{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,21]],"date-time":"2026-08-21T13:05:14Z","timestamp":1787317514867,"version":"build-2736575974"},"reference-count":88,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T00:00:00Z","timestamp":1777507200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T00:00:00Z","timestamp":1777507200000},"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":["Develop Med Child Neuro"],"abstract":"<jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To review barriers to ethical and equitable access to disease\u2010modifying therapies (DMTs) and newborn screening (NBS) for spinal muscular atrophy (SMA).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Method<\/jats:title>\n                    <jats:p>We searched PubMed, Scopus, Web of Science, EBSCOhost, the Cochrane Library, Google Scholar, and Primo for content on the ethics or equity of access to SMA DMTs or NBS from January 2014 to May 2025. Only evidence sources that had a primary focus on equity or ethics regarding access to either DMTs or NBS for children aged less than 18\u2009years were eligible.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Forty publications were included: all but one were published in English; three\u2010quarters were published after 2020 and were either narrative reviews (45%) or cross\u2010sectional studies (35%). The largest proportion were from North America (40%) and only three (7%) offered commentary on low\u2010 and middle\u2010income countries. Inequity in access to SMA DMTs and NBS were consistently reported to persist even within countries due to multiple factors, which include variation in health system resources and funding mechanisms. Lack of robust clinical data on the long\u2010term safety, efficacy, and cost\u2010effectiveness of DMTs hampers advocacy efforts, particularly in resource\u2010constrained settings. Questions remain around autonomy and distributive justice in diagnosis and care for this ultra\u2010rare disease.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation<\/jats:title>\n                    <jats:p>A decade after the approval of clinically transformative therapies, children with SMA continue to fall on the wrong side of an avoidable divide. Urgent and coordinated action is needed to establish regulated minimum clinical standards, generate robust real\u2010world data, and redesign funding models to ensure that NBS and treatment innovation serves all populations, not only those with politico\u2010economic advantage.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/dmcn.70297","type":"journal-article","created":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T18:08:40Z","timestamp":1777572520000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Ethics and equity in access to disease\u2010modifying therapies and newborn screening for spinal muscular atrophy: A scoping review"],"prefix":"10.1111","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4075-5072","authenticated-orcid":false,"given":"Serini","family":"Murugasen","sequence":"first","affiliation":[{"name":"Division of Paediatric Neurology, Department of Paediatrics and Child Health Red Cross War Memorial Children's Hospital  Cape Town South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariana","family":"Kruger","sequence":"additional","affiliation":[{"name":"Department of Paediatrics and Child Health Faculty of Medicine and Health Sciences, Stellenbosch University  Stellenbosch Western Cape South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc","family":"Blockman","sequence":"additional","affiliation":[{"name":"Division of Clinical Pharmacology, Department of Medicine, Faculty of Health Sciences University of Cape Town  Cape Town South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7328-1796","authenticated-orcid":false,"given":"Jo M.","family":"Wilmshurst","sequence":"additional","affiliation":[{"name":"Division of Paediatric Neurology, Department of Paediatrics and Child Health Red Cross War Memorial Children's Hospital  Cape Town South Africa"},{"name":"Neuroscience Institute University of Cape Town  Cape Town South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,4,30]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750\u20101172\u20106\u201071"},{"issue":"5","key":"e_1_2_9_3_1","doi-asserted-by":"crossref","first-page":"1111","DOI":"10.1016\/S1097-2765(01)00244-1","article-title":"SMN, the product of the spinal muscular atrophy gene, binds preferentially to dimethylarginine\u2010containing protein targets","volume":"7","author":"Friesen WJ","year":"2001","journal-title":"Mol Cell."},{"issue":"24","key":"e_1_2_9_4_1","doi-asserted-by":"crossref","first-page":"13312","DOI":"10.1073\/pnas.230364197","article-title":"Survival motor neuron protein modulates neuron\u2010specific apoptosis","volume":"97","author":"Kerr DA","year":"2000","journal-title":"Proc Natl Acad Sci U S A."},{"key":"e_1_2_9_5_1","first-page":"7","article-title":"Copy Number Variations in the Survival Motor Neuron Genes: Implications for Spinal Muscular Atrophy and Other Neurodegenerative Diseases","volume":"3","author":"Butchbach MER","year":"2016","journal-title":"Front Mol Biosci."},{"issue":"7","key":"e_1_2_9_6_1","article-title":"Spinal Muscular Atrophy Treatment in Patients Identified by Newborn Screening\u2014A","volume":"14","author":"Aragon\u2010Gawinska K","year":"2023","journal-title":"Systematic Review. 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