The European Medicines Agency (EMA) recently announced that it has validated the submission of a new indication for Translarna (ataluren) developed by PTC Therapeutics as a nonsense mutation cystic fibrosis (nmCF) treatment. PTC Therapeutics, a company that develops and assembles an integrated set of proprietary technologies focused on…
The International CF Modifier Consortium led by researchers from the University of North Carolina, the University of Toronto, Johns Hopkins University, Case Western University, and the University of Paris, along with some two dozen research institutions and departments in the United States, Canada, and France, are collaborating in the largest-ever…
In a recent study entitled “Comparative genomics of non-pseudomonal bacterial species colonising paediatric cystic fibrosis patients,” researchers performed a whole-genome profiling of bacterial strains, that are not Pseudomonas aeruginosa, from pediatric cystic fibrosis patients and determined their link to antibiotic resistance. The study was published in the open…
Vertex Pharmaceuticals, a global biotechnology company developing and commercializing innovative therapies for diseases such as cystic fibrosis, recently announced that the European Union Committee for Medicinal Products for Human Use (CHMP) has released a positive opinion and recommends Marketing Authorization for the company’s landmark CF combination therapy ORKAMBI (lumacaftor/ivacaftor). ORKAMBI…
Flemington, New Jersey based clinical stage biopharmaceutical company Arno Therapeutics, Inc. has announced data from a preclinical study demonstrating that its drug candidate AR-13, an analogue of Arno’s AR-12 infectious disease compound portfolio, shows promise as a potential new therapeutic option for patients with cystic fibrosis (CF) who are…
Two clinical-stage companies with investigational products for the cystic fibrosis treatment market have just announced their attendance and participation at the upcoming Leerink Partners’ 4th Annual Rare Disease Roundtable, taking place on Wednesday, September 30, 2015 at the Le Parker Meridien in New York, NY. The conference’s attendees will watch presentations on product and investment-related…
Concert Pharma recently announced successful results from a Phase 1 single ascending dose clinical trial for their lead investigational drug CTP-656 as a therapy for cystic fibrosis. Cystic fibrosis is a life-threatening disease characterized by a progressive lung function decline and is caused by mutations in the Cftr gene (cystic fibrosis transmembrane…
Not long after his birth in 2013, Paul Siebenthal‘s son Noah was diagnosed with cystic fibrosis (CF) and the new father became aware that his son would struggle with a chronic, life-threatening disease for his entire life. However, Paul Siebenthal did not accept that there is currently no cure for the…
Peter and Mary Frey are a young couple just like any other, starting a family together. However, there is one thing that makes their family different from the average American household. Mary suffers from cystic fibrosis (CF), a genetic and chronic disease that causes the excessive production of mucus in the lungs,…
The 2015 UK Cystic Fibrosis Conference (UKCFC), the United Kingdom’s most-anticipated annual conference for the cystic fibrosis community that brings together esteemed disease experts, researchers, health professionals, patients and family members, is set to take place on September 22-23, 2015. The two-day event is expected to attract over 300 attendees to Manchester, UK for…
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