The latest news from PTC

In honor of International Ataxia Awareness Day (IAAD), recognized on September 25th each year, we are joining forces with the global ataxia community in their efforts to bring awareness to this rare disease. The theme of this year’s day is “Ask Me About Ataxia” – encouraging people to learn more about ataxia by asking questions.…

Kailey is grateful for the Friedreich ataxia community and strives to raise awareness of FA.

Brittany encourages others with Friedreich ataxia to take their time accepting their diagnosis.

Tomáš wants to remain as independent and active as possible with FA for as long as possible.

PTC research highlights opportunities to improve the transition experience for young adults living with Duchenne In 2023, PTC supported a research project conducted by a patient-focused consultancy Redline Strategic exploring the experience for teenagers, young adults and caregivers living with Duchenne muscular dystrophy (Duchenne) as they transition from pediatric care to adult care. The research…

World Duchenne Awareness Day is on Sept. 7. In honor of this year’s theme, “raise your voice for Duchenne”, we are highlighting remarkable individuals in the Duchenne community who are doing just that: using their voices to raise awareness of Duchenne, advocating for those living with the condition and inspiring the community to join them…

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.