An experimental treatment for the first time slowed the devastating progression of Huntington’s disease, gene therapy company uniQure announced Wednesday, a rare hopeful advance against a cruel genetic disease that robs people of control of their bodies and minds in the prime of life.
People with Huntington’s often develop symptoms in their 40s and progress to severe disability and death over about two decades, ravaged by jerky and involuntary movements, behavior disruptions and cognitive decline. About 40,000 people in the United States have symptomatic Huntington’s, which is caused by a mutated gene.
After the gene that causes the disease was discovered in 1993, there was hope that better treatments were around the corner, but many interventions were tried and failed.
“Today is a really important day in the history of how we understand Huntington’s disease and possible approaches moving forward,” said Rachel Harding, a structural biologist at the University of Toronto who was not involved in the research.
This is what a REAL scientific breakthough looks like
— Neil Stone (@DrNeilStone) September 24, 2025
Not that Tylenol stuff pic.twitter.com/iUVUVRdjEb