{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T04:31:08Z","timestamp":1790742668381,"version":"4.1.0"},"reference-count":35,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2025,5,21]],"date-time":"2025-05-21T00:00:00Z","timestamp":1747785600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2025,5,21]],"date-time":"2025-05-21T00:00:00Z","timestamp":1747785600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2025,9]]},"abstract":"<jats:sec>\n            <jats:title>Background:<\/jats:title>\n            <jats:p>Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease associated with a significant burden of illness to both patients and caregivers; however, there is little evidence available regarding how patients and caregivers evaluate potential treatment benefit\u2013risk profiles. As access to SMA disease-modifying therapies increases, it is imperative to understand which treatment attributes drive treatment choices.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Objective:<\/jats:title>\n            <jats:p>To identify which treatment attributes drive treatment choices in adults with SMA and caregivers of children with SMA across nine countries in Latin America.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Methods:<\/jats:title>\n            <jats:p>A discrete choice experiment (DCE) survey was developed for market research using data collected via qualitative interviews and consultations with medical experts. Adults with Type 2\/3 SMA and caregivers of children with Types 1\u20133 SMA were recruited by patient advisory groups and physician referrals. Respondents completed a 30-min, online survey that collected patient demographics, disease-specific information, and quality of life data (via the EQ-5D-5L), and included the DCE, in which respondents were asked to choose between 14 sets of hypothetical treatment profiles. Data were pooled for analysis, as the country-level sample sizes were small. Raw data were aggregated in Microsoft Excel. Statistical testing was performed using data tables and SPSS (as appropriate). Demographic data were summarized descriptively.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Results:<\/jats:title>\n            <jats:p>A total of 143 respondents (45 adults with SMA and 98 caregivers) completed the online survey. Most respondents were from Argentina (35.0%) or Brazil (19.6%). Breathing function outcome was the most important treatment attribute for caregivers, while adults with SMA placed greater importance on motor function outcome. Both adults with SMA and caregivers placed the greatest importance on improvements in function compared with worse or stable outcomes.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Conclusions:<\/jats:title>\n            <jats:p>Understanding treatment attribute preferences at a regional level will improve shared medical decision-making for individuals with SMA.<\/jats:p>\n          <\/jats:sec>","DOI":"10.1177\/22143602251320267","type":"journal-article","created":{"date-parts":[[2025,5,21]],"date-time":"2025-05-21T06:34:36Z","timestamp":1747809276000},"page":"648-661","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Patient and caregiver spinal muscular atrophy treatment attribute preferences in Latin America"],"prefix":"10.1177","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4392-5885","authenticated-orcid":false,"given":"Victoria","family":"Saenz","sequence":"first","affiliation":[{"name":"Productos Roche SAQeI, Buenos Aires, Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-2144-4618","authenticated-orcid":false,"given":"Marijana","family":"Chlistalla","sequence":"additional","affiliation":[{"name":"F. Hoffmann-La Roche Ltd, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nayara","family":"Carlos","sequence":"additional","affiliation":[{"name":"Produtos Roche Qu\u00edmicos e Farmac\u00eauticos S\/A, S\u00e3o Paulo, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1981-7550","authenticated-orcid":false,"given":"Claudia","family":"Castiglioni","sequence":"additional","affiliation":[{"name":"Department of Neurology, Clinica Meds, School of Medicine, Universidad Finis Terrae, Santiago, Chile"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Soledad Monges","sequence":"additional","affiliation":[{"name":"Hospital Pediatria J.P. Garrahan, Buenos Aires, Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9270-4061","authenticated-orcid":false,"given":"Laurent","family":"Servais","sequence":"additional","affiliation":[{"name":"MDUK Oxford Neuromuscular Centre &amp; NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK"},{"name":"Division of Child Neurology, Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Li\u00e8ge and University of Li\u00e8ge, Li\u00e8ge, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4991-6760","authenticated-orcid":false,"given":"Edmar","family":"Zanoteli","sequence":"additional","affiliation":[{"name":"Department of Neurology, Medical School of the University of S\u00e3o Paulo (FMUSP), S\u00e3o Paulo, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,5,21]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ncl.2015.07.004"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-022-00380-8"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01695-7"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.15286"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1007\/s40273-022-01197-9"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.36469\/63185"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01667-3"},{"key":"e_1_3_5_9_2","first-page":"161865","article-title":"Disease modifying therapies for the management of children with spinal muscular atrophy (5q SMA): an update on the emerging evidence","author":"Hjartarson HT","year":"2022","unstructured":"Hjartarson HT, Nathorst-Boos K, Sejersen T. 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