{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T03:31:32Z","timestamp":1792812692310,"version":"4.5.0"},"reference-count":31,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2025,2,13]],"date-time":"2025-02-13T00:00:00Z","timestamp":1739404800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100014943","name":"Sarepta Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014943","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":[[2025,6]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Aims<\/jats:title><jats:p>There are few long\u2010term studies evaluating clinical outcomes and mortality among individuals with Duchenne muscular dystrophy (DMD); particularly using longitudinal health administrative claims data, reflecting populations managed in typical clinical practice. This study aimed to characterize DMD outcomes via a population\u2010based database.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Patients with DMD, diagnosed between 01\/1979 and 03\/2020 at \u2264\u200910\u2009years of age, were identified using the Manitoba Population Research Data Repository housed at the Manitoba Centre for Health Policy. De\u2010identified longitudinal administrative data from 1998 to 2020 were used to retrospectively assess frequencies and age at first observation of key DMD outcomes including scoliosis, cardiovascular\u2010related complications, severe respiratory\u2010related morbidities, and mortality. Survival analyses using Kaplan\u2013Meier curves were used to describe attrition and estimate probability of patients remaining observation\u2010free by age.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>This study included 198 patients with median (IQR) follow\u2010up of 9.6 (6.6\u201315.5) years. Corticosteroid use was observed in 26%, with a mean (SD) percentage of days covered of 31% (39%) from initiation to end of follow\u2010up. Scoliosis observations were captured in 18% (median[IQR] age 12 [11\u201315] years at first observation), severe respiratory\u2010related morbidities in 20% (14[6.5\u201318] years), and cardiovascular\u2010related complications in 32% of the cohort (12.5[2\u201320.5] years). Mortality was observed in 14% of the cohort. Kaplan\u2013Meier curves estimated 15% mortality by age 20\u2009years and 20% by 25\u2009years.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>In a population\u2010based data set with decades of follow\u2010up, these data provide longitudinal observations of the substantial burden of DMD, and insight into contemporary estimates of mortality and treatment patterns in Canada.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.28368","type":"journal-article","created":{"date-parts":[[2025,2,13]],"date-time":"2025-02-13T10:45:47Z","timestamp":1739443547000},"page":"955-962","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Longitudinal Outcomes Among Patients With Duchenne Muscular Dystrophy: A Canadian Retrospective Population\u2010Based Study"],"prefix":"10.1002","volume":"71","author":[{"given":"Christina","family":"Qian","sequence":"first","affiliation":[{"name":"Broadstreet Health Economics &amp; Outcomes Research  Vancouver British Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexa C.","family":"Klimchak","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shelagh M.","family":"Szabo","sequence":"additional","affiliation":[{"name":"Broadstreet Health Economics &amp; Outcomes Research  Vancouver British Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine L.","family":"Gooch","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roxana","family":"Dragan","sequence":"additional","affiliation":[{"name":"University of Manitoba  Winnipeg Manitoba Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather J.","family":"Prior","sequence":"additional","affiliation":[{"name":"University of Manitoba  Winnipeg Manitoba Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean K.","family":"Mah","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Cumming School of Medicine University of Calgary  Calgary Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,2,13]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23807"},{"key":"e_1_2_11_3_1","unstructured":"Parent Project Muscular Dystrophy \u201cThe Duchenne Registry \u201d(2019) https:\/\/www.duchenneregistry.org\/."},{"key":"e_1_2_11_4_1","unstructured":"Centers for Disease Control and Prevention \u201cMuscular Dystrophy Research and Tracking \u201d(2019) https:\/\/www.cdc.gov\/ncbddd\/musculardystrophy\/research.html."},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-221575"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30024-3"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/jpc.12868"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01862-w"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-220816"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.05.019"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000001950"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)32160-8"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-170280"},{"key":"e_1_2_11_14_1","unstructured":"University of Manitoba \u201cManitoba Population Research Data Repository \u2013 Overview \u201d(2018) http:\/\/umanitoba.ca\/faculties\/health_sciences\/medicine\/units\/chs\/departmental_units\/mchp\/resources\/repository\/index.html."},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.4045\/tidsskr.13.0836"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000012910"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.18553\/jmcp.2017.23.6.633"},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12325-024-02897-8"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70272-8"},{"key":"e_1_2_11_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_2_11_21_1","doi-asserted-by":"publisher","DOI":"10.1017\/S1047951118000288"},{"issue":"4","key":"e_1_2_11_22_1","doi-asserted-by":"crossref","first-page":"S388","DOI":"10.1016\/j.healun.2018.01.999","article-title":"Duchenne Muscular Dystrophy\u2010Related Acute Heart Failure: Multicenter Study of Hospitalizations and Outcomes","volume":"37","author":"Deshpande S.","year":"2018","journal-title":"Journal of Heart and Lung Transplantation"},{"key":"e_1_2_11_23_1","first-page":"1","article-title":"One in Five Patients With Duchenne Muscular Dystrophy Dies From Other Causes Than Cardiac or Respiratory Failure","volume":"37","author":"Wahlgren L.","year":"2021","journal-title":"European Journal of Epidemiology"},{"issue":"2","key":"e_1_2_11_24_1","first-page":"129","article-title":"Duchenne Muscular Dystrophy in a Developing Country: Challenges in Management and Genetic Counseling","volume":"25","author":"L\u00f3pez\u2010Hern\u00e1ndez L. B.","year":"2014","journal-title":"Genetic Counseling"},{"key":"e_1_2_11_25_1","doi-asserted-by":"publisher","DOI":"10.1097\/PHM.0000000000000228"},{"key":"e_1_2_11_26_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.brs.0000255062.94744.52"},{"key":"e_1_2_11_27_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26129"},{"key":"e_1_2_11_28_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073816687422"},{"key":"e_1_2_11_29_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.healun.2018.01.290"},{"key":"e_1_2_11_30_1","doi-asserted-by":"publisher","DOI":"10.1017\/S0317167100009926"},{"key":"e_1_2_11_31_1","volume-title":"Emerging Drugs for Duchenne Muscular Dystrophy","author":"Shawi F.","year":"2017"},{"key":"e_1_2_11_32_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10654-020-00613-8"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.28368","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,5,8]],"date-time":"2025-05-08T12:04:44Z","timestamp":1746705884000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.28368"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,2,13]]},"references-count":31,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2025,6]]}},"alternative-id":["10.1002\/mus.28368"],"URL":"https:\/\/doi.org\/10.1002\/mus.28368","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,2,13]]},"assertion":[{"value":"2024-04-04","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-01-29","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-02-13","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}