PTC THERAPEUTICS, INC. — Investor Relations & Filings

PTC Therapeutics, Inc. is a global biopharmaceutical company that discovers, develops, and commercializes clinically differentiated medicines for children and adults with rare and serious diseases. The company focuses on conditions with high unmet medical needs, leveraging its expertise across multiple scientific platforms. These platforms include RNA biology, with a focus on splicing modification to control protein production, and targeting inflammation and ferroptosis pathways for central nervous system diseases. PTC has brought several pioneering treatments to market, including the first approved therapy for Duchenne muscular dystrophy, the first gene therapy administered directly into the brain, and the first small molecule splicing modifier. The company maintains a diversified pipeline of innovative therapies.