Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies

OBJECTIVE: To report results of intrathecal nusinersen in children with later-onset spinal muscular atrophy (SMA). METHODS: Analyses included children from a phase 1b/2a study (ISIS-396443-CS2; NCT01703988) who first received nusinersen during that study and were eligible to continue treatment in th...

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Detalhes bibliográficos
Publicado no:Neurology
Main Authors: Darras, Basil T., Chiriboga, Claudia A., Iannaccone, Susan T., Swoboda, Kathryn J., Montes, Jacqueline, Mignon, Laurence, Xia, Shuting, Bennett, C. Frank, Bishop, Kathie M., Shefner, Jeremy M., Green, Allison M., Sun, Peng, Bhan, Ishir, Gheuens, Sarah, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C.
Formato: Artigo
Idioma:Inglês
Publicado em: Lippincott Williams & Wilkins 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6541434/
https://ncbi.nlm.nih.gov/pubmed/31019106
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000007527
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