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After 10 months, Alnylam still can't explain patient trial deaths

More than 10 months after disclosing that patients who received one of its experimental drugs in a late-stage trial had died at a far higher rate than those on placebo, Cambridge-based Alnylam Pharmaceuticals still cannot conclusively explain what went wrong. Alnylam (Nasdaq: ALNY) on Wednesday released the results of its investigation into the deaths of older patients with heart problems who took revusiran, a potential treatment for a deadly inherited disease called hATTR cardiomyopathy. In October,…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news

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This study explored experiences of HCM genetic testing to identify potential benefits and harms. Thirty-two individuals previously offered genetic testing for HCM were recruited. Semi-structured int erviews were conducted face-to-face or by phone, and transcribed audio-recordings were coded using framework analysis. Key themes were as follows: (1) helping the next generation, (2) misunderstanding risk, (3) discrepancy between actual/perceived impact. Participants described multiple psychologica l (shock, worry, uncertainty) and behavioural (career, sport, insurance, family planning) consequences, depending on perceived ris...
Source: Journal of Genetic Counseling - Category: Genetics & Stem Cells Source Type: research
Source: BJOG: An International Journal of Obstetrics and Gynaecology - Category: OBGYN Authors: Tags: BJOG Exchange Source Type: research
Written by Pendell Meyers, edits by Steve SmithThis will be too easy for most long-time readers, but if you are at that level, sit back and enjoy noticing how few milliseconds it takes to recognize this thanks to so many prior examples on this blog!I was sent this ECG from EMS with only the information that it belonged to a middle aged male with left shoulder pain.What do you think?There are hyperacute T-waves in leads V1-V6, as well as in leads II, III, aVF. The J-points are all at baseline with the exception of leads V2-V3 which show a small amount of STD (which makes de Winter morphology in the presence of hyperacute T-...
Source: Dr. Smith's ECG Blog - Category: Cardiology Authors: Source Type: blogs
Publication date: Available online 21 February 2018 Source:Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease Author(s): Yang Xiao, Qing Qing Wu, Ming Xia Duan, Chen Liu, Yuan Yuan, Zheng Yang, Hai Han Liao, Di Fan, Qi Zhu Tang Diabetic cardiomyopathy is associated with suppressed autophagy and augmented inflammation in the heart. The effects of Tax1 binding protein 1 (TAX1BP1) on both autophagy and inflammation suggest that it may participate in the progression of diabetic cardiomyopathy. Mice were injected with streptozotocin (STZ) to induce experimental diabetes. An adenovirus system was used to induce he...
Source: Biochimica et Biophysica Acta (BBA) Molecular Basis of Disease - Category: Molecular Biology Source Type: research
Authors: Wang L, Liu S, Zhang H, Hu S, Wei Y Abstract Arrhythmogenic cardiomyopathy (AC) is an inherited disorder that is predominantly present in the right ventricular myocardium. Mutations in the genes encoding the desmosomal protein are thought to underlie the pathogenesis of AC. Since AC is genetically heterogeneous and phenotypically diverse, modifier genes and environmental factors have an important role in disease expression. The aim of the present study was to identify AC-associated desmosomal gene variations, and examine the expression levels of intercalated disc proteins in AC patients who carry the varia...
Source: Experimental and Therapeutic Medicine - Category: General Medicine Tags: Exp Ther Med Source Type: research
AbstractBackgroundThe effect of levocarnitine supplementation has not been evaluated in children with dilated cardiomyopathy (DCM).ObjectiveThe aim of this study was to explore the effect of oral levocarnitine supplementation in pediatric patients with DCM.MethodsTwenty-nine children with DCM (17 male, 12 female, aged 1  month to 13 years) were divided into two groups according to a simple randomization: control group (n = 10) and experimental group (n = 19). All children were given oral hydrochlorothiazide, enalapril, and spironolactone; additionally, patients with cardiac function of NYHA gr...
Source: Pediatric Drugs - Category: Pediatrics Source Type: research
We present a case of a 61-year-old man with ischemic cardiomyopathy receiving sacubitril/valsartan (Entresto; Novartis, Cambridge, MA) who developed profound hypotension after HT. He was treated with intravenous methylene blue and high-dose vasopressors, but developed acute kidney injury requiring dialysis and a prolonged stay in the intensive care unit. This case supports a potent vasodilatory effect of sacubitril/valsartan, and if confirmed by other studies, might warrant consideration for withholding treatment while awaiting HT, particularly in patients with risk factors for vasoplegia.
Source: Canadian Journal of Cardiology - Category: Cardiology Source Type: research
SummarySurvival after childhood cancer has improved substantially; therefore, the number of childhood cancer survivors is increasing. This growing population of childhood cancer survivors, however, is at risk of a  spectrum of adverse health outcomes. Unfortunately, until now, there was a lack of comprehensive follow-up recommendations. The purpose of this article is to provide information on recently developed harmonized evidence-based guidelines on surveillance investigations to screen for the early detec tion of breast cancer, cardiomyopathy, male gonadotoxicity, and premature ovarian failure in childhood canc...
Source: Memo - Magazine of European Medical Oncology - Category: Cancer & Oncology Source Type: research
Conclusions Contemporary HCM in Portugal is characterized by relatively advanced age at diagnosis, and a high proportion of invasive treatment of obstructive forms. Long-term mortality is low; heart failure is the most common cause of death followed by sudden cardiac death. However, the burden of morbidity remains considerable, emphasizing the need for disease-specific treatments that impact the natural history of the disease.
Source: Revista Portuguesa de Cardiologia - Category: Cardiology Source Type: research
Publication date: January 2018 Source:Revista Portuguesa de Cardiologia (English Edition), Volume 37, Issue 1 Author(s): Gláucia Maria Moraes de Oliveira
Source: Revista Portuguesa de Cardiologia - Category: Cardiology Source Type: research
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