{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T21:08:24Z","timestamp":1773004104749,"version":"3.50.1"},"reference-count":16,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2025,8,15]],"date-time":"2025-08-15T00:00:00Z","timestamp":1755216000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2025,8,15]],"date-time":"2025-08-15T00:00:00Z","timestamp":1755216000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","award":["23ek0109611h0002"],"award-info":[{"award-number":["23ek0109611h0002"]}],"id":[{"id":"10.13039\/100009619","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","award":["25ek0109611h0004"],"award-info":[{"award-number":["25ek0109611h0004"]}],"id":[{"id":"10.13039\/100009619","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Neuropsychopharm Rep"],"published-print":{"date-parts":[[2025,9]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>This study aimed to evaluate the efficacy and safety of steroid therapy in patients with Fukuyama congenital muscular dystrophy (FCMD).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      This is a noncontrolled, nonblinded, multicenter collaborative phase 2 trial in patients with a definite diagnosis of 3\u2010kb insertion mutation homozygous or compound heterozygous FCMD based on genetic testing. The first part of the study evaluates efficacy; patients with homozygous FCMD were given oral prednisolone at a dose of 1\u2009mg\/kg on alternate days (0.5\u2009mg\/kg\/day) for 24\u2009weeks. The second part of the study evaluates safety; patients with homozygous and heterozygous FCMD were given oral prednisolone at a dose of 1\u2009mg\/kg on alternate days (0.5\u2009mg\/kg\/day) for 48\u2009weeks. Homozygous patients will be evaluated in the first part of the study for up to 24\u2009weeks after prednisolone administration, after which they will continue receiving prednisolone in the second part for an additional 24\u2009weeks. The primary endpoints were the changes in motor function evaluated using the gross motor function measure after treatment with prednisolone in the first part and the safety profiles based on the results of physical examination, vital signs, 12\u2010lead electrocardiography (ECG), echocardiography, ophthalmic testing, SpO\n                      <jats:sub>2<\/jats:sub>\n                      , laboratory tests, immunological tests, and adverse events in the second part.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Based on previous clinical research, prednisolone shows great potential as a therapeutic drug in patients with FCMD. To achieve this goal, we planned an investigator\u2010initiated trial to confirm the effectiveness and safety of prednisolone.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/npr2.70043","type":"journal-article","created":{"date-parts":[[2025,8,15]],"date-time":"2025-08-15T07:14:24Z","timestamp":1755242064000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Nonrandomized Allocation of Steroid Therapy in Patients With Fukuyama Congenital Muscular Dystrophy: Study Protocol for a Phase\n                    <scp>II<\/scp>\n                    Clinical Trial"],"prefix":"10.1002","volume":"45","author":[{"given":"Terumi","family":"Murakami","sequence":"first","affiliation":[{"name":"Department of Pediatrics Tokyo Women's Medical University School of Medicine  Shinjuku\u2010ku Tokyo Japan"},{"name":"Department of Clinical Research National Hospital Organization Higashisaitama Hospital  Hasuda Saitama Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Takatoshi","family":"Sato","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Tokyo Women's Medical University School of Medicine  Shinjuku\u2010ku Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3641-3328","authenticated-orcid":false,"given":"Takami","family":"Ishizuka","sequence":"additional","affiliation":[{"name":"Department of Clinical Research Support, Clinical Research and Education Promotion Division National Center of Neurology and Psychiatry  Kodaira Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Harumasa","family":"Nakamura","sequence":"additional","affiliation":[{"name":"Department of Clinical Research Support, Clinical Research and Education Promotion Division National Center of Neurology and Psychiatry  Kodaira Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hisateru","family":"Tachimori","sequence":"additional","affiliation":[{"name":"Department of Health Policy and Management Keio University School of Medicine  Shinjuku\u2010ku Tokyo Japan"},{"name":"Department of Information Medicine National Institute of Neuroscience, National Center of Neurology and Psychiatry  Kodaira Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hiroko","family":"Harada","sequence":"additional","affiliation":[{"name":"Department of Clinical Research Support, Clinical Research and Education Promotion Division National Center of Neurology and Psychiatry  Kodaira Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hideki","family":"Oi","sequence":"additional","affiliation":[{"name":"Department of Clinical Data Science, Clinical Research and Education Promotion Division National Center of Neurology and Psychiatry  Kodaira Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kenji","family":"Hatano","sequence":"additional","affiliation":[{"name":"Department of Clinical Data Science, Clinical Research and Education Promotion Division National Center of Neurology and Psychiatry  Kodaira Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mari S.","family":"Oba","sequence":"additional","affiliation":[{"name":"Department of Clinical Data Science, Clinical Research and Education Promotion Division National Center of Neurology and Psychiatry  Kodaira Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kumiko","family":"Ishiguro","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Tokyo Women's Medical University School of Medicine  Shinjuku\u2010ku Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Minobu","family":"Shichiji","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Tokyo Women's Medical University School of Medicine  Shinjuku\u2010ku Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuki","family":"Kihara","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Tokyo Women's Medical University School of Medicine  Shinjuku\u2010ku Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yasuhiro","family":"Takeshima","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Hyogo Medical University  Nishinomiya Hyogo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mariko","family":"Taniguchi\u2010Ikeda","sequence":"additional","affiliation":[{"name":"Department of Clinical Genetics Fujita Health University Hospital  Toyoake Aichi Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ayako","family":"Hattori","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Nagoya City University East Medical Ceter  Chikusa\u2010ku Nagoya Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuko","family":"Shimizu\u2010Motohashi","sequence":"additional","affiliation":[{"name":"Department of Child Neurology National Center Hospital National Center of Neurology and Psychiatry  Kodaira Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hiroyuki","family":"Awano","sequence":"additional","affiliation":[{"name":"Department of Clinical Genetics Tottori University Hospital  Yonago Tottori Japan"},{"name":"Department of Pediatrics Kobe University Graduate School of Medicine  Chuo\u2010ku Kobe Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ryosuke","family":"Bo","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Kobe University Graduate School of Medicine  Chuo\u2010ku Kobe Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Satoru","family":"Nagata","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Tokyo Women's Medical University School of Medicine  Shinjuku\u2010ku Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8435-7418","authenticated-orcid":false,"given":"Keiko","family":"Ishigaki","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Tokyo Women's Medical University School of Medicine  Shinjuku\u2010ku Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2025,8,15]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0387-7604(81)80002-2"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/28653"},{"issue":"3","key":"e_1_2_10_4_1","first-page":"196","article-title":"Recent Trends in the Development of Treatments for Fukuyama Congenital Muscular Dystrophy","volume":"55","author":"Ishigaki K.","year":"2023","journal-title":"No to Hattatsu"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2018.08.001"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.09.014"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature10456"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.880170405"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-021-03781-z"},{"issue":"18","key":"e_1_2_10_10_1","first-page":"197","article-title":"The Analysis of the Course of Motor Impairment in Fukuyama Type Congenital Muscular Dystrophy","volume":"22","author":"Okawa Y.","year":"1985","journal-title":"Journal of Rehabilitation Medicine"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.1989.tb04003.x"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.braindev.2020.02.006"},{"key":"e_1_2_10_13_1","first-page":"S330","article-title":"A Trial of Steroid Treatment for Fukuyama\u2010Type Congenital Muscular Dystrophy (Mild Type)","volume":"42","author":"Miyamoto A.","year":"2010","journal-title":"No to Hattatsu"},{"key":"e_1_2_10_14_1","first-page":"S245","article-title":"Effects of Oral Steroid Treatment on Ambulatory Fukuyama\u2010Type Congenital Muscular Dystrophy","volume":"47","author":"Toyono M.","year":"2015","journal-title":"No to Hattatsu"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0960-8966(02)00023-8"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.21006"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.braindev.2011.06.002"}],"container-title":["Neuropsychopharmacology Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/npr2.70043","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/npr2.70043","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/npr2.70043","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T18:27:43Z","timestamp":1772994463000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/npr2.70043"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,8,15]]},"references-count":16,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2025,9]]}},"alternative-id":["10.1002\/npr2.70043"],"URL":"https:\/\/doi.org\/10.1002\/npr2.70043","archive":["Portico"],"relation":{},"ISSN":["2574-173X","2574-173X"],"issn-type":[{"value":"2574-173X","type":"print"},{"value":"2574-173X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,8,15]]},"assertion":[{"value":"2025-02-24","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-08-04","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-08-15","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70043"}}