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NFL is a marker of treatment response in children with SMA treated with nusinersen

BACKGROUND: Recently, the anti-sense oligonucleotide drug nusinersen was approved for spinal muscular atrophy (SMA) and our aim was to find a response marker for this treatment. METHODS: Twelve children with SMA type 1 and two copies of the SMN2 gene were included in a consecutive single-center stud...

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Detalles Bibliográficos
Autores principales: Olsson, Bob, Alberg, Lars, Cullen, Nicholas C., Michael, Eva, Wahlgren, Lisa, Kroksmark, Anna-Karin, Rostasy, Kevin, Blennow, Kaj, Zetterberg, Henrik, Tulinius, Már
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6687695/
https://www.ncbi.nlm.nih.gov/pubmed/31123861
http://dx.doi.org/10.1007/s00415-019-09389-8

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