Tadnersen sodium, an antisense oligonucleotide (ASO), selectively targets C9ORF72 transcript variants 1 and 3 that carry the expansion.
References:
[1]. Smeyers J, et al. C9ORF72: What It Is, What It Does, and W It Matters. Front Cell Neurosci. 2021;15:661447. Published 2021 May 5.
[2]. Aoki Y, et al. Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases [published online ahead of print, 2021 Jun 3]. J Neuromuscul Dis. 2021;10.3233/JND-200560.
















