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 |
|---|---|---|---|---|
| Verotoxin | Rescues endogenous misfolded proteins |
Pre-clinical | Restore CFTR Function | |
| pGM169/GL67A | Plasmid DNA encoding the CFTR gene |
Phase Two Clinical Trial | Restore CFTR Function |
