Glucosamine is an amino sugar and a prominent precursor in the biochemical synthesis of glycosylated proteins and lipids. Supplemental glucosamine may help to r...
Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne M...
PPQ-102, an effective CFTR inhibitor, can completely inhibit CFTR chloride current (IC50: 90 nM). |
VX-661 is a small molecule that can be used as a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) gene function. |
CFTRinh-172 is a voltage-independent, selective CFTR inhibitor. |
GlyH-101 is a cell-permeable glycinyl hydrazone compound that blocks CFTR with Ki of 1.4 uM. |
IOWH032, a synthetic CFTR inhibitor, has been investigated for the treatment of cholera, diarrhea, and secretory diarrhea. |
Ivacaftor (VX-770) is a potentiator of CFTR targeting G551D-CFTR (EC50: 100 nM) and F508del-CFTR (EC50: 25 nM) in Fisher rat thyroid cells, respectively. |
VX-809 is a CFTR modulator that corrects the folding and trafficking of CFTR protein. It enhances F508del-CFTR protein maturation in FRT cells (EC50: 100 nM). |
KM11060 is a novel corrector of the F508del-CFTR trafficking defect, Correcting F508del-CFTR trafficking, increasing the amount of functional CFTR at the plasm...
GLPG1837 is an effective CFTR potentiator, with EC50s of 3 nM and 339 nM for F508del and G551D CFTR, respectively. |
FDL-169 is a novel and potent CFTR (Cystic fibrosis transmembrane conductance regulator) corrector for treating cystic fibrosis (CF) patients who carry the F50...
Elexacaftor is a modulator of cystic fibrosis transmembrane conductance regulator (CFTR) and also facilitates the processing and trafficking of CFTR to increase...
PG01 is effective on ΔF508 (Ka of 0.3 μM). PG01 is also an effective CFTR Cl- channel potentiator. PG01 increases ΔF508-CFTR Cl- current after adding Forskol...
PTI-428 is a specific amplifier of the cystic fibrosis transmembrane conductance regulator. |