As policymakers, researchers, and patient advocates gathered in Washington, D.C. on June 2nd, to discuss the future of rare disease clinical trials, CureGRN attended to ensure the GRN community's voice was part of the conversation at the U.S. Capitol.

CureGRN advocates Ben Adams and Heidi Hall attendance at the Rare Disease Congressional Caucus Town Hall served three key goals:

• To connect with fellow advocates and rare disease organizations facing the same critical roadblock that GRN patients face, namely, the FDA's requirement for placebo controls in Phase 3 clinical trials. For patients living with rare diseases who are in life-or-death situations, this requirement poses a profound ethical and practical barrier to access to potentially life-saving treatments.
• To learn firsthand what Congress is currently doing to address these systemic issues: including proposed legislation and policy initiatives aimed at creating more compassionate and effective clinical trial frameworks for rare disease communities.
• To listen directly from patients and families whose experiences highlight the urgency of reform and the very human stakes of this work. Hearing from those who are living with devastating diseases reinforces why advocacy and action cannot wait.

"One of the most meaningful parts of the town hall was hearing directly from patients and families affected by other rare diseases. Their experiences echoed so many of the challenges our GRN community faces. It was a powerful reminder that we are not alone in this fight and that our collective voices can help drive impactful policy change for clinical trials involving rare diseases."

—Heidi Hall, CureGRN Champion

About the Town Hall

Sponsored by the Huntington's Disease Society of America (HDSA) and moderated by Amy Gray, HDSA President and CEO, the event brought together scientists, patient advocates, biomedical researchers, financial analysts, and U.S. legislators — all united by a shared commitment to accelerating access to treatments for rare and terminal diseases.

Panel Speakers & Key Takeaways

Dr. Tamara Maiuri, Huntington's Disease Society of America (HDSA)

• A scientist with 15 years of dedicated Huntington's disease research, Dr. Maiuri provided valuable insight into the scientific landscape and the utmost need for trial design reform to better serve terminal rare disease patients.
• In a particularly moving moment, Dr. Maiuri shared that she has borne witness to a patient who was directly and devastatingly impacted by the FDA's current clinical trial guidelines, illustrating a real, human consequence of a policy not recognizing the urgency facing terminal rare disease communities.

Emily Gantman, PhD, CHDI Management
Vice President of CHDI Management, a privately funded, single-disease-focused nonprofit biomedical research organization dedicated exclusively to Huntington's disease.

• Dr. Gantman spoke from a biomedical research organization perspective about the unique challenges of funding and conducting research for rare diseases when there is no broad commercial incentive, and the power of focused, mission-driven science.

Kat Bryant Knudson, Patient Advocate, Limb-Girdle Muscular Dystrophy

• Bryant Knudson shared her personal experience living with Limb-Girdle Muscular Dystrophy and gave a powerful account of the logistical and physical burden that clinical trial participation places on patients with disabilities including the challenges of traveling to and from trial locations when a person’s body makes travel incredibly difficult.
• Her testimony underscored the need for decentralized trial options, home visits, and better accessibility infrastructure for rare disease patients.

Mont Stanford, Washington Analysis  

• Stanford spoke from an investor and policy analysis perspective, walking the audience through the difficult process of attracting investors to rare disease clinical trials.
• Her most resonant point: a patient's personal story is the number one selling point when it comes to winning over investors. Data matters but humanity closes the deal.

Dr. Lauren Moore, PhD, National Ataxia Foundation
Chief Scientific Officer and Vice President of Research

• Dr. Moore brought an intensely personal dimension to the conversation, sharing her experience watching her grandmother, her father, and now her sister suffer from Ataxia. She is more than a scientist; she is a daughter, a granddaughter, and a sister on the front lines of a disease that has followed her family for generations.
• Her story was a powerful reminder that behind every research paper, every trial design, and every piece of legislation is a family waiting for answers.

U.S. Congressional Representatives

Two members of Congress joined the panel and delivered a message that gave the rare disease community real cause for hope:

• Representative Jake Auchincloss and Representative Riley Moore both spoke with conviction about the urgent need for FDA reform when it comes to genetic and rare disease clinical trials.
• Both representatives agreed that these changes must be and can be a bipartisan effort. Patients suffering from rare diseases do not belong to one party. And the solution cannot either.

Looking Ahead

CureGRN will continue to show up in rooms like this: advocating alongside our rare disease community partners, amplifying the voices of GRN patients and families, and pushing for the policy changes that will make clinical trials more accessible, more humane, and more effective for everyone living with a terminal rare disease.

"Being in the room with patients, researchers, advocates, and lawmakers reminded me that progress happens when people come together around a shared purpose. The voices of families affected by rare diseases need to be heard, and I was proud to represent the GRN community in those conversations."

—Ben Adams, CureGRN Champion

Together, we are louder. Together, we are closer to a cure.