Cystic fibrosis drugs that have the potential to save lives are rejected for use in Scotland

The Scottish Medicines Consortium have rejected Orkambi and Symkevi after hearing 'powerful testimonies'. Both drugs cost about £100,000 per year per patient that is eligible.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news

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Conclusions: Liver US patterns in children with CF correlate with platelet count, spleen size and indices of liver fibrosis. Multivariable models of these biomarkers have excellent discriminating ability for NL versus NOD, and good ability to distinguish other US patterns, suggesting that US patterns correlate with clinically relevant liver disease.
Source: Journal of Pediatric Gastroenterology and Nutrition - Category: Gastroenterology Tags: Original Articles: Hepatology Source Type: research
Guidelines recommend breastfeeding in cystic fibrosis (CF) but breastfeeding rates decline rapidly in CF. We initiated a quality improvement project to improve breastfeeding in CF by incorporating International Board-Certified Lactation Consultants (IBCLC) into the initial CF-diagnosis visit to support mothers who were breastfeeding at diagnosis. In the preintervention group, only 8/14 (57%) continued to provide breast milk after the first visit, whereas postintervention, 16/17 (94%) mothers continued to do so (P = 0.03). The duration of any (or partial) breastfeeding increased to an average of 7.7 months from an avera...
Source: Journal of Pediatric Gastroenterology and Nutrition - Category: Gastroenterology Tags: Short Communication: Pancreatology Source Type: research
Publication date: Available online 21 August 2019Source: Progress in Lipid ResearchAuthor(s): Dušan Garić, Juan B. De Sanctis, Juhi Shah, Daciana Catalina Dumut, Danuta RadziochAbstractCeramides, the principal building blocks of all sphingolipids, have attracted the attention of many scientists around the world interested in developing treatments for cystic fibrosis, the most common genetic disease of Caucasians. Many years of fruitful research in this field have produced some fundamentally important, yet controversial results.Here, we aimed to summarize the current knowledge on the role of long- and very-long- cha...
Source: Progress in Lipid Research - Category: Lipidology Source Type: research
There is a great deal of interest in the airway microbiota, its diversity and the role of specific microbial taxa in the pathophysiology of lung disease [1]. Non-cystic fibrosis bronchiectasis is a significant public health problem in many countries, including New Zealand, where prevalence is high and morbidity and mortality are substantial [2–4]. A role for bacteria in the pathophysiology of bronchiectasis is widely accepted but poorly characterised due to inherent difficulties with lower airway sampling, especially in young children. Culture-based studies demonstrate associations with Haemophilus influenzae, Moraxe...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Original Articles: Research letters Source Type: research
This report highlights the usefulness of bronchoscopy in case of recurrent pneumonia with the same localization even in CF patients where the presence of bronchiectasis as promoting factor of infections could delay the diagnosis. AbstractThis report highlights the usefulness of bronchoscopy in case of recurrent pneumonia with the same localization even in CF patients where the presence of bronchiectasis as promoting factor of infections could delay the diagnosis.
Source: Clinical Case Reports - Category: General Medicine Authors: Tags: CASE REPORT Source Type: research
Publication date: Available online 20 August 2019Source: Stem Cell ResearchAuthor(s): Lena Engels, Ruth Olmer, Jeanne de la Roche, Gudrun Göhring, Saskia Ulrich, Ralf Haller, Ulrich Martin, Sylvia MerkertAbstractCFTR encodes for a chloride ion channel expressed primarily in secretory epithelia in the airways, intestine, liver and other tissues. Mutations in the CFTR gene have been identified in people suffering from Cystic Fibrosis. Here, we established a CFTR knock-in reporter cell line from a human iPSC line (MHHi006-A) using TALEN technology. The reporter enables the monitoring and optimization of the differentiati...
Source: Stem Cell Research - Category: Stem Cells Source Type: research
Stenotrophomonas maltophilia is an emerging opportunistic and nosocomial pathogen. S. maltophilia is also a risk factor for lung exacerbations in cystic fibrosis patients. S. maltophilia attaches to various mammalian cells, and we recently documented that the bacterium encodes a type II secretion system which triggers detachment-induced apoptosis in lung epithelial cells. We have now confirmed that S. maltophilia also encodes a type IVA secretion system (VirB/VirD4 [VirB/D4] T4SS) that is highly conserved among S. maltophilia strains and, looking beyond the Stenotrophomonas genus, is most similar to the T4SS of Xanthomonas...
Source: Infection and Immunity - Category: Infectious Diseases Authors: Tags: Cellular Microbiology: Pathogen-Host Cell Molecular Interactions Source Type: research
The clinical effects of Aspergillus fumigatus in the cystic fibrosis (CF) airway, with the exception of allergic bronchopulmonary aspergillosis, is unclear.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Source Type: research
Soft Matter, 2019, Accepted Manuscript DOI: 10.1039/C9SM00371A, PaperFrancesca Zuttion, Delphine Sicard, Lucie Dupin, G érard Vergoten, Camille Girard-Bock, Mimouna Madaoui, Yann Chevolot, Francois Morvan, Sebastien Vidal, Jean-Jacques Vasseur, Eliane Souteyrand, Magali Phaner-Goutorbe Pseudomonas aeruginosa is a human opportunistic pathogen responsible for lung infections in cystic fibrosis patients. The emergence of resistant strains and its ability to form a biofilm seems to give... The content of this RSS Feed (c) The Royal Society of Chemistry
Source: RSC - Soft Matter latest articles - Category: Chemistry Authors: Source Type: research
PMID: 31430187 [PubMed - as supplied by publisher]
Source: American Journal of Rhinology and Allergy - Category: ENT & OMF Authors: Tags: Am J Rhinol Allergy Source Type: research
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