MyoKardia ’s Phase II trial for oHCM drug meets endpoints

MyoKardia has reported positive top-line results from the first cohort of its Phase II PIONEER-HCM clinical trial of mavacamten in patients with symptomatic, obstructive hypertrophic cardiomyopathy (oHCM).
Source: Drug Development Technology - Category: Pharmaceuticals Source Type: news

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The letter by Dr Srilakshmi is a thoughtful representation of the current challenges we are all facing in treating patients with hypertrophic cardiomyopathy (HCM). These patients, when diagnosed or become symptomatic, pose a unique challenge in achieving better control of symptoms and minimizing the risk of sudden death. The issue raised by Dr Srilakshmi of risk prediction to better guide the use of an automatic implantable cardioverter-defibrillator is an interesting one. However, it remains theoretical only because no risk prediction model is perfect, let  alone one with such a small number of patients available.
Source: The Journal of Thoracic and Cardiovascular Surgery - Category: Cardiovascular & Thoracic Surgery Authors: Tags: Adult: Other: Letter to the Editor Source Type: research
We read the case series on electrical storms in patients with hypertrophic cardiomyopathy (HCM) with midventricular obstruction and apical aneurysm.1
Source: The Journal of Thoracic and Cardiovascular Surgery - Category: Cardiovascular & Thoracic Surgery Authors: Tags: Adult: Other: Letter to the Editor Source Type: research
In conclusion, the db/db mouse model recapitulates functional and histological features of human HFpEF associated with metabolic dysfunction. Development of fibrosis in db/db hearts, in the absence of myofibroblast conversion suggests that metabolic dysfunction may activate an alternative pro-fibrotic pathway associated with accentuated extracellular matrix protein synthesis. PMID: 30004258 [PubMed - as supplied by publisher]
Source: American Journal of Physiology. Heart and Circulatory Physiology - Category: Physiology Authors: Tags: Am J Physiol Heart Circ Physiol Source Type: research
ski A PMID: 29984817 [PubMed - in process]
Source: Polish Heart Journal - Category: Cardiology Authors: Tags: Kardiol Pol Source Type: research
Publication date: September 2018Source: Clinica Chimica Acta, Volume 484Author(s): Akiomi Yoshihisa, Takatoyo Kiko, Takamasa Sato, Masayoshi Oikawa, Atsushi Kobayashi, Yasuchika TakeishiAbstractThe differential diagnosis of cardiomyopathy is important. It has been recently reported that urinary titin N (U-TN) is increased in patients with muscular dystrophy (MD), and is associated with muscular damage. We aimed to clarify whether U-TN is useful as a diagnostic tool for distinguishing MD from various cardiomyopathies [e.g. dilated cardiomyopathy (DCM), hypertrophic cardiomyopathy (HCM)]. We measured and compared the U-TN/cr...
Source: Clinica Chimica Acta - Category: Laboratory Medicine Source Type: research
In conclusion, HIMF has a critical role in the development of cardiac hypertrophy, and targeting HIMF may represent a potential therapeutic strategy.
Source: Hypertension - Category: Cardiology Authors: Tags: Heart Failure, Hypertrophy Original Articles Source Type: research
Publication date: June 2018Source: Molecular Genetics and Metabolism Reports, Volume 15Author(s): Zuhair N. Al-Hassnan, Ola A. Khalifa, Dalal K. Bubshait, Sahar Tulbah, Maarab Alkorashy, Hamad Alzaidan, Mohammed Alowain, Zuhair Rahbeeni, Moeen Al-SayedAbstractInfantile-Onset Pompe Disease (IOPD) is an autosomal recessive disorder of glycogen metabolism resulting from deficiency of the lysosomal hydrolase acid α-glucosidase encoded by GAA gene. Affected infants present before the age of 12 months with hypotonia, muscle weakness, and hypertrophic cardiomyopathy. Enzyme replacement therapy (ERT) has been shown to impr...
Source: Molecular Genetics and Metabolism Reports - Category: Genetics & Stem Cells Source Type: research
We describe two brothers exhibiting the GLA p.N215S mutation, a variant most often conferring a late-onset disease confined to the myocardium.The proband was diagnosed aged 34, following investigation into proteinuria.Despite Enzyme Replacement Therapy, he progressed to end-stage renal failure, and subsequently received a renal transplant. He also developed hypertrophic cardiomyopathy.His sibling however, whose disease was detected aged 32 following screening, exhibits mild left ventricular hypertrophy, and no evidence of renal disease. He remains clinically asymptomatic.This case report details a discordant phenotype in b...
Source: Molecular Genetics and Metabolism Reports - Category: Genetics & Stem Cells Source Type: research
Publication date: Available online 2 June 2018Source: The Kaohsiung Journal of Medical SciencesAuthor(s): Ke Han, Yan-Ping LiAbstractHypertrophic cardiomyopathy (HCM) is the most common genetic cardiac disease and is an important cause of sudden death in patients of all ages. The aim of this study was to find out whether Toll-like receptor-4 (TLR4) polymorphism is associated with HCM. To explore the association between TLR4 gene polymorphisms and HCM, 486 HCM patients and 214 healthy controls were enrolled in a case–control study of Chinese Han population. Two single nucleotide polymorphisms (SNPs) in the promoter re...
Source: The Kaohsiung Journal of Medical Sciences - Category: Universities & Medical Training Source Type: research
Publication date: July 2018Source: Journal of Comparative Pathology, Volume 162Author(s): T.A. Donovan, N. Balakrishnan, I. Carvalho Barbosa, T. McCoy, E.B. Breitschwerdt, P.R. FoxSummaryEndomyocarditis is a commonly detected post-mortem finding in domestic cats presenting for sudden onset cardiovascular death, yet the aetiology remains unresolved. Cats are documented reservoir hosts for Bartonella henselae, the infectious cause of cat scratch disease in man. Various Bartonella spp. have been associated with culture-negative endocarditis, myocarditis and sudden death in man and animals. We hypothesized that Bartonella spp....
Source: Journal of Comparative Pathology - Category: Pathology Source Type: research
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