The latest news from PTC

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…

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…

Through recumbent trike riding, Kyle was able to approach the roadblock that was his FA diagnosis.