Washington, D.C. – – September 18, 2026 -- Advocates for limb-girdle muscular dystrophy (LGMD) held more than 60 meetings with congressional offices this week, representing patients from over 20 states, as part of The Speak Foundation's LGMD Day on the Hill.
No FDA-approved treatment exists for LGMD despite scientific progress
LGMD is a group of rare genetic diseases causing progressive muscle weakness, and no FDA-approved treatment currently targets the condition. Advocates urged lawmakers to increase federal investment in LGMD research, expand access to Department of Defense research funding, and push for clearer, more consistent regulatory pathways for rare-disease treatments.
BridgeBio nears potential first approval for an LGMD subtype
The event coincided with scientific advances bringing the community closer to a possible first FDA-approved treatment for LGMD 2I/R9, a subtype being developed by BridgeBio. "Patients are not a renewable resource. Every blood draw, muscle biopsy, tissue donation and research visit is a significant contribution from a very small community," said Kathryn Bryant Knudson, founder and CEO of The Speak Foundation, who lives with LGMD.
Advocates push for patient input before trial design begins
The Speak Foundation is advocating for patient engagement prior to trial design so perspectives can shape endpoints, eligibility criteria, visit schedules and outcomes relevant to daily life. Advocates also flagged the risk that inconsistent or late-changing regulatory expectations can stall rare-disease programs with limited funding, data and eligible patient populations, discouraging investment.
Two House lawmakers receive 2026 Congressional Champion awards
The Speak Foundation presented its 2026 Congressional Champion for Limb-Girdle Muscular Dystrophy Awards to Rep. John Joyce, M.D. (R-PA) and Rep. Jake Auchincloss (D-MA), both members of the House Energy and Commerce Subcommittee on Health. Auchincloss has worked to modernize clinical development and integrate research into patient care, while Joyce championed the bipartisan ORPHAN Cures Act, signed into law earlier this Congress to preserve incentives for rare-disease drug development.
"My bipartisan ORPHAN Cures Act... is a step toward making sure this community isn't left behind," Joyce said, adding that cross-party work can keep pushing research forward.