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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.

Three of PTC's scientists
The Latest on PTC

Making headlines

  • NEWSROOM

    PTC Celebrates 10 Years of Impact in Europe

    It has been 10 years since PTC entered the European market with a mission to improve the lives of people living with rare diseases. In the past decade, we have pioneered transformative progress and created possibilities for the rare disease community across Europe.
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  • PRESS RELEASE

    PTC Therapeutics Announces Validation of Sepiapterin European MAA

    PTC Therapeutics announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA. "The validation of the MAA is an important step towards making sepiapterin available to children and adults affected by PKU in Europe," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The European submission is the first of several global submissions planned in 2024."
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  • PRESS RELEASE

    PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation

    PTC Therapeutics announced today that the European Commission (EC) has decided not to adopt the CHMP's negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorization of Translarna™ (ataluren) and has returned the opinion to the CHMP for re-evaluation.
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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