The FDA and the EMA approval of nusinersen (Spinraza) for spinal muscular atrophy in 2016 and the FDA and the EMA approval of patisiran (Onpattro) for hereditary transthyretin-mediated amyloidosis in 2018 demonstrated the clinical efficacy of RNA therapeutics and accelerated research in this field [4]. Here, TTR is linked to proximal spinal muscular atrophy.