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HDClarity @hdclaritystudy.bsky.social · 6d

Following on from last weeks exciting news, please visit this link for more information on the AMT-130 gene therapy trial, which was found to slow disease progression in Huntington's disease by 75% eur01.safelinks.protection.outlook.com?url=https%3A...

Huntington's disease successfully treated for first time | BBC News

Huntington's disease has been successfully treated for the first time, say doctors. The disease runs through families, relentlessly kills brain cells and resembles a combination of dementia, Parkinson's and motor neurone disease. The new treatment is a type of gene therapy given during 12 to 18 hours of delicate brain surgery. An emotional research team became tearful as they described how data shows the disease was slowed by 75% in patients. Subscribe here: http://bit.ly/1rbfUog For more news, analysis and features visit: www.bbc.com/news #HuntingtonsDisease #Health #BBCNews

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HDClarity @hdclaritystudy.bsky.social · 13d

Patients on the high-dose treatment maintained independence for longer, with biomarkers also showing less brain cell damage.

This marks a huge milestone in HD research, as this is the first time a drug trial has reported continuing, statistically significant slowing of Huntington’s progression.

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HDClarity @hdclaritystudy.bsky.social · 13d

Researchers announced that a one time gene therapy, AMT-130 (developed by UniQure), slowed the progression of Huntington’s Disease by ~75% over three years.

Until now, no therapy could alter its course. AMT-130, delivered directly into the brain, silences the faulty gene driving the disease.

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HDClarity @hdclaritystudy.bsky.social · 13d

A major milestone was reached yesterday in neurodegenerative medicine, advancing hope for those affected by Huntington’s disease🧵

Huntington's disease successfully treated for first time

One of the most devastating diseases finally has a treatment that can slow its progression and transform lives, tearful doctors tell BBC.

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HDClarity @hdclaritystudy.bsky.social · 15d

Please visit hdclarity.net/request-samp... for more information on how to request HDClarity samples.

Request samples – HDClarity

hdclarity.net

HDClarity @hdclaritystudy.bsky.social · 15d

Sampling visits:
Since the end of June, a further 65 sampling visits have been completed, totalling 272 visits this year! This is by far our most active year yet since the study began in 2017, and we would once again like to thank our participants and dedication of all staff at our study sites.

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HDClarity @hdclaritystudy.bsky.social · 15d

We have now collected over 23 litres of cerebrospinal fluid (CSF)! This represents a huge number of samples available for researchers to request and study biomarkers in HD.

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HDClarity @hdclaritystudy.bsky.social · 15d

Exciting updates on the HDClarity study 🧵

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HDClarity @hdclaritystudy.bsky.social · 26d

HDClarity samples can also be used for method development, for example, validating new assays for HD biomarkers.

Any qualified investigator can request HDClarity samples for research purposes. Please visit hdclarity.net/request-samp... for more information.

Request samples – HDClarity

hdclarity.net

HDClarity @hdclaritystudy.bsky.social · 26d

Perhaps the most exciting use of HDClarity samples is their role in providing reference data for clinical trial endpoints, as well as serving as a comparison for evaluating therapeutic drug effects.

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HDClarity @hdclaritystudy.bsky.social · 26d

Research using HDClarity samples can also be used to understand disease progression specifically, due to the longitudinal nature of the study. Clinical measures can also be linked with biomarker data to support outcome prediction.

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HDClarity @hdclaritystudy.bsky.social · 26d

The samples requested by researchers can be used in many ways. The first is biomarker discovery and validation, which involves researchers identifying biomarkers involved in disease onset, progression and treatment response.

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HDClarity @hdclaritystudy.bsky.social · 26d

So far, over 12,000 vials of CSF, 7,000 vials of plasma and 3,000 vials of serum have been distributed to researchers around the world. These samples have been used across 36 projects, of which 9 projects are still ongoing and at various stages.

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HDClarity @hdclaritystudy.bsky.social · 26d

Since the study began in 2017, we have collected 76,016 vials of CSF, 64,180 vials of plasma, and 5,039 vials of serum. These samples can be requested by researchers to improve understanding of HD or to support the development of novel treatments.

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HDClarity @hdclaritystudy.bsky.social · 26d

The key aim of the HDClarity study is to collect high-quality cerebrospinal fluid (CSF) and blood samples over time in people with Huntington’s disease and healthy controls.

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HDClarity @hdclaritystudy.bsky.social · 26d

HDClarity samples: storage, distribution and utility 🧵

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