Innovation, fueled by science and a passion for patients
PTC is an established global biopharmaceutical company that delivers transformative therapies for people living with rare diseases. For 25 years, we have been harnessing our scientific platforms to create new therapies that address the underlying cause of the disease and deliver on our promise to create more moments for those who count on us.
Focused research platforms at work for patients
Challenging what's possible
Harnessing groundbreaking science for patients has led to many firsts – the first approved treatment for Duchenne in the world, the first approved gene therapy directly infused into the brain, and the first approved small molecule splicing modifier. Our innovative pipeline continues to expand as we discover and develop treatments using the newest technologies available.
Diseases in focus
Aromatic l-amino acid decarboxylase (AADC) deficiencyPatients with AADC deficiency lack dopamine, an essential neurotransmitter that controls movement
Duchenne muscular dystrophy (DMD)Rare and fatal genetic disorder resulting in progressive muscle weakness from early childhood
Phenylketonuria (PKU)Metabolic condition caused by mutations in PAH, a key digestive enzyme, that causes cognitive disabilities and seizures