Transforming Lives
Innovation, fueled by science and a passion for patients
PTC is a global biopharmaceutical company that discovers, develops, and commercializes clinically differentiated medicines for children and adults living with serious diseases of high unmet need. Our ability to identify innovative new therapies and globally commercialize products is the foundation that drives investment in a diversified pipeline.
Focused research platforms at work for patients
Rare Journeys
In the Rare Journeys hub, discover stories from people in the rare disease community.
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.
Clinical Trials
We are committed to making progress in rare disease through clinical trial research.
Working as OnePTC
As One PTC, we feel the urgent call to help those with significant unmet needs. We are motivated to provide treatments and new possibilities for our patients.
Doing things that no one’s ever done before is hard. That doesn’t deter us.
If you’re looking to take on rewarding work with inspiring colleagues, come grow with us.
Making headlines
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PRESS RELEASE
PTC Therapeutics Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
PTC Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU. -
PRESS RELEASE
Sephience™ (sepiapterin) Granted Marketing Authorization by the European Commission for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
PTC Therapeutics today announced that Sephience™ (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and disease severities. -
NEWSROOM
Fostering Inclusive Workspaces Though Empathy, Authenticity and Unity
Our employees bring a wealth of perspectives and ideas, driving innovation and fostering more dynamic teams. To ensure we have a broad range of voices where decisions are made, we encourage all to lead with authenticity and empathy, which allows people to feel comfortable being vulnerable and safe so they can ask questions. We also promote embracing curiosity and our various perspectives.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
Explore our investor relations section and sign up for alerts on important PTC developments