clinical-trial
-
Additional Clarity: What We Know 4 Weeks After the uniQure News
We’re 4 weeks out from uniQure’s positive update about AMT-130 that took the world by storm. Does the data match the media hype? Where do uncertainties remain? And what are some of the key questions from the Huntington’s disease community?
-
Huntington’s Disease Clinical Research Congress 2025 – Day 3
Catch up on all the latest HD news from Day 3 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
-
Huntington’s Disease Clinical Research Congress 2025 – Day 2
Catch up on all the latest HD news from Day 2 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
-
Huntington’s Disease Clinical Research Congress 2025 – Day 1
Catch up on all the latest HD news from Day 1 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
-
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