MyoKardia begins dosing in Phase I trial of MYK-491 to treat DCM

US-based clinical stage biopharmaceutical firm MyoKardia has begun dosing in its Phase I single ascending dose clinical study of MYK-491 to treat dilated cardiomyopathy (DCM).
Source: Drug Development Technology - Category: Pharmaceuticals Source Type: news

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Abstract Hypereosinophilic syndrome is characterized by sustained and marked eosinophilia leading to tissue damage and organ dysfunction. Morbidity and mortality occur primarily due to cardiac and thromboembolic complications. Understanding the cause and mechanism of disease would aid in the development of targeted therapies with greater efficacy and fewer side effects. We discovered a spontaneous mouse mutant in our colony with a hypereosinophilic phenotype. Mice develop peripheral blood eosinophilia, infiltration of lungs, spleen and heart by eosinophils, and extensive myocardial damage and remodeling. This ulti...
Source: American Journal of Physiology. Heart and Circulatory Physiology - Category: Physiology Authors: Tags: Am J Physiol Heart Circ Physiol Source Type: research
AbstractBackgroundThe benefits of implantable cardioverter ‐defibrillators (ICDs) for the prevention of sudden cardiac arrest (SCA) are well established. However, a significant knowledge gap remains regarding current indications and utilization of ICDs in real‐world settings in Asia.MethodsPatients who underwent ICD implantation in South Korea from 2007 to 2015 were identified using the Health Insurance Review and Assessment Service database. We investigated trends in use of ICD for prevention of SCA.ResultsA total of 4,649 ICDs were implanted during the 9 years. ICDs were implanted in 1,448 (31.2%) patients for primar...
Source: Pacing and Clinical Electrophysiology : PACE - Category: Cardiology Authors: Tags: DEVICES Source Type: research
The diagnosis of LGMD2A (calpainopathy) can be challenging due to genetic heterogeneity and to high similarity with other LGMDs or neuromuscular disorders. In this setting, NGS panels are highly recommended to perform differential diagnosis, identify new causative mutations and enable genotype-phenotype correlations. In this manuscript, the case of a patient affected by LGMD2A is reported, for which the application of a defined custom designed NGS panel allowed to confirm the diagnosis of calpainopathy linked with two heterozygous variants in CAPN3, namely c.550delA and c.1813G>C. The first variant has been extensively ...
Source: Frontiers in Neurology - Category: Neurology Source Type: research
Conclusion: In patients with DCM and ICD, genetic variants could be identified in a significant proportion of patients in several genes, highlighting the potential role of genetics in DCM SCD risk stratification. PMID: 31179125 [PubMed]
Source: Cardiology Research and Practice - Category: Cardiology Authors: Tags: Cardiol Res Pract Source Type: research
Conclusion: AHA have the greatest significance in the diagnosis of latent myocarditis in patients with “idiopathic” arrhythmias and DCM. The use of a complex of noninvasive criteria allows the probability of myocarditis to be estimated and the indications for EMB to be determined.Cardiology
Source: Cardiology - Category: Cardiology Source Type: research
ConclusionsML effectively predicted risk in patients with severe DCM in 1-year follow-up, and this may direct risk stratification and patient management in the future.
Source: European Journal of Radiology - Category: Radiology Source Type: research
Conclusion The exhaustive systematic evaluation of ECG has an incremental impact in the prognostication of a large cohort of DCM patients, also regarding the arrhythmic stratification.
Source: Journal of Cardiovascular Medicine - Category: Cardiology Tags: Research articles: Heart failure Source Type: research
This article is part of a Special Issue entitled: Stem Cells and Their Applications to Human Diseases edited by Hemachandra Reddy.
Source: Biochimica et Biophysica Acta (BBA) Molecular Basis of Disease - Category: Molecular Biology Source Type: research
This study aims to review the aetiology and associated outcomes of DCM. The median age at diagnosis was 6  months (0–42 months);n = 23 (43.3%) were idiopathic;n = 11 (20.9%) secondary to a viral infection;n = 12 (22.6%) genetic disorders andn = 7 (13.2%) as a result of vitamin D deficiency. There was a significant correlation between aetiology and mortality,r = 0.85, with a lower survival rate in idiopathic and genetic cohorts. Males were significantly less likely to survive to 1 year of age,p = 0.035. The age at diagno...
Source: European Journal of Pediatrics - Category: Pediatrics Source Type: research
Conclusion: Heterozygous Mylk3 knockout mice show mild reduction of cardiac contractility by 4 months of age, and proteins reduced by approximately 75% relative to the control wild-type mice. These mice partly resemble human with the heterozygous MYLK3 mutation, but the reduction in cardiac contractility was milder.
Source: Frontiers in Physiology - Category: Physiology Source Type: research
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