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Translating Science

Transforming Lives

Extending life’s moments for people living with rare diseases

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

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Splicing
Modulating splicing to control protein production
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brain-inflammation
Ferroptosis & Inflammation
Targeting oxidative stress and inflammation pathways to treat CNS diseases
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The PTC Pipeline

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.

Join Our Team

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.

PTC Lab Employees
The Latest on PTC

Making headlines

  • PRESS RELEASE

    PTC Therapeutics to Participate at Upcoming Investor Conferences

    PTC Therapeutics announced that the company will present a company overview at three upcoming investor conferences on Sept. 5, Sept. 6, and Sept. 17. The presentations will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics' website and will be archived for 30 days following the presentation.
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  • NEWSROOM

    Raising Voices for Duchenne

    World Duchenne Awareness Day is on Sept. 7. In honor of this year’s theme, “raise your voice for Duchenne”, we are highlighting remarkable individuals in the Duchenne community who are doing just that: using their voices to raise awareness of Duchenne, advocating for those living with the condition and inspiring the community to join them in amplifying their voices.
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  • NEWSROOM

    Checking In with Emerging Leaders at PTC

    The Emerging Leaders program was created specifically for individuals at PTC who demonstrate the passion and traits to become successful future leaders. Earlier this year, the company celebrated its third cohort of Emerging Leaders. We checked in with graduates from all three cohorts to learn more about their ELP experience, their biggest takeaways, and how the program prepared them for future leadership roles.
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Resources For
Healthcare Providers

PTC works closely with healthcare providers to support their needs

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Resources For
Patients & Caregivers

Support for the PTC patient, family, and caregiver community

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Information for
Investors

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