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Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Respiratory and sleep outcomes in children with SMA treated with nusinersen - real world experience

K Gonski1, S Chuang2, A Teng3

  • 1Department of Respiratory Medicine Departments, Sydney Children's Hospital, Randwick, Australia; Department of Sleep Medicine, Sydney Children's Hospital, Randwick, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Sydney, Randwick, Australia.

Neuromuscular Disorders : NMD
|June 8, 2023
PubMed

Keywords:
ChildrenNusinersenPaediatricsPolysomnographySpinal muscular atrophySpirometry

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