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 |
---|---|---|---|---|
1-EBIO | CFTR potentiator |
Pre-clinical | Restore CFTR Function | |
9-aminoacridine | Small molecule CFTR corrector |
Pre-clinical | Restore CFTR Function | |
ALLN | Proteasome inhibitor preventing premature CFTR breakdown |
Pre-clinical | Restore CFTR Function | |
ALLO | CFTR corrector and potentiator |
Pre-clinical | Restore CFTR Function | |
ALX-009 | Bactericidal combination of bovine lactoferrin and hypothiocyanite |
Phase One Clinical Trial | Anti-Infective | |
AP2M1 | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
Acebilustat | Leukotriene A4 hydrolase inhibitor reducing inflammation |
Phase Two Clinical Trial | Anti-Inflammatory | |
Alpha 1 Anti-Trypsin | Glassia® | Inhibitor of neutrophil elastase attenuating airway inflammation |
FDA-approved for Other Purpose | Anti-Inflammatory |
Amikacin | Arikayce® | Suppresses premature stop mutation |
FDA-approved for Cystic Fibrosis | Anti-Infective,Restore CFTR Function |
Amlexanox | NMD inhibitor leading to full-lenght protein synthesis from nonsense mutation-containing RNA |
FDA-approved for Other Purpose | Restore CFTR Function |