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 |
---|---|---|---|---|
KB001 | Targets a Pseudomonas aeruginosa virulence factor |
Phase Two Clinical Trial | Anti-Inflammatory | |
Levofloxacin | Quinsair® | Antibiotic |
FDA-/EMA-approved for Cystic Fibrosis | Anti-Infective |
Lipase | RELiZORB® | Digestive enzyme cartridge |
FDA-approved for Cystic Fibrosis | Nutritional-GI-Other |
Lumacaftor | Orkambi® (ivacaftor/lumacaftor) | CFTR corrector |
FDA-/EMA-approved for Cystic Fibrosis | Restore CFTR Function |
MCG1516A | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
MG-132 | Proteasome inhibitor preventing premature CFTR breakdown |
Pre-clinical | Restore CFTR Function | |
MGC16186 | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
MPB | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
Mannitol | Bronchitol® | Osmotic agent helping to moisten and thin the mucus in the airways |
EMA-approved for Cystic Fibrosis | Mucociliary Clearance |
Miglustat | Zavesca® | CFTR corrector |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function |