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10.1177/0883073815627882

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suck abstract from ncbi


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pmid26823478
      J+Child+Neurol 2016 ; 31 (7 ): 899-906
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  • Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience #MMPMID26823478
  • Haché M ; Swoboda KJ ; Sethna N ; Farrow-Gillespie A ; Khandji A ; Xia S ; Bishop KM
  • J Child Neurol 2016[Jun]; 31 (7 ): 899-906 PMID26823478 show ga
  • Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug administered intrathecally to treat spinal muscular atrophy. We summarize lumbar puncture experience in children with spinal muscular atrophy during a phase 1 open-label study of nusinersen and its extension. During the studies, 73 lumbar punctures were performed in 28 patients 2 to 14 years of age with type 2/3 spinal muscular atrophy. No complications occurred in 50 (68%) lumbar punctures; in 23 (32%) procedures, adverse events were attributed to lumbar puncture. Most common adverse events were headache (n = 9), back pain (n = 9), and post-lumbar puncture syndrome (n = 8). In a subgroup analysis, adverse events were more frequent in older children, children with type 3 spinal muscular atrophy, and with a 21- or 22-gauge needle compared to a 24-gauge needle or smaller. Lumbar punctures were successfully performed in children with spinal muscular atrophy; lumbar puncture-related adverse event frequency was similar to that previously reported in children.
  • |Adolescent [MESH]
  • |Child [MESH]
  • |Child, Preschool [MESH]
  • |Female [MESH]
  • |Humans [MESH]
  • |Injections, Spinal/adverse effects [MESH]
  • |Male [MESH]
  • |Muscular Atrophy, Spinal/*drug therapy [MESH]
  • |Neuromuscular Agents/*administration & dosage/adverse effects [MESH]
  • |Oligonucleotides/*administration & dosage/adverse effects [MESH]


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