Transforming Lives
Innovation, fueled by science and a passion for patients
PTC is an established global biopharmaceutical company that delivers transformative therapies for people living with rare diseases. For 25 years, we have been harnessing our scientific platforms to create new therapies that address the underlying cause of the disease and deliver on our promise to create more moments for those who count on us.
Focused research platforms at work for patients
Challenging what's possible
Harnessing groundbreaking science for patients has led to many firsts – the first approved treatment for Duchenne in the world, the first approved gene therapy directly infused into the brain, and the first approved small molecule splicing modifier. Our innovative pipeline continues to expand as we discover and develop treatments using the newest technologies available.
Clinical Trials
We are committed to making progress in rare disease through clinical trial research.
We love what we do
PTC employees are working to change patients’ lives every single day.
We are motivated by our patients and the drive for them to have more moments with their families, made possible in part by our research and therapies.
If you’re looking to take on rewarding work with inspiring colleagues, come grow with us.
Making headlines
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Alex’s Day-to-Day with FA
Alex lives with Friedreich’s ataxia (FA), a rare, genetic, and progressive neuromuscular disease that mainly affects the central nervous system and the heart. He proudly shared his story with us to help raise awareness about FA. -
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Kurt’s Journey in Filmmaking and Community Building
Meet Kurt, a dynamic documentary filmmaker living with phenylketonuria (PKU). In honor of PKU Awareness Month in the U.S., Kurt shares the challenges and triumphs of balancing his demanding career – including long, physically taxing video shoot days – with his unique dietary needs. -
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Taking Control of Her Huntington’s Disease Story
Evonne was motivated to undergo genetic testing for Huntington’s disease (HD) after her father’s diagnosis, seeking to avoid the struggles he faced in his HD journey.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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