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,…
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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.
lev radin / Shutterstock.com An initiative organized last Saturday to support the Cincinnati Children’s Hospital Medical Center in Ohio raised $2 million for its cystic fibrosis and pulmonary programs, a historic amount for the event. The 10th annual Celestial Ball at the Duke Energy Convention Center brought…
A drug derived from Norwegian seaweed may help increase the life expectancy of people with cystic fibrosis, which currently averages about 40 years. The Norwegian company AlgiPharma presented OligoG at the 37th European Cystic Fibrosis Conference in Gothenburg, Sweden recently, revealing…
Greek researchers at Athens University Medical School and Aghia Sophia Children’s Hospital uncovered psychosocial vulnerability in children and adolescents with cystic fibrosis. A study of 36 cystic fibrosis patients, aged 8-18 years, and 31 healthy, age-matched control participants revealed that cystic fibrosis patients have a greater difficulty establishing friendships and…
Bernstein Research analyst Geoff Porges advised investors about the probability of failure of the Vertex Pharmaceuticals’ experimental cystic fibrosis (CF) therapy, as he returned from the European Cystic Fibrosis Society conference in Sweden. Although test results of the combination of Kalydeco and VX-809 – Traffic and Transport has been eagerly…
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