As a consequence of the grave prognosis of ALS, a constantly enhanced TGFβ system activity in ALS patients, and the promising characteristics of ASO technology to downregulate critical targets, we developed a novel LNA-gapmer ASO targeting TGFBR2 (formerly TGF-βRII) mRNA (NVP-13). This evidence concerns the gene TGFB1 and amyotrophic lateral sclerosis.