News

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…

Proteostasis Therapeutics is eliminating 13 research positions in order to save $3 million as it “readjusts priorities to focus resources on researching and developing” its cystic fibrosis (CF) programs, including clinical trials for PTI-428, PTI-801 and PTI-808 small molecules. The R&D shift also includes its discovery-stage program in…

Vertex Pharmaceuticals recently presented data from several clinical trials of cystic fibrosis treatments showing positive outcomes for CF patients. Data focused on the approved therapies Orkambi and Kalydeco, and investigative treatments like tezacaftor/ivacaftor and several triple regimens. These findings were presented at the North American Cystic Fibrosis Conference (NACFC) that ran Nov.

A long-term clinical study showed that RELiZORB improves fatty acid absorption in patients suffering from fat malabsorption or pancreatic deficiencies, which is good news for cystic fibrosis (CF) patients suffering from insufficient lipase production. Alcresta Therapeutics, RELiZORB’s developer, presented data from its ASSURE study at the North American Cystic Fibrosis…

Vertex Pharmaceuticals, a clinical-stage biopharmaceutical company, recently announced that two Phase 3 studies evaluating its tezacaftor/ivacaftor combination therapy have shown statistically significant and clinically meaningful improvements in lung function in patients with certain mutations associated with cystic fibrosis (CF). The first study, called EVOLVE (NCT02347657), was conducted to…