Daniela Semedo, PhD,  —

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 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…

Keiser University has raised $50,000 to support the Cystic Fibrosis Foundation through the efforts of its staff and students across 17 campuses in Florida. This is the ninth year Keiser has taken part in a fundraising event for the foundation. The 2015 campaign adopted a “Terminator”-inspired movie theme for activities that raised money to further research and care into cystic fibrosis (CF),…

A team of specialists in the United Kingdom are investigating whether a novel hearing test could effectively detect early hearing loss in patients with cystic fibrosis (CF), a possible side effect of antibiotics used to treat recurrent disease-related chest infections. CF is genetic disorder that mostly affects the lungs, and patients are…

Contrary to previous research, in a recent study published in the journal BMC Pulmonary Medicine, scientists did not find any general elevated risk for anxiety and depression among patients with Cystic Fibrosis. In people with CF, a defective gene causes a thick buildup of mucus…

Galapagos NV, a clinical-stage biotechnology company specialized in the discovery and development of small molecule medicines with novel modes of action for treating diseases such as cystic fibrosis as well as inflammation and other indications, recently announced that its drug candidate GLPG2665 has been chosen to be developed as a next-generation…

Proteostasis Therapeutics, Inc. (PTI), a company that develops disease-modifying therapeutics for diseases involving protein processing, recently announced at a presentation during the 29th Annual North American Cystic Fibrosis Conference data on its transmembrane conductance regulator (CFTR) amplifier program for use in combined treatments for patients with cystic fibrosis (CF). In…

Alcresta, a company dedicated to developing and commercializing novel enzyme-based products that address the challenges faced by patients with gastrointestinal disorders and rare diseases such as cystic fibrosis recently presented at the 29th Annual North American Cystic Fibrosis Conference (NACFC) new clinical results reporting on its product and technology. The conference is taking…

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…

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…