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The latest news from PTC

  1. What You Need to Know About Ferroptosis & Friedreich Ataxia (FA)

    An Informative Q&A with Jonathan Cherry, Global Project Leader 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 Jonathan Cherry, Global Project Leader, to learn more about…
    Science & Innovation
    May 17, 2024
    2 minutes
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  2. 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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  3. 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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  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. AADCAware Registry: An Example of the Value that Disease Registries Bring to AADC Deficiency

    By Rafael Sierra, Executive Director, Global Medical Affairs – Global Gene Therapy Lead Rare and ultra rare diseases present unique challenges for research and healthcare, especially when patients are geographically dispersed, and the condition presents with high heterogeneity. Disease registries have emerged as a valuable tool to continue understanding the disease progression and the natural…
    Science & Innovation
    October 16, 2023
    2 minutes
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  6. PTC joins Screen4Care to shorten the diagnosis journey for people living with rare diseases

    For too many people living with rare diseases, getting a correct diagnosis is a lengthy process that negatively impacts patients and their family. Through an innovative research collaboration called Screen4Care, PTC will be working alongside 36 international partners from academia, industry, and patient advocacy organizations to find solutions to shorten the path to diagnosis and…
    Rare Disease Community, Science & Innovation
    July 12, 2022
    1 minute
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