TTR and Familial transthyretin-related amyloidosis: Similarly, Inotersen, also a PS gapmer with 2′-MOE-modified ends, targets transthyretin (TTR) mRNA to prevent the production of dysfunctional TTR proteins, which accumulate as deposits causing sensorimotor and autonomic neuropathy in hereditary transthyretin amyloidosis (hATTR) [74,75].