Translating Science
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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Our 2025 Corporate Social Responsibility Report
PTC’s 2025 Corporate Social Responsibility (CSR) Report captures a year defined by focus, execution and measurable progress toward our mission: delivering clinically differentiated medicines for children and adults living with rare disorders. -
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International Clinical Trials Day: Patients Lead the Way
On May 20, we recognize International Clinical Trials Day: a time to honor everyone who makes clinical research possible, especially the people living with rare diseases who choose to participate in clinical studies. -
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Finding Strength in the “Giant Family” of the FA Community
Kailey, who lives in Louisiana with her boyfriend and two dogs, was diagnosed with Friedreich’s ataxia (FA) after years of concerning symptoms and multiple kinds of testing. She describes FA as feeling like your brain and body aren’t communicating.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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