The latest news from PTC

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…

Navigating a rare disease diagnosis and treatment can be 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 and their families…

The PTC team in Brazil recently won three awards for their creative and impactful projects highlighting the rare disease community. First Up: The documentary series, “Living is Rare” (“Viver é Raro”) won the SABRE América Latina 2023 Award. The series was recognized with the award in the Public Education category. The world’s leading award in Public Relations, SABRE celebrates creative…

Navigating a rare disease diagnosis and treatment can be 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 and…

Felix Wu was four when he was diagnosed with Duchenne muscular dystrophy. Throughout his school career, he saw how Duchenne affected him, and how inclusive (or not) people could be. “People with disabilities have to think about so much more when we plan anything, just in terms of access,” Felix said. “At school, for example,…