{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,10]],"date-time":"2026-02-10T00:14:58Z","timestamp":1770682498628,"version":"3.49.0"},"reference-count":17,"publisher":"International Research and Cooperation Association for Bio & Socio-Sciences Advancement (IRCA-BSSA)","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IRDR"],"published-print":{"date-parts":[[2022,11,30]]},"DOI":"10.5582\/irdr.2022.01094","type":"journal-article","created":{"date-parts":[[2022,11,27]],"date-time":"2022-11-27T11:16:18Z","timestamp":1669547778000},"page":"189-195","source":"Crossref","is-referenced-by-count":6,"title":["Assessment of health-related quality of life in patients with spina muscular atrophy in China"],"prefix":"10.5582","volume":"11","author":[{"given":"Chengaxin","family":"Duan","sequence":"first","affiliation":[{"name":"China National Health Development Research Center; National Center for Drug and Technology Assessment, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dandan","family":"Ai","sequence":"additional","affiliation":[{"name":"China National Health Development Research Center; National Center for Drug and Technology Assessment, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Qian","family":"Xu","sequence":"additional","affiliation":[{"name":"China National Health Development Research Center; National Center for Drug and Technology Assessment, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Binyan","family":"Sui","sequence":"additional","affiliation":[{"name":"China National Health Development Research Center; National Center for Drug and Technology Assessment, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kun","family":"Zhao","sequence":"additional","affiliation":[{"name":"Vanke School of Public Health, Tsinghua University; Institute for Healthy China, Tsinghua University, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"3542","reference":[{"key":"1","doi-asserted-by":"crossref","unstructured":"1. Chambers GM, Settumba SN, Carey KA, Cairns A, Menezes MP, Ryan M, Farrar MA. Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy. Neurology. 2020; 95:e1-e10.","DOI":"10.1212\/WNL.0000000000009715"},{"key":"2","doi-asserted-by":"crossref","unstructured":"2. L\u00f3pez-Bastida J, Pe\u00f1a-Longobardo LM, Aranda-Reneo I, Tizzano E, Sefton M, Oliva-Moreno J. Social\/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain. Orphanet J Rare Dis. 2017; 12:141.","DOI":"10.1186\/s13023-017-0695-0"},{"key":"3","doi-asserted-by":"crossref","unstructured":"3. Pe\u00f1a-Longobardo LM, Aranda-Reneo I, Oliva-Moreno J, Litzkendorf S, Durand-Zaleski I, Tizzano E, L\u00f3pez-Bastida J. The economic impact and health-related quality of life of spinal muscular atrophy. An analysis across Europe. Int J Environ Res Public Health. 2020; 17:5640.","DOI":"10.3390\/ijerph17165640"},{"key":"4","doi-asserted-by":"crossref","unstructured":"4. Landfeldt E, Edstr\u00f6m J, Sejersen T, Tulinius M, Lochm\u00fcller H, Kirschner J. Quality of life of patients with spinal muscular atrophy: A systematic review. Eur J Paediatr Neurol. 2019; 23:347-356.","DOI":"10.1016\/j.ejpn.2019.03.004"},{"key":"5","doi-asserted-by":"crossref","unstructured":"5. Thimm A, Brakemeier S, Kizina K, Munoz Rosales J, Stolte B, Totzeck A, Deuschl C, Kleinschnitz C, Hagenacker T. Assessment of health-related quality of life in adult spinal muscular atrophy under nusinersen treatment-a pilot study. Front Neurol. 2022; 12:812063.","DOI":"10.3389\/fneur.2021.812063"},{"key":"6","unstructured":"6. Albrechtsen SS, Born AP, Boesen MS. Nusinersen treatment of spinal muscular atrophy - a systematic review. Dan Med J. 2020; 67:A02200100."},{"key":"7","doi-asserted-by":"crossref","unstructured":"7. Klug C, Schreiber-Katz O, Thiele S, Schorling E, Zowe J, Reilich P, Walter MC, Nagels KH. Disease burden of spinal muscular atrophy in Germany. Orphanet J Rare Dis. 2016; 11:58.","DOI":"10.1186\/s13023-016-0424-0"},{"key":"8","doi-asserted-by":"crossref","unstructured":"8. Yao M, Ma Y, Qian R, Xia Y, Yuan C, Bai G, Mao S. Quality of life of children with spinal muscular atrophy and their caregivers from the perspective of caregivers: a Chinese cross-sectional study. Orphanet J Rare Dis. 2021; 16:7.","DOI":"10.1186\/s13023-020-01638-8"},{"key":"9","doi-asserted-by":"crossref","unstructured":"9. Iannaccone ST, Hynan LS, Morton A, Buchanan R, Limbers CA, Varni JW; AmSMART Group. The PedsQL in pediatric patients with Spinal Muscular Atrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory Generic Core Scales and Neuromuscular Module. Neuromuscul Disord. 2009; 19:805-812.","DOI":"10.1016\/j.nmd.2009.09.009"},{"key":"10","doi-asserted-by":"crossref","unstructured":"10. Hu J, Jiang L, Hong S, Cheng L, Kong M, Ye Y. Reliability and validity of the Chinese version of the Pediatric Quality Of Life InventoryTM (PedsQLTM) 3.0 neuromuscular module in children with Duchenne muscular dystrophy. Health Qual Life Outcomes. 2013; 11:47.","DOI":"10.1186\/1477-7525-11-47"},{"key":"11","doi-asserted-by":"crossref","unstructured":"11. Mah JK, Thannhauser JE, Kolski H, Dewey D. Parental stress and quality of life in children with neuromuscular disease. Pediatr Neurol. 2008; 39:102-107.","DOI":"10.1016\/j.pediatrneurol.2008.04.011"},{"key":"12","doi-asserted-by":"crossref","unstructured":"12. Varni JW, Limbers CA, Neighbors K, Schulz K, Lieu JE, Heffer RW, Tuzinkiewicz K, Mangione-Smith R, Zimmerman JJ, Alonso EM. The PedsQL\u2122 Infant Scales: feasibility, internal consistency reliability, and validity in healthy and ill infants. Qual Life Res. 2011; 20:45-55.","DOI":"10.1007\/s11136-010-9730-5"},{"key":"13","doi-asserted-by":"crossref","unstructured":"13. Weaver MS, Hanna R, Hetzel S, Patterson K, Yuroff A, Sund S, Schultz M, Schroth M, Halanski MA. A prospective, crossover survey study of child- and proxy-reported quality of life according to apinal muscular atrophy type and medical interventions. J Child Neurol.<b> <\/b>2020; 35:322-330.","DOI":"10.1177\/0883073819900463"},{"key":"14","doi-asserted-by":"crossref","unstructured":"14. Vega P, Glisser C, Castiglioni C, Am\u00e9zquita MV, Quirola M, Barja S. Quality of life in children and adolescents with Spinal Muscular Atrophy. Rev Chil Pediatr. 2020; 91:512-520.","DOI":"10.32641\/rchped.v91i4.1443"},{"key":"15","doi-asserted-by":"crossref","unstructured":"15. Mix L, Winter B, Wurster CD, Platen S, Witzel S, Uzelac Z, Graf H, Ludolph AC, Lul\u00e9 D. Quality of life in SMA patients under treatment with nusinersen. Front Neurol. 2021; 12:626787.","DOI":"10.3389\/fneur.2021.626787"},{"key":"16","doi-asserted-by":"crossref","unstructured":"16. Farrar MA, Carey KA, Paguinto SG, Chambers G, Kasparian NA. Financial, opportunity and psychosocial costs of spinal muscular atrophy: an exploratory qualitative analysis of Australian carer perspectives. BMJ Open. 2018; 8:e020907.","DOI":"10.1136\/bmjopen-2017-020907"},{"key":"17","doi-asserted-by":"crossref","unstructured":"17. Callan A, Nallagangul TK, Jawla S, de Pommerol HJ, Risson V. Neurology. 2019; 92 (15 Supplement): P4.4- 016.","DOI":"10.1212\/WNL.92.15_supplement.P4.4-016"}],"container-title":["Intractable &amp; Rare Diseases Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/irdr\/11\/4\/11_2022.01094\/_pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,10,9]],"date-time":"2024-10-09T15:55:26Z","timestamp":1728489326000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/irdr\/11\/4\/11_2022.01094\/_article"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,11,30]]},"references-count":17,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2022]]}},"URL":"https:\/\/doi.org\/10.5582\/irdr.2022.01094","relation":{},"ISSN":["2186-361X","2186-3644"],"issn-type":[{"value":"2186-361X","type":"electronic"},{"value":"2186-3644","type":"print"}],"subject":[],"published":{"date-parts":[[2022,11,30]]},"article-number":"2022.01094"}}