| T0429 |
Glucosamine |
Glucosamine is an amino sugar and a prominent precursor in the biochemical synthesis of glycosylated proteins and lipids. Supplemental glucosamine may help to rebuild cartilage and |
| T1805 |
Ataluren |
Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in |
| T1874 |
PPQ-102 |
PPQ-102, an effective CFTR inhibitor, can completely inhibit CFTR chloride current (IC50: 90 nM). |
| T2263 |
VX661 |
VX-661 is a small molecule that can be used as a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) gene function. |
| T2355 |
CFTRinh-172 |
CFTRinh-172 is a voltage-independent, selective CFTR inhibitor. |
| T2451 |
GlyH-101 |
GlyH-101 is a cell-permeable glycinyl hydrazone compound that blocks CFTR with Ki of 1.4 uM. |
| T2486 |
IOWH-032 |
IOWH032, a synthetic CFTR inhibitor, has been investigated for the treatment of cholera, diarrhea, and secretory diarrhea. |
| T2588 |
Ivacaftor |
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. |
| T2595 |
Lumacaftor |
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). |
| T3135 |
KM11060 |
KM11060 is a novel corrector of the F508del-CFTR trafficking defect, Correcting F508del-CFTR trafficking, increasing the amount of functional CFTR at the plasma membrane. |
| T5312 |
FDL169 |
FDL-169 is a novel and potent CFTR (Cystic fibrosis transmembrane conductance regulator) corrector for treating cystic fibrosis (CF) patients who carry the F508del mutation. |
| T7083 |
GLPG1837 |
GLPG1837 is an effective CFTR potentiator, with EC50s of 3 nM and 339 nM for F508del and G551D CFTR, respectively. |
