Patricia Inácio, PhD, science writer —

Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly lowered several pro-inflammatory blood markers with as early as one month of use in cystic fibrosis (CF) patients carrying F508del — the most common CF-causing mutation — and in parallel with gains in lung function. These are the findings of a small study that suggest…

Vertex Pharmaceuticals and England’s National Health Service (NHS) have reached an agreement that allows all current and future eligible cystic fibrosis (CF) patients to access three of the company’s approved CFTR modulators at low or no out-of-pocket cost. Kaftrio (elexacaftor/tezacaftor/ivacaftor, sold as Trikafta in the U.S.), Symkevi (tezacaftor/ivacaftor,…

Most parents of newborns diagnosed with cystic fibrosis (CF) or a different disease as part of U.S. newborn screening programs said understanding the test results was difficult and about a third said they lacked support from their child’s healthcare team, according to a national survey.

The use of the approved medication Trikafta (elexacaftor/tezacaftor/ivacaftor) was shown to significantly increase weight in people with cystic fibrosis-related diabetes (CFRD) and a low body mass index, or BMI, a measure of body fat, in a new study. The researchers say the study’s findings, which also showed…

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…