Huntington’s disease (HD) is a fatal neurodegenerative disease characterized by uncoordinated and uncontrollable movements, cognitive deterioration, as well as behavioral and/or psychological problems. The classic onset of HD symptoms typically occurs in middle age, but the disease also manifests in children and the elderly. Disease progression is characterized by a gradual decline in motor control, cognition, and mental stability and generally results in death within 10-20 years of clinical diagnosis. Current treatments for HD aim to ease the burden of symptoms and to improve quality of life, and there is a very high level of unmet need for disease-modifying therapies for HD.

This report provides an overview of the HD market including a comprehensive analysis of current therapies and medical practice and opportunities for emerging therapies. The findings described in this report are derived from detailed interviews with expert U.S. and European neurologists, secondary research, and best-in-class epidemiological analysis. This report provides deep insights into this complex and evolving clinical space and includes a detailed analysis of specific opportunities for current and emerging therapies, including experts’ actionable recommendations to optimize development of new HD therapies

Questions Answered in This Report:

  • HD is a rare genetic disease affecting only a small percentage of the population. What is the size of the U.S. and EU5 (France, Germany, Italy, Spain, and the United Kingdom) HD patient population, and how will it change over the ten-year study period?

  • The pathophysiological basis of the onset and progression of neurodegeneration in HD is unclear, and multiple mechanisms have been proposed. How is experts’ understanding of the HD pathophysiological basis evolving? What are the current treatment landscape and medical practice in HD? What are the key drivers for prescribing in HD?

  • Unmet needs in HD are many and span a range of challenging issues. What are the most promising avenues of research and development? Which unmet needs do thought leaders expect to remain unaddressed by mid- to late-stage emerging therapies for HD?

  • Owing to the lack of disease-modifying treatment options for HD, the treatment goal of HD is to optimize patients’ quality of life through addressing symptoms and maximizing their ability to live and function independently for as long as possible. What potential approaches to developing a disease-modifying therapy are in study? What are thought leaders’ opinions about the directions of drug research in HD?


Market covered: United States, France, Germany, Italy, Spain, and United Kingdom.

Primary research: Eight country-specific interviews with thought-leader neurologists.

Epidemiology: Diagnosed prevalent cases of HD by country, by age, and by gender.

Emerging therapies:  Phase II: 4; Phase III/PR: 3; coverage of select preclinical and Phase I products.

Author(s): Seamus Levine-Wilkinson, Ph.D.