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
Apigenin

Flavonoid activator of CFTR

 Accessible Dietary Supplement Restore CFTR Function
AquADEKs® AquADEKs®

Antioxidant-enriched multivitamin supplement

 Accessible Dietary Supplement Nutritional-GI-Other
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
C18

Small molecule CFTR corrector

 Pre-clinical Restore CFTR Function
C3

Small molecule CFTR corrector

 Pre-clinical Restore CFTR Function
C4

Small molecule CFTR corrector

 Pre-clinical Restore CFTR Function