The latest news from PTC

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…

It has been 10 years since PTC entered the European market with a mission to improve the lives of people living with rare diseases.  In the past decade, we have pioneered transformative progress and created possibilities for the rare disease community across Europe. PTC was founded with the mission to leverage its novel drug discovery…

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 honor of Huntington’s disease (HD) Awareness Month, we are highlighting inspiring and impactful voices from the HD community. Watch the video below to learn more about this rare, hereditary, genetic disorder of the central nervous system, and hear from the global community about their hope for the future of HD.

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…

In this podcast episode we are discussing the topic of Nutrition and Duchenne with our guest speaker, Dr. Kindann Fawcett, Behavioral Health Scientist, Dietitian and Caregiver to her son with Duchenne Muscular Dystrophy.  Our conversation with Dr. Kindann Fawcett explores; Tune in to learn more about this topic. Visit the Take on Duchenne: North America…

Now 23, Luke Hains was 8 years old when his younger brother, Levi, was diagnosed with Duchenne muscular dystrophy. Looking after his brother and helping with his daily activities was part of life, even during college. While in the midst of figuring out the trajectory of his career during college, Luke realized that he could…

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…

Navigating a rare disease diagnosis and treatment can be time consuming and overwhelming, from getting a prescription, working through insurance coverage and getting the medication on time. For people living with rare diseases taking a PTC treatment, PTC Cares is here to help. PTC Cares helps take care of treatment coordination, so patients with a rare disease…

It is fair to say that Jessica Fabus Cheng is a very impressive woman who carries many crowns and titles including surgical nurse, sportswoman, mother, Duchenne carrier and cancer survivor, and Mrs. New York International 2023. She is also a former figure skater and in 2021 was listed as one of Taekwondo Life Magazine’s ‘10…