Huntingtons Disease: From Chorea to Cure 💃🧬💡
Dr RR Baliga's "Got Knowledge Doc" Podkast
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Show Notes
🚨 Breakthrough in Huntington's Disease 🧬🧠✨
Exciting results from the pivotal Phase I/II AMT-130 gene therapy trial show a 75% slowing of disease progression at 36 months compared to controls. Key functional and cognitive endpoints (cUHDRS, TFC, SWRT, SDMT) also demonstrated significant benefit, alongside biomarker reduction in CSF neurofilament light chain 📊🔑.
💉 Delivered via precision stereotactic neurosurgery, AMT-130 was well-tolerated and shows clear evidence of a dose–response effect. A BLA submission is planned for 2026, potentially marking a landmark shift in Huntington's disease care.
🌍 For patients and families living with HD, these results provide long-awaited hope.
#HuntingtonsDisease #GeneTherapy #ClinicalTrials #Neurology #PrecisionMedicine #Innovation 🚀