We’re All in This Together: A Brief Recap of the CTAX Conference and Amsterdam International Ataxia Patient Conference

The end of October 2025 was a busy time for the research team here at NAF. We had delegates at two different research conferences: the Clinical Trials in Inherited Ataxias (CTAX) conference and the Amsterdam International Ataxia Patient Conference. Here are a few highlights from each of the meetings.

The CTAX Conference was all about ataxia clinical trials. There were over 120 attendees, including researchers, industry members, regulators, and patient advocates. It was hosted by four organizations; NAF, FARA, Ataxia UK, and the Ataxia Global Initiative. Topics at this meeting included:

• Updates on progress on current ataxia clinical trials, from companies like Arrowhead Pharmaceuticals, VICO Therapeutics, Solid Biosciences, Neurocrine Bioscience, Lexeo Therapeutics, Biogen Inc., Biohaven Pharmaceuticals, IntraBio Inc., Solaxa Inc., and Larimar Therapeutics.
• Discussion about how Natural History data and Real-World Evidence can be used to convince regulators about the safety or efficacy of future ataxia therapies. Real-World Evidence is data collected about patient experiences with a medication separate from controlled clinical trials, such as electronic health record analysis or experience surveys. It’s data from the real world, instead of a controlled clinical environment.
• Conversations about the best way to measure ataxia symptoms and progression. This includes Biomarker measurement, clinical assessment like the SARA scale or mFARS, and patient-reported outcomes like the PROM-Ataxia. We talked about what measurements are suited for different contexts.
• Examining how to best design clinical trials for ataxia. There are special challenges that occur when you try to design a clinical trial for ataxia – these disorders are slowly progressing, there is variability between patients, and there are different symptoms to consider. Traveling to trial sites may also be challenging. Researchers need to take all these factors into account when designing a clinical trial.

The Amsterdam International Ataxia Patient Conference was all about how patient advocacy organizations can help support ataxia clinical trials. Attendees included NAF, FARA, Ataxia UK, and several other ataxia patient advocacy organizations from Europe, Canada, and the United States. This meeting was hosted by Euro-Ataxia, a consortium of European ataxia patient advocacy organizations. Topics at this meeting included:

• Examining the challenges of conducting ataxia clinical trials and logistics for gaining regulatory approval. Different pharmaceutical companies from the CTAX meeting shared updates, as well as difficulties they have faced. We also discussed the differences between regulatory agencies, such as the FDA and the European Medicines Agency.
• Discussing what ataxia patient advocacy organizations can do to advocate for patient access to new therapies. We talked about how advocacy organizations can help gather data to help measure the impact of living with ataxia. Three European ataxia groups also shared how they have been advocating to their governments for people with Friedreich Ataxia to access Omaveloxolone.

Attending both conferences back-to-back was a bit of a whirlwind. Lots of great discussions, but also a lot of work to do. The NAF team will be taking what we learned and applying it to our work back stateside.