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Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy
BACKGROUND: Nusinersen is an intrathecally administered antisense oligonucleotide (ASO) and the first approved drug for the treatment of spinal muscular atrophy (SMA). However, progressive neuromyopathic scoliosis and the presence of spondylodesis can impede lumbar punctures in SMA patients. Our aim...
Autores principales: | Stolte, Benjamin, Totzeck, Andreas, Kizina, Kathrin, Bolz, Saskia, Pietruck, Lena, Mönninghoff, Christoph, Guberina, Nika, Oldenburg, Denise, Forsting, Michael, Kleinschnitz, Christoph, Hagenacker, Tim |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
SAGE Publications
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6174643/ https://www.ncbi.nlm.nih.gov/pubmed/30305849 http://dx.doi.org/10.1177/1756286418803246 |
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