Huntington's Disease Defeated: 75% Progress## Translation NotesI've provided a direct translation of the Russian headline into English, maintaining the original format and meaning.

A breakthrough event has occurred in the medical world: for the first time, gene therapy has demonstrated the ability to significantly slow down the progression of Huntington's disease – one of the cruelest neurodegenerative pathologies. According to clinical trial data published in June 2025 by uniQure, the AMT-130 treatment, administered through brain surgery, reduces the rate of disease progression by 75%. This means that what typically takes a year now stretches to four years, giving patients additional decades of full life. The emotional team of researchers couldn't hold back tears when announcing the results at a press conference at the University of London. Professor Sarah Tabrizi, a leading Huntington's specialist, noted in an interview with BBC News: "We are giving hope to families who have been waiting for a breakthrough for decades."

Huntington's disease, a hereditary pathology affecting the brain and combining symptoms of dementia, Parkinson's disease, and amyotrophic lateral sclerosis, is diagnosed in 30 thousand new patients worldwide annually. It is caused by a mutation in the HTT gene, leading to the accumulation of toxic huntingtin protein that destroys neurons. Until 2025, treatment was limited to symptomatic therapy: antidepressants, antipsychotics, and physiotherapy. AMT-130 is a one-time gene therapy using viral vectors to reduce the production of the mutated protein. The procedure lasts 12-18 hours: the drug is administered to the brain through stereotactic surgery, minimizing the risk of infections. In phase 1b/2a trials involving 39 patients, the therapy showed safety and efficacy: in early stages, progress slowed by 75%, in later stages – by 50%, according to the UHDRS scale.

The FDA granted AMT-130 "breakthrough therapy" status in April 2025, accelerating its path to market. UniQure plans to submit an approval application in 2026, while Roche has begun phase 1 trials of its therapy RG6551. The European Medicines Agency (EMA) also noted the potential but emphasized the need for long-term data. In the UK, where Tabrizi lives, the NHS is preparing pilot programs for 500 patients. Globally, according to the Huntington's Disease Society of America, the therapy could affect 200 thousand people in the US and Europe.

Experts emphasize: AMT-130's success is the result of 30 years of research, from CRISPR editing to viral vectors. However, challenges remain: the cost of the procedure is $1-2 million, accessibility in developing countries, and ethical questions about mutations. Tabrizi warns: "This is not a cure, but a revolution – patients will be able to live, not just survive." Families fighting the disease are already calling the treatment a "miracle"