39_1","doi-asserted-by":"publisher","DOI":"10.1136\/archdischild\u20102014\u2010306366"},{"key":"e_1_2_12_40_1","doi-asserted-by":"publisher","DOI":"10.1038\/ejhg.2014.301"},{"key":"e_1_2_12_41_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002891"},{"key":"e_1_2_12_42_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cct.2017.04.008"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70327","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.70327","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70327","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T13:52:06Z","timestamp":1786629126000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.70327"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,7,8]]},"references-count":41,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,9]]}},"alternative-id":["10.1002\/mus.70327"],"URL":"https:\/\/doi.org\/10.1002\/mus.70327","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,7,8]]},"assertion":[{"value":"2025-01-10","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-06-23","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-07-08","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}