ABSTRACT
Familial amyloidotic polyneuropathy (FAP) is a fatal hereditary amyloidosis and it has been identified amyloidogenic mutated transthyretin (ATTR), apolipoprotein A-I and gelsolin as FAP amyloidogenic proteins. Among qualified 100 point of mutation or deletion in TTR gene, 13 mutations are nonpathologic forms. Other abnormal TTRs induce FAP which can be classified into several phenotypes, such as neuropathic, oculoleptomeningeal, and cardiac types. Several types of ATTR mutants do not show neuropathy although they are classified into FAP. Of various clinical symptoms, those in the autonomic dysfunction group have the greatest effect on patients’ lives, because they restrict the daily life of FAP patients. Stabilizing TTR in tetrameric form is one of the most important therapeutic strategies for preventing amyloid deposition in tissues of FAP patients. NSAID derivatives are one of the candidate agents that may be used to stabilize TTR in tetrameric form.
