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...

Descripción completa

Detalles Bibliográficos
Autores principales: Stolte, Benjamin, Totzeck, Andreas, Kizina, Kathrin, Bolz, Saskia, Pietruck, Lena, Mönninghoff, Christoph, Guberina, Nika, Oldenburg, Denise, Forsting, Michael, Kleinschnitz, Christoph, Hagenacker, Tim
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2018
Materias:
Original Research
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

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6174643/
https://www.ncbi.nlm.nih.gov/pubmed/30305849
http://dx.doi.org/10.1177/1756286418803246

Ejemplares similares