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The latest news from PTC

  1. 25 Remarkable Stories: Supporting the Supporters

    Patient advocacy organizations play a vital role in giving a voice to those affected by rare diseases. They serve their communities by providing much needed resources and support, raising awareness and funds. These organizations also provide critical perspectives and education to industry partners, by sharing insights into the lives of patients and what they need…
    Rare Disease Community
    September 20, 2023
    3 minutes
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  2. Luke Millington on the Ball

    We asked Luke Millington, our own PTC employee and wheelchair football player why he is passionate about the sport. Watch the video to learn why Luke has been playing for almost 12 years to date.
    Rare Disease Community
    September 19, 2023
    1 minute
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  3. PTC’s Top 3 Goals for the FIPFA Powerchair Football World Cup

    Football or soccer is one of the most exciting sports that unites people all over the world. Powerchair football is even more exciting as players with varying degrees of physical disability demonstrate immense capabilities and achieve incredible things. This is right ‘on the ball’ at PTC and our approach to helping improve the lives of…
    Rare Disease Community
    September 14, 2023
    4 minutes
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  4. Advocacy in Action: Diagnosis & Disease Awareness

    There are many ways that people living with rare diseases and their loved ones can be advocates! We connected with Anne Bruns, Senior Director, Clinical Patient Engagement, Global, on what it means to advocate on behalf of a diagnosis, how to advocate about a rare disease, the importance of participating in clinical trials and the…
    Rare Disease Community
    September 13, 2023
    3 minutes
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  5. Arzu’s Dedication to a Cause – Professionally and Personally

    Arzu Mulayim, Senior Director, Medical Affairs at PTC, is responsible for the Middle East and North Africa region. In her role, Arzu works to support healthcare professionals in delivering care to patients with rare diseases and improving the lives of families. Outside of work, Arzu has dedicated herself to improving the lives of individuals and…
    Rare Disease Community
    September 12, 2023
    2 minutes
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  6. Why It’s Imperative to Continue to Improve the Care of Adults Living With Duchenne

    By Christian Werner, Executive Director, Global Medical Affairs – Global Duchenne Muscular Dystrophy Lead at PTC Individuals living with Duchenne are now living longer lives thanks to marked improvements in disease management.1 This is a positive development, but it also presents new challenges for healthcare professionals (HCPs) and caregivers who must provide continuous care to…
    Rare Disease Community
    September 6, 2023
    3 minutes
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  7. 25 Remarkable Stories: Supporting and Empowering the Rare Disease Community

    When you speak with our Global Patient Engagement (PE) Team, you can feel the passion and care they have for the patients they serve. At PTC, we have dedicated PE teams covering the U.S., Latin America, and EMEA (Europe, Middle East and Africa). The PE team is an incredibly important function of PTC, as they…
    Rare Disease Community
    August 30, 2023
    3 minutes
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  8. My VIBE: Every Story Deserves to be Told

    Last year, the PTC U.S. Patient Engagement team created a new podcast called My VIBE: My Voice is Inspirational, Brave and Empowering – and the response from the rare disease community has been overwhelmingly positive.  My VIBE gives patients, caregivers, families and friends a forum to share their story and journey living with a rare disease. The…
    Rare Disease Community
    August 21, 2023
    2 minutes
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  9. Take on Duchenne Podcast: A Scientist’s Quest to Solve Duchenne Muscular Dystrophy

    Dr. Ellen Welch is the Chief Scientific Officer at PTC who has been researching and tackling Duchenne for more than 20 years. In this podcast episode, Dr. Welch reflects on scientific advances in the last two decades and how it has contributed to progress in the Duchenne space.    She also explains the growing appreciation…
    Rare Disease Community
    August 18, 2023
    2 minutes
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  10. What are Natural History Studies and Why are They Important?

    Knowing the course of a given disease over time provides important information to drug developers on how the disease progresses in the absence of intervention, providing clues on how best to treat it. This information is obtained through what is known as a natural history study: an observational study that follows a group of people…
    Rare Disease Community
    August 15, 2023
    3 minutes
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