A groundbreaking treatment has offered fresh hope to people living with Huntington’s disease, a devastating genetic condition that causes gradual loss of brain function and has long been considered untreatable.
The experimental therapy, called AMT-130, uses a viral vector to deliver microRNA directly into the brain, silencing the faulty gene that produces toxic proteins. In a clinical trial involving 17 participants, patients who received the treatment showed a 75 percent slower disease progression over three years compared to those who did not.
The procedure, delivered through brain surgery, marks the first time a therapy has shown the potential to alter the course of Huntington’s disease rather than just manage symptoms. Scientists say the findings could pave the way for a new era in treating neurodegenerative illnesses, though they stress the results are still early and require larger trials for confirmation.
Experts caution that while the therapy is promising, challenges remain around safety, accessibility, and affordability. Even so, the breakthrough offers renewed optimism for families who have waited generations for progress in combating the disease.
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