{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T05:01:46Z","timestamp":1787893306731,"version":"build-2784847793"},"reference-count":13,"publisher":"American Academy of Pediatrics (AAP)","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2020,9,1]]},"abstract":"<jats:sec>\n                  <jats:title>BACKGROUND AND OBJECTIVES:<\/jats:title>\n                  <jats:p>Historically, autosomal recessive 5q-linked spinal muscular atrophy (SMA) has been the leading inherited cause of infant death. SMA is caused by the absence of the SMN1 gene, and SMN1 gene replacement therapy, onasemnogene abeparvovec-xioi, was Food and Drug Administration approved in May 2019. Approval included all children with SMA age &amp;lt;2 years without end-stage weakness. However, gene transfer with onasemnogene abeparvovec-xioi has been only studied in children age \u22648 months.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>METHODS:<\/jats:title>\n                  <jats:p>In this article, we report key safety and early outcome data from the first 21 children (age 1\u201323 months) treated in the state of Ohio.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>RESULTS:<\/jats:title>\n                  <jats:p>In children \u22646 months, gene transfer was well tolerated. In this young group, serum transaminase (aspartate aminotransferase and alanine aminotransferase) elevations were modest and not associated with \u03b3 glutamyl transpeptidase elevations. Initial prednisolone administration matched that given in the clinical trials. In older children, elevations in aspartate aminotransferase, alanine aminotransferase and \u03b3 glutamyl transpeptidase were more common and required a higher dose of prednisolone, but all were without clinical symptoms. Nineteen of 21 (90%) children experienced an asymptomatic drop in platelets in the first week after treatment that recovered without intervention. Of the 19 children with repeated outcome assessments, 11% (n = 2) experienced stabilization and 89% (n = 17) experienced improvement in motor function.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>CONCLUSIONS:<\/jats:title>\n                  <jats:p>In this population, with thorough screening and careful post\u2013gene transfer management, replacement therapy with onasemnogene abeparvovec-xioi is safe and shows promise for early efficacy.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1542\/peds.2020-0729","type":"journal-article","created":{"date-parts":[[2020,8,25]],"date-time":"2020-08-25T12:25:24Z","timestamp":1598358324000},"source":"Crossref","is-referenced-by-count":125,"title":["Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes"],"prefix":"10.1542","volume":"146","author":[{"given":"Megan A.","family":"Waldrop","sequence":"first","affiliation":[{"name":"Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio;"},{"name":"Center for Gene Therapy and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cassandra","family":"Karingada","sequence":"additional","affiliation":[{"name":"Departments of Neurology and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mike A.","family":"Storey","sequence":"additional","affiliation":[{"name":"Pharmacy, Nationwide Children\u2019s Hospital, Columbus, Ohio;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brenna","family":"Powers","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Megan A.","family":"Iammarino","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie F.","family":"Miller","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy 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Ohio;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eileen","family":"Broomall","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology, Cincinnati Children\u2019s Hospital, Cincinnati, Ohio; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Goldstein","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics, Rainbow Babies and Children\u2019s Hospital, Cleveland, Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy","family":"Bass","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics, Rainbow Babies and Children\u2019s Hospital, Cleveland, Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda P.","family":"Lowes","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chang-Yong","family":"Tsao","sequence":"additional","affiliation":[{"name":"Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio;"},{"name":"Departments of Neurology and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerry R.","family":"Mendell","sequence":"additional","affiliation":[{"name":"Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio;"},{"name":"Center for Gene Therapy and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne M.","family":"Connolly","sequence":"additional","affiliation":[{"name":"Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio;"},{"name":"Center for Gene Therapy 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