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Exome-Sequencing Might Help Identify Genetic Cause Of Thousands Of Disorders For the first time, scientists have successfully used a method called exome sequencing to quickly discover a previously unknown gene responsible for a mendelian disorder. Mendelian disorders, such as cystic fibrosis and sickle cell disease, are the result of one or more mutations in a single gene, typically a gene that makes a protein. All of the regions that code for proteins taken together are called the exome. ...more	20 Nov 2009
Antioxidant Found In Vegetables Has Implications For Treating Cystic Fibrosis Scientists at the University of Pennsylvania School of Medicine discovered that a dietary antioxidant found in such vegetables as broccoli and cauliflower protects cells from damage caused by chemicals generated during the body's inflammatory response to infection and injury. The finding has implications for such inflammation-based disorders as cystic fibrosis (CF), diabetes, heart disease, and neurodegeneration. ...more	18 Nov 2009
Inspire Completes Patient Enrollment In Three Late-Stage Clinical Trials In Cystic Fibrosis, Dry Eye And Blepharitis Inspire Pharmaceuticals, Inc. (NASDAQ: ISPH) announced today patient enrollment is complete in three of its late-stage clinical trials. "We are pleased to be executing on our strategic plan with the achievement of these patient enrollment milestones in the clinical development programs for denufosol, PROLACRIA™ and AZASITE®, as this places us in a position to have top-line results from all our late-stage clinical programs within 18 months. ...more	10 Nov 2009
Call For Cystic Fibrosis Carrier Testing To Be Offered To All Couples Considering Pregnancy, Australia Cystic fibrosis carrier testing should be offered to all couples considering pregnancy and to women in early pregnancy and their partners, according to the authors of a study published in the Medical Journal of Australia. ...more	03 Nov 2009
New Clinical Guidelines For Exacerbations In Cystic Fibrosis The American Thoracic Society has released new clinical guidelines for the treatment of exacerbations in cystic fibrosis based on a review of the literature on current clinical practices. "This is the first such comprehensive and evidence-based systematic review of best practices for pulmonary exacerbation of cystic fibrosis," said Susanna McColley, M.D. ...more	24 Oct 2009
KaloBios' KB001 Antibody Shows Encouraging Safety And Activity In New Approach To Fighting Pseudomonas Infection In Cystic Fibrosis Patients Phase 1/2 clinical trial results with KB001, a Humaneered™, high-affinity antibody fragment under development by KaloBios Pharmaceuticals, Inc. in cystic fibrosis patients showed an acceptable safety profile as well as trends toward reducing tissue inflammation and clearance of the bacterium. Results of the multi-center, randomized, double-blind, single-dose, placebo-controlled study are being presented today at the North American Cystic Fibrosis Conference in Minneapolis, MN. ...more	18 Oct 2009
NanoBio's Nanoemulsion Kills Drug-Resistant Bacteria Found In Cystic Fibrosis Patients NanoBio Corporation today announced compelling preclinical data for NB-401, a nebulized nanoemulsion-based agent that kills highly drug-resistant strains of bacteria commonly found in cystic fibrosis patients. Currently there are limited treatment options available that effectively address these resistant bacteria. The study results are being presented today at the 2009 Annual North American Cystic Fibrosis Conference (NACFC) in Minneapolis, Minn. ...more	17 Oct 2009
University Of North Carolina Awarded $6.2 Million Renewal Grant By National Institutes Of Health Rare Diseases Research Network The University of North Carolina at Chapel Hill has been awarded a five-year, $6.2 million renewal grant to continue its work as part of the National Institutes of Health's Rare Diseases Clinical Research Network (RDCRN). "This additional funding will let us continue our discovery of rare disease-causing gene mutations, which has already culminated in a clinical genetic test, but which needs to be expanded and improved," said Michael Knowles, M.D. ...more	15 Oct 2009
NIH Director Francis Collins To Address 3,000 Cystic Fibrosis Experts At International Conference In Minneapolis Francis S. Collins, M.D., Ph.D., the new NIH director who led an international effort to map the human genome and co-discovered the gene responsible for cystic fibrosis, will address more than 3,000 leading scientists and clinicians from around the world at the 23rd annual North American Cystic Fibrosis Conference (NACFC). Held in Minneapolis from Oct. 15-17, 2009, the conference comes at a pivotal point in the history of cystic fibrosis, a fatal genetic disease. ...more	10 Oct 2009
Results Published In Journal Of Cystic Fibrosis Confirm CREON(R) (pancrelipase) Delayed-Release Capsules Improves Fat Absorption Solvay Pharmaceuticals, Inc. announced that Phase III data published in the Journal of Cystic Fibrosis showed that CREON® (pancrelipase) Delayed-Release Capsules, the most prescribed pancreatic enzyme replacement therapy (PERT) in the United States, significantly improves a key measure of fat absorption in patients with CF who suffer from exocrine pancreatic insufficiency (EPI). ...more	10 Oct 2009
DiscoveryBioMed, Inc. Awarded Phase 2 SBIR Grant By The NIH To Discover Hypertension And Cystic Fibrosis (CF) Drugs DiscoveryBioMed, Inc. (DBM) announced that it has been awarded a $750,000 Small Business Innovations Research (SBIR) Phase 2 grant by the National Institutes of Health (NIH) to continue the research into the discovery and development of small molecules to alleviate multiple chronic human diseases including cystic fibrosis (CF), hypertension and chronic kidney diseases with hypertension. ...more	29 Sept 2009
Nationwide Designated Translational Research Center In CF Foundation's National Clinical Trials Network Nationwide Children's Hospital has been selected to serve as one of 13 special translational research centers in a network of 77 clinical trial sites that are part of the Therapeutics Development Network (TDN) of the Cystic Fibrosis Foundation. The translational research centers will lead the newest Phase I clinical trials and provide scientific direction to the entire network. ...more	29 Sept 2009
Cleveland Indians Manager Eric Wedge Urges Fans To Fight Cystic Fibrosis Eric Wedge, manager of the Cleveland Indians, is joining the fight against cystic fibrosis (CF), a fatal genetic disease. Wedge and 17-year old Michaela Mullet will star in a public service announcement (PSA) to be aired during a Cleveland Indians game against the Chicago White Sox on Monday evening, September 28. Cystic fibrosis clogs the lungs with thick mucus, causing life-threatening infections and premature death. ...more	28 Sept 2009
Cystic Fibrosis Patient Emily Schaller Begins 2,000-Mile Quest To Get On "The Ellen DeGeneres Show" Emily Schaller, a 27-year-old drummer with cystic fibrosis (CF), begins a 2,000-mile journey today aboard a Vespa scooter - riding from Chicago to Burbank, California, in her quest to get on "The Ellen DeGeneres Show." Schaller, whose singular goal is to raise awareness about cystic fibrosis, has appeared in ...more	24 Sept 2009
Senate Introduces Bill To Boost Participation In Clinical Trials For Rare Diseases Four members of the United States Senate introduced legislation today to allow patients with rare diseases to participate in clinical drug studies without losing their eligibility for public healthcare coverage, echoing a move by the House of Representatives last month. The "Improving Access to Clinical Trials Act" is co-sponsored by Senators Ron Wyden (D-OR), Chris Dodd (D-CT), James Inhofe (R-OK) and Richard Shelby (R-AL). ...more	17 Sept 2009
The Making Of Mucus In Common Lung Diseases In the lung, mucus is produced by cells known as goblet cells, which are present in small numbers in the walls of the lungs and airways. Many inflammatory stimuli, including allergens, cigarette smoke, and chronic infections, increase the number and activity of these goblet cells. ...more	17 Sept 2009
Kamada Presents Data On Inhaled Alpha-1 Antitrypsin In Cystic Fibrosis Patients At The European Respiratory Society Meeting 2009, Vienna Kamada, a bio-pharmaceutical company engaged in the development, manufacturing and marketing of specialty life-saving therapeutics, announced that data on its next-generation alpha-1 antitrypsin (Inhaled-AAT) in cystic fibrosis patients was presented, at the Annual Congress of the European Respiratory Society (ERS) 2009, Vienna. As previously reported, the study showed that inhaled-AAT was safe and biologically effective. ...more	16 Sept 2009
Mpex Reports Positive Phase II Results With Aeroquin, Delivered By PARI Pharma's EFlow Technology Mpex Pharmaceuticals, Inc. reported positive top line results from a Phase 2b clinical trial with Aeroquin, a novel aerosol formulation of levofloxacin delivered by an optimized Investigational eFlow Nebulizer System from PARI Pharma. In the Mpex cystic fibrosis trial, nebulized Aeroquin met the primary endpoint of reducing bacterial counts of Pseudomonas aeruginosa in sputum after 28 days of dosing versus placebo. ...more	15 Sept 2009
Study Identifies Genetic Variation Associated With Increased Risk Of Liver Disease For Patients With Cystic Fibrosis A genetic analysis indicates that a certain gene variation in patients with cystic fibrosis may significantly increase their risk of developing severe liver disease, according to a study in the September 9 issue of JAMA. A small fraction (about 3 - 5 percent) of patients with cystic fibrosis (CF) develop severe liver disease characterized by cirrhosis with portal hypertension (CFLD; increase in blood pressure caused by obstruction in the liver). ...more	15 Sept 2009
UA Researchers Seek Safer Cystic Fibrosis Test Researchers from The University of Arizona Colleges of Pharmacy and Medicine are teaming up to try to invent a novel non-invasive lung test for cystic fibrosis sufferers. Eric Snyder, PhD, assistant professor at the UA College of Pharmacy, is the principal investigator on the study, "Quantification of Exhaled Condensate Using Bronchoalveolar Lavage in Cystic Fibrosis." Dr. ...more	11 Sept 2009
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