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 |
|---|---|---|---|---|
| Pancrelipase enzyme | Pertzye® | Digestive enzymes |
FDA-approved for Cystic Fibrosis | Nutritional-GI-Other |
| Pancrelipase enzyme | Creon® | Digestive enzymes |
FDA-approved for Cystic Fibrosis | Nutritional-GI-Other |
| Pancrelipase enzyme | Pancreaze® | Digestive enzymes |
FDA-approved for Cystic Fibrosis | Nutritional-GI-Other |
| Pancrelipase enzyme | Zenpep® | Digestive enzymes |
FDA-approved for Cystic Fibrosis | Nutritional-GI-Other |
| 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 | |
| Recombinant human deoxyribonuclease I | Pulmozyme® | Thins and loosens mucus in the airways |
FDA-/EMA-approved for Cystic Fibrosis | Mucociliary Clearance |
| Sildenafil | CFTR activator |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function | |
| Sodium 4-phenylbutyrate | Induces CFTR function |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function | |
| Tadalafil | CFTR activator |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function |
