Welcome to the August 2026 edition of The RFC1 Connection
Message from: Allan Tissenbaum, MD - President, RFC1 Foundation
Only a few months ago, the RFC1 Foundation existed as a vision—to unite patients, families, clinicians, researchers, and supporters around a shared mission of improving the lives of individuals affected by RFC1-related disease. Today, that vision is rapidly becoming reality.
I am pleased to report that our Foundation has grown to more than 100 members representing multiple countries, with new members joining us almost every week. While the majority of our members are individuals living with RFC1-related disorders, our community continues to expand through the involvement of family members, caregivers, healthcare professionals, researchers, and friends who believe that progress is possible through collaboration. One of our most exciting initiatives is the formation of the RFC1 Foundation Patient Advisory Committee. This committee will ensure that the experiences and priorities of patients remain central to every decision we make. Importantly, the committee will elect one of its members to serve on the Foundation's Board of Directors, ensuring that the patient voice is represented at the highest level of our organization.
This issue also introduces an important addition to The RFC1 Connection. Beginning with this edition, members of our Scientific Advisory Board will periodically share their perspectives on RFC1 disease, current research, and future therapeutic directions. These articles will bring our readers directly to the forefront of scientific discovery while helping explain complex topics in language that everyone can understand. We are deeply honored that our inaugural contribution comes from Dr. Puneet Opal, Director of the Denning Ataxia Center at Northwestern University Feinberg School of Medicine. His thoughtful article reflects both the remarkable progress that has been made in understanding RFC1 disease and the optimism shared by researchers around the world as they work toward meaningful therapies.
I also encourage you to join us on September 10 for our next educational webinar featuring Dr. Andrea Cortese and Dr. Peter Todd, two internationally recognized leaders whose work continues to transform our understanding of RFC1disease.
Whether you joined the Foundation yesterday or have been with us since the beginning, thank you. Every new member, every shared experience, and every scientific breakthrough strengthens our community and moves us closer to better treatments—and ultimately, a cure.
On behalf of our Board of Directors and Scientific Advisory Board, thank you for believing in this mission and for helping us build a Foundation that represents hope for families throughout the world.
With warm regards,
Allan Tissenbaum, MD
President, RFC1 Foundation