Researchers at Lawrence Berkeley National Laboratory have identified a previously overlooked mechanism that may contribute to nerve cell damage in Huntington’s disease (HD), raising the possibility of a new treatment strategy focused on protecting DNA rather than directly targeting the genetic mutation. Although much research has focused on the abnormal protein and expansion of DNA…
Category: Huntington’s disease
Study suggests protein clumps may help protect brain cells in Huntington’s disease
Protein clumps that accumulate in the brains of people with Huntington’s disease and other neurodegenerative disorders may play a protective rather than harmful role, according to new research that challenges a long-standing theory of disease progression. Published in Cell Death & Differentiation, the study found that so-called inclusion bodies, long regarded as toxic deposits that…
Sarah Tabrizi recognised in King’s Birthday Honours for Huntington’s disease research
Professor Sarah Tabrizi has been appointed a Commander of the Order of the British Empire (CBE) in the 2026 King’s Birthday Honours in recognition of her services to people with Huntington’s disease and her pioneering contributions to neurological research. Professor Tabrizi is a Group Leader at the UK Dementia Research Institute (UK DRI) at UCL,…
New guidance aims to improve mental health support for people with Huntington’s disease
New clinical resources have been developed to help healthcare professionals provide better mental health and cognitive support for people living with Huntington’s disease. The guidance, produced by the Huntington’s Disease Association in collaboration with clinical experts, is intended to address gaps in awareness and specialist knowledge among mental health services. The new publications include a…
Update on AMT-130 for Huntington’s disease
uniQure N.V, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced on November 3rd 2025 that it received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, an investigational gene therapy for Huntington’s disease (HD). Based on the discussions at…
Phase 3 path for Huntington’s disease drug SOM3355
SOM Biotech secures clear registrational path after FDA End‑of‑Phase 2 Meeting SOM Biotech has received formal confirmation from the US Food and Drug Administration (FDA) outlining a clear registrational path for its lead candidate, SOM3355, in Huntington’s disease. The positive outcome follows an End-of-Phase 2 meeting with the Agency, which endorsed the design of the…
Gene therapy for Huntington’s
Uniqure and UCL scientists announce positive results for new treatment Patients treated with the investigational gene therapy AMT-130 experienced a 75% reduction in disease progression over 36 months, compared with matched participants from the long-running Enroll-HD natural history study, according to new data released by uniQure. This is the first drug trial to report sustained,…
The role of DNA repair genes in Huntington’s disease
This is an AI summary of this press release. Recent research from UCLA Health has identified a crucial role for DNA mismatch repair (MMR) genes in Huntington’s disease (HD), revealing how they drive neuronal vulnerability and disease progression. Published in Cell, the study provides mechanistic insights linking genetic modifiers to selective neuronal degeneration, offering potential…
Accelerated Approval in Huntington’s disease?
uniQure announces alignment with FDA on key elements of Accelerated Approval pathway for AMT-130 in Huntington’s disease 10 December 2024: – uniQure N.V., a gene therapy company advancing therapies for patients with severe medical needs, has announced that the company reached agreement with the U.S Food and Drug Administration (FDA) on key elements of an…
Cognitive impairment in HD
Phase 2 DIMENSION Study fails to meet primary endpoint Sage Therapeutics announced that its Phase 2 DIMENSION Study of dalzanemdor (SAGE-718) for cognitive impairment in Huntington’s disease failed to meet its primary and secondary endpoints. Dalzanemdor showed no statistically significant or clinically meaningful improvement versus placebo in the Symbol Digit Modalities Test at Day 84…
