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They had no treatment\u2010related adverse events, normal acquisition of motor milestones, and normal neurological examination at 19\u2009months. Genotyping revealed 0 copies of <jats:italic>SMN1<\/jats:italic> and a single, hybrid <jats:italic>SMN2<\/jats:italic> gene containing the positive genetic modifier c.835\u201044A&gt;G. This was associated with full\u2010length <jats:italic>SMN2<\/jats:italic> blood mRNA expression levels similar to a 2 copy SMA infant. The observed favorable outcomes are likely due to the genetic modifier combined with early drug administration enabled by prematurity.<\/jats:p>","DOI":"10.1002\/acn3.52213","type":"journal-article","created":{"date-parts":[[2024,9,29]],"date-time":"2024-09-29T05:39:06Z","timestamp":1727588346000},"page":"3042-3046","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Onasemnogene\u2010abeparvovec administration to premature infants with spinal muscular atrophy"],"prefix":"10.1002","volume":"11","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1347-5908","authenticated-orcid":false,"given":"Stephen M.","family":"Brown","sequence":"first","affiliation":[{"name":"Department of Neurology Johns Hopkins University School of Medicine  Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aparna S.","family":"Ajjarapu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Iowa Carver College of Medicine  Iowa City Iowa USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Divya","family":"Ramachandra","sequence":"additional","affiliation":[{"name":"Department of Genetics Advocate Children's Hospital  Oak Lawn Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Blasco\u2010P\u00e9rez","sequence":"additional","affiliation":[{"name":"Department of Clinical and Molecular Genetics and Medicine Genetics Group, VHIR Hospital Vall d'Hebron  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mar","family":"Costa\u2010Roger","sequence":"additional","affiliation":[{"name":"Department of Clinical and Molecular Genetics and Medicine Genetics Group, VHIR Hospital Vall d'Hebron  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eduardo F.","family":"Tizzano","sequence":"additional","affiliation":[{"name":"Department of Clinical and Molecular Genetics and Medicine Genetics Group, VHIR Hospital Vall d'Hebron  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charlotte J.","family":"Sumner","sequence":"additional","affiliation":[{"name":"Department of Neurology Johns Hopkins University School of Medicine  Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine D.","family":"Mathews","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Iowa Carver College of Medicine  Iowa City Iowa USA"},{"name":"Department of Neurology University of Iowa Carver College of Medicine  Iowa City Iowa USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,9,28]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-022-00380-8"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1090-3798(99)80015-4"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-022-01866-4"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-230122"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2023.1230889"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27261"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-180304"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/humu.24200"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI124120"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddx166"},{"key":"e_1_2_8_13_1","volume-title":"Blood Groups and Red Cell Antigens","author":"Dean L","year":"2005"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/s004390000452"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.braindev.2020.09.005"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1212\/NXG.0000000000200175"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.abb6871"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073820928784"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.1017\/S0954579419001536"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.1044\/1092-4388(2013\/12-0350)"},{"key":"e_1_2_8_21_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2044.1979.tb06327.x"},{"key":"e_1_2_8_22_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-230136"},{"key":"e_1_2_8_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.14798"},{"key":"e_1_2_8_24_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.18.02132"},{"key":"e_1_2_8_25_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2024.1326528"},{"key":"e_1_2_8_26_1","unstructured":"Package Insert\u2010 ZOLGENSMA. 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