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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NEWSEROOM
The Importance of Sharing Your Story
On this International Ataxia Awareness Day, we're sharing Sam's journey living with Freidreich's ataxia (FA), how his life changed after diagnosis, and his message to the FA community. -
PRESS RELEASE
PTC Therapeutics to Participate at Upcoming Investor Conferences
Today PTC announced that its executives will speak at three upcoming conferences in September. -
PRESS RELEASE
PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2025 Financial Results
PTC announced a corporate update and financial results for the second quarter ended June 30, 2025.
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