title: Tafamidis (Vyndaqel): a light for FAP patients.
abstract: Slowing FAP progression: Tafamidis meglumine is a small molecule capable of stabilizing the transthyretin (TTR) tetramer. Tafamidis acts in a similar way to the natural hormone T4, prevents TTR amyloid fibril formation, and offers a potential alternative to liver transplantation for the treatment of patients with TTR familial amyloid polyneuropathies (TTR-FAP).
authors.affiliation: Dipartimento di Farmacia, Università di Pisa, Via Bonanno 6, 56126 Pisa (Italy). nencettis@farm.unipi.it.
authors.forename: Susanna
authors.initials: S
authors.lastname: Nencetti
authors.forename: Armando
authors.initials: A
authors.lastname: Rossello
authors.forename: Elisabetta
authors.initials: E
authors.lastname: Orlandini
mesh_terms: D028227:Amyloid Neuropathies, Familial / Q000188:drug therapy*; D001583:Benzoxazoles / Q000737:chemistry / Q000378:metabolism / Q000627:therapeutic use*; D001665:Binding Sites; D015195:Drug Design; D006801:Humans; D009154:Mutation; D011228:Prealbumin / Q000737:chemistry / Q000235:genetics / Q000378:metabolism; D011485:Protein Binding; D017434:Protein Structure, Tertiary
chemical_list: D001583:Benzoxazoles; D011228:Prealbumin; C547076:tafamidis
keywords: drug discovery; familial amyloid polyneuropathy; ligand design; tafamidis meglumine; transthyretin