Personalized Therapy for Individuals with Cystic Fibrosis

Cystic fibrosis, one of the most common fatal genetic diseases, causes difficulties in breathing, recurrent lung infections, and digestive disorders. Recent drug discoveries can address the underlying genetic defect, but only some patients respond positively to them. Given the side effects and cost ($300K+ per year per patient for a drug that needs to be administered lifelong), there is a pressing need for robust predictors of who will respond to the treatment. Dr. Felix Ratjen’s team is developing predictive tools to determine individualized treatment for navigating patient care, research goals, and ethics.