Sionna Therapeutics is rated Hold after SION-719 failed its Phase 2a endpoint in cystic fibrosis when combined with Trikafta.
A multicenter Italian study and an accompanying commentary provide real-world evidence that early lumacaftor/ivacaftor therapy improves CFTR function biomarkers and early lung physiology in preschool ...
A new mouse study shows the approved cystic fibrosis drug ivacaftor reduced pulmonary fibrosis by suppressing senescence in ...
Expanded CFTR Variant Coverage Designed to Align with Updated Professional Recommendations "With evolving CFTR variant recommendations, laboratories are reassessing panel content to ensure alignment ...
A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly ...
Cystic fibrosis is a hereditary disease caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR protein is an ion channel that carries salts into and out ...
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A study of 50 adult patients with cystic fibrosis (CF) has found that challenged urine bicarbonate excretion may offer a new, simple, and safe quantification of cystic fibrosis transmembrane ...