Huntingtons Disease: From Chorea to Cure 💃🧬💡 episode artwork

EPISODE · Sep 28, 2025 · 5 MIN

Huntingtons Disease: From Chorea to Cure 💃🧬💡

from Dr RR Baliga's "Got Knowledge Doc" Podkast · host MasterMedFacts LLC

🚨 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 🚀

Episode metadata supplied by the publisher feed · Published Sep 28, 2025

Embed this episode

NOW PLAYING

Huntingtons Disease: From Chorea to Cure 💃🧬💡

0:00 5:14

No transcript for this episode yet

We transcribe on demand. Request one and we'll notify you when it's ready — usually under 10 minutes.

No similar episodes found.

No similar podcasts found.

Frequently Asked Questions

How long is this episode of Dr RR Baliga's "Got Knowledge Doc" Podkast?

This episode is 5 minutes long.

When was this Dr RR Baliga's "Got Knowledge Doc" Podkast episode published?

This episode was published on September 28, 2025.

Can I download this Dr RR Baliga's "Got Knowledge Doc" Podkast episode?

Yes. Use the download control on the episode player to save the publisher-provided media file.
URL copied to clipboard!