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The latest news from PTC

  1. 25 Remarkable Stories: 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
  2. 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