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Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters
BACKGROUND: Nusinersen is an intrathecally administered antisense oligonucleotide (ASO) that improves motor function in patients with spinal muscular atrophy (SMA). In addition to efficacy, the safety of a therapy is the decisive factor for the success of the treatment. For some ASOs, various organ...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Berlin Heidelberg
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8563549/ https://www.ncbi.nlm.nih.gov/pubmed/33899154 http://dx.doi.org/10.1007/s00415-021-10569-8 |