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 |
|---|---|---|---|---|
| Curcumin | CFTR potentiator |
Accessible Dietary Supplement | Restore CFTR Function | |
| Cysteamine | Cystagon® | Proteostasis regulator, rescues the function of F508del-CFTR |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function |
| DMA | CFTR corrector and potentiator |
Pre-clinical | Restore CFTR Function | |
| Docosahexaenoic Acid | Lowers oxidative stress |
Accessible Dietary Supplement | Anti-Inflammatory | |
| ETX001 | TMEM16A potentiator |
Pre-clinical | Mucociliary Clearance | |
| Elexacaftor | Trikafta™ | CFTR corrector |
FDA-approved for Cystic Fibrosis | Restore CFTR Function |
| Eluforsen | 33-nucleotide antisense oligonucleotide designed to restore CFTR function |
Phase One Clinical Trial | Restore CFTR Function | |
| Endothelin 1 | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
| Epigallocatechin gallate | Flavonoid improving CFTR function |
Accessible Dietary Supplement | Restore CFTR Function | |
| Epoxomicin | Proteasome inhibitor preventing premature CFTR breakdown |
Pre-clinical | Restore CFTR Function |
