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2022: A Year of Many Firsts

December 15, 2022
  1. 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…

  2. HD Awareness Month: Ashley’s Caregiver Perspective

    May is Huntington’s Disease Awareness Month, and we caught up with Ashley Clarke, an HD caregiver from Northern Ireland, on what this month means to her, how we can best honor HD Awareness Month and her advice for others who are affected by this disease. PTC: What does HD Awareness Month mean to you? Ashley:…

  3. Introducing Insightful Moments™, a Rare Disease Patient Education Series

    En español haz clic aquí Living with a rare disease or caring for someone with a rare disease comes with many challenges. In addition to processing new and complicated information and making countless decisions, it can be tough to know where to turn to for help, or where to even begin. To help support patients…

  4. What’s in Store for PTC in 2022

    Last week, we presented at the 40th Annual J.P. Morgan Healthcare Conference, an annual healthcare investment symposium connecting healthcare industry leaders, innovators, and the broader healthcare industry. Even though this year’s meeting was (once again) virtual, the excitement for everything that’s to come in 2022 was palpable – especially for us at PTC, as 2022…

  5. PTC in 2021: A Year of Expansion

    For PTC, 2021 was about expansion – not just literally, as we grew our global reach and added team members, but what we can do for patients, too, through our awareness efforts and continued dedication to scientific innovation. Expansion by definition is the act of becoming larger or more extensive. And in fact, we had so much good news in 2021, it was hard for us to pick the highlights to include…

  6. Our Commitment to the Rare Disease Community

    PTC was founded in 1998 by a scientist with a desire to serve patients with rare diseases and unmet medical needs. Over the course of our history, we have grown to a team of more than a thousand employees and developed new innovative therapies. However, we’ve always maintained our foundational commitment to our patients and…

  7. Ashley Clarke: Remember the Caregivers

    Ashley Clarke is an HD caregiver from Northern Ireland. She has served as board member for the Huntington’s Disease Association of Northern Ireland (HDANI) and operates the #Imnotdrunk lifestyle blog for members of the HD community. Follow her on Instagram at @imnotdrunklifestyleblog, and Facebook @imnotdrunkblog. Do you have a rare disease story to share? Contact…

  8. B.J. Viau: Turning Hope into Help for the Huntington’s Disease Community

    B.J. Viau is a lifelong Huntington’s disease (HD) advocate and has served as Board Chairman of the Huntington’s Disease Youth Organization (HDYO). The following article was contributed to the PTC Newsroom to help spread awareness about HD. Follow him on Twitter @BJsView. Do you have a rare disease story to share? Contact our news team…

  9. Passing the Baton in a Multiyear Race – Beating Huntington’s Disease

    When the time came for biologist Anu Bhattacharyya, Ph.D. to hand off leadership duties for the PTC Huntington’s disease (HD) research project to clinical project leader Brian Beers, she felt like she was passing the baton in a relay race. As with such a race, your heart continues to beat furiously for a few moments…

  10. Our CEO Has Big Plans

    PTC founder and Chief Executive Officer, Stuart Peltz, Ph.D., was recently the subject of a MassBio profile. When asked about the future of PTC, he had this to say: PTC has been growing rapidly over the last two years and we will continue to grow and expand in the upcoming year. We plan to launch…