News

Self-screening Tool Developed for Nonwhite CF Populations

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…

Exon-Skipping Shows Potential to Treat Nonsense Mutations in CF

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, “…

Kaftrio Rescues Chloride, Bicarbonate Deficits in GI Tissues

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…