Title: Cystic FibrosisCategory: Diseases and ConditionsCreated: 12/31/1997 12:00:00 AMLast Editorial Review: 7/6/2018 12:00:00 AM
There ’s been an odd-sounding word circulating in the English headlines recently.The word is Orkambi; a drug created by Vertex that treats approximately 40% of people born with the genetic condition, cystic fibrosis. It is the second drug to market in a promising pipeline that targets the CFTR mutation. True gene therapy it isn’t, but it represents a near-first for a biotech firm, by successfully altering the shape and function of a protein. And priced at £105,000 per patient per year, it is the latest in a long line of medicines the NHS can’t afford.Orkambi has been licensed by the EMAsin...
A NEW, improved NHS offer for life-saving drugs was welcomed by cystic fibrosis sufferers last night. The Daily Express has been pressurising NHS England to strike a deal with US drugs giant Vertex for its pipeline of CF medicines like Orkambi.
Conditions: Cystic Fibrosis; Cystic Fibrosis-related Diabetes; Cystic Fibrosis Liver Disease; Pancreatic Steatosis; Hepatic Steatosis Interventions: Device: Frequently Sampled Oral Glucose Tolerance Test and CGM; Radiation: MRI Pancreas and Liver Sponsor: University Hospitals Cleveland Medical Center Recruiting
Distal intestinal obstruction syndrome (DIOS) is a common complication in patients with cystic fibrosis (CF) . DIOS occurs in 10 to 40% of all CF-patients. The risk of developing DIOS is considered higher in the early post-transplant period due to dehydration, immobility, intake of opiates and post-operative adynamic gut . Patients suffer from prolonged intestinal passage or even gastric paresis due to polymedication leading to chronic constipation long-term following lung transplant (LTX) .
Airways of Cystic Fibrosis (CF) patients are Nitric Oxide (NO) deficient which may contribute to impaired lung function and infection clearance. Mycobacterium abscessus (M. abscessus) infection prevalence is increasing in CF patients and is associated with increased morbidity and mortality. Here, we assess the safety and efficacy of intermittent inhaled NO (iNO) as adjuvant therapy in CF patients with refractory M. abscessus lung infection.
ConclusionOur results suggest that LCZ may provide effective prevention against colitis.
Authors: Vizzardi E, Sciatti E, Bonadei I, Cani DS, Menotti E, Prati F, Dallapellegrina L, Metra M, Berlendis M, Poli P, Padoan R Abstract Increasing survival from cystic fibrosis show untypical systems involvement, such as cardiocirculatory. In particular, the presence of CFTR in smooth muscle and endothelial cells, systemic inflammation and oxidative stress could explain vascular alterations in these patients. We aimed at noninvasely evaluating macro- and microvascular dysfunction in cystic fibrosis adults without cardiovascular risk factors. Twenty-twoadults affected by cystic fibrosis and 24 healthy volunteers ...