News

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

More than half of children and teens with cystic fibrosis (CF) who do not show breathing difficulties during physical exercise were found to have reduced cardiorespiratory fitness (CRF), a measure of oxygen supply to the muscles, according to a single-center study in the Netherlands. Notably, glucose intolerance and…

HIT-CF, a European Union-funded research project, is working to provide personalized disease-modifying treatments to cystic fibrosis (CF) patients with rare CFTR mutations who lack effective treatment options. “We hope that within five years, these patients will have new drugs,” Kors van der Ent, MD, PhD, professor in pediatric…

Risk factors for obstructive sleep apnea (OSA) — a condition in which airflow is blocked intermittently during sleep — differ between children and adults with cystic fibrosis (CF), according to a study. Upper airway conditions, such as enlarged tonsils and inflamed sinuses, appear to be important risk factors for…

A new tool has been developed to help people self-screen for symptoms of cystic fibrosis (CF), which may be particularly helpful for identifying the disease in nonwhites — namely in Black, Indigenous, and people of color (BIPOC), populations that are particularly vulnerable to delayed or incorrect diagnoses. Called the…

Note: This story has been updated to clarify that the Cystic Fibrosis Foundation has funded more than $100 million in various studies through its Infection Research Initiative, not that it has raised funding for future research.  The Cystic Fibrosis Foundation (CFF) has funded more than $100 million in various studies to…

The bacterial composition within the airways of children with cystic fibrosis (CF) differs from those with primary ciliary dyskinesia (PCD), a disorder also characterized by mucus buildup and lung infections, a study reported. Its researchers, who followed children with either disease for about one year, found airway bacterial diversity…

The Cystic Fibrosis Foundation (CFF) has appointed Steven Rowe, MD, from the University of Alabama at Birmingham, as its new chief scientific officer. Rowe is the director of the university’s Gregory Fleming James Cystic Fibrosis Research Center and currently holds the Nancy R. and Eugene C. Gwaltney family endowed…

A team of scientists in New York has created small molecules called antisense oligonucleotides that could be used to “skip over” the mutated part of the CFTR gene to treat cystic fibrosis (CF) patients with a specific “nonsense” mutation and no available therapies. Findings were published in the study, “…

The triple combination therapy Kaftrio restores the function of CFTR — the defective protein in cystic fibrosis (CF) — and associated chloride and bicarbonate flow in gastrointestinal “mini-organs” derived from a patient carrying the most common CF-causing mutation, a study shows. These findings highlight the benefits of Vertex…