Add to ORKGCystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene on chromosome 7, most commonly diagnosed at birth via heel prick testing. Patients with CF suffer from a variety of complications including lung disease, gastrointestinal disease and pancreatic dysfunction leading to malabsorption and secondary diabetes mellitus. To date, treatment has been largely symptomatic, but the recent development of a new class of drug, the CFTR modulator, may be a game-changer for children with CF. CFTR modulators target the underlying pathophysiological process of CF itself, either the misfolding of protein channels (correctors) or the length of time functional ...
Nauman Chaudary Division of Pulmonary Disease and Critical Care Medicine, Department of Medicine, V...
Alexandria M Arends,1 Rebecca S Pettit2 1Department of Pharmacy, Indiana University Health, 2Departm...
Rare diseases affect 400 million individuals worldwide and cause significant morbidity and mortality...
Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the cystic fi...
Cystic fibrosis (CF) is an autosomal recessive genetic condition that is caused by variants in the c...
Cystic fibrosis (CF) is an autosomal recessive disorder due to mutations in the Cystic Fibrosis Tran...
Cystic fibrosis (CF) is a genetic disorder that causes multiorgan morbidity and premature death, mos...
Cystic fibrosis (CF) is caused by genetic mutations that affect the cystic fibrosis transmembrane co...
Cystic fibrosis (CF) is the most common life-limiting inherited condition in Caucasians. It is a mul...
Cystic fibrosis (CF) is the most common life-limiting inherited disease in Caucasian populations, af...
Introduction: Cystic Fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane Condu...
Cystic fibrosis (CF) results from mutations in the CF transmembrane conductance regulator (CFTR) gen...
Cystic fibrosis (CF) is the most common life-limiting inherited disease in Caucasian populations, af...
With the discovery of the CFTR gene in 1989, the search for therapies to improve the basic defects o...
Cystic fibrosis (CF) is a recessive disorder caused by mutations in the gene that encodes the CF tra...
Nauman Chaudary Division of Pulmonary Disease and Critical Care Medicine, Department of Medicine, V...
Alexandria M Arends,1 Rebecca S Pettit2 1Department of Pharmacy, Indiana University Health, 2Departm...
Rare diseases affect 400 million individuals worldwide and cause significant morbidity and mortality...
Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the cystic fi...
Cystic fibrosis (CF) is an autosomal recessive genetic condition that is caused by variants in the c...
Cystic fibrosis (CF) is an autosomal recessive disorder due to mutations in the Cystic Fibrosis Tran...
Cystic fibrosis (CF) is a genetic disorder that causes multiorgan morbidity and premature death, mos...
Cystic fibrosis (CF) is caused by genetic mutations that affect the cystic fibrosis transmembrane co...
Cystic fibrosis (CF) is the most common life-limiting inherited condition in Caucasians. It is a mul...
Cystic fibrosis (CF) is the most common life-limiting inherited disease in Caucasian populations, af...
Introduction: Cystic Fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane Condu...
Cystic fibrosis (CF) results from mutations in the CF transmembrane conductance regulator (CFTR) gen...
Cystic fibrosis (CF) is the most common life-limiting inherited disease in Caucasian populations, af...
With the discovery of the CFTR gene in 1989, the search for therapies to improve the basic defects o...
Cystic fibrosis (CF) is a recessive disorder caused by mutations in the gene that encodes the CF tra...
Nauman Chaudary Division of Pulmonary Disease and Critical Care Medicine, Department of Medicine, V...
Alexandria M Arends,1 Rebecca S Pettit2 1Department of Pharmacy, Indiana University Health, 2Departm...
Rare diseases affect 400 million individuals worldwide and cause significant morbidity and mortality...