Cystic Fibrosis (CF) is caused by mutations in the Cftr gene (short for cystic fibrosis transmembrane conductance regulator). The CFTR protein is responsible for transporting chloride and sodium ions in and out of lung and intestinal epithelium cells, and so in the absence of CFTR functional protein, thick mucus…
News
A recent paper published in the European Respiratory Journal reports that Canadians suffering with cystic fibrosis (CF) are living nearly 20 years longer than they did two decades ago. In 1990, the median survival age for Canadian patients with cystic fibrosis on average was 31.9 years. That number increased to 49.7…
The Chief Executive of the Cystic Fibrosis Trust recently wrote a letter to the NHS England‘s National Director for Commissioning Operations to help ensure that budget responsibility and delivery of cystic fibrosis (CF) services are not shifted to local commissioners. Ed Owen expressed his concern…
Arcturus Therapeutics, Inc. recently presented encouraging non-human primate studies concerning its preclinical messenger RNA therapies for the potential treatment of genetic diseases such as cystic fibrosis at the 2nd International mRNA Health Conference held November 11-12 in Cambridge, Massachusetts. Arcturus’ presentation, “Potent Delivery of LUNAR™ Nanoparticles Containing Synthetic mRNA for…
New ways of treating cystic fibrosis are under development at the University of Dundee. Along with colleagues in Italy, France, and Scotland, Dr. Anil Mehta, a clinical researcher at Dundee’s Medical Research Institute, pioneered a new drug combination that gives hope to patients with cystic fibrosis. “This work shows…
A group of undergraduate researchers from Dundee University are the proud recipients of 3 prizes from a conference in Boston, Massachusetts for their breakthrough work on harnessing a fluorescent bacteria to detect a potentially dangerous lung infection in cystic fibrosis (CF) patients. “The Lung Ranger,”…
All over the country and throughout the world, local communities organize events to raise both awareness and funds to help patients suffering with cystic fibrosis (CF), as well as to support research dedicated to finding a cure for the life-threatening genetic disease. Approximately 70,000 people suffer from CF worldwide, 30,000 of which…
In a recent study entitled “Subnanometre-resolution electron cryomicroscopy structure of a heterodimeric ABC exporter” published in the online issue of Nature, the authors determined the structure of an ABC transporter protein with a sub nanometer resolution for the first time. The new findings has implications in…
Proteostasis Therapeutics, a company developing novel therapeutics for Cystic Fibrosis and other diseases to address defects in protein folding, trafficking and clearance, and Astellas Pharma Inc. (“Astellas”), a company dedicated to improving people’s health by providing innovative and reliable pharmaceuticals, announced a worldwide joint collaboration to research and…
Massachusetts-based Vertex Pharmaceuticals, Inc. announced yesterday that they have filed for a New Drug Application (NDA) with the US Food and Drug Administration, and a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for their pipeline combination lumacaftor–ivacaftor drug, indicated for the…
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