Is  TRIKAFTA® the current breakthrough therapy for CF??? 

Cystic Fibrosis (CF) is a common autosomal recessive genetic disease that affects the lungs, pancreas, and other organs causing problems with breathing and digestion by thickening the fluids of mucous, sweat, and digestive juices. It is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is widely expressed in epithelial cells that regulate salt and fluid homeostasis in many tissues. 3, 4 

CF signs and symptoms can vary, depending on the severity of the disease. Most signs and symptoms of CF affect the respiratory system, digestive system, and reproductive system. 3  

Respiratory Symptoms

The thick and sticky mucus can block the tubes that carry air in and out of the lungs, which can cause signs and symptoms of:


Respiratory complications:




Digestive Symptoms

The thick and sticky mucus can block tubes that carry digestive enzymes from the pancreas to the small intestines. With low to no production of digestive enzymes, that can lead to: 2, 3 


Digestive complications: 2, 3 


Reproductive Symptoms

Thickness of the mucus can also affect reproductive health complications: 1, 3 

References