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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...
Autores principales: | Haché, Manon, Swoboda, Kathryn J., Sethna, Navil, Farrow-Gillespie, Alan, Khandji, Alexander, Xia, Shuting, Bishop, Kathie M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
SAGE Publications
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4871174/ https://www.ncbi.nlm.nih.gov/pubmed/26823478 http://dx.doi.org/10.1177/0883073815627882 |
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