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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Bibliografiska uppgifter
I publikationen:Ther Adv Neurol Disord
Huvudupphovsmän: Stolte, Benjamin, Totzeck, Andreas, Kizina, Kathrin, Bolz, Saskia, Pietruck, Lena, Mönninghoff, Christoph, Guberina, Nika, Oldenburg, Denise, Forsting, Michael, Kleinschnitz, Christoph, Hagenacker, Tim
Materialtyp: Artigo
Språk:Inglês
Publicerad: SAGE Publications 2018
Ämnen:
Original Research
Länkar: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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