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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Dades bibliogràfiques
Publicat a:Ther Adv Neurol Disord
Autors principals: Stolte, Benjamin, Totzeck, Andreas, Kizina, Kathrin, Bolz, Saskia, Pietruck, Lena, Mönninghoff, Christoph, Guberina, Nika, Oldenburg, Denise, Forsting, Michael, Kleinschnitz, Christoph, Hagenacker, Tim
Format: Artigo
Idioma:Inglês
Publicat: SAGE Publications 2018
Matèries:
Original Research
Accés en línia: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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