Acoramidis
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Acoramidis
Acoramidis, sold under the brand name Attruby, is a medication used for the treatment of cardiomyopathy. It is a near-complete (>90%) transthyretin stabilizer, developed to mimic the protective properties of the naturally-occurring T119M mutation, to treat transthyretin amyloid cardiomyopathy. It is taken by mouth. The most common adverse reactions include diarrhea and upper abdominal pain. Acoramidis was approved for medical use in the United States in November 2024, and in the European Union in February 2025. Medical uses Acoramidis is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. ATTR-CM is a rare and serious disease that affects the heart muscle. In people with ATTR-CM, there is a build-up of protein deposits in the heart, causing the walls of the heart to become stiff, and making the left ventricle unable to pr ...
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Cardiomyopathy
Cardiomyopathy is a group of primary diseases of the heart muscle. Early on there may be few or no symptoms. As the disease worsens, shortness of breath, feeling tired, and swelling of the legs may occur, due to the onset of heart failure. An irregular heart beat and fainting may occur. Those affected are at an increased risk of sudden cardiac death. As of 2013, cardiomyopathies are defined as "disorders characterized by morphologically and functionally abnormal myocardium in the absence of any other disease that is sufficient, by itself, to cause the observed phenotype." Types of cardiomyopathy include hypertrophic cardiomyopathy, dilated cardiomyopathy, restrictive cardiomyopathy, arrhythmogenic right ventricular dysplasia, and Takotsubo cardiomyopathy (broken heart syndrome). In hypertrophic cardiomyopathy the heart muscle enlarges and thickens. In dilated cardiomyopathy the ventricles enlarge and weaken. In restrictive cardiomyopathy the ventricle stiffens. In ...
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Transthyretin Amyloid Cardiomyopathy
Amyloid cardiomyopathy ( stiff heart syndrome) is a condition resulting in the death of part of the myocardium (heart muscle). It is associated with the systemic production and release of many amyloidogenic proteins, especially immunoglobulin light chain or transthyretin (TTR). It can be characterized by the extracellular deposition of amyloids, foldable proteins that stick together to build fibrils in the heart. Symptoms Amyloid cardiomyopathy is associated with a number of symptoms: * diastolic dysfunction * congestive heart failure * arrhythmia * cardiac nervous conduction block * fatigue * dyspnea Pathophysiology Amyloid proteins are deposited in the myocardium. This limits ventricular filling during diastole, which increases end-diastolic volume. This can lead to a variety of cardiac issues, such as congestive heart failure, atrial arrhythmia, ventricular arrhythmia, and blocks to cardiac nervous conduction. Diagnosis Diagnosis is often delayed, because its symptoms a ...
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