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The latest news from PTC

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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  6. 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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  7. Jessica Fabus Cheng takes the DuchenneCAN Crown

    It is fair to say that Jessica Fabus Cheng is a very impressive woman who carries many crowns and titles including surgical nurse, sportswoman, mother, Duchenne carrier and cancer survivor, and Mrs. New York International 2023. She is also a former figure skater and in 2021 was listed as one of Taekwondo Life Magazine’s ‘10…
    Rare Disease Community
    November 21, 2023
    3 minutes
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  8. PTC Cares™: Providing Financial Assistance

    Navigating a rare disease diagnosis and treatment can be 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 and their families…
    Rare Disease Community
    November 21, 2023
    2 minutes
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  9. PTC Team in Brazil Earns Three Awards

    The PTC team in Brazil recently won three awards for their creative and impactful projects highlighting the rare disease community. First Up: The documentary series, “Living is Rare” (“Viver é Raro”) won the SABRE América Latina 2023 Award. The series was recognized with the award in the Public Education category. The world’s leading award in Public Relations, SABRE celebrates creative…
    Our Culture, Rare Disease Community
    November 20, 2023
    2 minutes
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  10. PTC Cares™: Helping to Navigate Insurance

    Navigating a rare disease diagnosis and treatment can be 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 and…
    Rare Disease Community
    November 17, 2023
    1 minute
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