Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy

OBJECTIVE: To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy of intrathecal nusinersen (previously ISIS-SMN(Rx)), an antisense oligonucleotide designed to alter splicing of SMN2 mRNA, in patients with childhood spinal muscular atrophy (SMA). METHODS: Nusinersen was...

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Detalhes bibliográficos
Publicado no:Neurology
Main Authors: Chiriboga, Claudia A., Swoboda, Kathryn J., Darras, Basil T., Iannaccone, Susan T., Montes, Jacqueline, De Vivo, Darryl C., Norris, Daniel A., Bennett, C. Frank, Bishop, Kathie M.
Formato: Artigo
Idioma:Inglês
Publicado em: Lippincott Williams & Wilkins 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4782111/
https://ncbi.nlm.nih.gov/pubmed/26865511
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000002445
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