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       February 10, 2023

Louise Vetter, HDSA’s President & CEO, provides statement in support of the FDA Listening Session on Juvenile Onset Huntington’s Disease

New York, New York — Juvenile onset Huntington’s disease (JoHD) is the tragedy of Huntington’s disease magnified by the loss of childhood and adulthood. Unlike adult onset Huntington’s disease, which robs function from an individual in the prime of life, JoHD steals lives early, denying them the hopes and dreams of independent lives.

The current tools for developing new treatments for Huntington’s disease have limited application to the JoHD community.  The symptoms of JoHD are different, challenging the utility of today’s clinical assessments. The disease progression is accelerated, requiring a unique risk-benefit ratio and shorter, more innovative approaches to identifying therapeutic solutions. Furthermore, the Huntington’s Disease Integrated Staging System (HD-ISS), which could have profound impact on identifying interventions for pre-symptomatic adults who inherit the HD gene expansion, has limited value in JoHD where stages 0 and 1 are unidentified due to how JoHD is diagnosed.

While all families affected by Huntington’s disease require the partnership of the US Food and Drug Administration to bring forward effective therapeutic solutions, the unique needs of the JoHD community demand special consideration and immediate attention. 

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Huntington’s disease (HD) is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities during their prime working years and has no cure. Every child of a parent with HD has a 50/50 chance of inheriting the faulty gene that causes HD. Today, there are approximately 41,000 symptomatic Americans and more than 200,000 at risk of inheriting the disease. The symptoms of HD are described as having, ALS, Parkinson’s and Alzheimer’s – simultaneously.

The Huntington’s Disease Society of America (HDSA) is the premier nonprofit organization dedicated to improving the lives of everyone affected by HD. From community services and education to advocacy and research, HDSA is the world’s leader in providing help for today, hope for tomorrow for people with HD and their families. To learn more about Huntington’s disease and the work of the Huntington’s Disease Society of America, visit www.HDSA.org or call (800) 345-HDSA.