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      Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy

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          Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy

          Transthyretin amyloid cardiomyopathy is caused by the deposition of transthyretin amyloid fibrils in the myocardium. The deposition occurs when wild-type or variant transthyretin becomes unstable and misfolds. Tafamidis binds to transthyretin, preventing tetramer dissociation and amyloidogenesis.
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            Nonbiopsy Diagnosis of Cardiac Transthyretin Amyloidosis.

            Cardiac transthyretin (ATTR) amyloidosis is a progressive and fatal cardiomyopathy for which several promising therapies are in development. The diagnosis is frequently delayed or missed because of the limited specificity of echocardiography and the traditional requirement for histological confirmation. It has long been recognized that technetium-labeled bone scintigraphy tracers can localize to myocardial amyloid deposits, and use of this imaging modality for the diagnosis of cardiac ATTR amyloidosis has lately been revisited. We conducted a multicenter study to ascertain the diagnostic value of bone scintigraphy in this disease.
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              Natural History of Wild-Type Transthyretin Cardiac Amyloidosis and Risk Stratification Using a Novel Staging System

              Wild-type transthyretin cardiac amyloidosis (ATTRwt) is increasingly recognized as an important cause of heart failure.
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                Author and article information

                Contributors
                (View ORCID Profile)
                Journal
                New England Journal of Medicine
                N Engl J Med
                Massachusetts Medical Society
                0028-4793
                1533-4406
                January 11 2024
                January 11 2024
                : 390
                : 2
                : 132-142
                Affiliations
                [1 ]From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) — both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon...
                Article
                10.1056/NEJMoa2305434
                38197816
                8b725222-99bb-457c-81a2-57f4753ed9a0
                © 2024

                http://www.nejmgroup.org/legal/terms-of-use.htm

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