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
Click the links below to learn more about our actively enrolling trials
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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NEWSROOM
Honoring International Women’s Day Through Inclusion and Pride
In honor of #InternationalWomensDay, we connected with women leaders across PTC to learn what makes them proud to be a female leader at PTC and how they inspire inclusion. -
PRESS RELEASE
PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2023 Financial Results
PTC today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2023. -
NEWSROOM
In it Together: Rare Disease Day 2024
With our deep experience in the rare disease space for more than 25 years and commitment to two key therapeutic areas – neurology and metabolic disorders – we’re proud to support the rare disease community on Rare Disease Day and every day, because at PTC, we’re in it together.