FDA Approves Treatments for Heart Failure Caused by Rare Disease

MONDAY, May 6, 2019 -- Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules have been approved to treat adults with cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM), the U.S. Food and Drug Administration announced...
Source: Drugs.com - Pharma News - Category: Pharmaceuticals Source Type: news

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CONCLUSION: discharge planning based on the NIC improves the NOC score and may interfere in the health outcomes. PMID: 32609201 [PubMed - in process]
Source: Revista Brasileira de Enfermagem - Category: Nursing Authors: Tags: Rev Bras Enferm Source Type: research
Abstract In recent years, home medical care has been strongly promoted. As a consequence, the conditions managed in home medical care have become increasingly diverse. Heart failure is one of the most common disorders after malignant diseases. Patients with chronic heart failure (CHF) are often forced into hospitalization because of the inability to control symptoms with oral medications, even though they hope to stay at home. Recently, we have experienced a case where the patient required continuous administration of dobutamine at home. In order to carry out CHF care at home successfully, it is necessary to adjus...
Source: Yakugaku Zasshi : Journal of the Pharmaceutical Society of Japan - Category: Drugs & Pharmacology Authors: Tags: Yakugaku Zasshi Source Type: research
Authors: S A, R V, P C, V I Abstract Ortners syndrome is a rare cause of recurrent laryngeal nerve palsy due to cardiac causes. After the description of this syndrome by Ortner in patients with mitral stenosis, it was described by many authors in multiple other cardiac conditions. Here we present a case of Ortner's syndrome in Dilated cardiomyopathy,which reverted completely after medical management of DCMP. PMID: 32610886 [PubMed - in process]
Source: Journal of the Association of Physicians of India - Category: General Medicine Tags: J Assoc Physicians India Source Type: research
1366Objectives: CURRENT ROLE OF PET IMAGING IN CARDIAC AMYLOIDOSIS Cardiac amyloidosis (CA) is a relatively rare disease characterized by extracellular deposition of abnormal protein fibrils in the heart, resulting in restrictive cardiomyopathy and heart failure with preserved ejection fraction. Majority of CA occurs due to deposition of two types of amyloid protein: monoclonal immunoglobulin light chains (AL amyloidosis) or transthyretin (ATTR), either wild or mutant type. Differentiating the two most common types of amyloid deposition in the heart is of paramount importance in guiding therapy and prognostication. Diagnos...
Source: Journal of Nuclear Medicine - Category: Nuclear Medicine Authors: Tags: Cardiovascular Sciences (Poster Session) Source Type: research
This article will review ATTR cardiac amyloidosis to provide physicians with the tools they need to establish a definitive diagnosis when there is a clinical suspicion of amyloidosis and provide the most appropriate care. Expert commentary: Increased awareness and improved diagnostic techniques will lead to earlier diagnosis and a greater understanding of the clinical presentation. The anticipated increases in the prevalence of this disease due to increased clinical awareness will require, and in-part, facilitate the development of new therapies to manage this patient population. PMID: 31478389 [PubMed - as supplied by publisher]
Source: Expert Review of Cardiovascular Therapy - Category: Cardiology Tags: Expert Rev Cardiovasc Ther Source Type: research
Rationale: Cardiac amyloidosis, considered for the last years to be a rare disease, is one of the determinants of HFpEF. The non-specific clinical presentation and the difficulties related to endomyocardial biopsy have made cardiac amyloidosis an underdiagnosed clinical entity. Improvement of non-invasive diagnostic techniques and the development of new therapies increased clinical awareness for this form of restrictive cardiomyopathy. We here summarize echocardiography and 99mTc-HDP scintigraphy findings in 6 cases of cardiac amyloidosis and review the literature data of this progressive and fatal cardiomyopathy. Patie...
Source: Medicine - Category: Internal Medicine Tags: Research Article: Clinical Case Report Source Type: research
Fight Aging! provides a weekly digest of news and commentary for thousands of subscribers interested in the latest longevity science: progress towards the medical control of aging in order to prevent age-related frailty, suffering, and disease, as well as improvements in the present understanding of what works and what doesn't work when it comes to extending healthy life. Expect to see summaries of recent advances in medical research, news from the scientific community, advocacy and fundraising initiatives to help speed work on the repair and reversal of aging, links to online resources, and much more. This content is...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Perhaps a score of the countless proteins in the human body misfold in large amounts in later life. The misfolded form is insoluble, leading to solid deposits of the protein in and around cells. These problem proteins are known as amyloids, and the accumulation of amyloids is one of the root causes of aging. Amyloidosis conditions arise from the presence of amyloid and the disruptive effect it has on cellular biochemistry. The best known form of amyloid is the amyloid-β thought to cause Alzheimer's disease, but the research community is beginning to appreciate that other forms may be just as big a problem over the cou...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
Fight Aging! provides a weekly digest of news and commentary for thousands of subscribers interested in the latest longevity science: progress towards the medical control of aging in order to prevent age-related frailty, suffering, and disease, as well as improvements in the present understanding of what works and what doesn't work when it comes to extending healthy life. Expect to see summaries of recent advances in medical research, news from the scientific community, advocacy and fundraising initiatives to help speed work on the repair and reversal of aging, links to online resources, and much more. This content is...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Transthyretin amyloidosis is a rare, life-threatening disease resulting from aggregation and deposition of transthyretin amyloid fibrils in various tissues. There are 2 predominate phenotypic presentations of the disease: transthyretin familial amyloid polyneuropathy, which primarily affects the peripheral nerves, and transthyretin cardiomyopathy (TTR-CM), which primarily affects the heart. However, there is a wide overlap with symptoms at presentation and disease course being highly variable and influenced by the underlying transthyretin mutation, age of the affected individual, sex, and geographic location. Treatment of ...
Source: Circulation: Heart Failure - Category: Cardiology Authors: Tags: Clinical Studies Advances in Clinical Trials Source Type: research
More News: Amyloidosis | Cardiology | Cardiomyopathy | Food and Drug Administration (FDA) | Heart | Heart Failure | Pharmaceuticals | Rare Diseases