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.
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.
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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NEWSROOM
Raise Your Voice for PKU
The Rare Disease Day 2026 theme of “More than you can imagine” reminds us that “rare diseases touch more lives, involve more conditions, and require more action than most people realize.” In recognition of this powerful global campaign, PTC honors the phenylketonuria (PKU) community by amplifying voices from across the globe. -
PRESS RELEASE
PTC Therapeutics to Participate at Upcoming Investor Conferences
PTC Therapeutics announced that its executives will speak at three upcoming conferences this March. -
NEWSROOM
Looking for Life’s Small Miracles
Tiffany, a mother of two who lives with Friedreich’s ataxia (FA), opens up about the challenges of navigating life with a physical disability while embracing her identity as a “whole person” – someone who is more than just her FA diagnoses.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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