News

Baby Bridget, born on June 27th, became the first baby conceived in Ireland using pre-implantation genetic diagnosis (PGD), a technique used to screen embryos for genetic mutations such as cystic fibrosis (CF). Despite the fact that both parents have genetics that, when combined, can lead to CF in their children, baby…

In the wake of recent promising clinical results, Vertex Therapeutics announced the submission of a supplemental New Drug Application to the U.S. Food and Drug Administration for the approval of the drug Kalydeco (ivacaftor) for patients with cystic fibrosis who are more than 18  years old and…

Hemolung Respiratory Assist System (RAS) is an innovative Pittsburgh-made, dialysis-like alternative or supplement to mechanical ventilation that is not approved by the U.S. Food and Drug Administration (FDA), but is gaining several positive opinions in the United States, following the promising results when used on an American terminal patient suffering from…

Drug developer Pharmaxis has announced that the company’s medication for cystic fibrosis, Bronchitol, was included in the shortlist for the UK Prix Galien Orphan Drug Award, an internationally-recognized prize that distinguishes technical, scientific, and clinical research skills necessary to develop innovative medicines. The therapies in the competition will be analyzed…

In an effort to alleviate symptoms in patients with nonsense mutation cystic fibrosis–a severe form that leads to little if any CFTR protein production–PTC Therapeutics, Inc. is initiating a global confirmatory Phase 3 clinical trial to test Translarna (ataluren) under the trial name ACT CF. In a previous,…

What happens when a cystic fibrosis charity and a biotechnology company team up through a partnership? The answer is a Phase IIa clinical trial in the United Kingdom put together by Cystic Fibrosis Trust and NovaBiotics. Also included in the effort are the University of Aberdeen, Health Science Scotland,…

In the wake of positive news about new combination therapies involving the Vertex drug Kalydeco (ivacaftor) for people with cystic fibrosis (CF), the drug recently received a positive recommendation for approval by the European Committee for Medicinal Products for Human Use (CHMP) for patients aged…

Sick children under 12 years old needing a lung transplant, and who were previously almost totally dependent on lung donations from other children, can now have broader access to donated organs from adults, thanks to a new decision from the Organ Procurement and Transplantation Network (OPTN). Adolescent donation protocol will…

Half of the adults who suffer from cystic fibrosis (CF) are infected with the aspergillus fungus, according to medical data from CF patients in 30 different countries. Not only is the discovery a surprise, as the findings may be relevant for the diagnosis and treatment of the rare genetic…

Vertex Pharmaceuticals Incorporated has been grabbing headlines recently related to its revolutionary cystic fibrosis drug Kalydeco (ivacaftor), which is currently approved for people with CF aged 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.