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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Neurology
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Lippincott Williams & Wilkins 2019
Aiheet:
Article
Linkit: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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