Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy.
Marianna FontanaJohn L BerkJulian D GillmoreRonald M WittelesMartha GroganBrian DrachmanThibaud DamyPablo Garcia-PaviaJorg TaubelScott D SolomonFarooq H SheikhNobuhiro TaharaJosé González-CostelloKenichi TsujitaCaroline MorbachZoltán PozsonyiMark C PetrieDiego DelgadoPeter Van der MeerAndrew JabbourAntoine BondueDarae KimOlga AzevedoSteen Hvitfeldt PoulsenAli YilmazEwa A JankowskaVincent AlgalarrondoAndrew SluggPushkal P GargKatherine L BoyleElena YurenevaNancy SillimanLilli YangJihong ChenSatish A EralyJohn VestMathew S Maurernull nullPublished in: The New England journal of medicine (2024)
Among patients with ATTR-CM, treatment with vutrisiran led to a lower risk of death from any cause and cardiovascular events than placebo and preserved functional capacity and quality of life. (Funded by Alnylam Pharmaceuticals; HELIOS-B ClinicalTrials.gov number, NCT04153149.).