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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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. -
PRESS RELEASE
PTC Therapeutics Provides Update at J.P. Morgan Annual Healthcare Conference
PTC provided an update on the Company's progress and its outlook for 2026. Matthew B. Klein, M.D., Chief Executive Officer, discusses these updates in the Company's presentation at the J.P. Morgan 2026 Healthcare Conference on Monday, January 12. -
PRESS RELEASE
PTC Announces Approval of its PKU Treatment in Japan
Sephience™ (sepiapterin) has been approved by the Japanese Ministry of Health, Labor and Welfare (MHLW) for the treatment of children and adults living with phenylketonuria (PKU).
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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