{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,18]],"date-time":"2026-06-18T07:05:38Z","timestamp":1781766338221,"version":"3.54.5"},"reference-count":14,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2024,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>In 2016, nusinersen became the first disease\u2010modifying medication approved by the U.S. Food and Drug Administration (FDA) for spinal muscular atrophy (SMA). With the later availability of risdiplam in 2020, individuals now have the option of switching from nusinersen to risdiplam. Limited published data exist to inform this decision. This study aims to evaluate the perceptions and experiences of adult participants and parents of minor participants who previously received nusinersen and switched to risdiplam for the treatment of SMA.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Institutional Review Board (IRB) approval was obtained from the Wake Forest IRB prior to the initiation of this study. A cross\u2010sectional, observational study, with qualitative and quantitative data gathered via questionnaire and medical record review, was performed. Inclusion criteria included (1) prior diagnosis of SMA, (2) previous treatment with nusinersen, and (3) change to treatment with risdiplam. No participants were excluded based on age.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Fourteen participants\u2014eight adults and six children\u2014were enrolled in the study. Respondents noted improvements in physical function with each medication. Overall, respondents reported worse satisfaction with the method of delivery of the intrathecally delivered nusinersen compared to the orally\u2010delivered risdiplam, but no respondent reported negative overall satisfaction with either medication. A majority (78.6%) of respondents reported that switching from nusinersen to risdiplam was the correct decision.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>These results suggest that most patients are satisfied when switching from nusinersen to risdiplam, with the method of delivery being a primary factor.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.28015","type":"journal-article","created":{"date-parts":[[2023,12,2]],"date-time":"2023-12-02T12:05:57Z","timestamp":1701518757000},"page":"179-184","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["A case series evaluating patient perceptions after switching from nusinersen to risdiplam for spinal muscular atrophy"],"prefix":"10.1002","volume":"69","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6403-0028","authenticated-orcid":false,"given":"Jeffrey C.","family":"Powell","sequence":"first","affiliation":[{"name":"Department of Neurology Wake Forest School of Medicine  Winston\u2010Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2725-1825","authenticated-orcid":false,"given":"James B.","family":"Meiling","sequence":"additional","affiliation":[{"name":"Department of Physical Medicine and Rehabilitation Mayo Clinic  Rochester Minnesota USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2026-6511","authenticated-orcid":false,"given":"Michael S.","family":"Cartwright","sequence":"additional","affiliation":[{"name":"Department of Neurology Wake Forest School of Medicine  Winston\u2010Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,12]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27358"},{"key":"e_1_2_12_3_1","unstructured":"TallasAR.Quality of life of children with spinal muscular atrophy: parents\u2019 perspectives in light of new treatments (Masters Thesis). Updated August 12 2020. Accessed April 3 2023 https:\/\/scholarcommons.sc.edu\/etd\/5747"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010020\u201001477\u20107"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.3233\/jnd\u2010180330"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.3390\/children8070604"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.3233\/jnd\u2010200563"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2021.02.014"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27116"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/ene.14902"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1177\/1756286421998902"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pmrj.2013.12.005"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.3171\/foc.2006.21.2.7"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1097\/aap.0000000000000251"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/ner.13167"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.28015","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,22]],"date-time":"2024-01-22T04:53:04Z","timestamp":1705899184000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.28015"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12]]},"references-count":14,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2024,2]]}},"alternative-id":["10.1002\/mus.28015"],"URL":"https:\/\/doi.org\/10.1002\/mus.28015","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,12]]},"assertion":[{"value":"2023-05-12","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-11-21","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-12-01","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}