Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.
Adams D, Tournev IL, Taylor MS, Coelho T, Planté-Bordeneuve V, Berk JL, González-Duarte A, Gillmore JD, Low SC, Sekijima Y, Obici L, Chen C, Badri P, Arum SM, Vest J, Polydefkis M; HELIOS-A Collaborators.
World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
Brito D, Albrecht FC, de Arenaza DP, Bart N, Better N, Carvajal-Juarez I, Conceição I, Damy T, Dorbala S, Fidalgo JC, Garcia-Pavia P, Ge J, Gillmore JD, Grzybowski J, Obici L, Piñero D, Rapezzi C, Ueda M, Pinto FJ.
Glob Heart. 2023 Oct 26;18(1):59. doi: 10.5334/gh.1262. eCollection 2023.
PMID:37901600
Amyloid Neuropathy: From Pathophysiology to Treatment in Light-Chain Amyloidosis and Hereditary Transthyretin Amyloidosis.
Chompoopong P, Mauermann ML, Siddiqi H, Peltier A.
Ann Neurol. 2024 Sep;96(3):423-440. doi: 10.1002/ana.26965. Epub 2024 Jun 24.
PMID:38923548
Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments.