The U.S. Food and Drug Administration (FDA) has agreed to review uniQure’s application seeking accelerated approval of AMT-130, a gene therapy candidate for Huntington’s disease that is now called ifezuntirgene inilparvovec. “The FDA’s acceptance of our [application] for review marks an important milestone for the Huntington’s disease community,”…
News
The experimental therapy brezosiran can reduce levels of the mutant huntingtin protein in people with Huntington’s disease, according to interim data from a small clinical trial. The results were shared in oral and poster presentations at the European Huntington’s Disease Network Clinical Research Congress 2026, held Oct.
Before the end of the year, Arexera Therapeutics is planning to launch a Phase 1 clinical trial testing its experimental therapy ATL-101 in people with Huntington’s disease. According to a company press release, the upcoming trial is expected to test one-time treatment with ATL-101 at various doses,…
Differences in brain connections for people who carry a mutation known to cause Huntington’s disease are detectable decades before the onset of motor symptoms, a study showed. The findings suggested that the ways these connections evolve over time is altered in Huntington’s mutation carriers. While the study was limited…
Up to four years after a single dose of the experimental gene therapy ifezuntirgene inilparvovec (formerly AMT-130), people with Huntington’s disease who received the high dose showed slower disease progression than would be expected without treatment. That’s according to data from two long-term Phase 1/2 clinical trials, one…
Time-restricted eating (TRE), a form of intermittent fasting that limits daily calorie intake to a six-to-eight-hour window, may help slow the progression of Huntington’s disease, according to results from a three-month pilot study. Study participants were able to maintain their body weight while showing reductions in disease severity measures…
In final 15-month study data, people with early-stage Huntington’s disease treated with SKY-0515, Skyhawk Therapeutics’ investigational oral therapy, showed improvements in function, movement, and cognition. That’s according to final results from Skyhawk’s Phase 1/2 clinical program, which included the registered Phase 1 trial (ACTRN12624000602527). The study included…
An early and progressive loss of a protein that helps maintain myelin, or the protective sheath around nerve fibers, may contribute to white matter damage in Huntington’s disease, a study suggests. In a mouse model of Huntington’s, levels of the myelin-associated glycoprotein (MAG) began falling before the loss of…
uniQure has submitted an application to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of AMT-130, the Netherlands-based company’s gene therapy candidate for Huntington’s disease. A similar request for marketing authorization in the U.K. has been made to the nation’s Medicines and Healthcare products Regulatory…
A real-world study shows that the approved chorea treatment Austedo (deutetrabenazine) reduces involuntary movements in adults with Huntington’s disease and is generally well tolerated in clinical settings. According to the researchers, most patients experienced a decrease in the severity…
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