Disparities, Uncertainties and Societal Cost: Precision Medicine and Transthyretin Amyloidosis

Elucidation of the biologic mechanisms underlying the pathogenesis of transthyretin (TTR) amyloidosis has led to the approval of therapies to treat both inherited (hATTR) and age-related or wild-type (wtATTR) forms of transthyretin amyloidosis manifesting as neuropathy and/or cardiomyopathy1-3. These developments have been hailed as a model of scientific and medical progress emanating from precision medicine, which can arrest or slow progression of fatal disorders and provide a potential pathway for progress in other complex clinical syndromes such as heart failure with preserved ejection fraction by endo-phenotyping.