News

The Geisel School of Medicine at Dartmouth announced that it has received a $10 million gift from an anonymous donor, plus a $5 million matching donation, also given anonymously, that will be used to accelerate its research into better treatments and, ultimately, a cure for cystic fibrosis (CF). The $15 million in…

A philanthropic donation to Emory University will contribute to the research of new therapies for cystic fibrosis (CF) at the laboratory of Dr. Eric J. Sorscher, a scientific leader in cystic fibrosis research. Sorscher heads a research program at Emory and Children’s Healthcare in Atlanta that focuses on discovering…

The drug ivacaftor appears to be safe for children 2 to 5 years old with specific types of a cystic fibrosis (CF) genetic mutation, according to recent U.K. research. The findings support the drug’s U.S. approval for youngsters in this age group, and also indicate a potential period in early life when organ…

A study reported that treating a mouse model of cystic fibrosis (CF) with the antibiotic streptomycin eased the animals’ airway hyper-responsiveness. The improvement in airway function was accompanied by an altered immune profile and changes in a certain bacteria in the gut. The study, titled “Streptomycin treatment…

Nivalis Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted its lead investigational drug, N91115, orphan drug status for the treatment of cystic fibrosis (CF). Drug candidate N91115 is a stabilizer of the CF transmembrane conductance regulator (CFTR) protein. Orphan drugs are generally those with a limited…

A recent study in the American Journal of Critical Care Medicine suggested a new therapeutic target for cystic fibrosis (CF), and reinforced the idea that inflammation is an acquired response unrelated to the CFTR genetic mutation. The study is titled “X-Box–Binding Protein 1 and Innate…

Vertex Pharmaceuticals, a biotech company that discovers, develops, and commercializes medicines for a series of diseases, currently with a clinical development program focused on cystic fibrosis (CF), recently announced its key business priorities for 2016. Vertex currently has two medicines approved for cystic fibrosis treatment: KALYDECO (ivafactor) and ORKAMBI (lumacaftor/ivacaftor).