By Leigh Farmer 

VCU Health is the first Limb-Girdle Muscular Dystrophy Center of Excellence in the country, marking a historic step forward in care, research and patient engagement for a rare disease with no cure and no federally-approved treatments. 

Limb-girdle muscular dystrophy (LGMD) is a collection of diseases marked by muscle weakness around the hips and shoulders and caused by mutations in more than 25 different genes.

The LGMD Center of Excellence at VCU Health, established in partnership with The Speak Foundation (TSF), will serve as the founding site of a planned national network of five LGMD Centers of Excellence across the United States. The Centers of Excellence were selected through a rigorous process for their expertise in neuromuscular care, commitment to multidisciplinary collaboration and leadership in advancing LGMD research – reflecting a shared dedication to improving patient outcomes and accelerating progress for the LGMD community. 

Each of the five Centers of Excellence will also designate a dedicated LGMD care coordinator to support patients and families in navigating multidisciplinary care, coordinating appointments across specialties and accessing the comprehensive services that define the Centers of Excellence model. In addition, each center commits to ongoing research, clinical trial readiness and participation in patient-centered care. 

This milestone is rooted in a powerful, full-circle story. Kathryn Bryant Knudson, founder of The Speak Foundation, started receiving care at VCU Health at age 7. Raised in Richmond and a graduate of the University of Virginia, her health journey started right here. For years, clinicians knew she had muscular dystrophy, but the genetic cause of her disease had not yet been discovered. 

Kathryn Bryant Knudson, CEO and founder of The Speak Foundation (right), with Nicholas Johnson, M.D. (left), a neuromuscular neurologist who leads VCU Health’s LGMD patient care and research efforts. (Children's Hospital Foundation)

In 2006, she was finally diagnosed with LGMD 2I/R9. The diagnosis spurred her to build a patient-driven foundation to elevate the lived experience of individuals with muscular dystrophy and accelerate progress in research and care. Today, she is reconnecting with VCU Health as a meaningful partner. 

“VCU Health was where myjourney began, long before science had answers,” Knudson said. “To now stand here, helping build a Center of Excellence that will change the future for families living with LGMD is deeply meaningful. This center ensures patients are no longer navigating this disease alone.” 

As the program expands, Knudson will also serve as the executive director under The Speak Foundation. 

The LGMD Center of Excellence at VCU Health will provide coordinated, multidisciplinary care while advancing clinical research, data generation and trial readiness. It will serve as a central hub for expertise in a disease that includes dozens of genetically distinct subtypes. 
 
VCU Health’s LGMD patient care and research efforts are led by Nicholas Johnson, M.D., a neuromuscular neurologist who directs the GRASP-LGMD consortium and a recognized leader in collaborative rare disease research. Johnson founded GRASP-LGMD in 2018, establishing an international, multi-site network focused on accelerating LGMD therapy development. 

“This Center of Excellence model allows us to deliver expert, coordinated care while building the research pathways necessary to bring safe and effective treatments to patients faster,” Johnson said. 
 
As the inaugural site, VCU Health will anchor a national LGMD Centers of Excellence network designed to expand access to expert care, accelerate research and ensure the patient’s voice remains central to every decision. 
 
For families who have waited decades for answers, the launch of the LGMD Center of Excellence at VCU Health represents a new era — where science, clinical care and lived experience come together to drive meaningful progress.

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