Moving into the Fast Lane: uniQure and the FDA Are on the Same Track for Accelerated Approval
On December 10, 2024 we received a regulatory update from uniQure about their drug, AMT-130, that they’re advancing through the clinic for Huntington’s disease (HD). On the call, uniQure announced alignment with the FDA on key elements to get accelerated regulatory approval. Of this program, Matt Kapusta, the CEO of uniQure, said that they are […]
Interim update from Vico Therapeutics on their CAG-targeting drug, VO659
Vico Therapeutics recently presented at several conferences to share an interim update on their Phase 1/2a clinical trial testing their drug called VO659, which targets the repeating C-A-Gs in people with Huntington’s disease (HD). These data suggest that VO659 may be able to reduce levels of the toxic HD protein in the small group of […]
Going boldly: First person treated in Phase 1 clinical trial by Alnylam Pharmaceuticals
On December 3, 2024 we received word that the very first person received a new drug, called ALN-HTT02, as part of a Phase 1 trial aiming to treat Huntington’s disease (HD). Going boldly as the first human ever to receive this drug, they’re charting a course that we all hope will one day lead to […]
Giving Thanks to the Huntington’s Disease Family Community for Advancing Research
In the spirit of gratitude, this Giving Tuesday, the HDBuzz editorial team would like to reflect on the unique and transformative role the Huntington’s disease (HD) family community plays in advancing scientific research. From their resilience in the face of this extremely challenging condition, to their extraordinary contributions to scientific studies, the HD family community […]
Self-determination on the HD journey: the role of Advanced Care Planning
Here, we cover a report on Advanced Care Planning (ACP) prepared by clinical researchers at the University College London, reminding us there are things that can be done right now to improve the lives of people living with HD. Until we have drugs that slow the progression of HD, peace of mind can be some […]
2024 HDBuzz Prize: Beyond nerve cells – who are the other players in the HD brain?
A new study led by researchers from Columbia University used postmortem brain samples to show that a special type of brain cell, called astrocytes, may play a role in how certain nerve cells are lost in Huntington’s disease (HD). This could have important implications for how we understand disease progression as well as for the […]
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