Translating science Transforming lives
Innovation, fueled by science and a passion for patients
PTC is a global biopharmaceutical company that discovers, develops, and commercialises clinically differentiated medicines for children and adults living with serious diseases of high unmet need. Our ability to identify innovative new therapies and globally commercialise products is the foundation that drives investment in a diversified pipeline.
Groundbreaking Science
Our approved therapies in the U.S. and other countries include a series of firsts: the first approved treatment for Duchenne muscular dystrophy in the world, the first approved gene therapy directly infused into the brain, and the first approved small molecule splicing modifier. We continue to invest in a diverse pipeline for conditions with significant unmet need.
THERAPEUTIC AREAS
Diseases in focus
Phenylketonuria (PKU)
Metabolic condition caused by mutations in Phenylalanine hydroxylase (PAH), a key digestive enzyme, that causes cognitive disabilities and seizures