Hope Grows for Huntington's Disease Treatments

Researchers make progress in understanding genetic roots and developing new therapies for the rare, inherited neurological disorder.

Mar. 31, 2026 at 2:42am

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Amy Aungst, who carries the genetic mutation for Huntington's disease, has seen the condition devastate her family. But she remains hopeful that advancements in research will lead to new treatments that can slow the progression of symptoms or even prevent the disease altogether, allowing her to live a full life and watch her children grow up.

Why it matters

Huntington's disease is a rare, inherited neurological disorder that affects about 30,000 people in the U.S. It causes progressive brain damage, leading to uncontrolled movements, emotional problems, and loss of thinking ability. Researchers are making progress in understanding the genetic roots of the disease and developing new therapies that could significantly improve quality of life for those affected.

The details

Huntington's disease is caused by a genetic mutation that leads to the production of an abnormal huntingtin protein, which can damage and kill brain cells. Symptoms typically appear between ages 30 and 50 and worsen over time, with most people living 15-20 years after diagnosis. Amy Aungst, 26, carries the genetic mutation but has not yet experienced symptoms. She has participated in research studies to help advance understanding and treatment of the condition, which has devastated her family. Researchers are exploring gene-based therapies to lower huntingtin protein levels, as well as medications to manage symptoms like involuntary movements and depression.

  • In 2015, Aungst found out she carries the genetic mutation for Huntington's disease at age 26.
  • For the past 8 years, Aungst has organized walks to raise awareness about Huntington's disease in her local community.

The players

Amy Aungst

A 26-year-old woman who carries the genetic mutation for Huntington's disease but has not yet experienced symptoms. She has participated in research studies to help advance understanding and treatment of the condition, which has devastated her family.

Jee Bang, M.D., M.P.H.

The clinical director of the Johns Hopkins Huntington Disease Center of Excellence, who shares that major research advancements, including recent FDA-approved medications for the treatment of chorea, are a result of partnerships with study participants like Aungst.

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What they’re saying

“I really want a different outcome for Huntington's disease. I've seen it affect my whole family my whole life.”

— Amy Aungst

“There are good reasons to have hope.”

— Jee Bang, M.D., M.P.H., Clinical Director, Johns Hopkins Huntington Disease Center of Excellence

What’s next

Researchers continue to study different aspects of Huntington's disease to understand its molecular underpinnings, how it affects people differently, and how this knowledge could inform the use of different treatments and their timing. The ultimate goal is to see if huntingtin-lowering therapies given at the earliest sign of rising protein levels could significantly delay or stop symptoms before they occur.

The takeaway

Advancements in research, including the development of gene-based therapies and new medications to manage symptoms, are providing hope for people like Amy Aungst who carry the genetic mutation for Huntington's disease. By participating in studies, Aungst and others are helping drive progress that could lead to treatments to slow the progression of this devastating neurological disorder and improve quality of life for those affected.