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The latest news from PTC

  1. 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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  2. 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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  3. Managing Complexity with Passion: PTC Latin America

    Latin America (also referred to as LATAM) is a vast region with almost 600 million people – and therefore, many people there are impacted by rare diseases. “Having a presence in LATAM is incredibly important for PTC, as it allows us to increase access to our treatments for people living with rare diseases in this…
    Rare Disease Community
    October 4, 2023
    3 minutes
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  4. Eat Without Fear

    Imagine being unable to experience the delight of eating food in a restaurant without fear. For people living with the rare disease familial chylomicronemia syndrome (FCS), this is their unfortunate reality. FCS, which affects one in 700,000 to one million people, prevents the body from breaking down fat, or triglycerides, leading to an accumulation in…
    Rare Disease Community
    June 7, 2023
    3 minutes
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  5. 2022: A Year of Many Firsts

    For PTC, 2022 was a milestone-reaching, patient-supporting, award-winning, employee-engaging, community-driven year. It was also another year of many firsts. The first approved disease-modifying treatment for AADC deficiency, and the first marketed gene therapy directly infused into the brain, was approved in Europe and the UK. We also had the first global regulatory approval for treatment…
    Science & Innovation
    December 15, 2022
    4 minutes
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