{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,3]],"date-time":"2026-05-03T10:41:15Z","timestamp":1777804875607,"version":"3.51.4"},"reference-count":10,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2025,3,2]],"date-time":"2025-03-02T00:00:00Z","timestamp":1740873600000},"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":["J Child Neurol"],"published-print":{"date-parts":[[2025,5]]},"abstract":"<jats:p>Early treatment in spinal muscular atrophy is widely recognized as critical for improving neurologic and respiratory outcomes, especially in presymptomatic infants. With the expansion of newborn screening, more infants are now diagnosed presymptomatically. Currently, there are no established treatment guidelines for preterm infants with spinal muscular atrophy, with only anecdotal reports available. Additionally, there is limited knowledge regarding the safety and efficacy of the different treatments in preterm infants. In Israel, although a newborn screening program for spinal muscular atrophy is not yet implemented, a significant portion of the population participates in genetic carrier screening. Here, we present a case of presymptomatic preterm twins, born at gestational age of 32\u2009+\u20092 weeks, birth weights of 1855 and 1740 g, respectively. They were treated with risdiplam followed by onasemnogene abeparvovec at a gestational age of 35 and 43 weeks, respectively. This case adds to the limited data on treatment options for preterm infants.<\/jats:p>","DOI":"10.1177\/08830738251322224","type":"journal-article","created":{"date-parts":[[2025,3,4]],"date-time":"2025-03-04T13:35:29Z","timestamp":1741095329000},"page":"371-373","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Early Treatment in Preterm Twins With Spinal Muscular Atrophy"],"prefix":"10.1177","volume":"40","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4963-5069","authenticated-orcid":false,"given":"Jacob","family":"Bistritzer","sequence":"first","affiliation":[{"name":"Pediatric Neurology Unit, Saban Children's hospital, Soroka University Medical Center, Beer-Sheva, Israel"},{"name":"Faculty of Health Science, Ben-Gurion University of the Negev, Beer-Sheva, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3284-2854","authenticated-orcid":false,"given":"Dekel","family":"Avital","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Unit, Saban Children's hospital, Soroka University Medical Center, Beer-Sheva, Israel"},{"name":"Faculty of Health Science, Ben-Gurion University of the Negev, Beer-Sheva, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6259-405X","authenticated-orcid":false,"given":"Inbal","family":"Golan-Tripto","sequence":"additional","affiliation":[{"name":"Faculty of Health Science, Ben-Gurion University of the Negev, Beer-Sheva, Israel"},{"name":"Pediatric Pulmonary Unit, Saban Children's Hospital, Soroka University Medical Center, Beer-Sheva, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6384-2577","authenticated-orcid":false,"given":"Ramy","family":"Abramsky","sequence":"additional","affiliation":[{"name":"Faculty of Health Science, Ben-Gurion University of the Negev, Beer-Sheva, Israel"},{"name":"Department of Neonatology, Saban Children's Hospital, Soroka University Medical Center, Be'er Sheva, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2916-7600","authenticated-orcid":false,"given":"Iris","family":"Noyman","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Unit, Saban Children's hospital, Soroka University Medical Center, Beer-Sheva, Israel"},{"name":"Faculty of Health Science, Ben-Gurion University of the Negev, Beer-Sheva, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2025,3,2]]},"reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60921-6"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24497"},{"key":"e_1_3_2_5_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-190468"},{"key":"e_1_3_2_6_2","doi-asserted-by":"publisher","DOI":"10.3390\/brainsci13101446"},{"key":"e_1_3_2_7_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27648"},{"key":"e_1_3_2_8_2","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2023.1230889"},{"key":"e_1_3_2_9_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27261"},{"key":"e_1_3_2_10_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-024-03362-z"},{"key":"e_1_3_2_11_2","unstructured":"Highlights of prescribing information for Zolgensma Durham: Novartis Gene Therapies Inc. 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