Patricia Inácio, PhD, science writer —

The European Commission has granted orphan drug designation to ARCT-032, an inhaled experimental treatment for cystic fibrosis (CF) developed by Arcturus Therapeutics. Orphan drug designation is designed to encourage developing therapies for rare and serious diseases, those affecting not more than 5 in every 10,000 people in the European Union…

People with cystic fibrosis (CF) can have abnormal levels or metabolism of three “essential nutrients” — vitamin D, calcium, and phosphate — even when using pancreatic enzyme replacement therapy (PERT) and taking dietary supplements, a small study suggests. “The findings of this study have many practical implications in…

Up to almost 30% of teenagers and adults with cystic fibrosis (CF) in the U.S. have smoked marijuana and/or cannabidiol (CBD), one of the active components of cannabis, according to an online survey. Use of e-cigarettes and traditional cigarettes was just as common among them.

An eight-week remote exercise program of moderate to high intensity increased leg muscle strength and reduced body fat in adults with cystic fibrosis (CF), a new study showed. While no differences were found in lung function and quality of life, the gains seen with the short-term duration of the exercise…

The Cystic Fibrosis Foundation has announced new funding of up to $15 million to Prime Medicine to further develop its gene-editing technology — called Prime Editing — for cystic fibrosis (CF). The award is part of the foundation’s $500 million Path to a Cure initiative, which aims…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease status to 4D-710, an inhaled gene therapy for people with cystic fibrosis (CF). The designation means 4D Molecular Therapeutics (4DMT) would be eligible to receive a priority review voucher from the FDA if its gene therapy…

Elevated levels of fecal calprotectin, measured over 1.5 years, significantly associated with poorer lung function in children with cystic fibrosis (CF), a European study reports. Levels of this protein, a biomarker of intestinal inflammation, also correlated with diarrhea in the young patients. Findings suggest that fecal calprotectin could originate…

In people with cystic fibrosis (CF), six months of treatment with Kaftrio — sold as Trikafta in the U.S. — significantly reduced the blood levels of molecules called chemokines that are involved in inflammation, new research shows. Noting that “exaggerated inflammation is a major driver of disease progression…

Restless legs syndrome (RLS), a disorder characterized by an irresistible urge to move legs or other body parts, especially during sleep, is highly prevalent among adults with cystic fibrosis (CF), a study shows. A higher percentage of hemoglobin A1c, or HbA1c, indicative of CF-related diabetes (CFRD), and…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ARCT-032, Arcturus Therapeutics’ inhaled experimental treatment for cystic fibrosis (CF). The designation is intended to encourage the development of therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides incentives,…