News

Galapagos dosed the first cystic fibrosis (CF) patient with GLPG2222, its novel CF corrector, in a Phase 2a clinical trial evaluating the drug as an add-on therapy to Kalydeco (ivacaftor) in CF patients harboring one F508del CFTR mutation and one gating mutation. The ALBATROSS Phase 2a clinical trial…

Guidelines for diagnosing cystic fibrosis (CF) were recently updated to give doctors firmer direction in determining signs of this disease, especially in those not diagnosed as newborns, and in being able to recommend the best treatment for each patient. The update, the work of 32 experts from nine countries, includes more information on mutations…

An Ohio cystic fibrosis (CF) patient was given an infusion of adult stem cells in a pioneering study that researchers hope will lead to therapies that reduce inflammation and infections linked to the disease. “I am hoping the future generations of CF patients can get better treatments and that eventually a cure…

The top 10 priorities for cystic fibrosis research — set by CF patients and specialists alike — range from learning more about treatments that delay or prevent disease progression, to simplifying therapies so they’re less burdensome and getting patients to keep using a prescribed medication, according to a survey exercise led by scientists at The University…

A significant number of cystic fibrosis (CF) patients do not receive timely lung transplant evaluations because of their preference and their doctors’ possibly too-narrow focus, according to the results of a survey. The study explaining the results, “Cystic fibrosis physicians’ perspectives on the timing of referral for lung transplant…

Drugs targeting a component of biofilm — the glue-like layer secreted by bacteria in cystic fibrosis (CF) patients’ lungs — could increase the bacteria’s vulnerability to antibiotics and natural immune reactions, according to a new study. Researchers discovered that a specific carbohydrate in biofilm — Psl — makes it stiffer and,…

Researchers at the University of Wisconsin–Madison have developed a technique that makes it possible to watch gene-editing processes in real time — which could help them understanding why editing attempts succeed or fail. The team, which received a $1.8 million grant from the National Institutes of Health (NIH), is now working to…

The Cystic Fibrosis Foundation (CFF) joined 72 other organizations, each advocating for patients with chronic, rare and life-threatening diseases, in an open letter to President Donald Trump and leaders in Congress. The letter asks that patients’ needs be taken into consideration as lawmakers revise current healthcare policies. Other groups joining this initiative include the Pulmonary Fibrosis…

A mouse model that reproduces the human symptoms of cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD) gives scientists a new way to study, and perhaps develop treatments for, the diseases, according to new research. Researchers at Kumamoto University in Japan developed the model, then tested it to see how…

The U.S. Food and Drug Administration (FDA) has designated Concert Pharmaceuticals‘ next-generation therapy, CTP-656, an Orphan Drug to treat cystic fibrosis (CF). CTP-656 is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that, according to the company’s website, was developed by applying deuterium chemistry to modify Kalydeco (ivacaftor).  Concert has said…