Researchers Probe Targets to Reduce Mucus Production

Yap and Taz — two proteins involved in lung injury responses and regeneration — suppress the maturation of mucus-producing goblet cells and limit mucus levels in the lungs, according to a study of mice. These findings suggest that promoting Yap/Taz’s activity may be a potential therapeutic approach to limit the…

Airway Inflammation Affects pH, Response to CFTR Therapies

Inflammation in the airways of people with cystic fibrosis (CF) regulates the pH of the surface liquid and increases the response to approved CFTR modulators, a cell-based study suggested. Airway inflammation may be a key determinant in response to CF medicines, the researchers noted. “To advance personalized CFTR…

Data From Phase 2 Trials of ELX-02 Expected This Year

Eloxx Pharmaceuticals announced it expects to share data later this year from the first four treatment groups of a Phase 2 program evaluating the investigational therapy ELX-02 in people with cystic fibrosis (CF) who have at least one G542X mutation. “Given the substantial efforts of our clinical team,…

Arrowhead Pauses ARO-ENaC Study Amid Safety Concerns

Arrowhead Pharmaceuticals has voluntarily paused new screening, enrollment, and dosing in its Phase 1/2 trial of ARO-ENaC, an investigational treatment for cystic fibrosis (CF). The decision came after unexpected signs of local lung inflammation occurred in an ongoing chronic toxicology study taking place in rats. “The safety of…

Low Aerobic Capacity Linked to Higher Risk of Exacerbations

Cystic fibrosis (CF) patients with lower aerobic exercise capacity — assessed with a non-invasive cardiopulmonary exercise test — have a 4.5 times higher risk of future pulmonary exacerbations, according to a small 10-year study in Greece. Aerobic capacity, or VO2peak, is the maximal amount of oxygen a person’s…

Featured Column

The Benefits of Being Sick

A banner for Lara's column, depicting a car on a road trip winding through a forest.
As columnist Lara Govendo reflects on her journey through CF and transplant, she realizes there are a surprising number of benefits.

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