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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PRESS RELEASE
PTC Therapeutics Provides Update on Commercial Performance and R&D Pipeline at 43rd Annual J.P. Morgan Healthcare Conference
PTC Therapeutics provided an update on the Company's progress and its outlook for 2025 at the 43rd Annual J.P. Morgan Healthcare Conference on January 13. A recording of the live webcast can be found on the Events and Presentations page under the Investors section of our website. -
PRESS RELEASE
PTC Therapeutics Announces Vatiquinone NDA Submission to FDA for the Treatment of Children and Adults Living with Friedreich's Ataxia
PTC Therapeutics announced today the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich's ataxia (FA) to the U.S. Food and Drug Administration (FDA). -
NEWSROOM
Pam and Kelsey’s PKU Story
Pam and her daughter, Kelsey, live with phenylketonuria (PKU), a rare, inherited metabolic disease which affects the brain. In honor of PKU Awareness Day, recognized on Dec. 3 each year in the U.S., we’re sharing Pam and Kelsey’s incredible PKU story.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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