this post was submitted on 17 Oct 2025
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A new treatment that proves to slow the progression of Huntington’s disease marks a breakthrough for a condition that has long been considered untreatable. Huntington’s is a rare, inherited neurodegenerative disorder that gradually deteriorates a person’s physical and mental abilities, often leading to death within 10 to 30 years of onset. Until now, treatments have only managed symptoms, rather than altering the disease’s course.

The research team, including Sung, tested a new treatment called AMT-130 on people who were in the early stages of HD. The treatment works by halting the part of the body’s gene production responsible for producing the toxic huntingtin protein. By targeting the messenger RNA that carries instructions from DNA to produce this protein, AMT-130 prevents its formation without altering the DNA itself — a key safety advantage over other genetic therapies.

After three years, those who received the higher dose showed a 75 percent slower disease progression compared to those who did not receive the treatment. The treatment helped trial participants maintain more of their movement, thinking and daily functioning abilities without causing significant side effects.

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[–] tynansdtm@lemmy.ml 22 points 2 days ago

My (half-) brother has Huntington's. It killed his father, so he knew it was possible and he's not having any children. He's just turned 30 and just had its presence confirmed, but apparently he'll be symptom-free until about 60. Hopefully we get more good news like this in the next 30 years.

[–] Imgonnatrythis@sh.itjust.works 47 points 3 days ago

This is the kind of good news we need right now. Contact your local representatives and let them know that science works and that basic research funding is a priority for you, your community, and humanity.

[–] DemBoSain@midwest.social 31 points 2 days ago (2 children)

A friend of mine carried the Huntington gene. He didn't get it, but all three of his kids are dead from it.

[–] BorgDrone@feddit.nl 16 points 2 days ago (1 children)

Was he aware of being a carrier of the gene before deciding to reproduce?

[–] AstralPath@lemmy.ca 7 points 2 days ago

Asking the real questions.

[–] _hovi_@lemmy.world 12 points 2 days ago

That's awful...

[–] daggermoon@lemmy.world 8 points 2 days ago

Finally, news that isn't depressing

[–] HubertManne@piefed.social 15 points 2 days ago

Seeing mrna technology to start to stride is one of the few solaces I have in this millenia.

[–] SabinStargem@lemmy.today 8 points 2 days ago

A treatment that will be refused by MAHA, I assume. 😒

[–] EnderLaw@lemmy.world 9 points 2 days ago

I knew a kid with that terrible disease. Wish it would have arrived 20 years ago.

[–] SaveTheTuaHawk@lemmy.ca 1 points 2 days ago (1 children)

This was not a clinical trial, it was a safety study of only 11 treated. No controls.

CEO of UniQure already dumped all his stock options.

[–] verdi@feddit.org 13 points 2 days ago

It was a phase I\II trial. Dafuq u on about?

Phase I\II determine efficacy and safety profiles.

In this case they are proceeding with a new cohort where the administration will be concurrent with immune suppression to counteract inflammation in the pos-op phase.

They will likely apply for a phase III after since this is an orphan drug.