SKY-0515 is a daily oral drug designed to lower both huntingtin, the protein that drives HD, and PMS1, a DNA repair protein linked to somatic CAG repeat expansion. Researchers hope this dual-target ...
In a recent study, scientists created a chemical that breaks down expanded huntingtin HTT clumps in HD cells and mouse models. This chemical finds HTT clumps and targets them to be broken down or ...
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