News

Ireland’s National Centre for Pharmacoeconomics (NCPE) stirred the pot this week with its decision that the cost of the new cystic fibrosis (CF) drug Orkambi is not justified by its acknowledged benefits, and should not be paid for by the Irish Health Service Executive. The regulatory agency, whose mission is…

The bacterium Burkholderia multivorans grows, evolves and adapts to life inside the lungs of patients with cystic fibrosis, according to the results of a study recently published in the journal mSystems. The study, “Long-Term Evolution of Burkholderia multivorans during a Chronic Cystic Fibrosis Infection Reveals Shifting Forces of Selection,”…

Researchers partially restored the function of the CFTR protein in lung cells isolated from cystic fibrosis (CF) patients by blocking a particular protein involved in its translation. The findings were published in the journal PLOS Biology, in the study “Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional…

The Boomer Esiason Foundation (BEF) and Johnson & Johnson recently announced the launch of the Johnson & Johnson TRU Heroes Cystic Fibrosis Nursing Program, a first-of-its-kind educational empowerment program for the nursing community focused on cystic fibrosis (CF). The Johnson & Johnson TRU Heroes program was designed to…

Walgreens is launching CF Champions: Navigating the Journey, Together, a program to support patients, caregivers and all who have been touched by cystic fibrosis (CF). The initiative runs throughout May, the Cystic Fibrosis Foundation’s National CF Awareness Month, and possibly through the end of this year. Walgreens partnered with four CF…

PTC Therapeutics, Inc. recently announced the recipients of its global STRIVE Awards (Strategies to Realize Innovation, Vision and Empowerment) program, which supports nonprofit associations assisting the cystic fibrosis (CF) community. The STRIVE Program recognizes the vital role patient advocacy groups play in giving voice to individuals affected by these rare…

ProMetic Life Sciences Inc. recently announced that Health Canada has approved the initiation of a clinical trial assessing the company’s anti-fibrotic lead drug candidate, PBI-4050, in patients with cystic fibrosis (CF). The Phase 2 study, which is expected to begin in shortly, aims to evaluate the drug’s effects on pancreatic and lung function…

Nivalis Therapeutics announced the dosing of a first patient in a Phase 2 clinical trial evaluating the efficacy and safety of its investigational drug N91115, in combination with ivacaftor (Kalydeco), in people with cystic fibrosis (CF). CF is a life-shortening genetic disease, characterized by mutations in the CFTR gene that lead to defective chloride…

Silencing a gene involved in the processing of the mutant CFTR protein in cystic fibrosis (CF) improved folding and increased the amount of functional protein that reached the plasma membrane — reaching clinically relevant levels when used in combination with the modulator drug VX-809 (lumacaftor). The results, from experiments in…

Researchers at the Queen’s University Belfast developed a new compound that may change how lung ailments in cystic fibrosis (CF) are treated. The potential medicine was shown in an early study to improve airway hydration and increase mucus clearance, results that suggest a mechanism to potentially delay or prevent lung disease independently of the…