FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin mediated amyloidosis

On May 3, the U.S. Food and Drug Administration approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. Vyndaqel and Vyndamax have the same active moiety, tafamidis, but they are not substitutable on a milligram to milligram basis and their recommended doses differ.
Source: World Pharma News - Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news

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The objective of this study was to compare the use of pulmonary regurgitation volume (PRV) or indexed PRV (PRVi) with that of pulmonary regurgitation fraction (PRF) in the assessment of patients with pulmonary regurgitation (PR) undergoing cardiac magnetic resonance (CMR) imaging. Materials and Methods: CMR of 176 patients with PR were retrospectively evaluated. Their right ventricular diastolic (end-diastolic volume index [EDVi]) and systolic (end-systolic volume index) volume indexes, stroke volume, and ejection fraction were obtained from cine CMR sequences, whereas phase-contrast flow sequences were analyzed to obta...
Source: Journal of Thoracic Imaging - Category: Radiology Tags: Original Articles Source Type: research
This article deals with the role of von Gierke as a persecuted and disenfranchised Jew. Accordingly, the focus is on von Gierkes’ repressive experiences in the Third Reich, which were quite different from other cases. It is based on (1) previously partly unnoticed archival sources and (2) a re-analysis of the relevant research literature.The paper shows that Edgar von Gierke was a double victim of Nazi Germany, even though he was able to maintain his professional position for a comparatively long time: In contrast to other Jews who were dismissed in 1933 on the basis of the „Aryan paragraph“, von Gierke b...
Source: Pathology Research and Practice - Category: Pathology Source Type: research
Condition:   Transthyretin‑Mediated Amyloid Cardiomyopathy (ATTR CM) Interventions:   Drug: AKCEA-TTR-LRx;   Drug: Placebo Sponsors:   Ionis Pharmaceuticals, Inc.;   Akcea Therapeutics Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Conditions:   Cardiovascular Disease Other;   Physical Activity Interventions:   Behavioral: Physical Activity Lifestyle Intervention;   Behavioral: Physical Activity Monitoring Sponsor:   Jamie Jackson Enrolling by invitation
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Authors: Lucreziotti S, Persampieri S, Belletti S, Carugo S Abstract PMID: 31633316 [PubMed - as supplied by publisher]
Source: Minerva Cardioangiologica - Category: Cardiology Tags: Minerva Cardioangiol Source Type: research
CLEVELAND – Two words dominated much of the program agenda for Cleveland Clinic’s 2019 Medical Innovation Summit (MIS) on Tuesday – Artificial Intelligence. From a keynote address from Craig Mundie, Senior Advisor to the CEO, and former Chief Research and Strategy Officer, Microsoft to a session discussing companies with non-traditional approaches to healthcare, the topic of AI was discussed. But is AI just the latest fad in healthcare? Is it fully-defined? And are there real-world examples to look to, to see AI in action? Richard Zane, MD CIO of University of Colorado ...
Source: MDDI - Category: Medical Devices Authors: Tags: Digital Health Source Type: news
We report a case of multifocal lymphangioendotheliomatosis with thrombocytopenia in a male infant with isolated GI involvement, diagnosed when he was 3 months old. The patient was managed with daily aminocaproic acid, octreotide drip, and corticosteroids for 13 months after diagnosis; he had complete resolution of symptoms by 2 years of age and showed adequate height and gain by 5 years of age. This case adds to the paucity of data in the literature pertaining to the disease’s phenotypic variability, long-term clinical course, and management of GI bleeding.
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research
By Mark HurleyWASHINGTON DC, Oct 23 2019 (IPS) New information published in the Morbidity and Mortality Weekly Report shows that action taken by just 11 countries – most of them low- or middle-income – has resulted in 20 million fewer adult tobacco users in 2017 compared with 2008. Seventy percent of the world’s tobacco users live in low- and middle-income countries. This promising progress is a testament to strong government action and its effects are far reaching. Between 2008 and 2017, over 53 million fewer adults were exposed to secondhand smoke in indoor public places like restaurants, government bu...
Source: IPS Inter Press Service - Health - Category: International Medicine & Public Health Authors: Tags: Development & Aid Editors' Choice Featured Global Global Governance Headlines Health Inequity IPS UN: Inside the Glasshouse Poverty & SDGs TerraViva United Nations Source Type: news
CHICAGO (AP) — Two centuries after its invention, the stethoscope — the very symbol of the medical profession — is facing an uncertain prognosis. It is threatened by hand-held devices that are also pressed against the chest but rely on ultrasound technology, artificial intelligence and smartphone apps instead of doctors’ ears to help detect leaks, murmurs, abnormal rhythms and other problems in the heart, lungs and elsewhere. Some of these instruments can yield images of the beating heart or create electrocardiogram graphs. Dr. Eric Topol, a world-renowned cardiologist, considers the stethoscope obs...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized technology Source Type: news
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
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