{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,19]],"date-time":"2026-05-19T06:19:39Z","timestamp":1779171579261,"version":"3.51.4"},"reference-count":13,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2024,8,13]],"date-time":"2024-08-13T00:00:00Z","timestamp":1723507200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2024,8,13]],"date-time":"2024-08-13T00:00:00Z","timestamp":1723507200000},"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":["Muscle and Nerve"],"published-print":{"date-parts":[[2024,11]]},"abstract":"Abstract<\/jats:title>\n \n Introduction\/Aims<\/jats:title>\n Following the approval of risdiplam, there are more possibilities for disease\u2010modifying therapy (DMT) in children with spinal muscular atrophy (SMA). Non\u2010treatment\u2010na\u00efve subjects with SMA involved in the JEWELFISH study, designed to evaluate the safety and tolerability of risdiplam, were required to undergo a washout period before receiving risdiplam. This study aims to investigate the safety of administering risdiplam in patients within 90\u2009days of receiving treatment with nusinersen.<\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n Data were collected on SMA patients who had undergone treatment with nusinersen, and who then received risdiplam within 90\u2009days of their last dose of nusinersen, including demographic characteristics, information on treatment with nusinersen and risdiplam, adverse events, and laboratory assessments in a follow\u2010up period of 90\u2009days, presented as median (range).<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n A total of 15 children with SMA were reported, including 8 males and 7 females. The median number of doses of previous nusinersen treatment received was 8 (6\u201317) doses, and the median age at first risdiplam treatment was 4.3 (1.9\u201311.2) years. Specifically, 8 children received risdiplam 30\u2009days or less after their most recent nusinersen treatment, 2 at 31\u201360\u2009days after nusinersen, and 5 at 61\u201389\u2009days post\u2010nusinersen. Adverse events of pyrexia, pneumonia, vomiting and rash were reported in 4 patients.<\/jats:p>\n <\/jats:sec>\n \n Discussion<\/jats:title>\n Our study showed good safety data on patients who received risdiplam following nusinersen within the washout period of 90\u2009days. This supplements the JEWELFISH study in the era of DMT, providing additional guidance for clinicians, but additional data from other centers is needed.<\/jats:p>\n <\/jats:sec>","DOI":"10.1002\/mus.28228","type":"journal-article","created":{"date-parts":[[2024,8,13]],"date-time":"2024-08-13T08:09:01Z","timestamp":1723536541000},"page":"1095-1098","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Safety of risdiplam in spinal muscular atrophy patients after short\u2010term treatment with nusinersen"],"prefix":"10.1002","volume":"70","author":[{"given":"Yue","family":"Yan","sequence":"first","affiliation":[{"name":"Department of Neurology, Children's Hospital Zhejiang University School of Medicine; National Clinical Research Center for Child Health Hangzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yijie","family":"Feng","sequence":"additional","affiliation":[{"name":"Department of Neurology, Children's Hospital Zhejiang University School of Medicine; National Clinical Research Center for Child Health Hangzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Liya","family":"Jiang","sequence":"additional","affiliation":[{"name":"Department of Neurology, Children's Hospital Zhejiang University School of Medicine; National Clinical Research Center for Child Health Hangzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jianing","family":"Jin","sequence":"additional","affiliation":[{"name":"Department of Neurology, Children's Hospital Zhejiang University School of Medicine; National Clinical Research Center for Child Health Hangzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6736-1407","authenticated-orcid":false,"given":"Shanshan","family":"Mao","sequence":"additional","affiliation":[{"name":"Department of Neurology, Children's Hospital Zhejiang University School of Medicine; National Clinical Research Center for Child Health Hangzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2024,8,13]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572\u2010022\u201000380\u20108"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2021.06.033"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40263\u2010022\u201000910\u20108"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/phar.2866"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27755"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51560"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120\u2010023\u201000444\u20101"},{"key":"e_1_2_13_9_1","unstructured":"Instructions for the use of risdiplam in China. National Healthcare Security Administration of China Web Site; (Accessed March 30 2024).http:\/\/www.nhsa.gov.cn\/attach\/Ypsn2023\/YPSN202300161\/YPSN202300161\u2010N2(PPT).pdf."},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(22)00339\u20108"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(21)00367\u20107"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ijsu.2020.11.005"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352\u20104642(21)00287\u2010X"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41434\u2010022\u201000341\u20106"}],"container-title":["Muscle & Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.28228","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.28228","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.28228","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T15:06:17Z","timestamp":1772982377000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.28228"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,8,13]]},"references-count":13,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2024,11]]}},"alternative-id":["10.1002\/mus.28228"],"URL":"https:\/\/doi.org\/10.1002\/mus.28228","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,8,13]]},"assertion":[{"value":"2024-01-30","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-07-24","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-08-13","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}