Last week, Emily Milligan, Executive Director, and Shelley Bowen, Director of Advocacy, spent days in Washington, D.C. advocating on behalf of the Barth Syndrome Foundation community. They had the honor of participating in a special hearing on the unmet medical needs faced by individuals with rare, progressive, and serious diseases led by Senator Mike Braun (R-IN), who serves as the Ranking Member of the Senate Special Committee on Aging. Before and after the meeting, Emily and Shelley spent significant time meeting with members of Congress to discuss our challenges with the FDA in more detail and to appeal to them for intervention.
During the hearing, Emily had the opportunity to share the ongoing advocacy work conducted by Barth Syndrome Foundation. She emphasized our commitment to urge the FDA to provide a fair, appropriate, and equitable review for elamipretide, and she reinforced the importance of standing together in the face of adversity, fostering a sense of understanding and support that transcends the inequitable, unjust, and illogical aspects of the current system. This shared experience has not only strengthened our resolve but has also reinforced the assurance that we are not alone in this journey.
"We see other drugs, other diseases, benefit from regulatory policies that are outside of our reach and for no good reason,” said Emily. “This was a unifying experience to be part of something bigger and join in solidarity, working together to fix parts of the FDA that aren't serving us or our loved ones well.”
Out of the 350 patient stories submitted to the congressional record, an impressive 21 came from the Barth Syndrome Foundation community. Patient stories have proven to be an incredibly powerful advocacy tool.
See Pink Sheet summary of the meeting available here.
"Pass the 'Promising Pathway Act' | Senator Braun" video available here.
Link to the Senate Hearing available here.