{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,19]],"date-time":"2026-06-19T10:07:25Z","timestamp":1781863645978,"version":"3.54.5"},"reference-count":26,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2024,3,21]],"date-time":"2024-03-21T00:00:00Z","timestamp":1710979200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100009438","name":"National Center of Neurology and Psychiatry","doi-asserted-by":"publisher","award":["29\u20134"],"award-info":[{"award-number":["29\u20134"]}],"id":[{"id":"10.13039\/501100009438","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100018651","name":"Toa Eiyo","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100018651","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002973","name":"Daiichi-Sankyo","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002973","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100018036","name":"Nippon Shinyaku","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100018036","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\/100010793","name":"Pfizer Japan","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100010793","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100019522","name":"JCR Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100019522","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":[[2024,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>Duchenne muscular dystrophy (DMD) presents with skeletal muscle weakness, followed by cardiorespiratory involvement. The need for longitudinal data regarding DMD that could serve as a control for determining treatment efficacy in clinical trials has increased notably. The present study examined the longitudinal data of Japanese DMD patients collectively and assessed individual patients with pathogenic variants eligible for exon\u2010skipping therapy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Patients with DMD who visited Kobe University Hospital between March 1991 and March 2019 were enrolled. Data between the patients' first visit until age 20\u2009years were examined.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Three hundred thirty\u2010seven patients were included. Serum creatine kinase levels showed extremely high values until the age of 6\u2009years and a rapid decline from ages 7\u201312\u2009years. Both the median 10\u2010m run\/walk velocity and rise\u2010from\u2010floor velocity peaked at the age of 4\u2009years and declined with age. The values for respiratory function declined from the age of 11\u2009years. The median left ventricular ejection fraction was &gt;60% until the age of 12\u2009years and rapidly declined from ages 13\u201315\u2009years. Examination of the relationship between pathogenic variants eligible for exon\u2010skipping therapy and longitudinal data revealed no characteristic findings.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>We found that creatine kinase levels and motor, respiratory, and cardiac functions each exhibited various changes over time. These findings provide useful information about the longitudinal data of several outcome measures for patients with DMD not receiving corticosteroids. These data may serve as historical controls in comparing the natural history of DMD patients not on regular steroid use in appropriate clinical trials.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.28073","type":"journal-article","created":{"date-parts":[[2024,3,21]],"date-time":"2024-03-21T04:28:35Z","timestamp":1710995315000},"page":"604-612","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Longitudinal data of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy"],"prefix":"10.1002","volume":"69","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9846-4142","authenticated-orcid":false,"given":"Hiroyuki","family":"Awano","sequence":"first","affiliation":[{"name":"Research Initiative Center, Organization for Research Initiative and Promotion Tottori University  Yonago Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshinori","family":"Nambu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Kobe University Graduate School of Medicine  Kobe Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chieko","family":"Itoh","sequence":"additional","affiliation":[{"name":"Division of Rehabilitation Medicine Kobe University Hospital  Kobe Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Akihiro","family":"Kida","sequence":"additional","affiliation":[{"name":"Division of Rehabilitation Medicine Kobe University Hospital  Kobe Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tetsushi","family":"Yamamoto","sequence":"additional","affiliation":[{"name":"Nagahama Institute of Bio\u2010Science and Technology  Nagahama 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