Vertex’s cystic fibrosis treatment makes it a hot company

Biotech group’s promising results help it defy broader investor fears over sector
Source: FT.com - Drugs and Healthcare - Category: Pharmaceuticals Source Type: news

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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
In this study, the aim was the development of LVX-loaded PLGA microspheres (MS) for pulmonary administration as a dry powder. MS were prepared, for the first time, by a modified double emulsion solvent evaporation method with premix membrane homogenization. Aqueous phases were saturated with LVX and a fatty acid (lauric acid) was added to avoid the drug escaping from the organic phase. MS were characterized in terms of size, drug content, morphology and in vitro release properties. X-ray diffraction, Fourier-transform infrared spectroscopy, differential and gravimetric thermal analysis, and cytotoxicity analyses were perfo...
Source: International Journal of Pharmaceutics - Category: Drugs & Pharmacology Source Type: research
STRUCTURE, GATING, AND REGULATION OF THE CFTR ANION CHANNEL. Physiol Rev. 2019 Jan 01;99(1):707-738 Authors: Csanády L, Vergani P, Gadsby DC Abstract The cystic fibrosis transmembrane conductance regulator (CFTR) belongs to the ATP binding cassette (ABC) transporter superfamily but functions as an anion channel crucial for salt and water transport across epithelial cells. CFTR dysfunction, because of mutations, causes cystic fibrosis (CF). The anion-selective pore of the CFTR protein is formed by its two transmembrane domains (TMDs) and regulated by its cytosolic domains: two nucleotide binding...
Source: Physiological Reviews - Category: Physiology Authors: Tags: Physiol Rev Source Type: research
Condition:   Cystic Fibrosis Interventions:   Drug: VX-121;   Drug: TEZ/IVA;   Drug: IVA;   Drug: Matched Placebo Sponsor:   Vertex Pharmaceuticals Incorporated Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Inhalation of hypertonic saline improves lung function and growth in infants with cystic fibrosis (CF), according to the results of the PRESIS randomized controlled trial.Reuters Health Information
Source: Medscape Pathology Headlines - Category: Pathology Tags: Pulmonary Medicine News Source Type: news
Nikhil T. Awatade, Sharon L. Wong, Chris K. Hewson, Laura K. Fawcett, Anthony Kicic, Adam Jaffe, Shafagh A. Waters
Source: Frontiers in Pharmacology - Category: Drugs & Pharmacology Source Type: research
Cystic fibrosis (CF) is a genetic disease characterized by chronic inflammation of the lungs that is ineffective at clearing pathogens. B-cell activating factor (BAFF), a cytokine involved in the development of B-cells, is known to be elevated in CF patients with subclinical infections. We postulate that the elevated BAFF levels in CF patients might be triggered by Pseudomonas aeruginosa infection and it might play a protective role in the regulation of lung responses to infection.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Source Type: research
Genetic counseling for cystic fibrosis (CF) is challenged by intricate molecular mechanisms, complex phenotypes, and psychosocial needs. CFTR variant interpretation has become critical; this manuscript examines variant nomenclature and classes, as well as opportunities and challenges posed by genetic technologies and genotype-directed therapies. With post-graduate training in medical genetics and counseling, genetic counselors educate patients and families, facilitate testing and interpretation, and help integrate genetic information into diagnosis and treatment.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Review Source Type: research
Cystic fibrosis (CF) is a recessive genetic disease that is characterised by airway mucus plugging and reduced mucus clearance. There are currently alternative hypotheses that attempt to describe the abnormally viscous and elastic mucus that is a hallmark of CF airways disease, including: 1) loss of CF transmembrane regulator (CFTR)-dependent airway surface volume (water) secretion, producing mucus hyperconcentration-dependent increased viscosity, and 2) impaired bicarbonate secretion by CFTR, producing acidification of airway surfaces and increased mucus viscosity. A series of experiments was conducted to determine the co...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: CF and non-CF bronchiectasis Original Articles: Cystic fibrosis Source Type: research
Cystic fibrosis (CF) is associated with the production of viscous and adherent mucus in the airway. Indeed, in some countries, CF is still called "mucoviscidosis". Mucus is a weak hydrogel exhibiting viscoelastic properties and these properties are important for normal function as they enable mucus to flow after secretion, to be effectively propelled by beating cilia (mucociliary clearance), and to provide a protective coating of the mucosal surfaces of our body that prevents dehydration and an exposure to external foreign substances and infectious agents. Mucus that is too thick or too thin can be difficult to t...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Editorials Source Type: research
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