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Huntington’s disease (HD) is a neurodegenerative disease inherited in an autosomal dominant manner. It is caused by an expansion of cytosine, adenine, guanine (CAG) repeats within the huntingtin (HTT) gene, which is located on chromosome 4. This pathological expansion of CAG repeats results in the production of a mutant huntingtin protein with an abnormally long polyglutamine […]

Giuseppe Lauria Pinter, WCN 2021: The Unfolded Protein Response in ALS: Results of a Phase 2 Trial

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Published Online: Nov 8th 2021

touchNEUROLOGY met up with Prof. Giuseppe Lauria Pinter (University of Milan, Milan, Italy) to discuss the efficacy and outcome measures of a phase 2 study investigating guanabenz for the treatment of amyotrophic lateral sclerosis.

The abstract entitled: ‘The unfolded protein response in Amyotrophic lateral sclerosis: Results of a Phase 2 Trial’ was presented at the XXV Biennial World Congress of Neurology (WCN) 2021, 3 Oct 2021 – 7 Oct 2021.

Questions:

  1. What is known about endoplasmic reticulum stress in the pathogenesis of amyotrophic lateral sclerosis (ALS)? (0:19)
  2. What is the mechanism of action of guanabenz? (3:46)
  3. What were the aims and design of your Phase II trial? (5:53)
  4. What were the efficacy and outcome measures and how well were they achieved? (6:42)
  5. What were the conclusions of the investigators and what future studies are planned? (10:53)

Disclosures: Giuseppe Lauria Pinter has nothing to disclose in relation to this video.

Support: Interview and filming supported by Touch Medical Media. Interview conducted by Katey Gabrysch

Filmed as a highlight of WCN, Virtual 2021

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