WASHINGTON, D.C. – During Rare Disease Week on Capitol Hill (February 24-26, 2025), the Barth Syndrome Foundation (BSF) recognized 16 members of Congress as “Champions of Progress” for their unwavering advocacy on behalf of individuals and families affected by Barth syndrome. These lawmakers have demonstrated leadership in advancing policies that support the rare disease community, improve research funding, and drive regulatory progress for life-changing therapies, including elamipretide for the treatment of Barth syndrome.
Among those honored were:
Senator Mark Kelly (AZ-D)
Senator Roger Wicker (MS-R)
Representative Jake Auchincloss (MA-04-D)
Representative Gus Bilirakis (FL-12-R)
Representative Buddy Carter (GA-01-R)
Representative Sharice Davids (KS-03-D)
Representative Mike Flood (NE-01-R)
Representative Michael Guest (MS-03-R)
Representative Morgan McGarvey (KY-03-D)
Representative Jim McGovern (MA-02-D)
Representative Doris Matsui (CA-06-D)
Representative Ralph Norman (SC-05-R)
Representative Paul Tonko (NY-20-D)
Representative William Timmons (SC-04-R)
Representative Lori Trahan (MA-03-D)
Representative Joe Wilson (SC-02-R)
“Rare Disease Week is a powerful reminder that strong public policy is essential to delivering treatments to families who urgently need them,” said Emily Milligan, Executive Director of BSF. “Our Champions of Progress understand the urgency of our fight, and we are grateful for their commitment to ensuring that the voices of those affected by Barth syndrome are heard.”
A key issue raised during Rare Disease Week was the ongoing FDA review of elamipretide, a potential treatment for Barth syndrome. Several members of Congress have continued to follow this critical process, emphasizing the need for transparency, expidiency, patient-centered decision-making, and the prioritization of rare disease innovation. The FDA is expected to make its decision on April 29, 2025, and BSF remains committed to working with regulators, researchers, and policymakers to ensure that families have access to safe and effective treatments as soon as possible.
BSF joins hundreds of advocates and patient organizations in urging Congress to advance policies that accelerate medical breakthroughs, expand access to rare disease treatments, and strengthen federal rare disease programs and research funding. The organization remains committed to partnering with lawmakers and federal agencies to create meaningful change for the Barth syndrome community.
Each of our Champions received a deck prism—a symbol of light and guidance. Once used in wooden sailing ships to illuminate dark spaces below deck, these prisms captured sunlight and shone it where it was needed most. It is a fitting tribute from our community, honoring the light our Champions have brought to those affected by Barth syndrome and other ultra-rare disorders.
