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Huntington’s disease successfully treated for the 1st time with gene therapy
Posted on 9/28/25 at 10:12 pm
Posted on 9/28/25 at 10:12 pm
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UniQure's (UQ1.F), opens new tab experimental gene therapy for Huntington's disease slowed progression of the brain disorder by 75% in an early-to-mid stage study, it said on Wednesday.
Huntington's is a rare inherited brain disorder, which steadily worsens and typically leads to death 10 to 30 years after symptoms begin. There are no FDA-approved treatments for the condition.
UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose, based on a widely used clinical scale.
It also slowed decline of functional abilities in patients by 60%, a key secondary goal of the trial.
"These groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease, where an urgent need persists," said Sarah Tabrizi, director of the University College London's Huntington’s Disease Center.
AMT-130 was generally well-tolerated, with no new serious side effects reported since late 2022, the company said.
UniQure plans to submit a marketing application to the U.S. Food and Drug Administration in early 2026, with hopes of launching the therapy later that year if approved.
8
Posted on 9/28/25 at 10:14 pm to hawgfaninc
Choreans
Posted on 9/28/25 at 10:15 pm to hawgfaninc
Something something big pharma something something would never allow a cure
Posted on 9/28/25 at 10:21 pm to hawgfaninc
Medical technology is the only technology that has actually made quality of life better for the human race.
Posted on 9/28/25 at 10:22 pm to hawgfaninc
I think the human species has moved beyond evolution. We’ll continue to learn but survival of the fittest doesn’t really seem to apply anymore.
Posted on 9/28/25 at 10:26 pm to hawgfaninc
This disease killed almost all of my dad’s siblings. I hope to see a future where we don’t have to worry about it.
Posted on 9/28/25 at 10:29 pm to hawgfaninc
Posted on 9/28/25 at 10:30 pm to rmnldr
quote:
This disease killed almost all of my dad’s siblings.
Damn…
Posted on 9/28/25 at 10:37 pm to hawgfaninc
But did they try hydroxychloroquine
Posted on 9/28/25 at 10:40 pm to rmnldr
quote:are you from Oklahoma/related to the Guthries?
This disease killed almost all of my dad’s siblings.
Posted on 9/29/25 at 6:51 am to BurningHeart
quote:
Medical technology is the only technology that has actually made quality of life better for the human race.
Without the advances of non-– medical technology, we would be hunter–gatherers running around, barefoot with spears. You can't possibly think that wouldn't be worse than what we have now.
On the other hand, this beautiful poem, supports your point of view.
quote:
The world is too much with us; late and soon, Getting and spending, we lay waste our powers;— Little we see in Nature that is ours; We have given our hearts away, a sordid boon! This Sea that bares her bosom to the moon; The winds that will be howling at all hours, And are up-gathered now like sleeping flowers; For this, for everything, we are out of tune; It moves us not. Great God! I’d rather be A Pagan suckled in a creed outworn; So might I, standing on this pleasant lea, Have glimpses that would make me less forlorn; Have sight of Proteus rising from the sea; Or hear old Triton blow his wreathèd horn.
This post was edited on 9/29/25 at 6:53 am
Posted on 9/29/25 at 7:03 am to BPTiger
quote:
I think the human species has moved beyond evolution. We’ll continue to learn but survival of the fittest doesn’t really seem to apply anymore.
The godless societies - China, America, Western Europe - are no longer reproducing enough to maintain their populations. Evolution?
Posted on 9/29/25 at 7:35 am to hawgfaninc
My wife’s aunt died from Huntington’s disease. Just a terrible disease.
Posted on 9/29/25 at 7:43 am to BurningHeart
quote:
Medical technology is the only technology that has actually made quality of life better for the human race.
Clean water and sanitation would be first and second.
Posted on 9/29/25 at 8:37 am to Aubie Spr96
My wife died from this horrible disease 10 years ago.
My two kids have a 50/50 chance of getting it also. In the end she didnt even know her kids.
I hate this F'ing disease with a passion. Whatever is needed to end it - END IT.
My two kids have a 50/50 chance of getting it also. In the end she didnt even know her kids.
I hate this F'ing disease with a passion. Whatever is needed to end it - END IT.
Posted on 9/29/25 at 8:43 am to BurningHeart
quote:
Medical technology is the only technology that has actually made quality of life better for the human race.
Wut? I’m not sure if this is a joke.
Posted on 9/29/25 at 9:30 am to BurningHeart
quote:
Medical technology is the only technology that has actually made quality of life better for the human race.
Never heard of the wheel have you...
Posted on 9/29/25 at 12:33 pm to klrstix
quote:
Never heard of the wheel have you...
MODERN technology would be a better way to convey the point.
Not referring to the technology of a stone age dude inventing a hammer
Posted on 9/29/25 at 12:37 pm to BurningHeart
quote:
MODERN technology would be a better way to convey the point.
It's all modern until it's not.
Posted on 9/29/25 at 12:38 pm to Aubie Spr96
quote:
Clean water and sanitation would be first and second.
Irrigation
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