Good news! However, it requires a long, complex brain surgery!
"A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington’s disease. ...
Huntington’s disease is a fatal brain disorder that runs in families, caused by a faulty gene that produces a protein called huntingtin. The disease typically begins to cause symptoms in people between 30 and 50 years old. ...
In this study, neurosurgeons delivered the treatment called AMT-130 directly into the brain using precise surgery guided by MRI scans. They targeted the striatum – the brain region most damaged by Huntington’s disease.
The treatment requires a single injection delivered during a complex 12- to 20-hour brain operation, which means it will probably be expensive.
AMT-130 uses a modified virus to carry genetic material that can reduce the amount of harmful huntingtin protein in brain cells. ...
The results showed that patients who received the high dose of AMT-130 (12 people) experienced significant benefits. The treatment appeared to slow disease progression by 75% over 36 months, compared with a carefully matched group of patients who didn’t receive the therapy and were from another study that investigates how the disease develops over time. ..."
"
- Pivotal study met primary endpoint; high-dose AMT-130 demonstrated statistically significant 75% disease slowing at 36 months as measured by cUHDRS compared to a propensity score-matched external control
- High-dose AMT-130 also demonstrated statistically significant slowing of disease progression as measured by TFC, a key secondary endpoint, and favorable trends across additional clinical measures
- Mean cerebrospinal fluid NfL levels were below baseline at 36 months
- AMT-130 continued to be generally well-tolerated with a manageable safety profile
- uniQure plans to submit a BLA in the first quarter of 2026, with anticipated U.S. launch later that year, pending approval
..."
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