HDSA 2026: Huntington’s community urged to step up advocacy efforts
Nonprofit exec stresses importance of lobbying for US legislative changes
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Every member of the Huntington’s disease community — patients, their family members, friends, and supporters — plays a crucial role in advocating for legislative and policy changes that will make a better future for people with the genetic disorder.
That’s the message from Phyllis Foxworth, senior manager of advocacy at the Huntington’s Disease Society of America (HDSA), who outlined ongoing advocacy efforts in a talk titled “Driving Change for the HD Community: From the FDA to the Act for ALS.” Foxworth was a key speaker at the 41st annual HDSA convention, held last week in Phoenix.
“The fight is still ongoing. … I am very optimistic that [due to] all the work that the community has done, not just your community, but the entire rare disease community, that things are moving forward in the right way,” Foxworth said, outlining advances made with U.S. regulators and Congress.
The convention’s webpage notes that the HDSA event was designed as “an inspiring gathering of patients, families, caregivers, researchers, and advocates all coming together to learn, share, and build hope for the future.”
According to Foxworth, the nonprofit’s advocacy program focuses on three areas: speeding therapeutic development, increasing access to care, and reducing financial burden.
Speaker notes advances with FDA, Congress
Foxworth said the HDSA works to achieve these goals with a strategic plan. The first step, she says, is to increase awareness — which supporters do, according to Foxworth, every time they participate in events, contact members of their local government, or speak with neighbors and other community members about Huntington’s.
“When we create awareness, it gives us the opportunity to drive the conversation,” Foxworth said. “We no longer are asking for a seat at the table. We are demanding that we set the table. … We’re setting the table with our own agenda instead of waiting to be asked.”
Foxworth pointed to the HDSA’s ongoing relationship with the U.S. Food and Drug Administration (FDA). In 2025, the team became concerned that, under new leadership, the FDA was making decisions that could negatively impact the development of new treatments for Huntington’s.
Congress has given the FDA broad authority to determine policies for rare disease drug development and clinical trial criteria. However, it was becoming unclear how regulators were making these decisions, according to Foxworth, hindering drug developers’ ability to design appropriate clinical trials and move toward regulatory approvals.
An example discussed is the unsteady path toward approval for AMT-130, a gene therapy candidate from uniQure. Regulators had previously said certain trial data could support an application for accelerated approval, but later reversed course, telling the company an entirely new trial would be needed.
Advocates decided to take the matter directly to U.S. legislators. “We were asking Congress to insist that the FDA give us transparency around the decisions that were being made,” Foxworth said. “If we don’t understand and if there’s no transparency … it affects everything else that’s coming up [in the pipeline].”
We no longer are asking for a seat at the table. We are demanding that we set the table… we’re setting the table with our own agenda instead of waiting to be asked.
Foxworth credits the Huntington’s community for showing up and speaking up. “Give yourselves a huge round of applause: You got into some good trouble,” Foxworth told the audience, quoting John Lewis, a famed U.S. representative from Georgia and civil rights activist.
Since last fall, advocates have sent thousands of petitions to the FDA and emails to Congress, and met with their representatives, Foxworth noted.
Advocacy needed to improve Huntington’s clinical trials
Earlier this year, the HDSA brought in clinician experts from its Centers of Excellence network to speak with Congress about clinical trial design and the importance of using data from natural history studies to inform drug development efforts.
Such studies are observational and track the natural course of a disease, typically involving patients not on interventional treatment but receiving standard care to manage symptoms. Natural history studies are used to assess how a potentially disease-modifying candidate therapy might alter the condition’s natural course.
“As a result of all your work, the director of the FDA’s center for biologics evaluation and review was dismissed, the commissioner of the FDA resigned, and the FDA reversed [its] decisions on accepting [an application] for accelerated approval for AMT-130,” Foxworth said.
Uniqure is now working on its application, which will be supported by data from clinical trials testing AMT-130 compared with data from natural history studies.
Amid applause, Foxworth noted that advocacy work is never done. Looking forward, one area of focus is updating the language in the Act for ALS, a 2001 piece of legislation enacted to accelerate treatments for people living with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases.
The problem, according to Foxworth, is that the way the bill is written, there’s no funding carved out for diseases other than ALS. The HDSA team drafted new language for the bill that would require new diseases to be included in the program, but now Congress has to pass it.
Foxworth called on the community, specifically the HDSA 2026 audience members who were in attendance, to take out their phones and call their members of Congress to ask for support to include Huntington’s in the Act. She pointed them to HDSA’s advocacy hub, where individuals can find contact information for their local representatives and a call script. The session ended with a chorus of audience advocates making their own calls on behalf of the Huntington’s community.
Note: The Huntington’s Disease News team is providing virtual coverage of the Huntington’s Disease Society of America’s annual conference June 25-27. Go here to see the latest stories from the conference.
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