{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T03:58:54Z","timestamp":1790913534353,"version":"4.1.0"},"reference-count":20,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2025,5,14]],"date-time":"2025-05-14T00:00:00Z","timestamp":1747180800000},"content-version":"vor","delay-in-days":133,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatrics International"],"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Spinal muscular atrophy (SMA) is a recessively inherited neurological disease resulting in motor neuron disorder. Onasemnogene abeparvovec is a gene replacement therapy used to treat patients with SMA. Cardiac toxicity was observed in animal studies on this therapy, and elevated cardiac troponin I levels were observed in clinical trials; however, the clinical importance of these findings is unknown. Therefore, this study aimed to reveal the cardiac toxicity of onasemnogene abeparvovec through careful investigation of cardiac function using cardiac strain analysis, which can detect early subtle abnormalities.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This study included patients with SMA treated with onasemnogene abeparvovec between June 2020 and November 2020. Echocardiography, including peak global longitudinal strain (GLS), and other laboratory test results were evaluated.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Case 1 showed a relative GLS decrease of 14.5% compared with that at the baseline (GLS reduction from \u221222.1% to \u221218.9%), elevation of N\u2010terminal prohormone B\u2010type natriuretic peptide levels from 227\u2009pg\/mL to 494\u2009pg\/mL, and elevated liver enzyme concentrations after gene replacement therapy without reduction of the left ventricular ejection fraction.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Subclinical myocardial dysfunction after infusion of onasemnogene abeparvovec was suggested by careful investigation of cardiac function. Cardiac toxicity may correlate with liver impairment following gene replacement therapy. Long\u2010term studies that allow for a higher number of patients and more extended observation periods should be conducted to confirm the cardiac toxicity of onasemnogene abeparvovec.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ped.70019","type":"journal-article","created":{"date-parts":[[2025,5,14]],"date-time":"2025-05-14T06:19:44Z","timestamp":1747203584000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Evaluation of cardiac function in patients with\n                    <scp>SMA<\/scp>\n                    after treatment with onasemnogene abeparvovec"],"prefix":"10.1111","volume":"67","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4275-2158","authenticated-orcid":false,"given":"Akihisa","family":"Horigome","sequence":"first","affiliation":[{"name":"Department of Pediatrics Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Satoshi","family":"Takasago","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Reiko","family":"Arakawa","sequence":"additional","affiliation":[{"name":"Department of Genomic Medicine Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9285-4649","authenticated-orcid":false,"given":"Katsuyoshi","family":"Shimozawa","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Child Health Nihon University School of Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masao","family":"Kaneshige","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"},{"name":"Department of Genomic Medicine Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keiji","family":"Goishi","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hideko","family":"Uryu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Junko","family":"Yamanaka","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hiroyuki","family":"Shichino","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ayumi","family":"Mizukami","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center Hospital of the National Center for Global Health and Medicine  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,5,14]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00001-6"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00251-9"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2018.01.003"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1086\/338627"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25101"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01356-1"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2020.11.054"},{"key":"e_1_2_8_10_1","unstructured":"U.S. Food & Drug administration. Washington DC.Zolgensma (onasemnogene abeparvovec) prescribing information. [Cited October 6 2022]. Available from:https:\/\/www.fda.gov\/media\/126109\/download"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2018.015"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1254\/fpj.21066"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-4642(21)00287-X"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.echo.2013.02.008"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1093\/eurheartj\/ehac244"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1253\/circj.CJ-18-0333"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00246-008-9258-4"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.76"},{"key":"e_1_2_8_19_1","first-page":"1184","article-title":"Study of effects of anthracycline drugs on myocardial function in breast cancer patients by quantitative analysis of layer\u2010specific strain via 2D\u2010STI technology","volume":"15","author":"Wu J","year":"2021","journal-title":"Am J Transl Res"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10554-018-1510-2"},{"key":"e_1_2_8_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2017.11.007"}],"container-title":["Pediatrics International"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/ped.70019","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/ped.70019","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/ped.70019","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T03:44:26Z","timestamp":1790912666000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/ped.70019"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1]]},"references-count":20,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2025,1]]}},"alternative-id":["10.1111\/ped.70019"],"URL":"https:\/\/doi.org\/10.1111\/ped.70019","archive":["Portico"],"relation":{},"ISSN":["1328-8067","1442-200X"],"issn-type":[{"value":"1328-8067","type":"print"},{"value":"1442-200X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,1]]},"assertion":[{"value":"2023-02-14","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-09-16","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-05-14","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70019"}}