Translating Science
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.
Our Scientific Platforms
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.
Working as OnePTC
Clinical Trials
We are committed to making progress in rare disease through clinical trial research.
Making headlines
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NEWSROOM
International PKU Day: Making Space for Mental Health Conversations
This International PKU Day, E.S.PKU is shining a light on as an aspect of PKU that is often left in the shadows: mental health. PTC is proud to join the campaign by elevating the voices of people in the PKU community and those who support them, and by making space for these important conversations. -
NEWSROOM
Our 2025 Corporate Social Responsibility Report
PTC’s 2025 Corporate Social Responsibility (CSR) Report captures a year defined by focus, execution and measurable progress toward our mission: delivering clinically differentiated medicines for children and adults living with rare disorders. -
NEWSROOM
International Clinical Trials Day: Patients Lead the Way
On May 20, we recognize International Clinical Trials Day: a time to honor everyone who makes clinical research possible, especially the people living with rare diseases who choose to participate in clinical studies.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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