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 …

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 …

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) …

Cystic fibrosis–Ten promising therapeutic approaches in the current era of care

R Somayaji, DP Nichols, SC Bell - Expert opinion on …, 2020 - Taylor & Francis
Introduction Cystic fibrosis (CF) is a genetic disease affecting multiple organ systems.
Research and innovations in novel therapeutic agents and health care delivery have …

Emerging drugs for cystic fibrosis

R Amin, F Ratjen - Expert opinion on emerging drugs, 2014 - Taylor & Francis
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 …

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 …

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 …

Cystic fibrosis: current therapeutic targets and future approaches

MM Rafeeq, HAS Murad - Journal of translational medicine, 2017 - Springer
Objectives Study of currently approved drugs and exploration of future clinical development
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 …

The effect of CFTR modulators on airway infection in cystic fibrosis

C Harvey, S Weldon, S Elborn, DG Downey… - International Journal of …, 2022 - mdpi.com
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 …