600 years ’ supply of cystic fibrosis drug destroyed in price row

8,000 packs of Orkambi go out of date during standoff between maker and NHSNearly 8,000 packs of Orkambi, the breakthrough medicine for cystic fibrosis, have been destroyed by the manufacturer while it has been in a stand-off with the NHS over the high price it wants to charge for the drug.The US company Vertex has distributed 80,000 packs of Orkambi around Europe from its base in the UK since the drug was licensed in 2015. Other European countries with smaller numbers of people affected by the genetic disease have agreed to a high price, sometimes in order to give access to patients before negotiating downwards.Continue reading...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Cystic fibrosis Society NHS Health Drugs Science UK news Source Type: news

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The U.S. Food and Drug Administration approved Vertex Pharmaceuticals Inc's combination treatment for a rare condition called cystic fibrosis, the agency said on Monday.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Boston-based Vertex Pharmaceuticals Inc. has achieved one of the biggest feats to date for cystic fibrosis patients — a treatment that could benefit 90 percent of patients with the rare lung disease. The FDA approved a combination of three drugs Monday called Trikafta, which targets a mutation in the F508del gene. That genetic error is found in approximately 90 percent of patients with the rare lung disease. The drug is set to be a blockbuster product for Vertex, which makes billions annually…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
CONCLUSION: A substantial proportion of infants diagnosed with CF after detection by NBS already showed evidence of lung disease. P. aeruginosa colonization was associated with increas ed Bhalla scores, highlighting the importance of this CF pathogen in early structural lung disease. The presence of bronchial wall thickening at such a young age may reflect the presence of airway inflammatory processes. The detection and quantification of structural abnormalities with the modified Bhalla score may aid in the identification of lung disease before it is clinically apparent.
Source: Clinics - Category: General Medicine Source Type: research
Review clinical reference information, guidelines, and medical news on cystic fibrosis--cystic fibrosis symptoms and cystic fibrosis treatment. Review causes of cystic fibrosis and cystic fibrosis carrier information.
Source: Medscape Today Headlines - Category: Consumer Health News Tags: Resource Center Source Type: news
Condition:   Cystic Fibrosis Interventions:   Behavioral: Focus group;   Behavioral: Individual interview Sponsor:   Assistance Publique - Hôpitaux de Paris Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Authors: Paterson SL, Barry PJ, Horsley AR Abstract Introduction: Cystic fibrosis (CF) is a complex, multi-system, genetic disease affecting over 70,000 people worldwide. The underlying defect is a mutation in the CFTR gene. Dysfunctional CFTR protein results in abnormal anion movement across epithelial membranes in affected organs. There has been a paradigm shift in CF treatment over the last decade with the advent of CFTR modulation, treatments which target this underlying genetic defect and have the potential to change the course of CF clinical disease. Areas covered: Available CFTR modulators in current clinica...
Source: Expert Review of Respiratory Medicine - Category: Respiratory Medicine Tags: Expert Rev Respir Med Source Type: research
Background: In US cystic fibrosis (CF) patients, methicillin-resistant Staphylococcus aureus (MRSA) rates have tripled in the past 2 decades. Known clinical risk factors include exposure to a healthcare setting, Pseudomonas aeruginosa and CF-related diabetes. Area-level socio-environmental exposures have not been evaluated. We explored the association of area-level deprivation with MRSA prevalence in a pediatric CF Center in the Southeastern United States. Methods: Patients’ residential addresses were geocoded and linked to a composite Area Deprivation Index and Rural-Urban Commuting Area scores. The association...
Source: The Pediatric Infectious Disease Journal - Category: Infectious Diseases Tags: Original Studies Source Type: research
Abstract Cystic fibrosis (CF) is the most common life-limiting hereditary condition of Caucasian populations and is characterised by chronic airways inflammation driving progressive structural lung damage. Despite tremendous advances in the treatment of CF and concomitant increased life expectancy for patients, chronic lung disease remains the main cause of morbidity and mortality among CF patients. While universal restoration of cystic fibrosis transmembrane conductance regulator activity remains a future hope, novel therapies aimed at reducing or preventing chronic airways inflammation and progressive structural...
Source: American Journal of Respiratory and Critical Care Medicine - Category: Respiratory Medicine Authors: Tags: Am J Respir Crit Care Med Source Type: research
Abstract In yeast Saccharomyces cerevisiae cells, some aberrant multimembrane-spanning proteins are not transported to the cell surface but form and are accumulated in endoplasmic reticulum (ER)-derived subcompartments, known as the ER-associated compartments (ERACs), which are observed as puncta under fluorescence microscopy. Here we show that a mutant of the cell surface protein Pma1, Pma1-2308, was accumulated in the ERACs, as well as the heterologously expressed mammalian cystic fibrosis transmembrane conductance regulator (CFTR), in yeast cells. Pma1-2308 and CFTR were located on the same ERACs. We also note ...
Source: Cell Structure and Function - Category: Cytology Authors: Tags: Cell Struct Funct Source Type: research
en-Foncea H Abstract During exercise, the human body maintains optimal body temperature through thermoregulatory sweating, which implies the loss of water, sodium (Na+), and other electrolytes. Sweat rate and sweat Na+ concentration show high interindividual variability, even in individuals exercising under similar conditions. Testosterone and cortisol may regulate sweat Na+ loss by modifying the expression/activity of the cystic fibrosis transmembrane conductance regulator. This has not been tested. As a first approximation, the authors aimed to determine whether basal serum concentrations of testosterone or cort...
Source: International Journal of Sport Nutrition and Exercise Metabolism - Category: Sports Medicine Authors: Tags: Int J Sport Nutr Exerc Metab Source Type: research
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