Austin (KXAN) – an exceptional and genetic disease It may be finally solution. Huntington’s disease affects one in twenty to twenty thousand Americans. This disease is a mixture of dementia and Parkinson’s, which causes a cognitive decrease and uncontrolled movements.
Genetic disorder, children with Huntington have a 50/50 chance of developing the disease as well. Once the symptoms develop, the average life usually lasts
“It is not great for anyone to see their family members, like a completely different person,” said Talia, a 15 -year -old teenager who suffers from a disease in her family. It has not yet been tested, worried about how to change the results in her life.
She said: “I feel that if I get a positive test, I will not be excited to continue studying, or perhaps just because I continue to try to build myself as a person.”
This week, the University of the Doctors of the London College (UCL) has announced hope. A new genetic treatment showed that it can reduce the development of the disease by 75 % over a three -year period.
“Just get a chance to succeed or just change your life, it will be amazing,” said Talia.
What is Huntington’s disease?
Huntington (HD) disease occurs when one gene, HTT, is repeated, part of its sequence over and over again. In most people, this sequence is repeated from 10 to 35 times. In people with HD, this section is repeated 36 to more than 120 times, According to the University of California San Francisco.
The symmetrical copies cause a long strand of protein, which is separated. The smaller part of the protein works as a toxin in the brain cells. The disease is presented in people between the ages of 30 and 70.
Talia’s mother said: “Often, people do not know that they are even in their families. Sometimes it strikes in the 1940s and fifties, and by that time, as you know, families have given birth to their children.”
Possible solution to Linington
The new treatment, which developed periods, uses genetic therapy to stop this symmetry. Treatment, AMT-130, is the first genetic treatment tested in people with HD.
The researchers tested 29 patients over 36 months, giving half a low dose of treatment and the other half gave a high dose. Patients saw a high dose of 75 % reduction.
Talia’s mother said: “I feel a little sweet and bitter, because for those who deepen in the midst of Huntington, we know that this treatment will most likely not help them, they went very far in the disease,” said Talia’s mother. “For those who are not yet symptoms, there is still hope for them, and the fact that it happens during our lives, we hope it will be a truly promising.”
The company is hoping for treatment to accelerate its release. They hope to obtain approval from the US Food and Drug Administration early next year, according to a statement. They will then try to obtain approval in the UK.