A Phase I Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of CT-G20 in Subjects With Obstructive Hypertrophic Cardiomyopathy
Condition: Cardiomyopathy, Hypertrophic Obstructive Interventions: Drug: CT-20; Drug: Placebo Sponsor: Celltrion Recruiting
Fabry cardiomyopathy can cause symptomatic left ventricular outflow tract obstruction. We review a case of Fabry cardiomyopathy mimicking hypertrophic cardiomyopathy on echocardiography with severe left ventricular outflow tract obstruction treated with ventricular septal myectomy.
ConclusionsThe presentation of our patients raises the possibility that hypertrophic cardiomyopathy may be an additional feature of the clinical syndrome associated withUCHL1 mutations, and highlights the importance of cardiac follow-up and treatment in neurodegenerative disease associated withUCHL1 mutations.
ConclusionASA has been shown to be a safe and reliable procedure; imaging techniques and dedicated multi ‐disciplinary teams can be used to select patients with HOCM. Though SM is recommended as gold standard treatment for drug refractory HOCM, ASA may play an increasing role in the near future due an ageing population; both ASA and SM can have a beneficial role in treating those who are affected by HOCM when the appropriate group of patients are selected for each intervention.
ConclusionThese preliminary data suggest that NOACs are at least as safe and effective as VKAs in patients with HCM undergoing catheter ablation for AF.This article is protected by copyright. All rights reserved.
To evaluate whether the Alfieri technique improves clinical and haemodynamic results and compare it with transaortic mitral valve secondary cord cutting in patients scheduled for septal myectomy for severely symptomatic hypertrophic obstructive cardiomyopathy.
AbstractObjectivesWe assessed whether an association exists between myocardial oxygenation and myocardial fibrosis in patients with hypertrophic cardiomyopathy (HCM), using blood-oxygen-level-dependent (BOLD) T2* cardiac magnetic resonance imaging (T2*-CMR) and T1 mapping.MethodsT1 mapping and T2*-CMR data were collected from 55 HCM patients using a 3-T MR and were prospectively analyzed. T2*-CMR was conducted using the black blood, breath-hold, multi-echo, and gradient echo sequence. Over 10 min, inhalation of oxygen at the flow rate of 10 L/min, T2* for mid-septum was measured following room-air and oxygen inh...
-Poyatos HM PMID: 32646796 [PubMed - as supplied by publisher]
Conclusions: Our study revealed the human cardiac pathogenesis in patient-specific iPSC-CMs from NS patients carrying biallelic variants in LZTR1 and identified a unique disease-specific proteome signature. In addition, we identified the intronic CRISPR repair as a personalized and in our view clinically translatable therapeutic strategy to treat NS-associated HCM. PMID: 32623905 [PubMed - as supplied by publisher]
AbstractChildren at high risk for sudden cardiac death (SCD) receive implantable cardioverter-defibrillators (ICD) for prevention, but the cost effectiveness of ICDs in children at intermediate risk is unclear. Our objective was to create a cost-effectiveness model to compare costs and outcomes in children at risk of SCD, with and without ICD. Utilizing hypertrophic cardiomyopathy as the proxy disease, a theoretical cohort of 8150 children was followed for 69 years. Model inputs were derived from the literature, with an incremental cost-effectiveness ratio (ICER) willingness-to-pay threshold of $100,000/quality-adjus...