{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,3]],"date-time":"2026-05-03T10:34:27Z","timestamp":1777804467539,"version":"3.51.4"},"reference-count":28,"publisher":"SAGE Publications","issue":"1","license":[{"start":{"date-parts":[[2021,11,9]],"date-time":"2021-11-09T00:00:00Z","timestamp":1636416000000},"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":["J Child Neurol"],"published-print":{"date-parts":[[2022,1]]},"abstract":"<jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>To identify factors parents considered in treatment decision making for children diagnosed with spinal muscular atrophy on newborn screening.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Participants were recruited through the University of Rochester or through flyers and Cure SMA social media outreach and asked to complete a telephone or online survey. Data were analyzed through mixed methods using descriptive statistics and theme identification in narrative responses.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Eighteen parents with children diagnosed with spinal muscular atrophy on newborn screening participated. Thirteen of 18 chose onasemnogene abeparvovec, 2 of 18 chose risdiplam, 1 of 18 chose nusinersen, and 2 of 18\u00a0did not receive treatment. The most commonly reported factors impacting treatment choice included treatment frequency and administration method. Seventeen (94.4%) parents felt that inclusion of spinal muscular atrophy on newborn screening was positive because it could allow for better outcomes with earlier treatment.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Treatment frequency and administration method were the most important factors for parents in determining spinal muscular atrophy treatment. Parents felt positively about newborn screening due to opportunity for earlier treatment.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/08830738211040292","type":"journal-article","created":{"date-parts":[[2021,11,9]],"date-time":"2021-11-09T22:17:10Z","timestamp":1636496230000},"page":"43-49","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":15,"title":["Parent Perceptions in Choosing Treatment for Infants With Spinal Muscular Atrophy Diagnosed Through Newborn Screening"],"prefix":"10.1177","volume":"37","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3265-6365","authenticated-orcid":false,"given":"Stella","family":"Deng","sequence":"first","affiliation":[{"name":"Department of Neurology, University of Rochester, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bo Hoon","family":"Lee","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of Rochester, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Emma","family":"Ciafaloni","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of Rochester, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2021,11,9]]},"reference":[{"key":"bibr1-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000290830.40544.b9"},{"key":"bibr2-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"bibr3-08830738211040292","doi-asserted-by":"publisher","DOI":"10.17265\/2159-5313\/2016.09.003"},{"key":"bibr4-08830738211040292","unstructured":"ZOLGENSMA (onasemnogene abeparvovec-xioi). Bannockburn, IL: AveXis, 2019. Revised May 2019. Accessed January 12, 2021. https:\/\/www.avexis.com\/us\/Content\/pdf\/prescribing_information.pdf"},{"key":"bibr5-08830738211040292","unstructured":"EVRYSDI (risdiplam). San Francisco, CA: Genentech, Inc. 2020. Revised August 2020. Accessed January 12, 2021. https:\/\/www.gene.com\/download\/pdf\/evrysdi_prescribing.pdf"},{"key":"bibr6-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"bibr7-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1710504"},{"key":"bibr8-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"bibr9-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2009965"},{"key":"bibr10-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1038\/s41436-020-0824-3"},{"key":"bibr11-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2017.152"},{"key":"bibr12-08830738211040292","unstructured":"Health and Resources and Services Administration, Maternal & Child Health. Newborn screening for spinal muscular atrophy: A summary of the evidence and advisory committee decision (2018)."},{"key":"bibr13-08830738211040292","doi-asserted-by":"publisher","DOI":"10.3233\/JND-180304"},{"key":"bibr14-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24864"},{"issue":"5","key":"bibr15-08830738211040292","first-page":"704","volume":"57","author":"Swoboda KJ","year":"2005","journal-title":"SMN2 copy number, and function. Ann Neurol"},{"key":"bibr16-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2019.09.007"},{"key":"bibr17-08830738211040292","volume-title":"Poster presented at: Annual Muscular Dystrophy Association Clinical and Scientific Conference","author":"Strauss KA"},{"key":"bibr18-08830738211040292","unstructured":"ClinicalTrials.gov [internet]. 2018 December 19. Identifier NCT03779334. A study of risdiplam in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (Rainbowfish). Available at: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03779334. Published February 9, 2021. Accessed March 1, 2021."},{"key":"bibr19-08830738211040292","volume-title":"Poster presented at: the Annual Muscular Dystrophy Association Clinical and Scientific Conference","author":"Darras BT"},{"key":"bibr20-08830738211040292","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S101821"},{"key":"bibr21-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1177\/0883073807305672"},{"key":"bibr22-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1186\/s12883-015-0473-3"},{"key":"bibr23-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.35756"},{"key":"bibr24-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1038\/ejhg.2014.147"},{"key":"bibr25-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24100"},{"key":"bibr26-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.38220"},{"key":"bibr27-08830738211040292","unstructured":"Kariyawasam DST, D\u2019Silva AM, Vetsch J, Wakefield C, Wiley V, Farrar MA. \u201cWe needed this\u201d: perspectives of parents and healthcare professionals involved in a pilot newborn screening program for spinal muscular atrophy. SSRN. https:\/\/ssrncom\/abstract=3682109."},{"key":"bibr28-08830738211040292","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.38031"}],"container-title":["Journal of Child Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/08830738211040292","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/08830738211040292","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/08830738211040292","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T20:40:13Z","timestamp":1777495213000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/08830738211040292"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,11,9]]},"references-count":28,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2022,1]]}},"alternative-id":["10.1177\/08830738211040292"],"URL":"https:\/\/doi.org\/10.1177\/08830738211040292","relation":{},"ISSN":["0883-0738","1708-8283"],"issn-type":[{"value":"0883-0738","type":"print"},{"value":"1708-8283","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,11,9]]}}}