TIMBERLINE study shows good safety and sweat chloride reductions with triple combo CFTR mo ...

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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 ...

Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...

Cystic fibrosis transmembrane conductance regulator (CFTR) is a cAMP-activated chloride channel expressed in a wide variety of epithelial cells, mutations of which are responsible for the hallmark ...

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 ...

Don't bet against Vertex Pharmaceuticals in cystic fibrosis.

To generate mice expressing CFTR specifically in ciliated cells, the EGFP cDNA in the previously described FOXJ1 construct 15 was replaced with the cDNA for human CFTR. 17 Transgenic mice were ...

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 ...

Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...

A preclinical study combining chemically modified suppressor tRNAs with inhaled lipid nanoparticles has restored functional ...