Tafamidis

Tafamidis is a prescription medication primarily used for the treatment of patients with Transthyretin (TTR) amyloidosis, a rare, progressive, and potentially life-threatening disorder. TTR amyloidosis is characterized by the buildup of abnormal proteins, known as amyloid, in various organs and tissues throughout the body.
Tafamidis is an oral, small-molecule medication that works by stabilizing the TTR protein, preventing the formation of toxic amyloid aggregates and reducing their accumulation in affected organs. This, in turn, helps to slow down the disease progression and alleviate symptoms.
The medication is specifically indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) in adults, a genetic disorder that affects multiple organ systems, including the nerves, heart, and eyes. The main symptoms of hATTR include neuropathy (nerve damage), cardiomyopathy (heart muscle disease), and ophthalmoplegia (eye muscle weakness).
Tafamidis has been shown to provide significant benefits for patients with hATTR, including improvements in their neurological and cardiac function. Additionally, the medication has been demonstrated to prolong the survival of patients with the disease, especially those with cardiac involvement.