Background Time has seen management for Cystic Fibrosis (CF) advance drastically, most
recently in the development of the disease-modifying triple combination therapy …

Cystic fibrosis (CF) is a life-shortening monogenic disease caused by mutations in the gene
encoding the CF transmembrane conductance regulator (CFTR) protein, an anion channel …

Tremendous progress has been made toward achieving pharmacologic restoration of cystic
fibrosis transmembrane conductance regulator (CFTR) function in people with CF (pwCF) …

Background While the clinical benefits of CFTR modulators are clear, their high prices
render them inaccessible outside of the world's richest countries. Despite this, there is …

Abstract Background Approximately 4–8% of the world suffers from a rare disease. Rare
diseases are often difficult to diagnose, and many do not have approved therapies. Genetic …

Mutation-agnostic treatments such as airway gene therapy have the potential to treat any
individual with cystic fibrosis (CF), irrespective of their CF transmembrane conductance …

Cystic fibrosis (CF) is an autosomal recessive monogenic disease caused by mutations in
the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. Although F508del …

Background Predictions based on patient-derived materials of CFTR modulators efficacy
have been performed lately in patient-derived cells, extending FDA-approved drugs for CF …

Objective: To determine the efficacy of the first triple CFTR protein modulators in children
and adolescents with cystic fibrosis. Methods: Systematic review and meta-analysis were …

Cystic fibrosis (CF) is a frequent genetic disease in Caucasians that is caused by the
deletion of F508 (ΔF508) in the nucleotide binding domain 1 (NBD1) of the CF …