{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,27]],"date-time":"2026-08-27T23:15:58Z","timestamp":1787872558761,"version":"build-2784847793"},"reference-count":31,"publisher":"SAGE Publications","issue":"4","license":[{"start":{"date-parts":[[2025,11,11]],"date-time":"2025-11-11T00:00:00Z","timestamp":1762819200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2025,11,11]],"date-time":"2025-11-11T00:00:00Z","timestamp":1762819200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"name":"Novartis Pharma AG"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:p>We evaluated the safety and effectiveness of onasemnogene abeparvovec (OA) for 34 patients with spinal muscular atrophy (SMA) and tracheostomies enrolled in the RESTORE registry. Most patients (76.5%) received other SMA treatments before and\/or after OA. Safety findings were consistent with OA's established profile. There were four fatalities, three caused by respiratory events unrelated to OA, one of unknown cause. Overall, patients exhibited positive motor outcomes, even in cases where the need for tracheostomy emerged after treatment. These real-world data support OA treatment for patients with SMA and tracheostomies and can inform future access, treatment, and care decisions.<\/jats:p>\n                  <jats:p>\n                    <jats:bold>Trial registration<\/jats:bold>\n                    : Clinicaltrials.gov NCT identifier NCT04174157, registration date September 12, 2019.\n                  <\/jats:p>","DOI":"10.1177\/22143602251395173","type":"journal-article","created":{"date-parts":[[2025,11,11]],"date-time":"2025-11-11T17:13:26Z","timestamp":1762881206000},"page":"788-794","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry"],"prefix":"10.1177","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0187-0580","authenticated-orcid":false,"given":"Yasemin","family":"Erba\u015f","sequence":"first","affiliation":[{"name":"SMA Europe"},{"name":"Spierziekten Vlaanderen, Borgerhout, Belgium"},{"name":"Tilburg University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurent","family":"Servais","sequence":"additional","affiliation":[{"name":"MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford"},{"name":"Department of Pediatrics, Neuromuscular Reference Center, University and University Hospital of Li\u00e8ge, Li\u00e8ge, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Perry B","family":"Shieh","sequence":"additional","affiliation":[{"name":"David Geffen School of Medicine at UCLA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie L","family":"Goedeker","sequence":"additional","affiliation":[{"name":"Washington University School of Medicine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Megan A","family":"Waldrop","sequence":"additional","affiliation":[{"name":"Nationwide Children's Hospital"},{"name":"The Ohio State University College of Medicine, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ryosuke","family":"Bo","sequence":"additional","affiliation":[{"name":"Kobe University Graduate School of Medicine, Kobe, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dheeraj","family":"Raju","sequence":"additional","affiliation":[{"name":"Novartis Pharma AG"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kamal","family":"Benguerba","sequence":"additional","affiliation":[{"name":"Novartis Pharma AG"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra P","family":"Reyna","sequence":"additional","affiliation":[{"name":"Novartis Pharma AG"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Wolff","sequence":"additional","affiliation":[{"name":"Novartis Pharma AG"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9351-7054","authenticated-orcid":false,"given":"Richard S","family":"Finkel","sequence":"additional","affiliation":[{"name":"St Jude Children's Research Hospital"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,11,11]]},"reference":[{"key":"e_1_3_3_2_2","volume-title":"GeneReviews\u00ae","author":"Prior TW","unstructured":"Prior TW, Leach ME, Finanger EL. 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Country performance: access to SMA medicines > Novartis \u2013 Zolgensma https:\/\/odysma.sma-europe.eu\/data-views\/zolgensma (2025 accessed 18 February 2025)."},{"key":"e_1_3_3_20_2","doi-asserted-by":"publisher","DOI":"10.1097\/PHM.0000000000002467"},{"key":"e_1_3_3_21_2","doi-asserted-by":"publisher","DOI":"10.1097\/PHM.0000000000002354"},{"key":"e_1_3_3_22_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-190451"},{"key":"e_1_3_3_23_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.11.014"},{"key":"e_1_3_3_24_2","doi-asserted-by":"publisher","DOI":"10.1177\/0883073811420294"},{"key":"e_1_3_3_25_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(99)70016-8"},{"key":"e_1_3_3_26_2","first-page":"808","article-title":"Onasemnogene abeparvovec gene therapy for spinal muscular atrophy: a cohort study from the United Arab Emirates","volume":"70","author":"Mundada V","year":"2024","unstructured":"Mundada V, Narayan O, Arora S, et al. 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