{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T15:45:03Z","timestamp":1790523903304,"version":"4.1.0"},"reference-count":29,"publisher":"SAGE Publications","issue":"10","license":[{"start":{"date-parts":[[2020,3,23]],"date-time":"2020-03-23T00:00:00Z","timestamp":1584921600000},"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":["Ann Pharmacother"],"published-print":{"date-parts":[[2020,10]]},"abstract":"<jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>To review the efficacy and safety of onasemnogene abeparvovec-xioi (Zolgensma) in the treatment of spinal muscular atrophy (SMA).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Data Sources:<\/jats:title>\n                    <jats:p>An English-language literature search of PubMed, MEDLINE, and Ovid (1946 to December 2019) was completed using the terms onasemnogene, AVXS-101, and spinal muscular atrophy. Manufacturer prescribing information, article bibliographies, and data from ClinicalTrials.gov were incorporated in the reviewed data.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Study Selection\/Data Extraction:<\/jats:title>\n                    <jats:p>All studies registered on ClinicalTrials.gov were incorporated in the reviewed data.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Data Synthesis:<\/jats:title>\n                    <jats:p>Onasemnogene is the first agent for SMA utilizing gene therapy to directly provide survival motor neuron 1 ( SMN1) gene to produce SMN protein. Four publications of 1 clinical trial, 1 comparison study of treatment effects, and 1 combination therapy case series have been published.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Relevance to Patient Care and Clinical Practice:<\/jats:title>\n                    <jats:p>Onasemnogene is a one time dose approved by the Food and Drug Administration for SMA patients &lt;2 years old who possess mutations in both copies of the SMN1 gene.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Onasemnogene appears to be an efficacious therapy for younger pediatric patients with SMA type 1. Concerns include drug cost and potential liver toxicity. Long-term benefits and risks have not been determined.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/1060028020914274","type":"journal-article","created":{"date-parts":[[2020,3,24]],"date-time":"2020-03-24T01:04:37Z","timestamp":1585011877000},"page":"1001-1009","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":73,"title":["Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy"],"prefix":"10.1177","volume":"54","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1655-5049","authenticated-orcid":false,"given":"Debra","family":"Stevens","sequence":"first","affiliation":[{"name":"Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie K.","family":"Claborn","sequence":"additional","affiliation":[{"name":"Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brooke L.","family":"Gildon","sequence":"additional","affiliation":[{"name":"Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tiffany L.","family":"Kessler","sequence":"additional","affiliation":[{"name":"Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4979-1873","authenticated-orcid":false,"given":"Cheri","family":"Walker","sequence":"additional","affiliation":[{"name":"Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2020,3,23]]},"reference":[{"key":"bibr1-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60921-6"},{"key":"bibr2-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-6-71"},{"key":"bibr3-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.005"},{"key":"bibr4-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2019.06.007"},{"key":"bibr5-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1177\/1060028018789956"},{"key":"bibr6-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"bibr7-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2019.05.005"},{"key":"bibr8-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.24203"},{"key":"bibr9-1060028020914274","doi-asserted-by":"publisher","DOI":"10.3233\/JND-190403"},{"key":"bibr10-1060028020914274","unstructured":"Spinraza [package insert]. Cambridge, MA: Biogen Inc; 2017."},{"key":"bibr11-1060028020914274","unstructured":"Zolgensma [package insert]. Bannockburn, IL: AveXis Inc; 2019."},{"key":"bibr12-1060028020914274","unstructured":"National Institutes of Health, US National Library of Medicine. Search results: Zolgensma. https:\/\/clinicaltrials.gov\/ct2\/results?cond=&term=zolgensma&cntry=&state=&city=&dist=. Accessed December 17, 2019."},{"key":"bibr13-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-019-01162-5"},{"key":"bibr14-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1007\/s12325-019-00923-8"},{"key":"bibr15-1060028020914274","unstructured":"US Food and Drug Administration. ZOLGENSMA\u00ae (onasemnogene abeparvovec-xioi): summary basis for regulatory action. https:\/\/www.fda.gov\/vaccines-blood-biologics\/zolgensma. Published May 24, 2019. Accessed December 18, 2019."},{"key":"bibr16-1060028020914274","unstructured":"US Department of Health and Human Services. Common terminology criteria for adverse events. Version 5.0. https:\/\/ctep.cancer.gov\/protocoldevelopment\/electronic_applications\/docs\/CTCAE_v5_Quick_Reference_8.5x11.pdf. Published November 27, 2017. Accessed December 5, 2019."},{"key":"bibr17-1060028020914274","unstructured":"US Food and Drug Administration. Drug-induced liver injury: premarketing clinical evaluation. https:\/\/www.fda.gov\/downloads\/Guidances\/UCM174090.pdf. Accessed December 5, 2019."},{"key":"bibr18-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.11.014"},{"key":"bibr19-1060028020914274","volume-title":"Bayley Scales of Infant and Toddler Development","author":"Bayley N.","year":"2006","edition":"3"},{"key":"bibr20-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1177\/15648265040251S106"},{"key":"bibr21-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21838"},{"key":"bibr22-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.283"},{"key":"bibr23-1060028020914274","doi-asserted-by":"publisher","DOI":"10.3233\/JND-180304"},{"key":"bibr24-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"bibr25-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1007\/s12325-019-01087-1"},{"key":"bibr26-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000008207"},{"key":"bibr27-1060028020914274","unstructured":"Institute for Clinical and Economic Review. Spinraza\u00ae and Zolgensma\u00ae for spinal muscular atrophy: effectiveness and value: final evidence report. https:\/\/icer-review.org\/wp-content\/uploads\/2018\/07\/ICER_SMA_Final_Evidence_Report_052419.pdf. Published April 3, 2019. Accessed December 16, 2019."},{"key":"bibr28-1060028020914274","doi-asserted-by":"publisher","DOI":"10.1080\/20016689.2019.1601484"},{"key":"bibr29-1060028020914274","unstructured":"Novartis. AveXis announces innovative Zolgensma\u00ae gene therapy access programs for US payers and families. https:\/\/www.novartis.com\/news\/media-releases\/avexis-announces-innovative-zolgensma-gene-therapy-access-programs-us-payers-and-families. Published May 24, 2019. 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