Familial Amyloidotic Polyneuropathy

(FAP), also called transthyretin-related hereditary amyloidosis, transthyretin amyloidosis, or Corino de Andrade’s disease, is caused by many different mutations in the protein transthyretin (TTR). FAP is a life-threatening disease, affecting predominantly the peripheral and autonomic nervous system. The disease has a wide geographic distribution with the largest populations in Portugal, Japan, and Sweden, and is estimated to affect ~5,000–10,000 patients worldwide. There are several drugs approved for treatment of FAP.