In fact, there are a number of diseases that are considered ‘undrugable’ by small molecules that can be corrected by targeting single-stranded RNA61—case in point are the recent development and approval of nusinersen (Spinraza) by Ionic Pharmaceuticals and patisiran (Onpattro) by Alnylam Pharmaceuticals for treatment of the respective spinal muscular atrophy and hereditary transthyretin-mediated amyloidosis. This evidence concerns the gene TTR and spinal muscular atrophy.