British researchers today announced that they have managed, for the first time, to slow down Huntington’s dance in patients, thanks to an innovative genetic therapy developed in collaboration with the US -Hellenic Biotechnology Society Uniqure.
Huntington’s disease or dance is a rare hereditary disease due to gene mutation. It is a neurodegenerative disorder that leads patients to complete loss of autonomy and death.
In the clinical trial performed by researchers at University College London (UCL), Huntington’s Disease Center and Uniqure, the development of the disease has slowed by 75%, over 36 months, in the 29 patients who received treatment compared to people who did not receive it.
About 8,000 people in Britain and 6,000 in France suffer from handington, according to health authorities.
Genetic therapy consists of the infusion, the cells of a patient, new “gene instructions” in the brain’s streak body, a structure particularly susceptible to Huntington’s dance. A single dose of this treatment, called AMT-130, is sufficient, the researchers who participated in Phases 1 and 2 of the clinical test are sufficient.
“For patients (treatment) enables them to maintain their functionality, stay at work for a longer period, and significantly slow down the progress of the disease,” said Professor Sarah Tabrizi, the scientific adviser to the clinical trial, describing the results.
“This is a great and promising progress,” said Zosia Mzibrodska, a professor of genetics at the University of Aberdeen. He noted, however, that it will still need many tests to determine whether the new treatment causes side effects or whether it is effective in the long run.
Uniqure intends to submit a request to the US Food and Drug Administration (FDA) in early 2026 to obtain permission to march its drug on the market with rapid procedures. Similar demands will follow in British and European authorities.
Source :Skai
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