Ataxia, Advocacy, and Accelerating Research: Inside the National Ataxia Foundation

Welcome back to Research Renaissance, presented by the Karen Toffler Charitable Trust. In this episode, host Deborah Westphal explores the cutting edge of rare disease research with special guests Andrew Rosen, CEO of the National Ataxia Foundation (NAF), and Dr. Lauren Moore, NAF’s Chief Scientific Officer.

Together, they unpack how the NAF has evolved from a small support-focused nonprofit into a strategic, science-driven organization that’s advancing treatments and reshaping what’s possible for people living with ataxia—a rare, progressive neurological condition affecting movement and coordination.

🔍 In this episode, we explore:

• The history and mission of the National Ataxia Foundation—from its 1957 founding to its current research initiatives
• What ataxia is, how it’s diagnosed, and why it’s so often misunderstood or misdiagnosed
• The diagnostic odyssey patients face and how genetic testing is both essential and still evolving
• How patient advocacy, family involvement, and rare-disease communities are accelerating the pace of scientific discovery
• Lauren’s personal journey from biomedical physics student to ataxia researcher—sparked by her father’s diagnosis
• Why wearable tech, remote trials, and AI are changing the game for neurological research
• The power of public-private partnerships and how the first FDA-approved ataxia treatment led to a $7.3B acquisition by Biogen
• Why “rare” doesn’t mean small—and why rare diseases deserve different standards of study, funding, and approval
  
  

🧠 Key Takeaway:

Treatments are on the horizon—but getting there takes relentless advocacy, strategic funding, and courageous participation from patients and families. As Andrew puts it: “We’d love to put ourselves out of business someday. Until then, we’re here to build the infrastructure that gets us there.”

🌐 Resources & Links:

• National Ataxia Foundation (NAF)
• Karen Toffler Charitable Trust
• Learn more about hereditary ataxias and ongoing research initiatives
• Support or participate in patient-driven research and advocacy

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 Available on Apple Podcasts, Spotify, and all major platforms. For more conversations at the intersection of neuroscience, technology, and purpose-driven science, follow us at tofflertrust.org or get in touch.

To learn more about the breakthroughs discussed in this episode and to support ongoing research, visit our website at tofflertrust.org.

Technical Podcast Support by Jon Keur at Wayfare Recording Co.