[HTML][HTML] CFTR modulators: shedding light on precision medicine for cystic fibrosis

M Lopes-Pacheco - Frontiers in pharmacology, 2016 - frontiersin.org
Cystic fibrosis (CF) is the most common life-threatening monogenic disease afflicting
Caucasian people. It affects the respiratory, gastrointestinal, glandular and reproductive …

[HTML][HTML] CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine

M Lopes-Pacheco - Frontiers in pharmacology, 2020 - frontiersin.org
Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …

Discovery of CFTR modulators for the treatment of cystic fibrosis

M Lopes-Pacheco, N Pedemonte… - Expert Opinion on Drug …, 2021 - Taylor & Francis
Introduction Cystic fibrosis (CF) is a life-threatening inherited disease caused by mutations
in the gene encoding the CF transmembrane conductance regulator (CFTR) protein, an …

New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls

SC Bell, K De Boeck, MD Amaral - Pharmacology & therapeutics, 2015 - Elsevier
With the discovery of the CFTR gene in 1989, the search for therapies to improve the basic
defects of cystic fibrosis (CF) commenced. Pharmacological manipulation provides the …

Personalized medicine in CF: from modulator development to therapy for cystic fibrosis patients with rare CFTR mutations

M Harutyunyan, Y Huang, KS Mun… - … of Physiology-Lung …, 2018 - journals.physiology.org
Cystic fibrosis (CF) is the most common life-shortening genetic disease affecting~ 1 in 3,500
of the Caucasian population. CF is caused by mutations in the CF transmembrane …

Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives

BZ Schmidt, JB Haaf, T Leal, S Noel - … : advances and applications, 2016 - Taylor & Francis
Mutations of the CFTR gene cause cystic fibrosis (CF), the most common recessive
monogenic disease worldwide. These mutations alter the synthesis, processing, function, or …

Modulators of CFTR. Updates on clinical development and future directions

E Bardin, A Pastor, M Semeraro, A Golec… - European journal of …, 2021 - Elsevier
Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the
Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane …

[HTML][HTML] Therapeutic approaches for patients with cystic fibrosis not eligible for current CFTR modulators

I Fajac, I Sermet - Cells, 2021 - mdpi.com
Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene encoding the CFTR protein, a …

[HTML][HTML] State of the Art on Approved Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators and Triple-Combination Therapy

A Meoli, V Fainardi, M Deolmi, G Chiopris, F Marinelli… - Pharmaceuticals, 2021 - mdpi.com
Cystic fibrosis (CF) is the most common life-limiting inherited disease in Caucasian
populations, affecting approximately 80,000 people worldwide. CF is a complex multi-organ …

Triplet CFTR modulators: future prospects for treatment of cystic fibrosis

N Chaudary - Therapeutics and clinical risk management, 2018 - Taylor & Francis
Cystic fibrosis (CF) is an autosomal recessive genetic disease characterized by mutations in
the cystic fibrosis transmembrane conductance regulator (CFTR). CFTR is a chloride …