Patisiran (Onpattro; Alnylam Pharmaceuticals, Cambridge, MA, USA) has been administered to patients with hereditary transthyretin (TTR) amyloidosis, an autosomal dominant disease in which the accumulation of misfolded TTR proteins in the body causes progressive neuropathy, cardiomyopathy, CKD and organ failure. Here, TTR is linked to autosomal dominant disease.