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
Never-Ending Support
In recognition of National PKU Awareness Month in May, we're sharing rare journey stories of those living with phenylketonuria (PKU). Amanda, who lives with PKU, and her mom, Jill, share what it’s like to manage the condition every day. -
PRESS RELEASE
PTC Therapeutics to Participate in Upcoming Investor Conferences
PTC Therapeutics announced that its executives will speak at three upcoming conferences in May and June. -
PRESS RELEASE
PTC Therapeutics Reports Positive Topline Results from Month 24 Interim Analysis of PIVOT-HD Extension Study of Votoplam
PTC Therapeutics reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease progression for Stage 2 Huntington's disease (HD) patients following 24 months of votoplam treatment compared to an external natural history cohort.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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