“The range of stuff that’s already in humans—people often ask about CRISPR and [such]—there’s tons of science going on but what’s impressive to me is that there’s a lot of stuff in actual people, really getting pushed forward in the now.”
A decade ago, a diagnosis of Huntington disease was essentially a sign that it was time to give up. With little to do in terms of treatments and a less-than-full pipeline for development, the future looked bleak, to say the least.

In the past few years, though, much of that has changed. Claudia Testa, MD, PhD, has watched this explosion of research herself. With multiple agents, including viral vector gene therapies, being developed, and now, making their way into human trials, the light at the end of the tunnel has gotten significantly brighter.

For Testa, it’s less about the one big step that’s being made and more about the slew of small steps that are truly altering the landscape of treatment for Huntington disease. Many are disease-modifying, having an effect on the progression of the condition itself.

The professor of neurology at Virginia Commonwealth University sat with NeurologyLive at the American Neurological Association’s 143rd Annual Meeting to discuss all the progress that’s been made in the space, and her thoughts on the advances.