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 |
|---|---|---|---|---|
| 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 | |
| Ataluren | Translarna™ | Suppresses premature stop mutation |
EMA-approved for Other Purpose | Restore CFTR Function |
| Azithromycin | Zithromax® | Antibiotic with anti-inflammatory properties |
FDA-approved for Cystic Fibrosis | Anti-Infective |
| Aztreonam | Cayston® | Antibiotic |
FDA-/EMA-approved for Cystic Fibrosis | Anti-Infective |
| Bortezomib | Velcade® | Proteasome inhibitor preventing premature CFTR breakdown |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function |
| Buserelin | Gonadotropin-Releasing Hormone analog restoring CFTR channel function |
EMA-approved for Other Purpose | Restore CFTR Function | |
| Ciclopirox | CFTR corrector |
FDA-approved for Other Purpose | Restore CFTR Function | |
| Cysteamine | Cystagon® | Proteostasis regulator, rescues the function of F508del-CFTR |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function |
