03/27/2026
Over the past two days, our Executive Director, Rachel DeConti, and young LGMD2D advocate Jacob were on for LGMD Policy Action Day — meeting with leaders across the House and Senate to address the realities of ultra-rare drug development.
Sharing Jacob’s story and the voices of our LGMD2D community, LGMD was positioned as a powerful case study — highlighting the urgent need to evolve how therapies are developed so patients can access treatment faster.
From the HELP and Aging Committees to the Doctor’s Caucus, and across key U.S. Food and Drug Administration and appropriations discussions, we helped ensure LGMD is being recognized at the highest levels of policymaking.
Thank you to The Speak Foundation for coordinating and leading this effort, to the many Offices who met with our community — especially Senator Richard Blumenthal, Senator Chris Murphy, Congresswoman Diana DeGette, and VCU Health Dr. Nick Johnson for bringing the critical scientific perspective.
We are always grateful to stand alongside our LGMD advocacy family in these moments, Curelgmd2i Foundation and Coalition to Cure Calpain 3.
The science is here. Our voices are being heard. Now the speed in treatment development and access needs to evolve.
