An Orphan Disease No More: Additional Treatment Options for Cardiac Amyloidosis

According to the Federal Drug Administration (FDA), an orphan disease is defined as “a condition that affects fewer than 200,000 people nationwide” [1]. This definition was an amendment to the 1983 Orphan Drug Act, which was passed to incentivize research and development into medications for treatment of orphan diseases through a combination of tax credits, grants, and market-e xclusivity rights regardless of patent status [2]. Transthyretin cardiac amyloidosis (ATTR), caused by misfolding and deposition of the transthyretin tetramer protein (TTR) in the myocardium, and is considered to be an orphan disease.
Source: Journal of Cardiac Failure - Category: Cardiology Authors: Tags: Editorial Source Type: research