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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Detalhes bibliográficos
Publicado no: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 em: SAGE Publications 2018
Assuntos:
Original Research
Acesso em linha: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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