Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience

Nusinersen (ISIS-SMN(Rx) 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 st...

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Pubblicato in:J Child Neurol
Autori principali: Haché, Manon, Swoboda, Kathryn J., Sethna, Navil, Farrow-Gillespie, Alan, Khandji, Alexander, Xia, Shuting, Bishop, Kathie M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: SAGE Publications 2016
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4871174/
https://ncbi.nlm.nih.gov/pubmed/26823478
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/0883073815627882
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