Marisa Wexler, MS,  senior science writer—

Vertex Pharmaceuticals is asking the U.S. Food and Drug Administration to approve a first triple combination therapy —  elexacaftor (VX-445) plus tezacaftor, and ivacaftor (Kalydeco) — to treat cystic fibrosis (CF) patients who cannot use its other disease-modifying treatments or don’t benefit as intended. Triple combinations include…

Poorer lung function in pregnant women with cystic fibrosis (CF) is associated with a lower birth weight for offspring, and a higher likelihood of cesarean section — but not with changes in lung or metabolic parameters, new research suggests. The study, titled “Pregnancy outcome in women…

AzurRx BioPharma has initiated a Phase 2 clinical trial testing its oral capsule MS1819-SD, combined with standard porcine pancreatic enzyme replacement therapy (PERT), in people with cystic fibrosis (CF) who have exocrine pancreatic insufficiency (EPI). PERT is the standard of care for EPI associated with…

Adding cystic-fibrosis-specific variables to the lung allocation score allows for more accurate identification of patients who are likely to benefit from a lung transplant, according to a new study. The study, titled “Effect of Including Important Clinical Variables on Accuracy of the Lung Allocation Score for Cystic…

The diagnostics company BillionToOne has launched its first product for commercial and clinical use — a blood test designed to help diagnose hereditary diseases in fetuses. The new product, called UNITY, tests cell-free fetal DNA to aid in the diagnosis of cystic fibrosis (CF), spinal muscular atrophy,…

The experimental treatment eluforsen is safe for adults with cystic fibrosis (CF) caused by a mutation called F508del, a new study reports, supporting further investigation and development of the treatment. The study, titled “Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic…