Transthyretin stabilization activity of the catechol-O-methyltransferase inhibitor tolcapone (SOM0226) in hereditary ATTR amyloidosis patients and asymptomatic carriers: proof-of-concept study - Gamez, Josep; Salvadó, María; Reig, Núria; Suñé, Pilar; Casasnovas, Carles; Rojas-Garcia, Ricard; Insa, Raúl (Amyloid, 2019)
Keywords: Adult; Aged; Amyloid Neuropathies, Familial - drug therapy; Amyloid Neuropathies, Familial - metabolism; Amyloidogenesis inhibitor; catechol O-methyltransferase inhibitors; Catechol O-Methyltransferase Inhibitors - therapeutic use; drug repositioning; drug repurposing; Female; hereditary ATTR amyloidosis; Humans; Male; Middle Aged; Mutation, Missense; Prealbumin - genetics; Prealbumin - metabolism; Proof of Concept Study; proof-of-concept; Protein Aggregation, Pathological - prevention & control; tolcapone; Tolcapone - pharmacology; Tolcapone - therapeutic use; transthyretin; TTR aggregation; TTR stabilization; drug repurposing; Amyloidogenesis inhibitor; tolcapone; transthyretin; TTR stabilization; proof-of-concept; catechol O-methyltransferase inhibitors; hereditary ATTR amyloidosis; drug repositioning; TTR aggregation