Lumacaftor

Lumacaftor is a medication used to treat cystic fibrosis, a genetic disorder that affects the lungs and digestive system. It works by helping the faulty protein caused by the disease to fold correctly, allowing it to reach the cell surface and function properly. Specifically, Lumacaftor targets the defective CFTR protein, improving its activity and helping to restore some normal function of mucus clearance and salt balance in the body. It is often combined with another drug called ivacaftor to enhance overall effectiveness in improving lung function and reducing symptoms in patients with certain genetic mutations.