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

CFTR potentiator

Pre-clinical Restore CFTR Function

Small molecule CFTR corrector

Pre-clinical Restore CFTR Function

Proteasome inhibitor preventing premature CFTR breakdown

Pre-clinical Restore CFTR Function

CFTR corrector and potentiator

Pre-clinical Restore CFTR Function

Bactericidal combination of bovine lactoferrin and hypothiocyanite

Phase One Clinical Trial Anti-Infective

CFTR corrector

Pre-clinical Restore CFTR Function

Leukotriene A4 hydrolase inhibitor reducing inflammation

Phase Two Clinical Trial Anti-Inflammatory
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

NMD inhibitor leading to full-lenght protein synthesis from nonsense mutation-containing RNA

FDA-approved for Other Purpose Restore CFTR Function