Advances in the cystic fibrosis drug development pipeline
C Esposito, M Kamper, J Trentacoste, S Galvin… - Life, 2023 - mdpi.com
Cystic fibrosis is a genetic disease that results in progressive multi-organ manifestations with
predominance in the respiratory and gastrointestinal systems. The significant morbidity and …
predominance in the respiratory and gastrointestinal systems. The significant morbidity and …
Emerging pharmacotherapies in cystic fibrosis
OJ McElvaney, C Gunaratnam… - Expert Review of …, 2018 - Taylor & Francis
Introduction: Cystic fibrosis (CF) is an autosomal dominant chloride channelopathy caused
by mutations in the CF transmembrane conductance regulator (CFTR) gene that results …
by mutations in the CF transmembrane conductance regulator (CFTR) gene that results …
Treatment options for cystic fibrosis: state of the art and future perspectives
EH Sears, EJ Gartman… - Reviews on recent clinical …, 2011 - ingentaconnect.com
Cystic fibrosis (CF) is an autosomal recessive disorder characterized by chronic lung and
sinus disease, impaired mucociliary clearance (leading to recurrent pulmonary infection) …
sinus disease, impaired mucociliary clearance (leading to recurrent pulmonary infection) …
Cystic fibrosis–Ten promising therapeutic approaches in the current era of care
Introduction Cystic fibrosis (CF) is a genetic disease affecting multiple organ systems.
Research and innovations in novel therapeutic agents and health care delivery have …
Research and innovations in novel therapeutic agents and health care delivery have …
Emerging drugs for cystic fibrosis
Introduction: Cystic fibrosis is an autosomal recessive disease, which is the result of a
genetic defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene …
genetic defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene …
Cystic fibrosis: exploiting its genetic basis in the hunt for new therapies
JL Kreindler - Pharmacology & therapeutics, 2010 - Elsevier
Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis
transmembrane conductance regulator (CFTR), an anion channel expressed in epithelial …
transmembrane conductance regulator (CFTR), an anion channel expressed in epithelial …
Unmet needs in cystic fibrosis
BK Rubin - Expert opinion on biological therapy, 2018 - Taylor & Francis
Introduction: Cystic fibrosis (CF) is a multisystem illness caused by abnormalities in the CF
transmembrane conductance regulator (CFTR) gene and protein. CFTR is an ion channel …
transmembrane conductance regulator (CFTR) gene and protein. CFTR is an ion channel …
Cystic fibrosis: current therapeutic targets and future approaches
Objectives Study of currently approved drugs and exploration of future clinical development
pipeline therapeutics for cystic fibrosis, and possible limitations in their use. Methods …
pipeline therapeutics for cystic fibrosis, and possible limitations in their use. Methods …
The future of cystic fibrosis treatment: from disease mechanisms to novel therapeutic approaches
SY Graeber, MA Mall - The Lancet, 2023 - thelancet.com
With the 2019 breakthrough in the development of highly effective modulator therapy
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …
The effect of CFTR modulators on airway infection in cystic fibrosis
The advent of Cystic fibrosis transmembrane receptor (CFTR) modulators in 2012 was a
critical event in the history of cystic fibrosis (CF) treatment. Unlike traditional therapies that …
critical event in the history of cystic fibrosis (CF) treatment. Unlike traditional therapies that …