Turning down huntingtin: Alnylam’s brezosiran shows promising early result
An experimental treatment for HD developed by Alnylam has shown long-lasting huntingtin lowering after just one dose, with encouraging early safety data. A pivotal trial is now planned for 2027 to figure out if this drug, brezosiran, can slow HD. Read more
Picking MSH3’s Pocket: New Insights into CAG Repeat Expansion
Scientists discovered a pocket on a protein called MSH3 that helps it grab DNA containing CAG repeats. By blocking this interaction, they slowed the growth of CAG repeats in cells, without removing the protein entirely. Read more
What Pigs Revealed that Mice Missed in Huntington’s Disease
A change in one gene can disrupt thousands of others in HD. A study in pigs suggests an explanation: expanded huntingtin interferes with chemical marks on DNA. The same effect wasn’t seen in mice, showing that the animal model matters. Read more
Mending the fabric of your genes
Cells constantly mend tears and rips in the fabric of your DNA, like a cherished pair of jeans. Sometimes this mending can add C-A-Gs by mistake. A group of scientists examined whether other kinds of rips in the genetic fabric may be occurring in HD. Read more
Could rewiring the brain’s “brakes” improve movement in Huntington’s disease?
Scientists discovered that a specific type of brain cell becomes unusually quiet in Huntington’s disease. By restoring the activity of these cells in mice, they were able to rebalance brain circuits and improve movement. Read more
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