Filter the drugs by the phase of research, how they help and the responsive mutations.
Cystic fibrosis is a genetic disease caused by mutations in the CFTR gene. Browse the
database of
drugs treating specific mutations compiled by the AIM tool.
| Drug | Marketed as | Description | Phase of research | How it helps |
|---|---|---|---|---|
| Paromomycin | Aminoglycoside capable of translational readthrough |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function | |
| Polymyxin B | Antibiotic |
FDA-/EMA-approved for Other Purpose | Anti-Infective | |
| Quercetin | Flavonoid activator of CFTR |
Accessible Dietary Supplement | Restore CFTR Function | |
| RDR1 | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
| RDR3 | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
| Recombinant human deoxyribonuclease I | Pulmozyme® | Thins and loosens mucus in the airways |
FDA-/EMA-approved for Cystic Fibrosis | Mucociliary Clearance |
| Resveratrol | Natural phenolic antioxidant, rescues CFTR expression |
Accessible Dietary Supplement | Restore CFTR Function | |
| SAPK substrate protein 1 | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
| SPX-101 | Blocks the function of sodium channel in lungs, maintaining fluid to improve mucus clearance |
Phase Two Clinical Trial | Mucociliary Clearance | |
| STAT1 | CFTR corrector |
Pre-clinical | Restore CFTR Function |
