Huntington’s

An experimental drug for Huntington’s disease has been granted an Innovation Passport, giving its developer access to coordinated support from UK regulators, health services and medicines assessment bodies. Brezosiran, developed by Alnylam, is one of three investigational treatments selected by the Medicines and Healthcare products Regulatory Agency (MHRA) for the UK’s Innovative Licensing and Access [...]

The FDA has granted priority review to a Huntington’s disease gene therapy developed by uniQure, with a regulatory decision expected by 1 May 2027. The US Food and Drug Administration (FDA) has accepted the company's application seeking accelerated approval of AMT-130, now known as ifezuntirgene inilparvovec. Priority review shortens the standard regulatory assessment period from [...]

Gene editing has improved movement and reduced harmful protein build-up in mice with Huntington’s disease, according to preclinical research. Researchers found that shortening repeated sections of DNA associated with the condition improved motor function and restored healthier patterns of gene activity in brain cells. The findings suggest the technique could offer an alternative to conventional [...]

A 12-week intermittent fasting pilot involving 20 people with early-stage Huntington’s disease reported improvements in disease markers without unintended weight loss. Participants followed a time-restricted eating schedule, limiting food intake to a six-to-eight-hour window each day. Researchers observed improvements in measures of disease severity, a blood marker associated with nerve cell damage and cellular energy [...]

DNA damage findings could point to a new treatment route for Huntington's disease after an antioxidant reduced neuronal damage and symptoms in mice. Researchers found a marked increase in breaks across DNA strands in a mouse model of the inherited neurodegenerative condition. Treatment with an investigational antioxidant reduced these breaks, prevented motor deficits and reduced [...]

A new imaging technique could help track Huntington's disease by detecting microscopic changes in brain tissue that conventional scans may miss. Researchers found the approach could identify abnormalities in brain regions affected by the inherited neurological condition. The findings could eventually help monitor disease progression and assess experimental treatments, although larger and longer-term studies are [...]

Gene editing reduced toxic protein fragments and Huntington’s symptoms in mice, suggesting a possible new approach to treatment. Unlike other gene-based treatments that aim to switch off the gene, the experimental method changes how cells read it. The tool alters a specific point in the huntingtin gene so the cell skips a small section prone [...]

A study of more than 2.5m adults has identified a genetic link between fibromyalgia and Huntington’s disease. The research found an association between the long-term condition, which causes brain fog, fatigue and widespread pain, and the inherited neurological disorder. Around 7,000 people in the UK are living with Huntington’s, a progressive condition that destroys nerve [...]

The first patient has received an experimental stem cell therapy in a clinical trial for Huntington’s disease. The phase 1b/2a trial will assess the safety of hNSC-01 neural stem cells delivered directly to the brain using a specialised targeting system in an MRI suite. Neural stem cells can develop into several types of cells found [...]

New guidance aims to help health professionals improve Huntington’s care across mental health, cognition and wider support. The resources have been developed by the Huntington’s Disease Association (HDA) with experts including Dr Sarah Gunn, from the University of Leicester, and Dr Akshay Nair. Huntington’s disease is a rare inherited condition that gradually damages nerve cells [...]