Sarah Tabrizi
Lead researcher at University College London who led the Huntington's disease trial described in the episode and who explains the gene therapy that slows disease progression.
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Mar 6, 2026 • 1h 35min
Current Events - uniQure and the FDA
Professor Sarah Tabrizi, neurologist and Huntington's researcher, and Dr. Victor Sung, clinician-researcher on AMT-130, discuss AMT-130 gene therapy delivery and trial design. They debate use of EnrollHD as a propensity-matched external control, surgical and imaging details, ethical issues around long blinding, FDA skepticism, and regulatory pathways.
