{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T21:32:11Z","timestamp":1772227931765,"version":"3.50.1"},"reference-count":24,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2020,12,19]],"date-time":"2020-12-19T00:00:00Z","timestamp":1608336000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2021,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>This study aimed to examine the early experience of nusinersen for spinal muscular atrophy (SMA) from the patient and caregiver perspective.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A 54\u2010item online survey was administered to adult patients and caregivers of pediatric patients diagnosed with SMA.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Overall, respondents (56 patients and 45 caregivers) were satisfied with nusinersen. Satisfaction was highest on changes in energy, stamina, and motor function and lowest on treatment administration and overall time commitment. Differences were noted for treatment effect sustained over time as reported by adult patients vs caregivers reporting on behalf of pediatric patients. Respondents reported insurance approval as a key barrier to access, particularly among adult patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Despite therapeutic advances, there remain significant unmet needs for SMA. Challenges with administration and barriers to access potentially limit the number of patients treated or delay treatment. Continued efforts are needed to develop more treatment options and to improve access to treatments.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27116","type":"journal-article","created":{"date-parts":[[2020,11,13]],"date-time":"2020-11-13T05:31:03Z","timestamp":1605245463000},"page":"311-319","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers"],"prefix":"10.1002","volume":"63","author":[{"given":"Er","family":"Chen","sequence":"first","affiliation":[{"name":"Genentech Inc San Francisco California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stacy","family":"Dixon","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Colorado School of Medicine Aurora Colorado USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rupali","family":"Naik","sequence":"additional","affiliation":[{"name":"Noesis Healthcare Technologies, Inc San Francisco California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Josh M.","family":"Noone","sequence":"additional","affiliation":[{"name":"Ipsos Healthcare New York New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8247-0342","authenticated-orcid":false,"given":"J. Daniel","family":"Buchenberger","sequence":"additional","affiliation":[{"name":"Ipsos Healthcare New York New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sarah M.","family":"Whitmire","sequence":"additional","affiliation":[{"name":"Ipsos Healthcare New York New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rosalina","family":"Mills","sequence":"additional","affiliation":[{"name":"Ipsos Healthcare New York New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"William","family":"Arnold","sequence":"additional","affiliation":[{"name":"Department of Neurology The Ohio State University Columbus Ohio USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2020,12,19]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0724-z"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.004"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.005"},{"key":"e_1_2_9_5_1","unstructured":"Food and Drug Administration.FDA approves first drug for spinal muscular atrophy.2016.https:\/\/www.fda.gov\/newsevents\/newsroom\/pressannouncements\/ucm534611.htm. Accessed June 20 2019."},{"key":"e_1_2_9_6_1","unstructured":"Cure SMA.AveXis files for FDA approval of gene therapy for spinal muscular atrophy type I.2018.http:\/\/www.curesma.org\/news\/avexis-fda-approval-type-i.htmlAccessed June 20 2019."},{"key":"e_1_2_9_7_1","unstructured":"Novartis.AveXis receives FDA approval for Zolgensma\u00ae the first and only gene therapy for pediatric patients with spinal muscular atrophy(SMA).2019.https:\/\/www.novartis.com\/news\/media-releases\/avexis-receives-fda-approval-zolgensma-first-and-only-gene-therapy-pediatric-patients-spinal-muscular-atrophy-sma. Accessed June 25 2019."},{"key":"e_1_2_9_8_1","unstructured":"CureSMA.The Voice of the Patient Report for Spinal Muscular Atrophy2018."},{"key":"e_1_2_9_9_1","volume-title":"Clinical Experience of Spinal Muscular Atrophy (SMA) Treatment: A Combination of Perspectives from a Large Survey. 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