Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.
Mathew S MaurerParag KaleMarianna FontanaJohn L BerkMartha GroganFinn GustafssonRebecca R HungRobert L GottliebThibaud DamyAlejandra González-DuarteNitasha SarswatYoshiki SekijimaNobuhiro TaharaMark S TaylorMilos KubanekErwan DonalTomas PalecekKenichi TsujitaW H Wilson TangWen-Chung YuLaura ObiciMarcus SimõesFábio FernandesSteen Hvitfeldt PoulsenIgor DiembergerFederico PerfettoScott D SolomonMarcelo Di CarliPrajakta BadriMatthew T WhiteJihong ChenElena YurenevaMarianne T SweetserPatrick Y JayPushkal P GargJohn VestJulian D Gillmorenull nullPublished in: The New England journal of medicine (2023)
In this trial, administration of patisiran over a period of 12 months resulted in preserved functional capacity in patients with ATTR cardiac amyloidosis. (Funded by Alnylam Pharmaceuticals; APOLLO-B ClinicalTrials.gov number, NCT03997383.).