{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,20]],"date-time":"2026-06-20T05:50:51Z","timestamp":1781934651836,"version":"3.54.5"},"reference-count":24,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T00:00:00Z","timestamp":1778457600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T00:00:00Z","timestamp":1778457600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100011022","name":"Vertex Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100011022","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007723","name":"Takeda Pharmaceuticals U.S.A.","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007723","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000038","name":"U.S. Food and Drug Administration","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000038","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100003975","name":"Myotonic Dystrophy Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100003975","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":[[2026,7]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aim<\/jats:title>\n                    <jats:p>The Myotonic Dystrophy Health Index (MDHI) is a patient\u2010reported outcome measure assessing disease burden in DM1. In prior cross\u2010sectional studies, the MDHI correlated with disability status and with several functional measures and activities of daily living. To aid its use and interpretation, we investigated the longitudinal performance of MDHI.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Patients with genetically confirmed DM1 were enrolled as part of an observational longitudinal study within the Myotonic Dystrophy Clinical Research Network (DMCRN). Internal consistency and construct validity were evaluated to confirm previous findings. Both the Minimal Detectable Change (MDC) and the Minimal Clinically Important Difference (MCID) were calculated using distribution\u2010 and anchor\u2010based methods, respectively. Sensitivity to change and responsiveness were also investigated, along with potential factors associated with the progression of overall burden.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      451 DM1 patients were assessed, with 147 completing 24\u2010month follow\u2010up. The MDHI showed excellent internal consistency (\n                      <jats:italic>\u03b1<\/jats:italic>\n                      \u2009=\u20090.95) and confirmed construct validity. MDC for the total score ranged from \u00b14.24 (effect size change\u2010based approach) to \u00b19.72 (Standard Error Method\u2010based approach) points, while MCID ranged from small changes (\u22120.12 improvement\/+1.25 worsening) to large changes (\u22122.17 improvement\/+4.71 worsening). Changes in MDHI total and subscale scores were statistically significant at individual\u2010 or subgroup\u2010levels and consistently mirrored changes in corresponding clinical measures.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>The MDHI demonstrates robust sensitivity and responsiveness to individual and subgroup\u2010level changes in disease burden, offering valuable insights into patient\u2010perceived progression. These findings support MDHI use over time and as a potential additional outcome measure at subgroup levels in clinical trials.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70277","type":"journal-article","created":{"date-parts":[[2026,5,12]],"date-time":"2026-05-12T02:03:40Z","timestamp":1778551420000},"page":"187-197","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Longitudinal Psychometric Properties of the Myotonic Dystrophy Health Index in a Large Multicenter Cohort of People Living With Myotonic Dystrophy Type 1"],"prefix":"10.1002","volume":"74","author":[{"given":"Valeria A.","family":"Sansone","sequence":"first","affiliation":[{"name":"The NeMO Clinical Center in Milan  Milan Italy"},{"name":"Neurorehabilitation Unit, Milan, University of Milan Italy  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8875-9665","authenticated-orcid":false,"given":"Andrea","family":"Lizio","sequence":"additional","affiliation":[{"name":"The NeMO Clinical Center in Milan  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-8621-2161","authenticated-orcid":false,"given":"Carola R.","family":"Ferrari Aggradi","sequence":"additional","affiliation":[{"name":"The NeMO Clinical Center in Milan  Milan Italy"},{"name":"Neurorehabilitation Unit, Milan, University of Milan Italy  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucia C.","family":"Greco","sequence":"additional","affiliation":[{"name":"The NeMO Clinical Center in Milan  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2829-8470","authenticated-orcid":false,"given":"Cynthia","family":"Gagnon","sequence":"additional","affiliation":[{"name":"Universit\u00e9 de Sherbrooke, CIUSSS Saguenay\u2010Lac\u2010St\u2010Jean  Qu\u00e9bec Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sub","family":"Subramony","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Florida College of Medicine  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard H.","family":"Roxburgh","sequence":"additional","affiliation":[{"name":"The University of Auckland, Centre for Brain Research Neurogenetics Clinic  Auckland New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8487-9478","authenticated-orcid":false,"given":"Karlien","family":"Mul","sequence":"additional","affiliation":[{"name":"Department of Neurology Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center  Nijmegen the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4851-7637","authenticated-orcid":false,"given":"Johanna","family":"Hamel","sequence":"additional","affiliation":[{"name":"Department of Neurology, Neuromuscular Disease and Department of Pathology and Laboratory Medicine University of Rochester Medical Center  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kate","family":"Eichinger","sequence":"additional","affiliation":[{"name":"Department of Neurology, Neuromuscular Disease and Department of Pathology and Laboratory Medicine University of Rochester Medical Center  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2827-3740","authenticated-orcid":false,"given":"Man","family":"Hung","sequence":"additional","affiliation":[{"name":"Roseman University of Health Sciences, University of Utah  Salt Lake City Utah USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey M.","family":"Statland","sequence":"additional","affiliation":[{"name":"University of Kansas Medical Center  Kansas City Kansas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"Turner","sequence":"additional","affiliation":[{"name":"The Institute of Neurology, University College London and NHNN, Queen Square, UCLH  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bakri","family":"Elsheikh","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Diseases The Ohio State University Wexner Medical Center  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2757-8131","authenticated-orcid":false,"given":"Benedikt","family":"Schoser","sequence":"additional","affiliation":[{"name":"Friedrich\u2010Baur\u2010Institute, Department of Neurology, LMU Clinic  Munich Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Ragole","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Colorado, Anschutz Medical Campus  Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma","family":"Matthews","sequence":"additional","affiliation":[{"name":"Atkinson\u2010Morley Neuromuscular Centre, St George's University Hospitals NHS  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacinda","family":"Sampson","sequence":"additional","affiliation":[{"name":"Department of Neurology &amp; Neurological Services Stanford University  Palo Alto California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masanori P.","family":"Takahashi","sequence":"additional","affiliation":[{"name":"Clinical Neurophysiology, Department of Clinical Laboratory and Biomedical Sciences The University of Osaka Graduate School of Medicine  Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Wicklund","sequence":"additional","affiliation":[{"name":"UT Health  San Antonio Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Swenson","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Iowa  Iowa City Iowa USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chamindra G.","family":"Laverty","sequence":"additional","affiliation":[{"name":"Department of Neurosciences University of California  San Diego California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Perry","family":"Shieh","sequence":"additional","affiliation":[{"name":"University of California  Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3053-4057","authenticated-orcid":false,"given":"Ericka P.","family":"Greene","sequence":"additional","affiliation":[{"name":"Houston Methodist Neurological Institute  Houston Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Beretta","sequence":"additional","affiliation":[{"name":"The NeMO Clinical Center in Milan  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacopo L.","family":"Casiraghi","sequence":"additional","affiliation":[{"name":"The NeMO Clinical Center in Milan  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeanne","family":"Dekdebrun","sequence":"additional","affiliation":[{"name":"Department of Neurology, Neuromuscular Disease and Department of Pathology and Laboratory Medicine University of Rochester Medical Center  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Raymond","sequence":"additional","affiliation":[{"name":"Department of Neurology Virginia Commonwealth University  Richmond Virginia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruby","family":"Langeslay","sequence":"additional","affiliation":[{"name":"Department of Neurology Virginia Commonwealth University  Richmond Virginia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erin","family":"DeSpain","sequence":"additional","affiliation":[{"name":"Department of Neurology Virginia Commonwealth University  Richmond Virginia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles A.","family":"Thornton","sequence":"additional","affiliation":[{"name":"Department of Neurology, Neuromuscular Disease and Department of Pathology and Laboratory Medicine University of Rochester Medical Center  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":"Department of Neurology, Neuromuscular Disease and Department of Pathology and Laboratory Medicine University of Rochester Medical Center  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3917-4257","authenticated-orcid":false,"given":"Nicholas E.","family":"Johnson","sequence":"additional","affiliation":[{"name":"Department of Neurology Virginia Commonwealth University  Richmond Virginia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"Myotonic Dystrophy Clinical Research 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&amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70277","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.70277","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70277","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,6,20]],"date-time":"2026-06-20T05:03:32Z","timestamp":1781931812000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.70277"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,5,11]]},"references-count":24,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,7]]}},"alternative-id":["10.1002\/mus.70277"],"URL":"https:\/\/doi.org\/10.1002\/mus.70277","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,5,11]]},"assertion":[{"value":"2025-12-05","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication 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