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Pridopidine Hits a Roadblock: EMA Says No to Approval for Huntington’s Disease Treatment
The EMA has rejected pridopidine for HD treatment in Europe. While disappointing, this outcome aligns with the trial data. Prilenia plans further studies, and despite setbacks, HD research in 2025 continues to bring momentum and hope.
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When the Brain’s Orchestra Falls Out of Tune: A New Map of Huntington’s Disease Progression
New research maps Huntington’s disease progression like a symphony unraveling. The brain first overcommunicates, then loses sync, and finally falls silent, each stage driven by different biological mechanisms.
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Stopping the Genetic Snowball: How a simple genetic interruption slows Huntington’s disease
The Huntington’s disease mutation worsens over time like a dangerous snowball. By harnessing the power of the gene editing tool CRISPR, scientists may have found a way to interrupt the HD mutation and stall disease onset.
By AJ Keefe -
Unsung Heroes: Could Glial Cells Treat Huntington’s Disease?
Transplanting healthy human glial cells into HD mouse brains improved movement, memory, and survival. Even more strikingly, the glia coaxed diseased neurons to behave more like healthy ones, offering a potential new path for treating HD.
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Energy off balance: How Huntington’s disease influences the cell’s powerhouseÂ
Scientists used 3D mini-brains grown from stem cells to study Huntington’s disease. They found early developmental changes linked to mitochondrial stress, suggesting that energy imbalance may play a role in HD, even before neurons fully form.
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Peeking at huntingtin and learning from a PET study
Scientists tested a new tool to measure harmful HTT protein in HD. It wasn’t perfect, but offered key insights to guide better tools already in the works. A big step toward tracking HD non-invasively, from inside the brain!
By Dr Leora Fox -
June 2025: This Month in Huntington’s Disease Research
June was filled with exciting research! Ranging from clinical trial updates to irritability, we’ve rounded up the most exciting Huntington’s disease research from this month.
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Trouble on the Block: When the Neighborhood Loses Its Best Firefighter
In HD, a key brain protector, PKD1, is missing. Without this “molecular firefighter,” neurons are vulnerable. Knowing why it disappears may help us uncover how the brain’s defense break down, and how to reinforce it.
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Spark ignited: first HD patient dosed in new Roche gene therapy trial
Spark and Roche issued a joint community letter to share that the first patient has been dosed in a new HD gene therapy trial.
By Dr Leora Fox -
When a Short Fuse Becomes a Storm: Understanding Irritability in Huntington’s Disease
For people living with HD, irritability can erupt like a storm, sudden and overwhelming. In a new study, researchers spoke directly to people with HD to understand what irritability truly feels like.