Huntington’s
An oral therapy for Huntington's disease has been deemed eligible for a fast-track approval pathway by Australia's medicines regulator. The therapy, SKY-0515, is a small molecule designed to target RNA, the genetic instructions cells use to produce proteins involved in the disease. Huntington's disease is a rare inherited neurodegenerative disorder that causes progressive damage to [...]
The US Food and Drug Administration says early trial data for a Huntington's gene therapy are not sufficient to support a marketing application. Gene therapy company uniQure said final meeting minutes from a Type A meeting with the FDA on 30 January 2026 confirmed the regulator's position on AMT-130. AMT-130 is an investigational gene therapy [...]
Harness Therapeutics has named HRN001 as its lead Huntington’s disease drug candidate and set out plans to advance the programme towards clinical trials. Huntington’s disease is an inherited neurodegenerative condition that causes progressive problems with movement, thinking and mental health, with death often occurring within 15 years of symptoms starting. There are currently no approved [...]
Nine months of an investigative treatment appears to slow Huntington's progression in early-stage patients, interim Phase 1 data from an Australian substudy suggests. Huntington's is a progressive disorder caused by a genetic change that produces a toxic form of huntingtin, a protein that harms nerve cells. New interim results indicate the oral candidate SKY-0515 slowed [...]
Northern Scotland has one of the world's highest Huntington's disease rates, more than five times the estimated global average, new research shows. For the first time in 35 years, scientists have accurately quantified the number of people in the region carrying the gene that causes the condition. Using NHS family-based records, researchers identified more than [...]
A man who lost three family members to Huntington's disease is completing a 100km challenge to raise awareness and break stigma around the condition. Mark Barrett, 38, from Howwood, Scotland, is taking on four half marathons and a triathlon over nine weeks after losing his father Frank, grandfather John and uncle Raymond to the inherited [...]
Gene therapy slowed Huntington's disease progression by 75 per cent in a recent trial, in what researchers describe as the first effective treatment for the condition. The therapy means that decline expected in one year would instead take four years, potentially giving patients decades of better quality life, researchers said. Researchers from University [...]
Children of people with Huntington's disease are more likely to experience trauma growing up, which may contribute to mental health problems in later life. A study found that emotional abuse in childhood was the strongest predictor of psychological distress in adulthood among those from Huntington’s-affected families. Huntington’s disease is a progressive genetic brain disorder that [...]
A man living in Worcestershire has fulfilled one of his lifelong ambitions to spend an afternoon driving around the Malvern Hills in a beautiful classic sports car, despite living with a complex neurological condition. Mark, 59, who is a resident at the Inspire Neurocare service in Worcester, asked if it would be possible to make [...]
Transplanting healthy human brain support cells into adult mice with Huntington’s disease slowed motor and cognitive decline and extended lifespan, researchers have found. The study suggests an alternative approach to treating Huntington’s – a hereditary condition that damages nerve cells – by replacing diseased glial cells rather than focusing solely on preserving neurons. Researchers observed [...]
