Onasemnogene‐abeparvovec administration to premature infants with spinal muscular atrophy
Onasemnogene‐abeparvovec administration to premature infants with spinal muscular atrophy
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Publisher
United States: John Wiley & Sons, Inc
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Language
English
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Publisher
United States: John Wiley & Sons, Inc
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Twin girls born at 30 weeks' gestation with spinal muscular atrophy (SMA) received onsasemnogene‐abeparvovec (OA) at 3.5 weeks of life. They had no treatment‐related adverse events, normal acquisition of motor milestones, and normal neurological examination at 19 months. Genotyping revealed 0 copies of SMN1 and a single, hybrid SMN2 gene containing...
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Full title
Onasemnogene‐abeparvovec administration to premature infants with spinal muscular atrophy
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TN_cdi_doaj_primary_oai_doaj_org_article_d5dc5d12e75c4010864e8957627976d6
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_d5dc5d12e75c4010864e8957627976d6
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ISSN
2328-9503
E-ISSN
2328-9503
DOI
10.1002/acn3.52213
