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Translating Science

Transforming Lives

Delivering transformative medicines to children and adults with serious diseases of high unmet need.

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

Splicing
Modulating splicing to control protein production
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Inflammation & Ferroptosis
Targeting oxidative stress and inflammation pathways to treat CNS diseases
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Rare Journeys

In the Rare Journeys hub, discover stories from people in the rare disease community.

Our Products

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.

Join Our Team

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.

PTC Lab Employees
The Latest on PTC

Making headlines

Resources For
Healthcare Providers

PTC works closely with healthcare providers to support their needs

Resources For
Patients & Caregivers

Support for the PTC patient, family, and caregiver community

Information for
Investors

Explore our investor relations section and sign up for alerts on important PTC developments