Translating science 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
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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Reaching for the Stars
Tracy, who lives with phenylketonuria (PKU), is a professional astrophysicist and works at NASA. In her opinion, living with PKU requires discipline to keep on top of managing the condition. -
PRESS RELEASE
PTC Therapeutics to Host R&D Day
PTC Therapeutics announced that it will host an R&D Day to discuss its pipeline on Dec. 2, 2025, at 9:30 a.m. EST in New York City. The event will also be webcast. -
NEWSROOM
In This Together
Sarah Chamberlin, the mother of a child with phenylketonuria (PKU) and a passionate advocate, shares her journey of turning a challenging diagnosis into an opportunity to create meaningful change for the PKU community.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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