According to the Cystic Fibrosis Foundation (CFF), these drugs work by improving the production, function, or stability of the CFTR protein, helping reduce the symptoms of cystic fibrosis.

The first CF rat model, CFTR tm1sage (termed CFTR-/- or KO), was designed in collaboration with SAGE (now Horizon Discovery) and subsequently characterized at UAB and distributed to the CF community.

CFTR mutations lead to CF by causing CFTR protein ... Interrupt TRIKAFTA for significant elevations in LFTs or in the event of signs or symptoms of liver injury. Consider referral to a hepatologist.