Stem cells could open trikafta to thousands with rare CF mutations

This study gave Trikafta, a cystic fibrosis (CF) drug, to 42 people with rare CF gene changes not normally treated with this medicine. Researchers checked if the drug improved lung function and sweat chloride levels. They also used skin or blood samples to grow stem cells in the lab, turning them into airway cells to see if they could predict who would benefit from Trikafta. The goal was to find a faster way to match rare CF mutations to existing treatments.