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health1d ago

https://www.bbc.co.uk/news/articles/cevz13xkxprohttps://www.bbc.com/news/articles/cevz13xkxprohttps://www.theguardian.com/science/2025/sep/24/huntingtons-disease-treated-successfully-for-first-time-in-gene-therapy-trial
Bbc.co.uk and 16 more
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Highlights
  • A one-time gene therapy can markedly slow the progression of Huntington's disease, potentially paving the way for the first ever treatment to alter the course of this rare, inherited brain disorder.
  • In a small trial of 29 people in early Huntington's decline, a high-dose therapy delivered into the brain slowed the disease by 75% over three years versus untreated controls.
  • The therapy uses a harmless virus to deliver a microRNA that suppresses the mutant huntingtin gene, reducing production of the toxic protein in brain cells.

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References

Huntington's disease successfully treated for first time

https://www.bbc.co.uk/news/articles/cevz13xkxproBbc.co.uk
Huntington's disease successfully treated for first time

Huntington's disease successfully treated for first time

https://www.bbc.com/news/articles/cevz13xkxproBbc.com
Huntington's disease successfully treated for first time

Huntington’s disease treated successfully for first time in gene therapy trial

https://www.theguardian.com/science/2025/sep/24/huntingtons-disease-treated-successfully-for-first-time-in-gene-therapy-trialTheguardian.com
Huntington’s disease treated successfully for first time in gene therapy trial

New gene therapy slows progression of Huntington's disease by 75%

https://news.sky.com/story/new-gene-therapy-slows-progression-of-huntingtons-disease-by-75-13437292News.sky.com
New gene therapy slows progression of Huntington's disease by 75%

Huntington’s treatment slows disease for first time in major breakthrough

https://www.independent.co.uk/news/health/huntingtons-disease-treatment-cure-breakthrough-ucl-b2832800.htmlIndependent.co.uk
Huntington’s treatment slows disease for first time in major breakthrough

Breakthrough Gene Therapy Slows Huntington's Disease by 75%

https://www.sciencealert.com/breakthrough-gene-therapy-slows-huntingtons-disease-by-75Sciencealert.com
Breakthrough Gene Therapy Slows Huntington's Disease by 75%

Huntington’s disease breakthrough: what to know about the gene therapy

https://www.newscientist.com/article/2497677-huntingtons-disease-breakthrough-what-to-know-about-the-gene-therapy/Newscientist.com
Huntington’s disease breakthrough: what to know about the gene therapy

Huntington’s disease is treated successfully for the first time

https://www.thetimes.com/uk/healthcare/article/huntingtons-disease-news-treatment-uniqure-fxtlw2g3lThetimes.com
Huntington’s disease is treated successfully for the first time

Experimental gene therapy found to slow Huntington’s disease progression, company says | CNN

https://www.cnn.com/2025/09/24/health/huntingtons-disease-slow-progression-uniqure-trialCnn.com
Experimental gene therapy found to slow Huntington’s disease progression, company says | CNN

Huntington’s disease treated for first time using gene therapy

https://www.nature.com/articles/d41586-025-03139-9Nature.com
Huntington’s disease treated for first time using gene therapy

Doctors successfully treat one of the world's most devastating diseases for the first time

https://www.unilad.com/news/health/doctors-huntingtons-disease-treatment-first-846736-20250924Unilad.com
Doctors successfully treat one of the world's most devastating diseases for the first time

UniQure's therapy slows Huntington's disease progression in trial; Shares more than triple

https://www.cnbc.com/2025/09/24/uniqures-therapy-slows-huntingtons-disease-progression-in-trial.htmlCnbc.com
UniQure's therapy slows Huntington's disease progression in trial; Shares more than triple

Hope for sufferers of devastating brain disease as new treatment has 75% success rate

https://nypost.com/2025/09/25/health/new-huntingtons-disease-treatment-has-75-success-rate/Nypost.com
Hope for sufferers of devastating brain disease as new treatment has 75% success rate

A gene therapy success in Huntington's and Trump's autism announcement

https://www.statnews.com/2025/09/25/readout-loud-podcast-huntingtons-uniqure-autism-trump-tylenol-leucovorin/Statnews.com
A gene therapy success in Huntington's and Trump's autism announcement

Has a groundbreaking trial found a cure for Huntington's?...

https://www.dailymail.co.uk/health/article-15129201/Has-groundbreaking-trial-CURE-Huntingtons-Researchers-successfully-slow-development-cruel-disease-75-CENT.htmlDailymail.co.uk
Has a groundbreaking trial found a cure for Huntington's?...

Breakthrough as cruel disease that kills brain cells treated for first time ever

https://www.ladbible.com/news/health/huntingtons-disease-successfully-treated-breakthrough-596407-20250924Ladbible.com
Breakthrough as cruel disease that kills brain cells treated for first time ever

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