Tailored CF drug trials offer hope for patients with rare mutations

This study aims to find effective treatments for people with cystic fibrosis who have rare gene mutations not covered by current drugs. Researchers will test FDA-approved CF drugs on nasal cells in the lab, then confirm the best option in a personalized N-of-1 trial. Up to 50 participants aged 6 and older with rare CFTR variants will be enrolled. The goal is to improve lung function by at least 5%.