Although this drug combination is not a perfect fix, it helps the mutant CFTR protein to move some chloride. This movement of chloride reduces the symptoms of CF. Watch the webcast (starting at 3:02) ...

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.

Although modulators can't yet completely restore proper chloride flow, they can improve the flow enough to relieve symptoms for people with CF. The CFTR protein is shaped like a tunnel that can be ...

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.

CF can cause symptoms soon after a baby is born ... in the gene that makes cystic fibrosis transmembrane regulator (CFTR) protein. To have CF, a baby must get two copies of the CF gene, one from each ...

Until recently, drug development efforts have emphasised therapies treating downstream signs and symptoms resulting from the underlying CF biological defect: reduced function of the CF transmembrane ...

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.

Background The effect of complex alleles in cystic fibrosis (CF) is poorly defined for the lack of functional studies. Methods We studied 70 homozygous, compound heterozygous or heterozygous for CFTR ...