News

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

Scientists at the University of North Carolina (UNC) School of Medicine developed a model of cystic fibrosis (CF) that may help to test current and future CF therapies. This model, showing how airway cells respond to environmental factors, also brought new insights into the thick mucus observed in CF patients. The…

Proteostasis Therapeutics is expecting clinical trial results on three of its cystic fibrosis therapies in the next few months, and plans to start additional trials in 2018. CF is a genetic disease caused by mutations in the sequence of the CFTR gene, which leads to impaired activity of the…

Spyryx Biosciences recently presented preclinical data supporting the clinical benefits of its cystic fibrosis (CF) treatment candidate SPX-101, intended to improve lung function in all patients regardless of their underlying disease-causing mutation. The data, presented in four posters at the recent North American Cystic Fibrosis Conference (NACFC) in Indianapolis,…

News that you’ve just been rejected for a necessary double-lung transplant rarely brings out the best in people. But Caleigh Haber, 27, a self-described fighter since being diagnosed with cystic fibrosis at birth, simply “wasn’t ready to wave the white flag,” she said in an interview with Cystic Fibrosis News…

More adults than children are living with cystic fibrosis for the first time ever, says the Cystic Fibrosis Foundation (CFF). This means more patients are encountering issues they have never had to face — such as saving for their children’s college and their own retirement. They are also having to deal…

Italian researchers have discovered a molecule that prevents cystic fibrosis patients’ anti-inflammatory and anti-bacterial defenses from working as well as they should. Scientists may be able to target the molecule, microRNA-181, to develop CF therapies. The team published their study in the journal Nature. The title is  “MicroRNA-181b…

The European Union’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for Orkambi (ivacaftor/lumacaftor) as a therapy for cystic fibrosis (CF) in children. The ruling is specifically for pediatric patients aged 6 to 11 with two copies of the F508del mutation in the…

BIOC51, a potential nitric oxide-based treatment of chronic pulmonary infections due to Pseudomonas aeruginosa bacteria in patients with cystic fibrosis (CF),was awarded special status to promote and speed its development, Novoclem Therapeutics announced. BIOC51 was granted what is known as Qualified Infectious Disease Product (QIDP) status by the U.S. Food and Drug Administration (FDA), whose incentives include priority review…

Vertex Pharmaceuticals announced it will double the number of “All in for CF Scholarships” this year to help more people with cystic fibrosis (CF) and their families. This increase in number of scholarships, the company said, is due to the positive response the program received last year when it was…