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 […]
Bucharest 2025
EHA CONFERENCE IN BUCHAREST 2025 The 2025 EHA Conference, held in Bucharest, Romania, from September 25th to 28th, brought together the HD community under the inspiring theme “Shaping our future together – we are the change!”. Participants from across Europe and all over the world gathered to share experiences, exchange knowledge, and discuss new developments […]
Recent results for AMT-130: a step towards treatments that could slow Huntington’s disease
Recent results for AMT-130 A step towards treatments that could slow Huntington’s disease In September 24th, the company UniQure announced the results of its clinical trial after 36 months with AMT-130, a gene therapy for Huntington’s disease (HD). For the first time in a study of this kind, the published results suggest that in a […]
Encouraging Phase 1 Results for Skyhawk’s Huntington’s Disease Investigational Treatment
Encouraging Phase 1 Results for Skyhawk’s Huntington’s Disease Investigational Treatment Skyhawk Therapeutics has shared a press release with positive news about SKY-0515, the experimental drug they developed and are studying for Huntington’s disease (HD). This first study was a Phase 1 trial, which mainly checks whether a drug is safe and how it works in […]
The EHA Team Visits Lund
The EHA Team Visits Lund: Learning from Sweden’s Multidisciplinary HD Care During our visit to Sweden for our annual meeting, the EHA Team had the opportunity to go to Lund to meet with Professor Åsa Petersén, a psychiatrist, researcher, head of the HD Center, and Deputy Chief of the EHDN, as well as Camilla Svensson, […]
SIMPLIFYING SCIENCE: WHAT CAUSES HUNTINGTON’S DISEASE?
SIMPLIFYING SCIENCE: WHAT CAUSES HUNTINGTON’S DISEASE? In this new series, we want to take a deep dive into scientific concepts in a simple way, with the goal of eventually bringing research concepts closer to all of you. This first article will be dedicated to understanding the hereditary nature of HD. It is common to have […]