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The latest news from PTC

  1. What You Need to Know About Ferroptosis

    Our research efforts at PTC are focused on two scientific platforms – splicing and ferroptosis and inflammation – where PTC has unique expertise to discover and advance innovative therapies to the clinic. We recently sat down with Jeff Trimmer, Senior VP, Research Site Head, to learn more about ferroptosis, how it relates to inflammation and…
    Science & Innovation
    March 20, 2024
    3 minutes
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  2. Honoring International Women’s Day Through Inclusion and Pride

    When asked why it’s important to recognize Women’s History Month (WHM) in March, women leaders at PTC agree: WHM is a significant moment in time to honor the achievements and impact of women across history. “Women’s History Month demonstrates the vast contributions women have made across all areas – politics, music, fine arts, drama, literature,…
    Our Culture
    March 8, 2024
    2 minutes
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  3. In it Together: Rare Disease Day 2024

    Rare Disease Day, which takes place every year on the last day of February, is a global movement aimed at raising awareness of the 7,000+ rare diseases and more than 300 million people worldwide who live with a rare disease. With our deep experience in the rare disease space for more than 25 years and…
    Rare Disease Community
    February 21, 2024
    3 minutes
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  4. Take on Duchenne Podcast: Nutrition and Duchenne

    In this podcast episode we are discussing the topic of Nutrition and Duchenne with our guest speaker, Dr. Kindann Fawcett, Behavioral Health Scientist, Dietitian and Caregiver to her son with Duchenne Muscular Dystrophy.  Our conversation with Dr. Kindann Fawcett explores; Tune in to learn more about this topic. Visit the Take on Duchenne: North America…
    Rare Disease Community
    December 19, 2023
    2 minutes
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  5. A Whole Team in One Molecule: PTC’s Splicing Platform

    There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them. Here, “splicing” refers to PTC’s scientific platform that takes aim at modulating pre-messenger RNA (pre-mRNA) splicing to control protein production. This scientific platform together with the technology that powers it, enables the identification of small molecules, which…
    Science & Innovation
    December 18, 2023
    2 minutes
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  6. PTC’s New Horizons in Kazakhstan

    PTC’s geographical footprint continues to expand as we fulfill our mission to provide access to best-in-class treatments for more patients around the world. Our recent expansion into Kazakhstan is a further achievement in this mission. In the first quarter of 2023, PTC officially opened its affiliate office in Almaty and hired the first employees.  Today,…
    Business & Finance, Our Culture
    December 13, 2023
    2 minutes
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  7. 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…
    Rare Disease Community
    December 5, 2023
    3 minutes
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  8. An Important Job and Rewarding Career

    It’s Calum Sinclair’s job to help ensure patient safety. As the Vice President, Global Pharmacovigilance (PV) at PTC, Calum leads a global team who collect, assess, and monitor the use and safety of PTC’s medicines, from clinical to commercial setting.   It’s a highly important role that Calum candidly enjoys and has throughout his extensive career…
    Science & Innovation
    December 5, 2023
    2 minutes
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  9. 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. Kevin started by producing the video “My PKU…
    Rare Disease Community
    December 3, 2023
    4 minutes
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  10. PTC Cares™: Supporting Specialty Pharmacy

    Navigating a rare disease diagnosis and treatment can be time consuming and overwhelming, from getting a prescription, working through insurance coverage and getting the medication on time. For people living with rare diseases taking a PTC treatment, PTC Cares is here to help. PTC Cares helps take care of treatment coordination, so patients with a rare disease…
    Rare Disease Community
    November 28, 2023
    2 minutes
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