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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6541434/
https://www.ncbi.nlm.nih.gov/pubmed/31019106
http://dx.doi.org/10.1212/WNL.0000000000007527

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6541434/
https://www.ncbi.nlm.nih.gov/pubmed/31019106
http://dx.doi.org/10.1212/WNL.0000000000007527

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