The latest news from PTC

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 –…

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…

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…

Latin America (also referred to as LATAM) is a vast region with almost 600 million people – and therefore, many people there are impacted by rare diseases. “Having a presence in LATAM is incredibly important for PTC, as it allows us to increase access to our treatments for people living with rare diseases in this…

Imagine being unable to experience the delight of eating food in a restaurant without fear. For people living with the rare disease familial chylomicronemia syndrome (FCS), this is their unfortunate reality. FCS, which affects one in 700,000 to one million people, prevents the body from breaking down fat, or triglycerides, leading to an accumulation in…