The Speak Foundation today announced the launch of the LGMD Centers of Excellence, a national network of leading academic medical institutions designed to improve coordinated, multidisciplinary care for individuals living with limb-girdle muscular dystrophy (LGMD) and to strengthen the clinical infrastructure needed to accelerate therapeutic development.
LGMD represents a group of more than thirty genetically distinct muscular dystrophies that progressively weaken skeletal muscles and currently have few treatment options. As multiple therapies targeting LGMD subtypes begin to advance through clinical development—including small molecules, pathway-modifying drugs, and gene therapies—the need for coordinated clinical infrastructure has become increasingly urgent.
The LGMD Centers of Excellence initiative brings together internationally recognized neuromuscular specialists and academic medical centers to create a collaborative platform for multidisciplinary care and clinical trial readiness.
“LGMD has historically faced significant barriers to therapeutic development due to fragmented care infrastructure and extremely small patient populations,” said Kat Bryant Knudson, founder of the Speak Foundation and an individual living with LGMD. “By creating a coordinated network of clinical centers with a dedicated LGMD Care Coordinator, we are helping prepare the field for the next phase of drug development and ensuring that patients have greater access to the healthcare they deserve.”
“As clinicians, we see firsthand the burden that fragmented care places on families. The LGMD Centers of Excellence model prioritizes coordination, genetic diagnosis where possible, and long-term follow-up — ensuring that patients receive comprehensive care that evolves alongside scientific discovery,” said Dr. Katherine Mathews, University of Iowa Health Care.
The inaugural LGMD Centers of Excellence include several of the nation’s leading neuromuscular programs. Participating institutions include University of Iowa Health Care, where Dr. Katherine Mathews leads neuromuscular research and clinical care initiatives; VCU Health, home to the nationally recognized LGMD program and founder of GRASP-LGMD, led by Dr. Nicholas Johnson; University of Florida College of Medicine, where Dr. Barry Byrne has pioneered gene therapy approaches for muscular dystrophy; University of Minnesota, where Dr. Peter Kang leads the Greg Marzolf Jr. Muscular Dystrophy Center; and The University of Texas at San Antonio, where Dr. Matthew Wicklund leads clinical care and research programs for neuromuscular disease. The network is expected to expand to additional academic centers where expertise in LGMD care and research already exists.
“The launch of the LGMD Center of Excellence at VCU Health represents a meaningful step forward for patients and families living with limb-girdle muscular dystrophy. Our goal is to deliver coordinated, multidisciplinary care while strengthening our readiness for clinical trials and access to emerging therapies. By partnering with the Speak Foundation, we are aligning clinical excellence with patient-centered infrastructure in a way that truly meets the needs of this community,” said Dr. Nicholas Johnson, neuromuscular neurologist at VCU Health.
The launch of the network comes at a pivotal moment for the LGMD field. Several therapies targeting specific LGMD subtypes are advancing through late-stage clinical development, including treatments for FKRP-related LGMD that may soon represent the first approved therapy for a form of the disease.
“Building strong clinical infrastructure is one of the most important steps in accelerating drug development for rare diseases,” said Knudson. “The LGMD Centers of Excellence represent a significant step forward in aligning patients, clinicians, and researchers as true partners as new therapies emerge. We are also pleased to announce our care partners, CureLGMD2i and LGMD Awareness Foundation, who are joining us in advancing this mission.”
Over the past 17 years, the Speak Foundation has worked to build the broader ecosystem supporting LGMD research and therapeutic development. The organization has convened scientific workshop meetings with the U.S. Food and Drug Administration, launched the International LGMD Conference, established LGMD Day on the Hill to engage policymakers, and developed LGMD News Magazine to elevate both patient voices and scientific progress.
The LGMD Centers of Excellence initiative represents the next phase of that effort, helping ensure that clinical infrastructure keeps pace with the rapidly evolving therapeutic pipeline.
About The Speak Foundation
Founded in 2008, The Speak Foundation is the first patient-led nonprofit dedicated to amplifying the voices of individuals living with all forms of Limb-Girdle Muscular Dystrophy (LGMD), accelerating research, and improving access to coordinated care. The foundation works across the research, clinical, and policy landscape to foster collaboration between patients, scientists, clinicians, and industry partners while advocating for stronger research infrastructure and therapeutic development. For more information on the LGMD Centers of Excellence, please visit thespeakfoundation.com.
About Virginia Commonwealth University and VCU Health
Virginia Commonwealth University is a major, urban public research university with national and international rankings in sponsored research. Located in downtown Richmond, VCU enrolls more than 29,000 students in more than 200 degree and certificate programs in the arts, sciences and humanities across VCU’s 12 schools and three colleges. The VCU Health brand represents the VCU health sciences academic programs, the VCU Massey Comprehensive Cancer Center and the VCU Health System, which comprises VCU Medical Center (the only academic medical center in the region), Community Memorial Hospital, Tappahannock Hospital, Children’s Hospital of Richmond at VCU, and MCV Physicians. The clinical enterprise includes a collaboration with Sheltering Arms Institute for physical rehabilitation services. For more, please visit vcu.edu and vcuhealth.org.
About the University of Minnesota Medical School
The University of Minnesota Medical School is at the forefront of learning and discovery, transforming medical care and educating the next generation of physicians. Our graduates and faculty produce high-impact biomedical research and advance the practice of medicine. We acknowledge that the U of M Medical School is located on traditional, ancestral and contemporary lands of the Dakota and the Ojibwe, and scores of other Indigenous people, and we affirm our commitment to tribal communities and their sovereignty as we seek to improve and strengthen our relations with tribal nations. Learn more at med.umn.edu.
About University of Iowa Health Care
University of Iowa Health Care is a leading academic medical center with a nationally recognized neuromuscular program, The LGMD program is led by Dr. Katherine Mathews, joined by neuromuscular specialists Dimah Saade, Katie Lutz, Christopher Nance, Andrea Swenson and Michael Shy. The center provides comprehensive multidisciplinary care for individuals living with muscular dystrophies and other neuromuscular disorders. Investigators at UI Health Care are actively involved in clinical research and therapeutic development aimed at advancing new treatments for LGMD. As part of the LGMD Centers of Excellence network, the team is committed to improving coordinated care and strengthening the clinical infrastructure needed to accelerate therapies.
About The University of Texas at San Antonio
The University of Texas at San Antonio is the 3rd largest university in Texas and in 2024 was rated the 6th fastest growing research university in biological sciences. The muscle disease team at UT San Antonio seeks to provide multidisciplinary care, clinical and translational research, and patient engagement for people living with muscular dystrophy. With goals of excellence in focused, personalized, and coordinated care along with groundbreaking research, our center serves patients arriving from Texas, the United States, and internationally. As one of the LGMD Centers of Excellence, we yearn to break down barriers, propel science, validate methodologies, comprehensively care for patients, and catapult treatments into the marketplace.
About University of Florida
UF Health Shands Hospital is a private, not-for-profit academic health center located in Gainesville, Florida. It serves as the primary teaching hospital for the University of Florida College of Medicine and is part of the broader UF Health system, which is one of the Southeast’s most comprehensive academic health centers.
The University of Florida pairs world class medical expertise with cutting edge advancements in gene therapies as home to the Powell Gene Therapy Center (PGTC). Led by Dr. Barry Byrne, the PGTC is working to bring innovation to care in LGMD, as well as many other inherited diseases. The team has been instrumental in administering gene therapies for LGMD and in the development of next generation therapies. At the University of Florida, patients and their families are an integral part of the process and can trust that the PGTC is relentless in pursuing the best possible outcomes for their patients – even ones yet to be imagined.
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