{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T11:21:07Z","timestamp":1788520867066,"version":"build-2803163510"},"reference-count":35,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,12,24]],"date-time":"2024-12-24T00:00:00Z","timestamp":1734998400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82271735"],"award-info":[{"award-number":["82271735"]}],"id":[{"id":"10.13039\/501100001809","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":[[2025,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>The rising use of disease\u2010modifying therapy is progressively impacting the health\u2010related quality of life (HRQoL) of patients with spinal muscular atrophy (SMA) in their daily lives. This study aimed to evaluate the changes in HRQoL and independence in children with later\u2010onset SMA receiving longitudinal treatment with nusinersen.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Forty\u2010nine pediatric patients with later\u2010onset SMA (symptom onset after 6\u2009months of age) and their caregivers were enrolled. The HRQoL of patients evaluated by the proxy\u2010reported Pediatric Quality of Life Inventory 3.0 Neuromuscular Module (PedsQL NMM) and the independence level determined by the SMA Independence Scale\u2013Upper Limb Module (SMAIS\u2013ULM) were assessed. Caregiver HRQoL was assessed using the Pediatric Quality of Life Inventory Family Impact Module (PedsQL FIM). Motor function was recorded using the Hammersmith Functional Motor Scale Expanded (HFMSE) and the Revised Upper Limb Module (RULM), with subsequent analysis of the correlation between motor function, HRQoL, and independence scores.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A significant difference was observed across all domains of the proxy\u2010reported PedsQL NMM and in the independence assessment over the 18\u2010month follow\u2010up period (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001). A positive correlation was identified between RULM and total PedsQL NMM scores (Pearson\u2010r\u2009=\u20090.539, <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001), as well as SMAIS\u2010ULM scores (Spearman\u2010rho\u2009=\u20090.507, <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001). Scores in all modules of the PedsQL FIM improved over time (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001).<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>This study demonstrates the longitudinal effects of nusinersen treatment on multifaceted aspects of SMA patients, as captured by patient\u2010reported outcome measures (PROMs). The inclusion of PROMs should be considered as part of the SMA multidisciplinary assessment.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.28311","type":"journal-article","created":{"date-parts":[[2024,12,24]],"date-time":"2024-12-24T12:32:05Z","timestamp":1735043525000},"page":"368-376","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Longitudinal Efficacy of Nusinersen Treatment on Health\u2010Related Quality of Life and Independence in Children With Later\u2010Onset Spinal Muscular Atrophy"],"prefix":"10.1002","volume":"71","author":[{"given":"Siyi","family":"Huang","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":[{"vocabulary":"crossref","role":"author"}]},{"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":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dongming","family":"Zhou","sequence":"additional","affiliation":[{"name":"Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health  Hangzhou China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yue","family":"Yan","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":[{"vocabulary":"crossref","role":"author"}]},{"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":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yicheng","family":"Yu","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":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mei","family":"Yao","sequence":"additional","affiliation":[{"name":"Department of Infectious Diseases Children's Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health  Hangzhou China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Feng","family":"Gao","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":[{"vocabulary":"crossref","role":"author"}]},{"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":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,12,24]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-022-00380-8"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01701-y"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-021-10954-3"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-023-11711-4"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2021.626787"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01638-8"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-221595"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2014.304"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1080\/13548506.2018.1510131"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.3390\/brainsci13010110"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.09.009"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.2147\/CEOR.S214084"},{"issue":"3","key":"e_1_2_12_14_1","first-page":"390","article-title":"Findings Regarding Emotion Regulation Strategies and Quality of life's Domains in Families Having Children With Spinal Muscular Atrophy","volume":"14","author":"Morcov M. 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