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October 17, 2025
5 min read
Key takeaways:
- Approximately 5,000 people in the United States have Friedreich’s ataxia.
- Advocates virtually met with legislators on Sept. 17.
- Priorities included timely and robust funding for federal health agencies.
Limited treatment options and access to care often challenge patients with Friedreich’s ataxia. So, on Sept. 17, the Friedreich’s Ataxia Research Alliance and National Ataxia Foundation met with lawmakers to advocate on their behalf.
Healio spoke with Brigid Brennan, director of advocacy at FARA, to find out more about United Against Ataxia Hill Day and FARA’s advocacy goals.
Advocate Kim Simpson (top left) and Brigid Brennan, director of advocacy at FARA (bottom), meet virtually with Emily Kane of the office of Senator Maggie Hassan (D-NH) to discuss legislative priorities. Photo: Friedreich’s Ataxia Research Alliance
Healio: Could you give us some background about Friedreich’s ataxia?
Brennan: Friedreich’s ataxia (FA) is a genetic, progressive neuromuscular disease. People with FA experience issues with balance and coordination of movement that lead to life-altering loss of mobility. Other common symptoms can include fatigue, serious heart conditions, scoliosis and diabetes. FA is life-shortening and affects an estimated 5,000 individuals in the United States and 15,000 worldwide. It also is estimated that one in 100 people with European ancestry are carriers.
There is currently only one approved treatment for adults with FA that is shown to slow progression (but does not address the root cause of the disease), and there are no approved treatments for children with FA. For more treatments to be approved in the U.S., there needs to be robust funding of the FDA and NIH.
Healio: Could you give us some background about FARA and why advocacy is necessary?
Brennan: FARA is a nonprofit organization dedicated to the pursuit of scientific research leading to treatments and a cure for FA. FARA’s mission is to marshal and focus the resources and relationships needed to cure FA by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies and other organizations dedicated to curing FA and related diseases.
Brigid Brennan
FARA also aims to support the FA scientific, pharmaceutical, and medical communities with the tools necessary to execute their best work and improve patient outcomes. FARA can assist healthcare professionals when it comes to diagnosis, participation in research, the latest results of clinical trials, and contact information for Friedreich’s ataxia clinicians.
Lawmakers and agencies have incredible influence over the laws and regulations guiding research and clinical care. Yet most lawmakers are not experts on science, medicine or specific diseases, and they need insight from people who will be affected by legislation and regulations to guide decision-making.
This is where advocacy comes in. Patients, scientists and providers have the expertise that lawmakers are missing, whether it’s lived experience or clinical training. It is critical that this expertise be brought to legislators and centered in the policymaking process, and that cannot happen without advocacy.
Advocacy has become a critical tool in advancing FARA’s mission. So much of the vital scientific research infrastructure is funded and maintained by congressional appropriations and legislation. FARA advocates to ensure that these agencies like the FDA and NIH have the resources they need to fulfill their missions, and, in turn, FARA works to secure available federal grant opportunities for FA researchers.
Advocacy isn’t limited to the legislative branch. Stakeholders also provide input to regulatory agencies on proposed rules and other regulatory guidance, such as speaking at hearings for Medicaid pharmacy and therapeutics or drug utilization reviews, which can have widespread implications for patient care and research.
Healio: What can you tell us about United Against Ataxia Hill Day?
Brennan: United Against Ataxia Hill Day is an event jointly organized by both FARA and the National Ataxia Foundation with the intention of educating lawmakers on ataxia and the needs of the ataxia community, as well as advocating for legislative changes that would better support the community.
During the event, advocates met with two to four congressional offices to share their stories and asked lawmakers to take action to better support ataxia patients. The event was fully virtual to ensure that as many advocates as possible were able to participate.
This year was the seventh annual United Against Ataxia Hill Day. In a typical year, over 100 advocates from across the country come together. This year we had a record turnout with 169 advocates from 38 states covering 99 meetings. Our hope is that the event not only educated legislators on ataxia and the needs of the ataxia community but also allowed advocates to foster longer-term relationships with their elected officials.
Healio: Are there specific concerns that FARA brought up with legislators?
Brennan: This year, FARA had a number of specific legislative asks.
The primary ask was timely and robust funding for federal public health agencies, namely the FDA and NIH. Research is the foundation of effective treatments and clinical care, and support for federal public health agencies is integral to ensuring that research and progress towards new approved treatments continues. The current government shutdown directly impacts research and delays treatments to patients.
Next, FARA asked for cosponsors for the Give Kids A Chance Act (H.R. 1262/S. 932). This bill would, among other things, reauthorize the Rare Pediatric Disease Priority Review Voucher program, which is a critical incentive for development of treatments for rare pediatric diseases. The program designates that any company receiving an FDA approval for a product indicated for a pediatric rare disease will receive a Priority Review Voucher, which would allow them to get an expedited approval for a subsequent product.
The voucher can be used on any type of subsequent product, not just another rare disease product. But most importantly, it’s incredibly valuable to biotechnology companies, making it an effective incentive. However, the longstanding program expired at the end of December 2024 when it was not included in the Dec. 19 continuing resolution. In addition to its importance for the rare disease community as a whole, it is particularly important to the ataxia community, given that it helped yield the first FDA approved treatment for FA.
FARA also advocated for the Accelerating Kids’ Access to Care Act (H.R. 1509/S. 752). There are very few specialists in rare diseases, and this means that patients are often forced to seek care out-of-state to be diagnosed and treated. However, this becomes complicated for patients on Medicaid.
Medicaid programs generally do not cover out-of-state care unless a provider is enrolled in the other state’s Medicaid program, and the process of enrolling in out-of-state Medicaid plans can be burdensome to providers. This bill seeks to provide a voluntary, streamlined process for providers to enroll in out-of-state Medicaid programs, hopefully alleviating administrative burden and improving access to care for patients.
Healio: At the practice level, is there anything that physicians can do to mitigate these concerns?
Brennan: Clearly document condition in medical records, including appropriate ICD-10 codes. Engage with patients on available clinical trials. Share clinical challenges with legislators and the FARA advocacy team.
Healio: How can physicians who are interested get more involved?
Brennan: If you’re a physician hoping to get involved in advocacy, we encourage you to engage with patient advocacy groups, and most importantly, reach out to your members of Congress to discuss the issues impacting your practice and your patients.
For more information:
Brigid Brennan can be reached at brigid.brennan@curefa.org.
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