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For decades, families affected by Huntington’s disease (HD) have faced a relentless neurological illness with no treatment to slow or stop its progression. Today, that may be changing, but whether scientific progress becomes real hope will depend on federal leadership.
On Huntington’s Disease Day of Action, April 13, advocates nationwide are urging policymakers to act with the urgency this moment demands.
Huntington’s disease is a rare, inherited, and ultimately fatal brain disorder that often strikes in midlife.
From symptom onset to death, it typically progresses over 10 to 20 years, steadily robbing individuals of their ability to think, move, and live independently. Each child of an affected parent has a 50 percent risk of inheriting the disease. As symptoms worsen, many individuals and caregivers are forced to leave the workforce, creating devastating financial consequences for families across generations.
After decades of setbacks, the HD community now stands on the brink of potential breakthroughs. A growing pipeline of novel therapies driven by advances in gene-targeting science and sustained research investment aims to slow disease progression.
People living with HD have made clear what progress means to them. In a 2024 Huntington’s Disease Society of America survey, nearly three-quarters of respondents said they would accept treatment risks for five years without disease progression.
Congress must act now. Lawmakers should urge the FDA to fully utilize the accelerated approval authorities Congress has provided — taking into account patients’ tolerance for risk — and ensure that all promising therapies in the Huntington’s disease pipeline receive timely, flexible, and science-based review grounded in the totality of the evidence.
For HD families, time matters. With strong federal leadership, hope can finally move from the lab to everyday life.
Nancy Patterson
Ellsworth
