PTC Therapeutics, Inc. is a biopharmaceutical company that discovers, develops, and commercializes medicines for children and adults with serious diseases that have high unmet needs. The company focuses on conditions such as phenylketonuria (PKU), Duchenne muscular dystrophy (DMD), and Friedreich’s ataxia (FA). PTC's product portfolio includes the first approved treatment for DMD and the first approved gene therapy infused directly into the brain. The company operates globally and invests in a diverse pipeline of therapies.
