clinical-trial
-
The First Domino Falls: AMT-130 Gene Therapy Slows Huntington’s in Landmark Trial
In an update from uniQure, they report that their experimental gene therapy, AMT-130, has the potential to slow Huntington’s disease progression in key clinical study.
-
SKY-0515 Lowers Huntingtin In People With Huntington’s Disease In Trial Update
SKY-0515, an oral drug, safely lowers huntingtin in people with HD and may also reduce PMS1. This could offer a possible two-pronged approach to treat HD. SKY-0515 is now being tested in a larger Phase 2/3 trial.
-
PROOF in the Publishing: Slicing the Data on Pridopidine
Pridopidine has been tested as a potential HD treatment over decades. The large PROOF-HD trial did not meet its main goals, but its results are now published. Publication makes the full data available, helping the HD community learn from the study.
By Dr Leora Fox -
Pridopidine Hits a Roadblock: EMA Says No to Approval for Huntington’s Disease Treatment
The EMA has rejected pridopidine for HD treatment in Europe. While disappointing, this outcome aligns with the trial data. Prilenia plans further studies, and despite setbacks, HD research in 2025 continues to bring momentum and hope.
-
Cracking the Case: How a Smartphone “Detective” is Helping Track Huntington’s Disease Progression
Smartphones can help scientists calculate a new clinical measure of HD, called the HD Digital Motor Score. Easier data collection could means less clinic trips, fewer people needed for studies, and a better understanding of HD.Â
-
Spark ignited: first HD patient dosed in new Roche gene therapy trial
Spark and Roche issued a joint community letter to share that the first patient has been dosed in a new HD gene therapy trial.
By Dr Leora Fox -
Full Steam Ahead: uniQure’s On Track With Hope for Accelerated Approval of Huntington’s Disease Drug
uniQure has aligned with the US FDA on next steps to achieve accelerated approval of AMT-130. If data remain positive, they will be on track to market the first HD gene therapy.
-
Roche provides an update on tominersen: What’s next for this huntingtin-lowering drug?
Roche gave an update this week about GENERATION HD2, testing the HTT-lowering drug tominersen in people with HD. The trial is continuing, but only the higher dose will move forward. What does this update mean for the HD community and this trial?
-
Huntington’s Disease Clinical Expert Prof. Ed Wild Shares Hopeful View For 2025
The HDBuzz team caught up with editor emeritus and Huntington's disease specialist, Prof Ed Wild to hear his thoughts on what 2025 has in store for the Huntington's disease community
-
2024: Year in Review
As we begin 2025, we look back on all of the Huntington’s disease research news and progress the field has made in the last 12 months.