Over the past two days, I had the privilege of representing Coalition to Cure Calpain 3 in my advocacy chair role at LGMD Policy Action Day on Capitol Hill. Our community came together for a full schedule of meetings across both the House and Senate, ensuring that the realities of ultra-rare drug development remain front and center in policy conversations.
LGMD Policy Action Day uniquely brings forward both scientific expertise and lived experience, a combination that deeply resonates with policymakers. As an advocate, I had the opportunity to help elevate the voices of our community while reinforcing a critical message: ultra-rare drug development must evolve if we are to deliver meaningful treatments to those who urgently need them.
Throughout our meetings, LGMD was consistently positioned as a case study, illustrating the complex and often overlooked challenges faced by ultra-rare disease communities. From discussions with the HELP Committee to engagement with the Doctor’s Caucus, and from FDA-related conversations to key appropriations priorities like the PRMRP and LGMD Centers of Excellence, our presence ensured that LGMD is being recognized at the highest levels of policymaking.
I am especially grateful to the Doctor’s Caucus for inviting the The Speak Foundation to share LGMD as a case study for ultra-rare disease, and to Co-Chairs Congressman Greg Murphy, MD and Congressman John Joyce for creating space for this important dialogue.
I’d like to thank The Speak Foundation for the passion and vision to bring this Action Day to life, as well as extend sincere appreciation to Dr. Nick Johnson for bringing a powerful scientific perspective into every conversation. He helped policymakers better understand what it truly takes to advance therapies in a space defined by small patient populations, progressive disease, and urgent unmet need. Special thanks to my #mommasonamission who teach me daily how to be a better advocate: Curelgmd2i Foundation and LGMD2D Foundation.
The science is here. Our voices are being heard. Now policy must evolve.
Melina Garza, C3 Advocacy Chair
