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...

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Detalles Bibliográficos
Publicado en:Ther Adv Neurol Disord
Main Authors: Stolte, Benjamin, Totzeck, Andreas, Kizina, Kathrin, Bolz, Saskia, Pietruck, Lena, Mönninghoff, Christoph, Guberina, Nika, Oldenburg, Denise, Forsting, Michael, Kleinschnitz, Christoph, Hagenacker, Tim
Formato: Artigo
Idioma:Inglês
Publicado: SAGE Publications 2018
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Original Research
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6174643/
https://ncbi.nlm.nih.gov/pubmed/30305849
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1756286418803246
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