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AMT-130 at Four Years: Hopeful Signs, Complex Controls
Latest News
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September 2026: This month in Huntington’s disease research
September brought progress across the HD research landscape. From brain waves and visual thinking to DNA repeats, nutrition, inclusive care, new technologies, and encouraging SKY-0515 data, we round up a busy month in Huntington’s disease research.
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The Great Care Conundrum: What “Good” Care Looks Like in Huntington’s Disease
⏱️8 min read | What does “good care” look like in Huntington’s Disease? A new Dutch study asked familial and professional caregivers, and uncovered how differently it’s experienced in practice.
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A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
⏱️ 6 min read | While the US regulatory path for AMT-130 remains complicated, uniQure announced plans to seek approval for its HD gene therapy in the UK. Here’s what that means, and what might be to come.
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Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
⏱️10 min read | PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to 52% slowing of disease progression. Here’s what the data show, what’s still missing, and the roll out of the Phase 3 trial.
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April 2026: This Month in Huntington’s Disease Research
⏱️ 8 min read | April 2026 showed us a toxic HTT fragment is center stage in mouse research, CRISPR is showing promise in mouse studies, AI and wearables move closer to the clinic, and 2 studies shine a light on the weight carried by HD caregivers.
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The Number on the Scale: What the TFC Score Measures, and Why It Matters Right Now
⏱️ 10 min read | The Total Functional Capacity score has been used in Huntington’s disease research for decades. Here’s what it measures, what it misses, & why it’s at the center of an anticipated clinical trial.
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Dancing With a Stranger: Understanding Apathy in Huntington’s Disease Through Caregivers
⏱️5 min read | Apathy in HD isn’t just “not caring.” It can affect relationships, routines, and emotional connection. A small study of caregivers reveals the heavy toll of apathy and the value of safe spaces to share experiences.
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Fund the Buzz: The $8,000 Reason HDBuzz Can Tell You the Truth
⏱️ 6 min read | HDBuzz is launching Fund the Buzz, our spring fundraising campaign! We’re independent, pharma funding-free, and run by scientists who know HD. Help us keep the lights on (and stay lawsuit-free).
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Cutting to the chase with CRISPR
⏱️ 6 min read | A new study uses “self-switching” genetic scissors to target the root cause of Huntington’s disease in mice – even after symptoms begin.
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Artificial Intelligence enters the HD space as a diagnostic tool
⏱️9 min read | From predicting symptom onset to tracking movement changes via smartwatch, artificial intelligence tools are being used in research. Here’s where we are, and why Huntington’s disease is a strong candidate for these approaches.
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Blocking a toxic fragment: new mouse study highlights the importance of HTT1a in Huntington’s disease
⏱️ 9 min read |New research in a HD mouse model points to a key culprit: a small fragment called HTT1a. Lowering HTT1a levels successfully delayed disease signs in mice that model HD, perhaps shaping the next generation of HTT-lowering therapies.
HDBuzz Trial Tracker
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Sites for Phase 3 trial for votoplam, INVEST-HD, now open. Updates on recruitment and first dosing imminent.
REGEN4HD, Phase 1 stem cell implantation study commences
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
