News, talk and articles
The latest news from PTC
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PKU Reimagined Series: Watch Emma’s Inspiring Story
Emma shares her story about living with PKU and her advice for others. -
Pam and Kelsey’s PKU Story
Pam and her daughter, Kelsey, live with phenylketonuria (PKU), a rare, inherited metabolic disease which affects the brain. -
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… -
Owning Your Rare Disease Story
Kevin, who lives with PKU, shares his passion for storytelling.