Huntington’s disease was successfully treated for the first time

Doctors say that one of the most devastating diseases – Huntington – has been successfully treated for the first time, says doctors.

The disease passes through families, kills the brain cells unabated and is similar to a mixture of dementia, Parkinson’s disease and motor nerve cells.

The emotional research team has become tears because they described how the data shows that the disease may slow 75 % in patients.

This means that the decline you usually expect in one year will take four years after treatment, giving patients contracts of “good quality life”.

New treatment is a type of genetic treatment given within 12 to 18 hours of sensitive brain surgery.

Huntington’s first symptoms tend to appear in the thirties or forties of life, usually fatal within two decades – opening the possibility that previous treatment can prevent symptoms from emerging ever.

Professor Tabrizi, Director of the University of Huntington University Diseases at the University of London Huntington, described the results as “amazing”.

She said, “We have never expected in the fiercest of our dreams slowing 75 % of clinical progress,” she said.

None of the patients treated, but one medical pension was retired and returned to work. Others still walk walking although they are expected to need a wheelchair.

The treatment is likely to be very expensive. However, this is a moment of real hope in a disease that strikes people in their destroyed families and destroys families.

Huntington runs through the Jack May Davis family. He has a defective gene that causes the disease, as his father did, unique, and Joyce found him.

Jack said he was “really horrific and terrible” monitoring his father’s retreat.

The first symptoms appeared in the late thirties of the last century, including changes in behavior and the way you move. He eventually needed tasting care around the clock throughout the week before his death at the age of 54, in 2016.

Jack is 30 years old, a lawyer writer, who recently participates with Chloe and participated in the research at the University of California, Los Angeles, to turn his diagnosis into positive.

But he always knew that he was able to share the fate of his father, to this day.

Now he says that “incredible hacking” has left it “steeped” and is able to look at the future “seems to be a little brighter, it allows me to believe that my life may be much longer.”

Huntington’s disease occurs due to an error in part of our DNA called Huntingtin.

If one of your parents has Huntington’s disease, there is a 50 % chance that you inherit the variable gene and will eventually develop Huntington.

This mutation turns into a natural protein required in the brain – called a hunter protein – into a killer of neurons.

The goal of treatment is to reduce the levels of this toxic protein permanently, at one dose.

Optimized genetic medicine is used to combine genetic therapy with genetic silence techniques.

It begins with a safe virus changed to contain a specially designed sequence of DNA.

It is immersed in the depth of the brain using the actual time magnetic resonance survey of Microcatheter to two areas of the brain – the nucleus of the meteor and the two. This takes 12 to 18 hours of neurosurgery.

The virus then acts as a microscopic mail – presents a new DNA inside the brain cells, where it becomes active.

This turns neurons into a factory to make the treatment to avoid their death.

Cells produce a small part of the genetic materials (called Microrna) designed to intercept and disable instructions (called Messenger RNA) that are sent from the DNA cells to build a mutant fishing.

This leads to a decrease in hunting levels in the brain.

The results of the experiment – which included 29 patients – were released in a statement issued by the company UNIQURE, but it has not been fully published yet for review by other specialists.

The data showed that after three years of surgery there was a slowdown in 75 % of the disease based on a measure that combines perception, kinetic function and the ability to manage in daily life.

Data also shows that treatment provides brain cells. The levels of nerve factors in the spinal fluid – a clear sign of the brain cells that die – should increase by a third if the disease continues to advance, but it is less than the beginning of the experiment.

“This is the result that we were waiting for,” said Professor Ed Wilde, a UCLH Neur Hospital, UCLH.

“There was every opportunity that we will never see as a result, so we live in a world we know that this is not only possible, but the actual size of the effect is to breathe, it is very difficult to wrap feelings completely.”

He said he was “little tears” considering the effect he could have on families.

Treatment was considered safe, although some patients have been infected with the virus that caused headaches and confusion that is solved or needed for cervicid.

Professor Wilde expects that the treatment “must last for life” because the brain cells are not replaced by the body in the same way that the blood, bones, and skin are constantly renewed.

Nearly 75,000 people suffer from Huntington’s disease in the United Kingdom, the United States and Europe with hundreds of thousands carrying a mutation, which means that they will develop the disease.

UNIQURE says she will apply for a license in the United States in the first quarter of 2026 with the aim of later later to launch the drug. The talks with the authorities in the United Kingdom and Europe will start next year, but the initial focus on the United States.

Dr. Wildi Abi SABB, chief medical official at the University of Uniuor, said that he is “incredibly excited” about what the results mean for families, adding that the treatment is “the ability to convert” Huntington’s disease mainly.

However, the drug will not be available to everyone due to very complex surgery and expected cost.

“It will definitely be charged,” says Professor Wilde.

There is no official drug price. Often genetic remedies are expensive, but their long -term effect means that it can still be affordable. In the United Kingdom, NHS is paid for the treatment of genes of 2.6 million pounds for the standards of hemophilia B.

Professor Tabrizi says this genetic therapy “is the beginning” and will open the gates for treatments that can reach more people.

She praised the “truly brave” volunteers who participated in the trial, saying that she was “overwhelmed with patients and families.”

It already works with a group of young people who know that they have a gene, but they have no symptoms yet – known as the Zerity Huntington – and aims to do the first prevention experience to see if the disease can be delayed significantly or even completely stopping.