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Huntington’s Disease Clinical Research Congress 2025 – Day 1
Latest News
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Controlling DNA Scanning Machines to Slow Expansion of CAG Repeats
A new study identifies small molecules that target a DNA repair machine, opening a potential path to delay Huntington’s disease onset.
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2025 HDBuzz Prize: When Good Huntingtin Goes Missing: A Step Toward Designing Safe HD Treatments
We’re proud to announce Gravity Guignard as a 2025 HDBuzz Prize winner! A new study points to one of the reasons why past HD trials might have failed. The good news? This shows the path to safer drugs, getting us closer to effective therapies.
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2025 HDBuzz Prize: On the Frontlines: Trusting the Hall Monitors of the Cell
We’re proud to announce Chloe Langridge as a 2025 HDBuzz Prize winner! A protein called SGTA shows promise as a target in HD therapeutics. Researchers find that increasing levels of SGTA in the cell helps reduce signs of HD.
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September 2025: This Month in Huntington’s Disease Research
September was a landmark month in Huntington’s disease research: a gene therapy shows signs of slowing progression, plus new biomarkers, mechanistic insights and mental health advances. The field has come far and exciting directions lie ahead.
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The First Domino Falls: AMT-130 Gene Therapy Slows Huntington’s in Landmark Trial
In an update from uniQure, they report that their experimental gene therapy, AMT-130, has the potential to slow Huntington’s disease progression in key clinical study.
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2025 HDBuzz Prize: When the Repair Crew Breaks Down: How Expanded Huntingtin Disrupts DNA Repair
We’re proud to announce Mustafa Mehkary as a 2025 HDBuzz Prize winner! New findings reveal that the huntingtin protein is a key player in the cell’s DNA repair crew. In HD, the crew falls apart leaving DNA damage and alarm bells ringing in its wake.
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SKY-0515 Lowers Huntingtin In People With Huntington’s Disease In Trial Update
SKY-0515, an oral drug, safely lowers huntingtin in people with HD and may also reduce PMS1. This could offer a possible two-pronged approach to treat HD. SKY-0515 is now being tested in a larger Phase 2/3 trial.
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2025 HDBuzz Prize: MR-“why” am I feeling this way? How MRI is helping us understand why people with HD may sometimes be unaware of their symptoms
We’re proud to announce Jenna Hanrahan as a 2025 HDBuzz Prize winner! People with HD may be unaware of their symptoms, not out of denial but due to real changes in the brain. MRI scans give new clues about what is going on.
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PROOF in the Publishing: Slicing the Data on Pridopidine
Pridopidine has been tested as a potential HD treatment over decades. The large PROOF-HD trial did not meet its main goals, but its results are now published. Publication makes the full data available, helping the HD community learn from the study.
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2025 HDBuzz Prize: ACTing on HD: Exploring Acceptance and Commitment Therapy to Improve Mental Health in People with Huntington’s Disease and Their Caregiver
We’re proud to announce Nicolo Zarotti as a 2025 HDBuzz Prize winner! A new case study shows Acceptance & Commitment Therapy (ACT) can improve mood and boost quality of life for people with HD, and help caregivers too.