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. This evidence concerns the gene TTR and cardiomyopathy.