Harness Therapeutics Nominates HRN001 Drug Candidate for HD and Launches clinical Advisory Board
Community Update Harness Therapeutics Nominates HRN001 Drug Candidate for HD and Launches Clinical Advisory Board HRN001 is an experimental treatment being developed by Harness Therapeutics for people with Huntington’s disease. Most experimental treatments for Huntington’s aim to lower the harmful huntingtin protein directly. HRN001 takes a different approach. Instead of targeting huntingtin itself, it is designed to increase […]
Novartis announces the new clinical trial INVEST-HD
Community Update Novartis announces the new clinical trial INVEST-HD Super exciting news for the HD community: Novartis´s announcement of the new the global Phase 3 trial for Votoplam will be called INVEST-HD. The trial will test how well Votoplam works and the safety in a larger group of participants. This is the next step in […]
The Not-So-Basic Basics of Huntington’s Disease
The Not-So-Basic Basics of Huntington’s Disease Huntington’s Disease (HD) is often described as a rare neurodegenerative disorder. But for caregivers and family members, this description turns out to be vague and the “basics” of HD quickly prove far from basic. HD is very complex; its symptoms change over time, and its impact extends far beyond […]
Roche POINT-HD
Great news for the HD community towards the end of 2025 This week Roche announced that recruitment has started for a new trial called POINT-HD. 40 participants will be included in this Phase I trial which means that the main objective is to test if the drug is safe and tolerated. Nevertheless, the trial will […]
New research grant to improve mental health support for people affected by Huntington’s disease
New research grant to improve mental health support for people affected by Huntington’s disease Huntington’s disease can have significant impacts on mental wellbeing for everybody in affected families. While the physical and cognitive impacts of Huntington’s for people who carry the gene expansion are perhaps more well recognised, those who have Huntington’s or support somebody […]
The Impact of Economic Evaluations in HTAs: Why Access to Therapies May Vary Across Countries
The Impact of Economic Evaluations in HTAs: Why Access to Therapies May Vary Across Countries In the first article of this section dedicated to Health Technology Assessments (HTAs), we examined what HTAs are, careful reviews that governments and health authorities conduct before deciding whether a new treatment should be made available and reimbursed in their […]
uniQure Provides Regulatory Update on AMT-130
The regulators in the US require more data from Uniqure to evaluate their AMT-130 therapy We would like to share an important update released today by uniQure regarding AMT-130 after their recent meeting with the FDA, the regulatory agency in the United States. The purpose of the meeting was to discuss plans for submitting a […]
Beyond numbers: The Impact of Iceland’s HD Association
Community Spotlight: Iceland 🇮🇸 Beyond numbers: The Impact of Iceland’s HD Association Iceland is one of the smallest countries in Europe, which represents both a challenge and a catalyst to make Huntington’s disease visible. The HD Association of Iceland was founded at the beginning of 2022 and currently has around 30 members. We had the […]
EHA at the Orphan Drug Conference in Amsterdam
EHA at the Orphan Drug Conference in Amsterdam Together Towards Fair Access for Rare Disease Therapies President Astri Arnesen attended the Orphan Drug Conference in Amsterdam to discuss how the new regulations on EU Joint Clinical Assessments (JCAs) can be a tool to ensure faster and equitable access to new therapies for Rare Diseases (RD). […]
SOM Biotech secures clear registrational path for SOM3355 in HD after FDA End‑of‑Phase 2 Meeting
SOM3355: An «old» drug showing positive effect on psychiatric and motor symptoms in HD patients SOMBiotech is a company based in Europe that tests drugs for new purposes, so-called re-purposing. A medicine that has been used for another condition (mild hypertension) for several decades, now named SOM3355 has been tested in 129 Huntington patients. Recently […]