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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.

Pioneers in Modulating Gene and Protein Expression

  1. Nonsense Suppression

    Bypassing translation mutations to enable protein production
  2. Splicing

    Modulating splicing to control protein production
  3. Bio-e

    Targeting oxidative stress and inflammation pathways to treat CNS diseases and cancer
Working in the lab Working in the lab Working in the lab
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

Click the links below to learn more about our actively enrolling trials

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

  • DUCHENNECAN

    Luke & Elliott: Two Paths to Duchenne

    Now 23, Luke Hains was 8 years old when his younger brother, Levi, was diagnosed with Duchenne muscular dystrophy. Looking after his brother and helping with his daily activities was part of life, even during college. While in the midst of figuring out the trajectory of his career during college, Luke realized that he could…
    READ MORE
  • PRESS RELEASE

    PTC Therapeutics Provides Updates on Translarna™ Regulatory Activities

    On December 5, PTC Therapeutics, Inc. shared an update on Translarna™ (ataluren) regulatory activities in Europe and the United States.
    READ MORE
  • NEWSROOM

    Kevin Alexander: Owning Your Rare Disease Story

    Kevin Alexander lives with phenylketonuria (PKU) and has been an advocate for PKU, newborn screening, and rare disease awareness for over a decade. He’s Vice President of the Louisiana Metabolic Disorders Coalition, a volunteer for the National PKU Alliance, and a member of the International Society for Neonatal Screening.
    READ MORE
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Resources For
Healthcare Providers

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Resources For
Patients & Caregivers

Support for the PTC patient, family, and caregiver community

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Information for
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