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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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PMID: 28819957 [PubMed - in process]
Source: Polish Heart Journal - Category: Cardiology Authors: Tags: Kardiol Pol Source Type: research
AbstractFor the past few decades, the transaortic septal myectomy (Morrow ’s procedure) has been the gold standard for treating severe left ventricular outflow tract obstruction in hypertrophic obstructive cardiomyopathy (HOCM) patients. 30-day mortality has been reported at less than 1% in dedicated centers. However, in a subgroup of patients, the interventricular sept al obstruction is localized very distally, below the aortic valve plane, and the transaortic approach can be very challenging. A subset of these patients can present with residual obstruction after surgery, due to inadequate length of septal excision,...
Source: General Thoracic and Cardiovascular Surgery - Category: Cardiovascular & Thoracic Surgery Source Type: research
Condition:   Hypertrophic Cardiomyopathy Intervention:   Device: Insertable cardiac monitor Sponsors:   Region Gävleborg;   St. Jude Medical;   Norrlands University Hospital Recruiting - verified August 2017
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
We report the clinical, morphological and molecular features of two patients with autosomal recessive SLC25A4 (ANT1) gene mutations. Furthermore, all previously published cases are reviewed to identify valuable features for future diagnosis. Patients present a common phenotype with exercise intolerance, hyperlactatemia, and hypertrophic cardiomyopathy. Muscle biopsies show wide sub-sarcolemmal mitochondrial aggregates, and increased activities of all respiratory chain complexes. The phenotype of recessive SLC25A4 (ANT1) mutations although rare, is homogenous and easily recognizable and could help orientate the molecular an...
Source: Mitochondrion - Category: Biochemistry Source Type: research
Authors: Brochado B, Cabral S, Pinheiro-Vieira A, Carvalho H, Torres S PMID: 28803523 [PubMed - as supplied by publisher]
Source: Acta Cardiologica - Category: Cardiology Tags: Acta Cardiol Source Type: research
Condition:   Cardiomyopathy, Hypertrophic Interventions:   Drug: Sodium Nitrate;   Drug: Placebo;   Diagnostic Test: Phosphorous Magnetic Resonance Spectroscopy;   Diagnostic Test: Exercise Stress Transthoracic Echocardiogram Sponsors:   University of East Anglia;   Norfolk and Norwich University Hospitals NHS Foundation Trust;   British Medical Research Council Recruiting - verified August 2017
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
The objectives of this article are to review the genetic basis for HCM and discuss clinical presentations of HCM in adolescents, so that general practitioners: develop confidence in requesting investigations in adolescents with suspected or proven HCM consider early referral to a paediatric cardiology department for any adolescent with left ventricular hypertrophy understand family screening guidelines for HCM. DISCUSSION: HCM is a complex cardiac disease with marked heterogeneity. Management strategies should be individually tailored, including avoidance of competitive sports, but encouragement of lower intensity phy...
Source: Australian Family Physician - Category: Primary Care Authors: Tags: Aust Fam Physician Source Type: research
Conclusions— Men with classic or later-onset FD caused by GLA missense mutations developed prominent and similar cardiovascular disease at similar ages, despite markedly different α-GalA activities.
Source: Circulation: Cardiovascular Genetics - Category: Cardiology Authors: Tags: Genetics, Cardiomyopathy, Hypertrophy Original Articles Source Type: research
Publication date: Available online 10 August 2017 Source:Revista Española de Cardiología (English Edition) Author(s): Ares Pasipoularides
Source: Revista Espanola de Cardiologia - Category: Cardiology Source Type: research
Source: Clinical Epidemiology - Category: Epidemiology Tags: Clinical Epidemiology Source Type: research
More News: Cardiology | Cardiomyopathy | Clinical Trials | Heart | Hypertrophic Cardiomyopathy | Pharmaceuticals