The latest news from PTC

Tiffany, a mother of two who lives with Friedreich’s ataxia (FA), opens up about the challenges of navigating life with a physical disability while embracing her identity as a “whole person” – someone who is more than just her FA diagnoses. In these videos, she highlights the importance of community in overcoming obstacles and finding…

For Sam, publishing his first book was nerve-wracking – it chronicled his Friedreich’s ataxia (FA) diagnosis and was therefore very personal. He wasn’t sure what people would think, but he was pleased with the positive reception of his book. Sam is a passionate writer and has since published five more books. In this video, Sam…

Alex, who lives with Friedreich’s ataxia (FA), encourages others in the FA community to get involved.

Andrea lives with Friedreich’s ataxia (FA), a rare, genetic, and progressive neuromuscular disease that mainly affects the central nervous system and the heart. She has shared her story with PTC to help spread awareness about FA.  “My name is Andrea Garcia, and I was diagnosed with FA when I was five years old.  Around the…

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's ataxia community and strives to raise awareness of FA.

Brittany encourages others with Friedreich's 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.

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 –…

An Informative Q&A with Jonathan Cherry, Global Project Leader Our research efforts at PTC are focused on two scientific platforms – splicing and inflammation and ferroptosis – where PTC has unique expertise to discover and advance innovative therapies to the clinic. We recently sat down with Jonathan Cherry, Global Project Leader, to learn more about…