[1][2][3] The aggregation of one precursor protein leads to peripheral neuropathy and/or autonomic nervous system dysfunction.

[4] Due to the rareness of the other types of familial neuropathies, transthyretin amyloidogenesis-associated polyneuropathy should probably be considered first.

[8] Fibrinogen, apolipoprotein A1, and lysozyme are associated with a closely related condition, familial visceral amyloidosis.

Liver transplantation has proven to be effective for ATTR familial amyloidosis due to Val30Met mutation.

[10] The FDA rejected the application for marketing approval in the US in 2012 on the basis that the clinical trial data did not show efficacy based on a functional endpoint, and the FDA requested further clinical trials.