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

Rescues endogenous misfolded proteins

Pre-clinical Restore CFTR Function

Plasmid DNA encoding the CFTR gene

Phase Two Clinical Trial Restore CFTR Function