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AAN 2026 | Unmet needs in Huntington’s disease and advances toward disease-modifying therapy

Victor Sung, MD, University of Alabama, Birmingham, AL, discusses the current treatment landscape for Huntington’s disease, highlighting the focus on symptomatic treatment for motor, cognitive, and behavioral symptoms. He notes disease-modifying therapies currently being investigated, with various mechanisms being explored. This interview took place at the 78th American Academy of Neurology (AAN) Annual Meeting in Chicago, IL.

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Transcript

So the current treatment landscape for Huntington’s disease is really focused on symptomatic treatment across the HD symptom triad, which is going to be motor, cognitive, and behavioral or psychiatric symptoms. There are multiple approved motor treatments for the chorea specifically. There’s a lot of off-label use of medications for the behavioral symptoms, but there are really no treatments that are either approved or effective for the cognitive symptoms...

So the current treatment landscape for Huntington’s disease is really focused on symptomatic treatment across the HD symptom triad, which is going to be motor, cognitive, and behavioral or psychiatric symptoms. There are multiple approved motor treatments for the chorea specifically. There’s a lot of off-label use of medications for the behavioral symptoms, but there are really no treatments that are either approved or effective for the cognitive symptoms. So from a symptomatic standpoint, that’s an area of unmet need is the cognitive symptoms of HD. And then there are also certain behavioral symptoms where we don’t have a lot of treatments, particularly apathy in Huntington’s disease. And then obviously disease-modifying therapy. So we are in a world where disease-modifying therapies do not yet exist, although there are many in active investigation. There are a few other attempts at disease modification that are being presented in different forms at this meeting. And I think that’s the exciting wave of the future in Huntington’s disease is the wave of Huntingtin-lowering therapies and disease-modifying therapies overall in the disease, which is a much-needed space. We don’t have anything currently. So there are multiple mechanisms from oral splicing modifiers to intrathecal antisense oligonucleotides to intracerebrally injected gene therapies and things across that spectrum. But there’s multiple of those that are being presented at this meeting. I think it’s all exciting. I think it’s a wave, as I said. There will be multiple of these crossing the finish line in the coming years, and it’s an exciting time.

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Disclosures

Dr. Sung has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Teva Neuroscience. Dr. Sung has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech. Dr. Sung has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neurocrine. Dr. Sung has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UniQure.