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Huntingtons Disease: From Chorea to Cure 💃🧬💡

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Manage episode 508872973 series 2740117
Content provided by MasterMedFacts LLC and RR Baliga MD. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by MasterMedFacts LLC and RR Baliga MD or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://podcastplayer.com/legal.

🚨 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 🚀

  continue reading

697 episodes

Artwork
iconShare
 
Manage episode 508872973 series 2740117
Content provided by MasterMedFacts LLC and RR Baliga MD. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by MasterMedFacts LLC and RR Baliga MD or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://podcastplayer.com/legal.

🚨 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 🚀

  continue reading

697 episodes

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