Patient-led organization urges Congress to strengthen LGMD research and help promising rare-
disease science reach patients faster

WASHINGTON, Sept. 16, 2026 /PRNewswire/ -- Advocates living with limb-girdle muscular dystrophy (LGMD) traveled from across the country to Washington this week, bringing their lived experience directly to the lawmakers shaping rare-disease research, regulation and treatment development.

LGMD refers to a group of rare genetic diseases that cause progressive muscle weakness and loss of function. Despite growing scientific progress, there is currently no FDA-approved treatment specifically for LGMD.

During The Speak Foundation's LGMD Day on the Hill, advocates representing more than 20 states participated in more than 60 meetings with congressional offices. They urged lawmakers to support federal investment in LGMD research, expand access to applicable Department of Defense research funding and encourage clearer, more consistent regulatory pathways for rare-disease treatments.

The event comes at a pivotal moment for LGMD, as scientific advances are bringing the community closer to the possibility of its first FDA-approved treatment for a subtype called LGMD 2I/R9, developed by BridgeBio.

"For people living with a progressive rare disease, time is measured differently. Research and clinical trials can take years while patients continue to lose strength and function," said Kathryn Bryant Knudson, founder and CEO of The Speak Foundation, who lives with LGMD. "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, and every one of those contributions should move the science forward."

Advocates asked Congress to support policies that recognize both the scientific challenges of studying rare diseases and the expertise patients bring to the process. The Speak Foundation supports engaging patients before trials are designed so their perspectives can inform endpoints, eligibility criteria, visit schedules and outcomes that matter in daily life.

Advocates also discussed with their representatives the need for greater regulatory predictability. Rare-disease development programs often operate with limited funding, data and eligible patient populations. When regulatory expectations are unclear, change late in development or are applied inconsistently, promising programs can stall and investment can disappear.

The Speak Foundation Honors Congressional Champions

As part of LGMD Day on the Hill activities, 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 advanced efforts to modernize clinical development, make trials more accessible and better integrate research into patient care. Joyce, a physician, has championed policies intended to preserve incentives for continued rare-disease drug development, including the bipartisan ORPHAN Cures Act.

"Hearing directly from those living with rare diseases, such as Limb-Girdle Muscular Dystrophy, puts in perspective why this work matters," said Congressman John Joyce, M.D. "Too many people living with rare diseases face few answers and even fewer treatment options, and they're counting on Congress to draw attention to the unique challenges that they face. My bipartisan ORPHAN Cures Act, which was signed into law earlier this Congress, is a step toward making sure this community isn't left behind. By working across the aisle, we can keep pushing research forward, leading to medical breakthroughs in treatments and greater hope for LGMD patients and their families."

"The progress families affected by rare diseases need will require sustained bipartisan leadership," Knudson said. "Representatives Joyce and Auchincloss understand that medical innovation is meaningful only when it reaches the people waiting for it. We are grateful for their willingness to listen to patients and work across party lines to strengthen rare-disease research and treatment development."

"We are entering a new era for LGMD," Knudson said. "Now we need policy to keep pace with the science."

About Speak Foundation

The Speak Foundation is a patient-led nonprofit advancing care, research, advocacy and innovation for people living with limb-girdle muscular dystrophy (LGMD) and other neuromuscular rare diseases. Founded in 2008 as the first patient-led nonprofit dedicated to all forms of LGMD, the organization brings authentic lived experience to efforts to improve care, research, drug development and policy. It brings patients and families together with clinicians, researchers, industry and policymakers to expand access to expert care, strengthen drug development and ensure patients help shape the decisions that affect their lives. Through initiatives including the LGMD Centers of Excellence, Speak Foundation is building patient-designed solutions that can serve as a model for the broader rare-disease community.

www.TheSpeakFoundation.com 

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SOURCE The Speak Foundation