SKY-0515, developed by Skyhawk Therapeutics, is an oral drug taken as a once-daily pill that works by targeting the genetic ...
AMT-130 is a gene therapy designed to lower levels of the huntingtin protein in the brain. It uses a harmless virus package, called an adeno-associated virus (AAV), to contain microRNA – genetic ...
8 min read | February 2026 HD research recap: published trial data from branaplam trial; new insights into the genetics of the HD “grey zone”; plus worms, brain circuits, eye tracking, and the hidden ...
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