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…

There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them. Here, “splicing” refers to PTC’s scientific platform that takes aim at modulating pre-messenger RNA (pre-mRNA) splicing to control protein production. This scientific platform together with the technology that powers it, enables the identification of small molecules, which…

PTC’s geographical footprint continues to expand as we fulfill our mission to provide access to best-in-class treatments for more patients around the world. Our recent expansion into Kazakhstan is a further achievement in this mission. In the first quarter of 2023, PTC officially opened its affiliate office in Almaty and hired the first employees.  Today,…

The bond between Luke and Elliott, who lives with Duchenne, is the foundation for new life paths.

It’s Calum Sinclair’s job to help ensure patient safety. As the Vice President, Global Pharmacovigilance (PV) at PTC, Calum leads a global team who collect, assess, and monitor the use and safety of PTC’s medicines, from clinical to commercial setting.   It’s a highly important role that Calum candidly enjoys and has throughout his extensive career…

Kevin, who lives with PKU, shares his passion for storytelling.

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…

Jessica uses her platform and titles, including Mrs. NY International, to advocate for Duchenne.

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…