Huntington's disease treated for first time
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Re: Huntington's disease treated for first time
#2More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas...
> AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT)
and the actual announcement: https://uniqure.gcs-web.com/news-releases/news-release-detai...
> 75% slowing of disease progression as measured by Unified Huntington’s Disease Rating Scale (p=0.003)
> 60% slowing of disease progression as measured by Total Functional Capacity (p=0.033)
> 88% slowing of disease progression as measured by Symbol Digit Modalities Test (p=0.057)
> 113% slowing of disease progression as measured by Stroop Word Reading Test (p=0.0021)
> 59% slowing of disease progression as measured by Total Motor Score (p=0.1741)
Re: Huntington's disease treated for first time
#3This is incredible, what a miraculous thing for sufferers and their families. Feels rare to see such a such an unquestionably good news story these days.
Re: Huntington's disease treated for first time
#4Quick skimmed, is there a peer reviewed paper?
Re: Huntington's disease treated for first time
#5More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas... > AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT) and the actual announcement: https://uniqure.gcs-web.com/news-…
What do those progression numbers mean in terms of outlook? For example, if someone is treated before showing symptoms (as they know they inherit it) is the progression slowing enough to give them a normal life expectancy and quality of life?
Re: Huntington's disease treated for first time
#6More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas... > AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT) and the actual announcement: https://uniqure.gcs-web.com/news-…
Why does it have to be delivered through brain surgery?
Re: Huntington's disease treated for first time
#7More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas... > AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT) and the actual announcement: https://uniqure.gcs-web.com/news-…
What do those progression numbers mean in terms of outlook? For example, if someone is treated before showing symptoms (as they know they inherit it) is the progression slowing enough to give them a normal life expectancy and quality of life?
It’s covered in the article
>It means the decline you would normally expect in one year would take four years after treatment, giving patients decades of "good quality life", Prof Sarah Tabrizi told BBC News.
>The first symptoms of Huntington's disease tend to appear in your 30s or 40s and is normally fatal within two decades – opening the possibility that earlier treatment could prevent symptoms from ever emerging.
Re: Huntington's disease treated for first time
#8More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas... > AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT) and the actual announcement: https://uniqure.gcs-web.com/news-…
Why does it have to be delivered through brain surgery?
Guessing: bypassing the blood-brain barrier.
Re: Huntington's disease treated for first time
#9More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas... > AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT) and the actual announcement: https://uniqure.gcs-web.com/news-…
Why does it have to be delivered through brain surgery?
I’m assuming the viral vector can’t pass the blood-brain barrier.
Re: Huntington's disease treated for first time
#10Wow. Just an incredible medical development