In a rare and encouraging development against a severe hereditary disease that deprives people of control over their bodies and brains in their prime, gene therapy startup UniQure revealed Wednesday that an experimental treatment had for the first time slowed the fatal progression of Huntington’s disease.

Doctors say they have successfully treated Huntington’s disease, one of the most terrible and debilitating illnesses, for the first time. The illness is familial, persistently destroys brain cells, and resembles a cross between Parkinson’s, motor neurone disease, and dementia.

Cure for Deadly Huntington’s Disease

As they explained how data indicates the disease was halted by 75% in patients, an emotional study team started crying. According to Prof. Sarah Tabrizi, this indicates that patients would have decades of “good quality life” after therapy, as opposed to the decrease that would often occur in a year.

During 12 to 18 hours of delicate brain surgery, a form of gene therapy is administered as the new treatment.

The most horrible hereditary condition is probably Huntington’s disease. Involuntary jerks, trouble eating and speaking, memory loss, difficulty focusing, melancholy, anxiety, mood swings, impatience, and personality changes are some of the symptoms.

The patient eventually passes away. However, on September 24th, a potential remedy was revealed: a kind of genetic material known as a microrna that prevents the production of the protein that causes it.

A Gene Therapy

Given that Huntington’s disease often kills a person within 20 years of the onset of symptoms, which typically start in the 30s or 40s, early treatment may be able to stop symptoms altogether. The findings were deemed “spectacular” by Prof. Tabrizi, director of the University College London Huntington’s Disease Centre.

“We never in our wildest dreams would have expected a 75% slowing of clinical progression,” she stated. Although none of the individuals receiving treatment are being named, one has returned to work after being medically retired. Even though they are supposed to require a wheelchair, other study participants are still walking.

Huntington’s disease is caused by a mistake in our DNA known as the huntingtin gene. If one of your parents has Huntington’s disease, you have a 50% risk of inheriting the mutated gene and later developing the disease.

This mutation converts the huntingtin protein, a normal protein found in the brain, into a neuron-killer. The medication aims to permanently reduce levels of this harmful protein in a single injection. The therapy employs cutting-edge genetic medicine, combining gene therapy and gene silencing techniques.

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