Casgevy is the first CRISPR-based drug to make its way through the approval process, all but curing Sickle Cell Disease and it’s paving the way for similar drugs targeting other diseases. Is Huntington’s disease next?
Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?
Many diseases are caused by repetitive DNA sequences. Understanding the regulation of those repetitive sequences may hold the key for unlocking therapeutics for Huntington’s disease. A team from Toronto has just advanced our understanding.
The HD pipeline is rich and varied. Let's talk about some out-of-the-box approaches for developing drugs for HD that don't involve huntingtin lowering.
Researchers used plants to study how to stop the Huntington’s disease protein from forming toxic clumps
The gene MSH3 is getting a lot of attention in HD research lately. New findings suggest MSH3 lowering could halt CAG repeat expansions, offering a new therapeutic avenue.
A new study from researchers at Thomas Jefferson University delves into the details of how genetic modifiers of Huntington’s disease work.
The FDA has approved valbenazine, also known as INGREZZA, as a treatment for the movement symptoms of Huntington’s disease
Replacing cells with HD in the brain could be an effective treatment strategy. Recent work shows that glia injected into mouse brains take over and oust the older cells, but for a surprising reason - because of age, not HD!
Both PTC Therapeutics and uniQure have shared updates from their respective clinical trials, testing different huntingtin-lowering approaches. We explore the data presented from both of these studies and what this means for HD family members.
Check out research updates from Day 3 of the 2022 HD Therapeutics Conference #HDTC2023