Tafamidis: A Review in Transthyretin Amyloidosis with Polyneuropathy

Transthyretin amyloidosis with polyneuropathy (ATTR-PN), a rare and progressive hereditary disorder, results from mutations in the gene coding for the transthyretin (TTR) protein that destabilize the protein’s tetrameric structure. In over 40 countries worldwide, tafamidis (Vyndaqel®) is approved for the treatment of TTR amyloidosis in adults with stage 1 symptomatic polyneuropathy, to delay peripheral neurological impairment. Tafamidis is...