On October 28th-29th 2025, Euro-ataxia hosted the International Ataxia Patient Conference in Amsterdam. This two-day event was attended by representatives from 18 ataxia patient organisations from Europe and North America. We were also joined by representatives from six pharmaceutical companies, and a number of ataxia researchers.
We heard updates on clinical programmes on a range of ataxias, from Lexeo Therapeutics, Biogen, Biohaven, IntraBio, Solaxa and Larimar. We were joined by specialists in accessing treatments, who presented information on the new Joint Clinical Assessment route being rolled out in the EU. We heard experiences from those working in Muscular Dystrophy field, who recently undertook a range of projects aimed at getting access to treatment. As potential ataxia treatments are now reaching the drug regulatory and reimbursement processes, we can learn a lot from other conditions that have worked on access to treatments in recent years. To end day one of the meeting, two researchers described their work on ensuring that ataxia rating scales (such as SARA and mFARS) are clinically meaningful to people living with ataxia.
On day two of the meeting, we heard about burden of illness studies being conducted by Biohaven (on SCAs) and Biogen (on FA) in collaboration with patient groups and ataxia clinical experts. Representatives of patient organisations in the US (National Ataxia Foundation, NAF), Greece (HEFAA), the UK (Ataxia UK), and France (AFAF) presented their experiences around drug development, from the NAF Drug Development Collaborative, to supporting the Health Technology Assessment (HTA) process in France by collecting information from people living with FA. The Health Technology Assessment is the process by which individual countries decide whether to provide a treatment on their health service. Although the process is individual to each country, the information required and lessons learned are applicable across countries and across different types of ataxia.
To end the meeting, we discussed the next steps that patient organisations need to take to ensure we are ready for future ataxia trials and potential treatments. This includes harmonising the information collected across different countries, so we can gather high quality data with which to represent the ataxia community.
The event was a great success, and we look forward to continuing our work with the international ataxia community to amplify the voice and experiences of people living with and affected by ataxia.
We are grateful to the sponsors of this event: Biogen, Lexeo Therapeutics and Biohaven.
You can view the full programme and presentations below:
Tuesday:
Session 1 – Chair – Barry Hunt, Ataxia UK
2pm Welcome – Sue Millman, Ataxia UK and Secretary of Euro-ataxia
2:05pm CTAX Highlights – Prof Paola Giunti, UCL/UCLH
2:15pm Updates on clinical programmes
- Greg Aubert, Lexeo Therapeutics, An Update on Friedreich Ataxia Cardiomyopathy Gene Therapy Clinical Programs
- Annemarie Drenth, Medical Lead, Biogen
- Melissa Beiner, Biohaven
- Sara Alabaf, Head of Clinical Development, IntraBio, A phase III, randomised, placebo-controlled crossover trial with N-acetyl-L-Leucine for Ataxia-Telangiectasia
- Mary Hogan, Solaxa, SCA27B Clinical Trial Update
- Rusty Clayton, Larimar
3pm Overcoming challenges in ataxia trials, regulatory approvals and HTAs
- Greg Aubert, Lexeo Therapeutics, Overcoming Challenges in Ataxia Trials, Regulatory Approvals, & HTAs
- Jon Cherry, PTC Therapeutics, Overcoming Challenges in Registrational Ataxia Trials: Balancing the choice of endpoints, selection of patient population, and trial duration
Session 2 – Chair – Sue Millman, Ataxia UK and Secretary of Euro-ataxia
3:40pm Navigating the new Joint Clinical Assessment route in Europe – Sheela Upadhyaya, Life Science Advisor in Rare Diseases
4:05pm What other conditions can teach us about preparing for patient access – Josie Godfrey, Realise Advocacy
4:30pm The AGI SARA qualification programme – Prof Thomas Klockgether, DZNE, Bonn
4:45pm What’s in an mFARS score? Linking clinical rating scales to daily function – Dr Christian Rummey, Clinical data science
5pm Closing remarks and discussion – Vassilis Karatzias, HEFAA
Wednesday:
Session 3 – Chair – Stéphan Rouillon, AFAF and Euro-ataxia Board member
9am Burden of illness studies
- Laura Heller, Biohaven
- Annemarie Drenth, Biogen, Burden of Illness Study
9:45am NAF Drug Development Collaborative – Andrew Rosen, National Ataxia Foundation
10am Surveys and experiences with regulators or HTA process from patient groups
- Greek HTA experience with the approval of Omaveloxelone for Friedreich Ataxia patients – Vassilis Karatzias, HEFAA
- Collecting information to support ataxia research and the HTA process – Emily Cutting, Ataxia UK
- Gathering FA patients’ perceptions for HTA processes – example of France – Cécilia Estrella, AFAF