Advancing Research on Ataxia for Development of Therapeutics and Patient Education with Andrew Rosen NAF

Nov 13, 2025

Andrew Rosen, CEO of the National Ataxia Foundation, champions the fight against hereditary ataxias, focusing on both research and patient support. He discusses the challenges in drug development due to the absence of reliable biomarkers and outlines the Foundation's role in funding vital research projects. Rosen emphasizes the importance of genetic testing and the need for regulatory flexibility to accelerate treatment approvals. He also highlights the Foundation's efforts to connect patients, clinicians, and researchers to enhance awareness and care.

Ask episode

AI Snips

Chapters

Transcript

Episode notes

INSIGHT

NAF's Twofold Mission

The National Ataxia Foundation (NAF) pursues dual goals: accelerate treatments and support patients worldwide.
NAF funds researchers, translational work, and hosts the largest annual ataxia patient conference to unite community and science.

INSIGHT

Ataxia: Disease Versus Symptom

'Ataxia' can mean a hereditary disease or simply a symptom of poor coordination.
The hereditary ataxias form a large, evolving family of genetic movement disorders with varied presentations.

INSIGHT

Testing Exists But Biomarkers Lag

Genetic testing exists for many SCAs, now numbered up to SCA52 and available via panels.
A major challenge remains: lack of reliable biomarkers forces reliance on subjective clinical scoring.

Get the Snipd Podcast app to discover more snips from this episode

Get the app

Andrew Rosen, Chief Executive Officer of the National Ataxia Foundation, has a dual mission of accelerating research for treatments and cures for Ataxia while supporting the patients affected by this rare disease. Ataxia describes both a group of hereditary genetic diseases and symptoms of lack of coordination seen in other conditions. While genetic tests exist for hereditary Ataxia, a significant challenge in drug development has been the lack of a reliable biomarker. To encourage drug development, NAF has funded translational research and the world's most extensive study for SCAs, Spinocerebellar Ataxias, providing new data on the progression of the disease and for the design of future trials.

Andrew explains, "NAF has been around for a long time. We were founded in the late 1950s by a neurologist in western Minnesota who had a disease called Ataxia in his family. And NAF's mission has really been twofold ever since that. Our formal statement is that we are trying to accelerate the development of treatments and a cure while working to improve the lives of those living with Ataxia. And so we really focus on research and support. We do a lot in the research world. We fund grants for researchers who are looking into the various types of Ataxia. You'd be hard pressed to find an Ataxia researcher in the world, I think I can say now at this point that hasn't received a grant from NAF at one time or another in their career. We also do a lot of translational-type research. We fund the largest natural history study in Ataxias."

"So, the term ataxia itself is even complex because it both describes a genetic disease, and I think of that as capital 'A' - Ataxia, but it's also a symptom of many other conditions. So small 'a', if you will. Ataxia just means a lack of coordination. It comes from a Greek word. And so what I mean by both hereditary and other things is if you, for instance, have too much to drink, you would show signs of Ataxia. You might stumble when you walk, and your speech might be slurred. Those are the two classic symptoms of hereditary Ataxia. Right. So that's really the Ataxias that we at NAF focus on. So hereditary, as in passed from parent to child, there are several hundred forms of hereditary Ataxia, and they continue to find more as more specific genetic mutations are discovered year after year now."

#NationalAtaxiaFoundation #Ataxia #PatientAdvocacy #RareDisease #SCA

ataxia.org

Download the transcript here