Huntington's Disease: Medical Treatments

We recognise the significant challenges faced by those living with rare diseases such as Huntington’s disease. Although there is currently no cure for the disease, there are efforts to ensure that innovative treatments and research opportunities reach patients and improve outcomes for those affected by Huntington’s disease. The Department supports research into Huntington’s disease through the National Institute for Health and Care Research (NIHR). The NIHR invests not only in individual studies but also in world-class research centres, a dedicated delivery workforce, and specialist facilities across the National Health Service. This infrastructure supports pioneering research into Huntington's, including the positive preliminary results for a novel gene therapy reported last month. The NIHR’s Be Part of Research service helps people find and participate in relevant research. There are eight Huntington’s disease studies currently recruiting.

The UK Rare Diseases Framework identifies four main priorities, including improving access to specialist care, treatments, and drugs. In February 2025, we published the England annual action plan, which outlines the steps we have taken to advance this priority.