Cystic Fibrosis boy gets new drug after NHS discount deal

Negotiations with US pharmaceutical company means that some 5,000 NHS patients will benefit Related items fromOnMedica Drug firm urged to cut £100,000 price of cystic fibrosis drug Life-changing cystic fibrosis drug cost 'should be lowered' Health-focused line on drugs misuse would save lives and money TB could be cured by four months ’ treatment in many patients Statins of small and uncertain benefit in primary prevention
Source: OnMedica Latest News - Category: UK Health Source Type: news

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Publication date: Available online 13 February 2020Source: Pharmacology &TherapeuticsAuthor(s): Kylie B.R. Belchamber, Louise E. DonnellyAbstractChronic pulmonary conditions now account for 1 in 15 deaths in the US and mortality is increasing. Chronic obstructive pulmonary disease (COPD) is due to become the 3rd largest cause of mortality by 2030 and mortality from other respiratory conditions such as asthma, idiopathic pulmonary fibrosis and cystic fibrosis are not reducing. There is an urgent need for novel therapies to address this problem as many of the current strategies targeting inflammation are not sufficient. ...
Source: Pharmacology and Therapeutics - Category: Drugs & Pharmacology Source Type: research
Publication date: Available online 8 January 2020Source: Diabetes &Metabolic Syndrome: Clinical Research &ReviewsAuthor(s): Zahra Gorji, Mohammadreza Modaresi, Saeed Yekanni-Nejad, Maryam MahmoudiPurposeLow glycemic index diets seem to be potentially effective to improve glycemic control and reduce lipid profiles. Hence, this study aimed to evaluate the effect of a low glycemic index/high fat, high-calorie diet on glycemic status and lipid profiles of patients with cystic fibrosis.MethodsIn this randomized clinical trial, 44 children and adolescents with cystic fibrosis were randomized to receive for three months e...
Source: Diabetes and Metabolic Syndrome: Clinical Research and Reviews - Category: Endocrinology Source Type: research
In the article “The Relative Contribution of Food Groups to Macronutrient Intake in Children with Cystic Fibrosis: A European Multicenter Assessment” by Calvo-Lerma and colleagues published in the August 2019 issue of the Journal of the Academy of Nutrition and Dietetics (pp 1305-1319), Table 3 on p 1310 stat es “Cholesterol” as a variable. This should read “Carbohydrate”, not cholesterol. The authors regret this error.
Source: Journal of the American Dietetic Association - Category: Nutrition Tags: From the Academy Source Type: research
Authors: Liu X Abstract The transport of specific molecules across lipid membranes is an essential function of all living organisms. The processes are usually mediated by specific transporters. One of the largest transporter families is the ATP-binding cassette (ABC) family. More than 40 ABC transporters have been identified in human, which are divided into 7 subfamilies (ABCA to ABCG) based on their gene structure, amino acid sequence, domain organization, and phylogenetic analysis. Of them, at least 11 ABC transporters including P-glycoprotein (P-GP/ABCB1), multidrug resistance-associated proteins (MRPs/ABCCs), a...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
Cosmeri Rizzato1, Javier Torres2, Elena Kasamatsu3, Margarita Camorlinga-Ponce2, Maria Mercedes Bravo4, Federico Canzian5 and Ikuko Kato6* 1Department of Translation Research and of New Technologies in Medicine and Surgery, University of Pisa, Pisa, Italy 2Unidad de Investigación en Enfermedades Infecciosas, Unidades Médicas de Alta Especialidad Pediatría, Instituto Mexicano del Seguro Social, Mexico City, Mexico 3Instituto de Investigaciones en Ciencias de la Salud, National University of Asunción, Asunción, Paraguay 4Grupo de Investigación en Biología del C&aacut...
Source: Frontiers in Microbiology - Category: Microbiology Source Type: research
Abstract The use of high-dose ibuprofen as an anti-inflammatory therapy in cystic fibrosis has been shown to be an effective intervention though use is limited due to potential adverse events. Identifying the mechanism of ibuprofen efficacy would aid in the development of new therapies that avoid these adverse events. Previous findings demonstrated that ibuprofen treatment restores the regulation of microtubule dynamics in cystic fibrosis (CF) epithelial cells through a 5' adenosine monophosphate-activated protein kinase (AMPK)-dependent mechanism. The goal of this study is to define the AMPK pathway that leads to...
Source: American Journal of Physiology. Lung Cellular and Molecular Physiology - Category: Cytology Authors: Tags: Am J Physiol Lung Cell Mol Physiol Source Type: research
In this study, we hypothesize that acetyl CoA carboxylase (ACC) is an important intermediate in Cystic fibrosis (CF) inflammatory signaling cascade. Here, we demonstrate that ACC inhibition mimics the cellular effects of ibuprofen promoting both redistribution of intracellular cholesterol and increased rates of microtubule reformation, while decreasing RhoA expression in CF epithelial cell models. Inhibiting ACC polymerization with citrate increases RhoA expression and decreases microtubule reformation rates in wild-type epithelial cells, suggesting pro-inflammatory signaling. Together, these findings demonstrate a novel m...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Homo sapiens Source Type: research
The USA has the highest drug costs in the world, and they are increasing at the fastest rate too. With one in five Americans saying they have been forced to leave a prescription unfilled due to high costs, the situation is clearly unsustainable.Payers and pharmacy benefit managers (PBMs), themselves facing increased criticism for blocking access to important medicines, are taking action by demanding that pharma companies justify the cost – and value – of their new medicines.“There is a point where a government, an employer or a healthcare plan has a finite amount of resources to spend,” says Brian H...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news
Previous studies have demonstrated that CF epithelial cells exhibit increased cholesterol content at the plasma membrane compared to wild type controls as measured by electrochemical methods. Microtubule dysregulation that impacts intracellular transport has also been identified in CF cells and is reversible with histone deacetylase 6 (HDAC6) inhibition, a regulator of tubulin acetylation. The hypothesis of this study is that increased membrane cholesterol content in CF cells is dependent on HDAC6 regulation.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Original Article Source Type: research
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Poster Sessions Source Type: research
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