UniQure gene therapy slowed Huntington’s disease for four years — STAT News

UniQure’s experimental gene therapy AMT-130 continued to slow the progression of Huntington’s disease four years after it was surgically implanted in patients’ brains. STAT News reports on the results announced by the company in its article.

Results of the four-year analysis

According to UniQure’s new analysis, a high dose of the one-time gene therapy AMT-130 slowed disease progression by 44% compared with matched participants in an external control group.

This group was formed based on a large natural history study of the disease. The difference between the indicators was not statistically significant.

More current news is available on the UA.News Telegram channel Telegram.

Comparison with the previous assessment

The scale of the therapy’s benefit decreased compared with a similar analysis conducted a year earlier. As STAT News notes, this may raise questions about the duration of the treatment’s effect.

The new data were released as the Food and Drug Administration begins reviewing UniQure’s application for authorization to sell AMT-130.