Clinical Trials

A clinical trial is a research study done to evaluate new potential treatments in people. At PTC, we are committed to bringing new treatments to patients with rare and life-threatening diseases. Ataluren  is our lead product candidate for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. Ataluren is currently in clinical trials for patients with nonsense mutation Duchenne muscular dystrophy and nonsense mutation cystic fibrosis. For information on ataluren for genetic disorders, click here.