{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,13]],"date-time":"2026-09-13T11:13:40Z","timestamp":1789298020087,"version":"build-2803163510"},"reference-count":79,"publisher":"Informa UK Limited","issue":"6","funder":[{"name":"Roche Colombia"}],"content-domain":{"domain":["www.tandfonline.com"],"crossmark-restriction":true},"short-container-title":["Expert Review of Pharmacoeconomics &amp; Outcomes Research"],"published-print":{"date-parts":[[2023,7,3]]},"DOI":"10.1080\/14737167.2023.2206569","type":"journal-article","created":{"date-parts":[[2023,4,25]],"date-time":"2023-04-25T08:33:48Z","timestamp":1682411628000},"page":"671-682","update-policy":"https:\/\/doi.org\/10.1080\/tandf_crossmark_01","source":"Crossref","is-referenced-by-count":4,"title":["Burden of disease of spinal muscular atrophy linked to chromosome 5q (5q-SMA) in Colombia"],"prefix":"10.1080","volume":"23","author":[{"given":"Yaneth","family":"Gil-Rojas","sequence":"first","affiliation":[{"name":"Real World Insights (RWI), IQVIA, Bogot\u00e1, Colombia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fernando","family":"Su\u00e1rez-Obando","sequence":"additional","affiliation":[{"name":"Instituto de Gen\u00e9tica Humana, School of Medicine, Pontificia Universidad Javeriana, Bogot\u00e1, Colombia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Devi","family":"Amaya-Granados","sequence":"additional","affiliation":[{"name":"Real World Insights (RWI), IQVIA, Bogot\u00e1, Colombia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Prieto-Pinto","sequence":"additional","affiliation":[{"name":"Evidence Generation, Roche, Bogot\u00e1, Colombia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Samac\u00e1-Samac\u00e1","sequence":"additional","affiliation":[{"name":"Real World Insights (RWI), IQVIA, Bogot\u00e1, Colombia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Blair","family":"Ortiz","sequence":"additional","affiliation":[{"name":"Universidad de Antioquia, Hospital San Vicente Fundaci\u00f3n, Medell\u00edn, Colombia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fabi\u00e1n","family":"Hern\u00e1ndez","sequence":"additional","affiliation":[{"name":"Real World Insights (RWI), IQVIA, Bogot\u00e1, Colombia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"301","published-online":{"date-parts":[[2023,4,28]]},"reference":[{"key":"cit0001","unstructured":"European Medicine Agency (EMA). Briefing document to the clinical trial readiness in Spinal Muscular Atrophy (SMA) SMA Europe, TREAT-NMD and European Medicines Agency Meeting. 2016 [cited 1-59 p]. https:\/\/www.ema.europa.eu\/en\/documents\/other\/briefing-document-clinical-trial-readiness-spinal-muscular-atrophy-sma-sma-europe-treat-nmd-european_en.pdf"},{"key":"cit0002","first-page":"1","volume":"29","author":"Bodamer O.","year":"2022","journal-title":"UpToDate"},{"key":"cit0003","unstructured":"Cure SMA. Best practices for physical therapists & clinical evaluators in spinal muscular atrophy (SMA). Recommendations to support the effective conduct of clinical trial in SMA. Families of Spinal Muscular Atrophy; 2021. https:\/\/www.curesma.org\/wp-content\/uploads\/2021\/09\/Clinical-Evaluators-Best-Practices-13-August-2021.pdf"},{"key":"cit0004","doi-asserted-by":"publisher","DOI":"10.1016\/j.ncl.2015.07.004"},{"key":"cit0005","doi-asserted-by":"publisher","DOI":"10.1515\/medgen-2020-2033"},{"key":"cit0006","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.005"},{"key":"cit0007","doi-asserted-by":"publisher","DOI":"10.1177\/08830738030180030201"},{"key":"cit0008","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000172860.41953.12"},{"key":"cit0009","doi-asserted-by":"publisher","DOI":"10.1016\/j.braindev.2020.05.004"},{"key":"cit0010","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awp236"},{"key":"cit0011","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-017-8549-1"},{"key":"cit0012","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000290830.40544.b9"},{"key":"cit0013","doi-asserted-by":"publisher","DOI":"10.1177\/0883073811420152"},{"key":"cit0014","doi-asserted-by":"publisher","DOI":"10.3233\/JND-170292"},{"key":"cit0015","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000009248"},{"key":"cit0016","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-021-00307-7"},{"key":"cit0017","doi-asserted-by":"publisher","DOI":"10.46531\/sinapse\/AO\/210058\/2021"},{"key":"cit0018","doi-asserted-by":"publisher","DOI":"10.1007\/s40273-021-01105-7"},{"key":"cit0019","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01695-7"},{"key":"cit0020","doi-asserted-by":"publisher","DOI":"10.22379\/2422402270"},{"key":"cit0021","unstructured":"Bola\u00f1os Camacho XE Caracterizaci\u00f3n funcional de pacientes con Atrofia Muscular Espinal valorados en instituto de rehabilitaci\u00f3n de Bogot\u00e1, Colombia, entre los a\u00f1os 2009 a 2019. 2020."},{"key":"cit0022","doi-asserted-by":"publisher","DOI":"10.7705\/biomedica.6178"},{"key":"cit0023","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2021.03.007"},{"key":"cit0024","unstructured":"Choconta L, Cortes A, Pardo N, et al. Lineamientos para el desarrollo de una estrategia de uso adecuado de Nusinersen en Atrofia Muscular Espinal (incluyendo el perfil de seguridad). Instituto de Evaluaci\u00f3n Tecnol\u00f3gica en Salud; 2019."},{"key":"cit0025","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2022.09.643"},{"key":"cit0026","unstructured":"World Health Organization (WHO). In: WHO methods and data sources for global burden of disease estimates 2000-2019. Geneva: World Health Organization; 2020."},{"key":"cit0027","doi-asserted-by":"publisher","DOI":"10.1016\/S0960-8966(06)80015-5"},{"key":"cit0028","doi-asserted-by":"publisher","DOI":"10.1016\/S1090-3798(99)80012-9"},{"key":"cit0029","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(95)91835-3"},{"key":"cit0030","unstructured":"Ministerio de Salud y de la Protecci\u00f3n Social (MSPS). Resoluci\u00f3n 2048 de 2015. 2015."},{"key":"cit0031","unstructured":"Departamento Administrativo Nacional de Estad\u00edstica (DANE). Serie Nacional de poblaci\u00f3n por \u00e1rea sexo y edad para el periodo 2005-2070 2021 [Oct 3, 2021]. Available from: https:\/\/www.dane.gov.co\/index.php\/estadisticas-por-tema\/demografia-y-poblacion\/proyecciones-de-poblacion"},{"key":"cit0032","unstructured":"Ministerio de Salud y de la Protecci\u00f3n Social (MSPS). Sistema Integrado de Informaci\u00f3n de la Protecci\u00f3n Social (SISPRO) 2022 [updated Jul 26, 2022]. Available from"},{"key":"cit0033","unstructured":"Bogeda de datos Sispro: Cubo de Registro Nacional de Enfermedades Hu\u00e9rfanas (RNEH) [Internet]. 2022. Available from: https:\/\/www.sispro.gov.co\/Pages\/Home.aspx"},{"key":"cit0034","unstructured":"Instituto Nacional de Salud (INS). Informes de Evento: Enfermedades hu\u00e9rfanas-raras, 2018-2021 2022 [Mar 18, 2022]. Available from: https:\/\/www.ins.gov.co\/buscador-eventos\/Paginas\/Info-Evento.aspx"},{"key":"cit0035","unstructured":"Instituto Nacional de Salud (INS). Protocolo de vigilancia en Salud P\u00fablica de enfermedades hu\u00e9rfanas-raras. Versi\u00f3n 5. 2022 [cited Jun 15, 2022]. Available from: https:\/\/www.ins.gov.co\/buscador-eventos\/Paginas\/Fichas-y-Protocolos.aspx"},{"key":"cit0036","unstructured":"Instituto Nacional de Salud (INS). Anexo Protocolo de vigilancia en Salud P\u00fablica de enfermedades hu\u00e9rfanas-raras. 2022 [cited Jun 15, 2022]. Available from"},{"key":"cit0037","volume-title":"4.5","author":"Rohatgi AW","year":"2021"},{"key":"cit0038","doi-asserted-by":"publisher","DOI":"10.1186\/s12874-021-01308-8"},{"key":"cit0039","unstructured":"Departamento Administrativo Nacional de Estad\u00edstica (DANE). Estimaciones del cambio demogr\u00e1fico: Tablas de vida completas y abreviada por sexo y \u00e1rea a nivel nacional 2018-2070 y departamental 2018-2050 2022 [updated Oct 9, 2020]. Available from: https:\/\/www.dane.gov.co"},{"key":"cit0040","doi-asserted-by":"publisher","DOI":"10.1186\/1478-7954-1-4"},{"key":"cit0041","doi-asserted-by":"publisher","DOI":"10.1177\/2329048X211008725"},{"key":"cit0042","doi-asserted-by":"publisher","DOI":"10.5772\/30752"},{"key":"cit0043","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000010540"},{"key":"cit0044","doi-asserted-by":"publisher","DOI":"10.1016\/j.apmr.2013.01.014"},{"key":"cit0045","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01367-y"},{"key":"cit0046","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.1989.tb04029.x"},{"key":"cit0047","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000007742"},{"key":"cit0048","doi-asserted-by":"publisher","DOI":"10.1111\/ene.14587"},{"key":"cit0049","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.01.019"},{"key":"cit0050","doi-asserted-by":"publisher","DOI":"10.1002\/jbmr.2072"},{"key":"cit0051","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000796"},{"key":"cit0052","unstructured":"Global Burden of Disease Collaborative Network. Global Burden of Disease Study 2019 (GBD 2019) Disability Weights. Seattle, United States of America: Institute for Health Metrics and Evaluation (IHME); 2020 [Jan 30, 2021]. Available from: https:\/\/ghdx.healthdata.org\/record\/ihme-data\/gbd-2019-disability-weights"},{"key":"cit0053","doi-asserted-by":"publisher","DOI":"10.1016\/S2214-109X(15)00069-8"},{"key":"cit0054","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(20)30925-9"},{"key":"cit0055","doi-asserted-by":"publisher","DOI":"10.1186\/s11689-021-09355-4"},{"key":"cit0056","doi-asserted-by":"publisher","DOI":"10.1155\/2019\/6357171"},{"key":"cit0057","doi-asserted-by":"publisher","DOI":"10.1007\/s00402-010-1096-1"},{"issue":"3","key":"cit0058","first-page":"429","volume":"72","author":"Murray CJ","year":"1994","journal-title":"Bull World Health Organ"},{"key":"cit0059","volume-title":"GBD Compare Data Visualization","author":"Institute for Health Metrics and Evaluation (IHME)","year":"2022"},{"key":"cit0060","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30404-6"},{"key":"cit0061","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0210574"},{"key":"cit0062","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-016-0424-0"},{"key":"cit0063","doi-asserted-by":"publisher","DOI":"10.3390\/ijerph17238989"},{"key":"cit0064","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-genom-102319-103602"},{"key":"cit0065","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2004-0668"},{"key":"cit0066","doi-asserted-by":"publisher","DOI":"10.1111\/jpc.12291"},{"key":"cit0067","doi-asserted-by":"publisher","DOI":"10.1159\/000096128"},{"key":"cit0068","doi-asserted-by":"publisher","DOI":"10.3390\/ijns7030045"},{"key":"cit0069","doi-asserted-by":"publisher","DOI":"10.1159\/000275492"},{"key":"cit0070","doi-asserted-by":"publisher","DOI":"10.1016\/j.braindev.2018.07.016"},{"key":"cit0071","doi-asserted-by":"publisher","DOI":"10.1159\/000026221"},{"key":"cit0072","doi-asserted-by":"publisher","DOI":"10.1038\/ejhg.2011.134"},{"key":"cit0073","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0671-8"},{"key":"cit0074","doi-asserted-by":"publisher","DOI":"10.1038\/sj.ejhg.5201821"},{"key":"cit0075","doi-asserted-by":"publisher","DOI":"10.3233\/JND-190466"},{"key":"cit0076","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-019-1125-2"},{"key":"cit0077","unstructured":"(WHO) WHO. ICD-11: International Classification of Diseases 11th Revision 2022 [Jun 24, 2022]. Available from: https:\/\/icd.who.int\/en"},{"key":"cit0078","unstructured":"Bodega de datos Sispro: Cubo de prescripciones Mipres [Internet]. 2022 [cited Jun 21, 2022]. Available from"},{"key":"cit0079","doi-asserted-by":"publisher","DOI":"10.1097\/PCC.0b013e3182388ad1"}],"container-title":["Expert Review of Pharmacoeconomics &amp; Outcomes Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1080\/14737167.2023.2206569","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,6,22]],"date-time":"2023-06-22T05:35:44Z","timestamp":1687412144000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.tandfonline.com\/doi\/full\/10.1080\/14737167.2023.2206569"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,4,28]]},"references-count":79,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2023,7,3]]}},"alternative-id":["10.1080\/14737167.2023.2206569"],"URL":"https:\/\/doi.org\/10.1080\/14737167.2023.2206569","relation":{},"ISSN":["1473-7167","1744-8379"],"issn-type":[{"value":"1473-7167","type":"print"},{"value":"1744-8379","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,4,28]]},"assertion":[{"value":"The publishing and review policy for this title is described in its Aims & Scope.","order":1,"name":"peerreview_statement","label":"Peer Review Statement"},{"value":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ierp20","URL":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ierp20","order":2,"name":"aims_and_scope_url","label":"Aim & Scope"},{"value":"2023-01-10","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-04-17","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-04-28","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}