A New Era in the Treatment of Amyloidosis?

Amyloidosis is a diverse group of diseases caused by extracellular accumulation of protein in a highly ordered, abnormal, insoluble fibrillar form. These diseases can be hereditary or acquired and localized or systemic. Most are progressive and fatal. Among the almost 30 different proteins that have been found to form amyloid in humans, transthyretin, a carrier molecule of thyroxine and vitamin A, is of particular interest because it may cause both hereditary and acquired amyloidosis with a diverse range of symptoms. In addition, transthyretin amyloid may be more prevalent than previously thought, and several potential new treatments are on the horizon. . . .