FDA OKs Two Tafamidis Formulations for Rare Cardiomyopathy FDA OKs Two Tafamidis Formulations for Rare Cardiomyopathy

These are the first FDA-approved treatments for transthyretin-mediated amyloidosis cardiomyopathy, a rare and often fatal disease caused by deposition of amyloid fibrils in the myocardium.FDA Approvals
Source: Medscape Cardiology Headlines - Category: Cardiology Tags: Cardiology News Alert Source Type: news

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AbstractPurpose of ReviewTo review the uses of echocardiography in patients with cancer and how it has expanded beyond the typical monitoring of systolic function during potentially cardiotoxic cancer therapeutics.Recent FindingsIn addition to myocardial strain imaging being a predictor of subsequent left ventricular dysfunction, it can be used for pattern recognition to help identify patients with cardiac amyloidosis or Takotsubo cardiomyopathy. Echocardiography is essential for diagnosis and planning of intervention for aortic stenosis in radiation-induced valvular disease, for which transcutaneous aortic valve replaceme...
Source: Current Cardiology Reports - Category: Cardiology Source Type: research
Conclusion Cardiac involvement is common and has significant prognostic implications in the evaluated patients with p.Glu89Gln mutation. Heart failure and rhythm disturbances are the main causes of death. An earlier identification of the disease is crucial to improve prognosis.
Source: Journal of Cardiovascular Medicine - Category: Cardiology Tags: Research articles: Heart failure Source Type: research
Since the diagnosis of cardiac amyloidosis (CA) is often delayed, echocardiographic findings are frequently indicative of advanced cardiomyopathy. We aimed to describe early echocardiographic features in patients subsequently diagnosed with CA. Pre-amyloid diagnosis echocardiographic studies were screened for structural and functional parameters and stratified according to the pathogenetic subtype (immunoglobulin light-chain (AL) or amyloid transthyretin (ATTR)). Abnormalities were defined based on published guidelines.
Source: The American Journal of Cardiology - Category: Cardiology Authors: Source Type: research
AbstractEstablishing an accurate diagnosis of amyloid subtype in patients with coexistent cardiac amyloidosis and monoclonal gammopathy is crucial due to treatment and prognostic implications. Here, we discuss a case of coexistent diagnoses of transthyretin amyloid cardiomyopathy and smoldering multiple myeloma, highlighting the challenges associated with the possibility of several disease combinations and the limitations of diagnostic testing. In addition, the importance of clinical clues such as disease course and progression, patient preference, and multidisciplinary collaboration should not be discounted in the diagnos...
Source: Journal of Nuclear Cardiology - Category: Nuclear Medicine Source Type: research
Authors: N M, Soni P, Anand R, Bali S, Hari Abstract Amyloidosis is a conglomeration of diseases due to production and deposition of amyloid, a proteinaceous substance, into organs, tissues, nerves and other places in the body affecting their normal function. This case report is of a 65 year old gentleman, resident of Bihar admitted with a short history of two months. He came with chief complaints of swelling in both lower limbs associated with heaviness in legs, shortness of breath, dizziness, fatigue and passage of frothy urine for two months. He was investigated and found to have proteinuria, low voltage ECG, Ec...
Source: Journal of the Association of Physicians of India - Category: General Medicine Tags: J Assoc Physicians India Source Type: research
člu Y Abstract Cardiac amyloidosis (CA) is a progressive infiltrative cardiomyopathy. Amyloid fibrils in the form of misfolded endogenous proteins accumulate in the heart, as well as the kidneys, liver, and gastrointestinal tract. The most common forms of CA are transthyretin (TTR) and immunoglobulin light chain amyloidosis (AL). CA has long been thought to be a rare disease. However, recent reports have suggested that 13% of heart failure patients with a preserved ejection fraction and 16% of advanced-age patients with severe aortic stenosis have TTR-CA. Patients with TTR-CA have a poor prognosis, with a median s...
Source: Turk Kardiyoloji Dernegi arsivi - Category: Cardiology Authors: Tags: Turk Kardiyol Dern Ars Source Type: research
The original article contained incorrect terminology for one of the cardiac measures; throughout the manuscript and supplementary information ‘intraventricular septum wall thickness’ should have been given as ‘interventricular septum wall thickness’. Corrections should also be noted for Tables 1 and 4: in the Table 1 legend‘Low risk – Neither above at baseline’ should read‘Low risk – Neither above threshold at baseline’; in Table 4, the rows‘Mild: eGFR> 60 to 30 to
Source: Cardiovascular Drugs and Therapy - Category: Cardiology Source Type: research
CONCLUSION: Molecular imaging represent a sensitive tool for early assessment of both amyloid burden and cardiac innervation, to differentiate between subtypes and to monitor disease burden, disease progression, and potential response to therapy. PMID: 32525789 [PubMed - as supplied by publisher]
Source: Current Radiopharmaceuticals - Category: Radiology Tags: Curr Radiopharm Source Type: research
Abstract Transthyretin amyloid cardiomyopathy (ATTR-CM) results in a restrictive cardiomyopathy caused by extracellular deposition of transthyretin, normally involved in the transportation of the hormone thyroxine and retinol-binding protein, in the myocardium. Enthusiasm about ATTR-CM has grown as a result of 3 simultaneous areas of advancement: Imaging techniques allow accurate noninvasive diagnosis of ATTR-CM without the need for confirmatory endomyocardial biopsies; observational studies indicate that the diagnosis of ATTR-CM may be underrecognized in a significant proportion of patients with heart failure; an...
Source: Circulation - Category: Cardiology Authors: Tags: Circulation Source Type: research
Contributors : Gian L Vita ; M'Hammed Aguennouz ; Francesca Polito ; Rosaria Oteri ; Massimo Russo ; Luca Gentile ; Cristina Barbagallo ; Marco Ragusa ; Carmelo Rodolico ; Rosa M Di Giorgio ; Antonio Toscano ; Giuseppe Vita ; Anna MazzeoSeries Type : Non-coding RNA profiling by arrayOrganism : Homo sapiensTransthyretin variant amyloidosis (ATTRv) is a rare autosomal dominant disease characterized by the accumulation of amyloid in many organs, mostly causing a sensory-motor neuropathy, cardiomyopathy, and dysautonomia. The aim of the study was to report microRNAs (miRNAs) expression profile identified in the blood of ATTRv ...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Non-coding RNA profiling by array Homo sapiens Source Type: research
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