Originally published on 9/25/25

For decades, so many families have watched loved ones suffer from Huntington’s disease, a neurodegenerative condition that progressively worsens over time. But now, there is finally a light at the end of the tunnel. A team of scientists recently shared that they have discovered a treatment for Huntington’s that may slow the disease by a groundbreaking 75 percent. We take you through exactly how it works, and when the cure might become available to the general public below. 

What is Huntington’s disease? 

Huntington’s disease is a genetic condition that breaks down brain cells, causing them to lose function and diet off. It’s caused by a DNA glitch that affects the HTT gene, which produces a protein called huntingtin that’s crucial for proper nerve cell function. In people with Huntington’s disease, these proteins destroy healthy nerve cells instead of supporting them. 

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New treatment may reduce Huntington’s progression by 75% 

While there’s no way to prevent Huntington’s disease, a team of scientists at University College London have been working toward a potential cure. It comes in the form of a type of gene therapy called AMT-130—a treatment that works by delivering corrective genetic material directly to the brain—and is given to patients during a 12- to 18-hour brain surgery. The results from the one-time treatment are meant to last a lifetime.

In preliminary trials on a small group of 29 patients, the treatment reduced disease progression by 75 percent over three years in those given a high dose compared to those on a standard care regimen. “This result changes everything,” said UCL professor Ed Wild in a statement. He even noted that a patient who was forced to retire due to the disease has since been able to return to work. 

“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 professor Sarah Tabrizi, lead scientific advisor on the trial, in the press release. “For patients, AMT-130 has the potential to preserve daily function, keep them in work longer and meaningfully slow disease progression.” 

When will Huntington’s disease treatment be available? 

The trial’s sponsor, uniQure, announced they plan to seek FDA approval in the first quarter of 2026, meaning the drug won’t be publicly available until next year at the earliest.

“We are incredibly excited about these topline results and what they may represent for individuals and families affected by Huntington’s disease,” said Walid Abi-Saab, MD, chief medical officer of uniQure, in a statement. “These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease.” 

Huntington’s disease symptoms to watch for

Anyone can develop Hungtinton’s, though most people begin to be affected in their 30s or 40s. That said, there’s often a hereditary component. If at least one of your parents has Huntington’s disease, there’s a 50 percent chance you’ll develop the disease as well. According to the Mayo Clinic, symptoms of Hungtinton’s disease may include:. 

• Difficulty walking
• Involuntary movements
• Trouble speaking, such as slurred speech
• Memory loss or trouble focusing
• Irritability or depression

Oftentimes these symptoms end up mimicking dementia, making the diagnosis that much harder and complex for patients and their family members. Currently, the average life expectancy for Huntington’s is 15 to 20 years after the initial diagnosis. For people who develop symptoms before the age of 20, though, that number shrinks to 10 to 15 years. 

Most physicians recommend a variety of medicines and therapy to help ease symptoms, though there’s been no way to stop, slow or even reverse the progression of the disease—until now. With a potential treatment on the horizon, Huntington’s patients may one day be able to dramatically improve their health and longevity.

Link to original: https://www.womansworld.com/wellness/new-huntingtons-disease-treatment-reduces-progression