News, talk and articles
The latest news from PTC
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Debunking Myths About PKU
In honor of International PKU Awareness Day, June 28, members of the community helped us to debunk common myths about PKU. Watch the video below to hear from PKU advocates, healthcare professionals, a person living with PKU and a caregiver as they share the facts: International PKU Awareness Day is a global day hosted by… -
Navigating Clinical Trials
Clinical trials provide the medical community with valuable research and data that helps to facilitate the development of new and potentially life-saving therapies. By participating in a clinical trial, you are taking an active role in the development and approval of new medicines and treatments. There is a lot to learn about clinical trials –… -
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… -
Kevin Alexander: Owning Your Rare Disease Story
Kevin Alexander lives with phenylketonuria (PKU) and has been an advocate for PKU, newborn screening, and rare disease awareness for over a decade. He’s Vice President of the Louisiana Metabolic Disorders Coalition, a volunteer for the National PKU Alliance, and a member of the International Society for Neonatal Screening. Kevin started by producing the video “My PKU… -
Roadmap to Newborn Screening Changes: Q&A With Judit Illes
Judit Illes, Director, Government Affairs & Public Policy at PTC recently contributed to a newly published white paper, “Pioneering the New Era of Newborn Screening.” We connected with Judit to learn more about newborn screening, why it is so important and what she hopes people take away from this comprehensive paper on proposed reforms to… -
Improving the Lives of Others
Neil Smith, Global PKU Project Leader for PTC Therapeutics, has always been driven to improve the lives of other people. Originally trained as a pharmacist, Neil has worked for more than two decades in the pharmaceutical industry, stepping into various roles at both large pharmaceutical companies and small biotech firms, and building a diverse skill… -
What’s So Special About 2023?
2023 is a meaningful, unique and thrilling year for PTC. It’s the company’s 25th anniversary, and it is poised to be a year of momentum as we anticipate significant trial readouts in multiple disease areas. We’re also working toward our ambitious goal to produce a therapy every two to three years based on our robust… -
National Family Caregivers Month: Thank You, Caregivers
National Family Caregivers Month (NFCM), which takes place each November, is a time to recognize and express gratitude for the more than 50 million Americans who provide care and medical assistance to their loved ones, including parents, children and siblings. We’d like to take this time to thank caregivers in the U.S. for their selfless… -
Creating My VIBE: My Voice is Inspirational, Brave and Empowering
At its core, PTC’s Patient Engagement team is deeply committed to understanding the patient experience. The team knows firsthand the importance of making direct connections and building relationships with people in the rare disease community. Working closely with patients, caregivers and patient advocacy groups, their goal is to offer support, connection, and resources that address… -
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…