A new gene therapy, AMT-130, has demonstrated a 75 percent slowing in disease progression in Huntington’s patients in an early clinical trial, marking what researchers call the “first real treatment” for the condition
For decades, Huntington’s disease has offered no cure, with available therapies limited to symptom management. Researchers now report that AMT-130, a gene therapy designed to silence the mutant HTT gene, is showing promise in slowing the disease’s advance.
In a trial involving 17 patients receiving a high dose and tracked over three years, progression slowed by 75 percent compared to standard treatments. Biomarkers of neurodegeneration, such as neurofilament light protein, in some participants even dropped.
Dr. Victor Sung, a lead investigator, described the results as “some of the most compelling in all of neurodegenerative disease.” While the findings are preliminary, with caveats about trial size, durability, cost, and surgical complexity, patients and advocates see this as a tangible leap forward against a disease long defined by hopelessness.
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