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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...

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Detalles Bibliográficos
Autores principales: Stolte, Benjamin, Nonnemacher, Michael, Kizina, Kathrin, Bolz, Saskia, Totzeck, Andreas, Thimm, Andreas, Wagner, Bernd, Deuschl, Cornelius, Kleinschnitz, Christoph, Hagenacker, Tim
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2021
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