To follow up the hypothesis of a dysregulated TGFβ-system underlying the detrimental disease course of neurodegenerative disorders like ALS, we developed a novel antisense oligonucleotide (NVP-13) in a locked nucleic acid (LNA)-gapmer design, targeting TGFβ-RII mRNA. This evidence concerns the gene TGFB1 and amyotrophic lateral sclerosis.