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    Canadian adults with CF living longer

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    CFF Sells Drug Rights for $3.3 Billion

    Big News today from the Cystic Fibrosis Foundation who just sold its drug royalty rights for $3.3 Billion to Royalty Pharma. The CFF plans to reinvest the money into new research efforts. “These new funds give us a tremendous opportunity to supercharge our efforts to develop lifesaving new...
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    Sweat Sensor plus Mobile Device for diagnosis of CF

    Electronic patch technology is rapidly gaining momentum in the medical device research arena. By analyzing sweat, an electronic patch maybe able to diagnose cystic fibrosis and may one day be used for monitoring blood glucose levels for diagnosis and treatment of diabetes. The new patch-type...
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    Kalydeco Approved in Australia

    Kalydeco just got approved to be added to the list of the Pharmaceutical Benefits Scheme (PBS) in Australia, and will become available on December 1st 2014, as announced by Australian Health Minister Pete Dutton. This means that the costs of Kalydeco will be subsidized by the Australian...
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    P. aeruginosa uses host cells to kill S. aureus in CF patients

    A very interesting and potentially significant study was published by a french group at the Institute Pasteur demonstrating how a bacterium manipulates its host to eradicate an opponent. They showed that a particular enzyme produced by the airways of cystic fibrosis patients is instrumental in...
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    Kalydeco Approved for R117H Mutation

    The Pulmonary-Allergy Drugs Advisory Committee (Ad Comm) votes 13 - 2 in favor of a label expansion for Vertex Pharmaceuticals' cystic fibrosis med Kalydeco (ivacaftor) to include the R117H mutation. This expands the approved CF mutation list to ten. (PR source: http://t.co/514NrU3K9Q ) There...
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    Nanotech lung infection detection device for Cystic Fibrosis

    Researchers at UC Ivine have developed a low-cost, disposable breath analysis nanotechnology device for people with cystic fibrosis. The device sends data to a smartphone in the event of a lung infection. The device can detect tiny quantities of infection in a small sample of breath. The device...
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    Staphylococcus aureus Sharing Nutrients to Increase Virulence during Infection

    An interesting study performed at the Vanderbilt University demonstrated the ability of bacteria, in this case Staphylococcus aureus, to work together in a mixed population, collectively sharing nutrients to resist drugs like antibiotics. There are many ways bacteria can develop resistance to...
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    New insights into bacterial growth in CF lung infection

    Researchers from the University of Copenhagen have shown for the first time how bacteria can grow directly in the lungs of Cystic fibrosis patients, giving them the opportunity to get tremendous insights into bacteria behavior and growth in chronic infections. The study also discovered the...
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    Median age of survival for CFers surpasses 50 years of age!

    In a press release from the Canadian Cystic Fibrosis Foundation on Oct. 15, 2014: We all saw this coming and expected these announcements to start hitting the news wires. Canada is leading the announcements and it will not be long before other foundations like the CFF start to release new...
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    Some doubts for Kalydeco in R117H patients

    Vertex would like to add patients with R117H mutations into the Kalydeco patient pool. But they are facing some resistance from the FDA. R117H mutation "results in a functionally different chloride channel than with the G551D mutation", Kalydeco's original indication. In a trial to examine...
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    Parion Sciences Announces $15.6 Million in Grant Support for Pulmonary Research

    Parion Sciences, a company dedicated to the development of novel treatments for pulmonary and ocular diseases, announced today that the National Institutes of Health (NIH) has awarded up to $15.6 million over 5 years in grants to The University of North Carolina, Chapel Hill (UNC-CH) and The...
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    Tannic acid could help treat bacterial lung infections

    Staphylococcus aureus is a common cause of lung infection in CFers. These bacteria secrete an enzyme called SMase C that impact negatively on CFTR activity and also impair the function of white blood cells important in fighting off infection. Scientists have discovered that SMase C also...
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    Boler-Parseghian Center for Rare & Neglected Diseases

    Thanks to generous donations from the Boler and Parseghian families, the University of Notre Dames's Center for Rare and Neglected Diseases is renamed to the Boler-Parseghian Center for Rare & Neglected Diseases. A donation of $10 million dollars to the center will go towards funding further...
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    Promising News from Vertex for the Cystic Fibrosis Community

    Vertex trial results recently presented at the NACFC, spurred several stock analysts to increase expectations for the company’s shares. 22 out of 34 analysts covering Vertex now have a positive rating on the stock. Vertex recently provided data showing its experimental drug, lumacaftor, and its...
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    CSA-13 Against Psuedomonas Aeruginosa Biofilms

    N8 Respiratory presented data at the NACFC demonstrating antimicrobial efficacy of CSA-13 against Psuedomonas aeruginosa (PA) biofilms. Specifically, a preclinical study was presented demonstrating its lead compound CSA-13's efficacy as an antimicrobial peptide mimic against Pseudomonas...
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    Fixing Faulty Genes with CRISPR/Cas9

    Jennifer Doudna and her colleagues found an enzyme in bacteria that makes editing DNA in animal cells much easier. For this discovery, Jennifer Doudna maybe in line to win a Nobel Prize in the coming decade. Until now, the tools for fixing or replacing a gene in animals were cumbersome, if...
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    Vertex Reviews Recent CF R&D Progress at NACFC

    Here are the highlights from Vertex's CF R&D programs presented at the NACFC. 1. Interim analysis of rollover study following the Phase 3 TRAFFIC and TRANSPORT studies showed sustained improvements in lung function through 48 weeks of treatment with lumacaftor in combination with ivacaftor in...
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    Novel corrector / potentiator combinations for restoring CFTR function

    Galapagos, a Belgium biotechnology company recently presented novel corrector / potentiator combination drug candidates for restoring CFTR function at the NACFC. Pre-clinical data was shown in which these novel corrector molecules in combination with existing CFTR modulators restored at least...
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    Use of Kalydeco in Children Ages 2 to 5

    Use of Kalydeco in Children Ages 2 to 5: Vertex today announced the submission of an NDA in the United States for the approval of Kalydeco in children with CF ages 2 to 5 who have one of the following nine mutations in the CFTR gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P...
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