Specific Therapy in Transthyretin Amyloid Cardiomyopathy
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a relatively prevalent cause of morbidity and mortality. Over the recent years, development of disease-modifying treatments has enabled stabilization of the circulating transthyretin tetramer and suppression of its hepatic production, resulting in a remarkable improvement in survival of patients with ATTR-CM. Second-generation drugs for silencing are currently under investigation in randomized clinical trials. In vivo gene editing of transthyretin has been achieving unanticipated suppression of hepatic production in ATTR-CM. Trials of antibodies inducing the active removal of transthyretin amyloid deposits in the heart are ongoing, and evidence has gathered for exceptional spontaneous regression of ATTR-CM .
Source: Heart Failure Clinics - Category: Cardiology Authors: Riccardo Saro, Valentina Allegro, Marco Merlo, Franca Dore, Gianfranco Sinagra, Aldostefano Porcari Source Type: research