The latest news from PTC

Mark Rance, Senior Director, Clinical Development, PTC Therapeutics, has helped people living with the rare cancer leiomyosarcoma (LMS) in his work as a surgeon, and has seen firsthand the high unmet need for a treatment to improve the quality of life for this community. LMS is a rare and aggressive cancer found in smooth muscle.…

Judit Illes, Director, Government Affairs & Public Policy at PTC recently contributed to a newly published white paper, “Pioneering the New Era of Newborn Screening.” We connected with Judit to learn more about newborn screening, why it is so important and what she hopes people take away from this comprehensive paper on proposed reforms to…

In any conversation about patient advocacy for Duchenne muscular dystrophy, Pat Furlong’s name is bound to come up. Pat is the driving force behind Parent Project Muscular Dystrophy (PPMD), a patient advocacy organization founded by Pat and a group of parents of children with Duchenne in 1994. Since its founding, PPMD has played a pivotal…

Patient advocacy organizations play a vital role in giving a voice to those affected by rare diseases. They serve their communities by providing much needed resources and support, raising awareness and funds. These organizations also provide critical perspectives and education to industry partners, by sharing insights into the lives of patients and what they need…

We asked Luke Millington, our own PTC employee and wheelchair football player why he is passionate about the sport. Watch the video to learn why Luke has been playing for almost 12 years to date.

Football or soccer is one of the most exciting sports that unites people all over the world. Powerchair football is even more exciting as players with varying degrees of physical disability demonstrate immense capabilities and achieve incredible things. This is right ‘on the ball’ at PTC and our approach to helping improve the lives of…

There are many ways that people living with rare diseases and their loved ones can be advocates! We connected with Anne Bruns, Senior Director, Clinical Patient Engagement, Global, on what it means to advocate on behalf of a diagnosis, how to advocate about a rare disease, the importance of participating in clinical trials and the…

Arzu Mulayim, Senior Director, Medical Affairs at PTC, is responsible for the Middle East and North Africa region. In her role, Arzu works to support healthcare professionals in delivering care to patients with rare diseases and improving the lives of families. Outside of work, Arzu has dedicated herself to improving the lives of individuals and…

By Christian Werner, Executive Director, Global Medical Affairs – Global Duchenne Muscular Dystrophy Lead at PTC Individuals living with Duchenne are now living longer lives thanks to marked improvements in disease management.1 This is a positive development, but it also presents new challenges for healthcare professionals (HCPs) and caregivers who must provide continuous care to…

When you speak with our Global Patient Engagement (PE) Team, you can feel the passion and care they have for the patients they serve. At PTC, we have dedicated PE teams covering the U.S., Latin America, and EMEA (Europe, Middle East and Africa). The PE team is an incredibly important function of PTC, as they…