Kalydeco - A Cystic Fibrosis Treatment Kalydeco has been approved by the Food And Drug Administration (FDA) to treat a vicious type of Cystic Fibrosis (CF). CF is a deadly recessive disease which targets the lungs, but can also harm the liver, pancreas, and intestine. It occurs from the unusual transport of chloride and sodium across the epithelium, causing mucus buildup in the lungs, and thick secretions...
Chemists Unlock Potential Target For Drug DevelopmentA receptor found on blood platelets whose importance as a potential pharmaceutical target has long been questioned may in fact be fruitful in drug testing, according to new research from Michigan State University chemists. A team led by Dana Spence of MSU's Department of Chemistry has revealed a way to isolate and test the receptor known as P2X1...
Lung Function In CF Improved By Long-Term Inhaled Dry Powder MannitolAdding inhaled dry powder mannitol to standard therapy for cystic fibrosis produced sustained improvement in lung function for up to 52 weeks, according to a new study. Along with the treatment's efficacy and good safety profile, the convenience and ease of administration of mannitol treatment may improve adherence with therapy in these patients...
Cystic Fibrosis - Inhaling Mannitol Plus Standard therapy Improves Lung FunctionA new study found that the combination of inhaled dry powder mannitol with standard therapy for cystic fibrosis resulted in maintained improvement in lung function for 12 months. In addition to being effective and safe, the easy administration of the treatment might help enhance adherence with treatment in individuals suffering with the condition...
Inhaled Dry Powder Mannitol Improves Lung Function In CFAdding inhaled dry powder mannitol to standard therapy for cystic fibrosis produced sustained improvement in lung function for up to 52 weeks, according to a new study. Along with the treatment's efficacy and good safety profile, the convenience and ease of administration of mannitol treatment may improve adherence with therapy in these patients...
Adults With Hemophilia B Benefit From Gene TherapySymptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K...
The Tale Of An Outbreak's Evolution Told By Bacterial GenesResearchers at Harvard Medical School and Children's Hospital Boston have retraced the evolution of an unusual bacterial infection as it spread among cystic fibrosis patients by sequencing scores of samples collected during the outbreak, since contained...
New Therapy Marks A Milestone In Fight Against Cystic FibrosisResults of the pivotal Phase 3 clinical trial published in the Nov. 3, 2011 New England Journal of Medicine, find that the oral medication ivacaftor (VX-770) provides major, sustained improvement in lung function, growth and other signs and symptoms for cystic fibrosis (CF) patients...
New Medication Effectively Treats Underlying Cause Of Cystic FibrosisA new study has confirmed that the drug, ivacaftor (VX-770), significantly improves lung function in some people with cystic fibrosis (CF). The results of the phase III clinical trial study, "A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation," led by Bonnie W...
'Vampire' Bacteria Has Potential As Living AntibioticA vampire-like bacteria that leeches onto specific other bacteria - including certain human pathogens - has the potential to serve as a living antibiotic for a range of infectious diseases, a new study indicates...
Link Between Smoking Cigarettes And Cystic FibrosisNew research in The FASEB Journal suggests that cigarette smoke interferes with the Cystic Fibrosis Transmembrane Conductance Regulator protein, leading to dry, sticky mucus and increased infections. If you smoke cigarettes, you have more in common with someone who has cystic fibrosis than you think...