Patient-led group launches network of clinics to improve LGMD care

Patient-led nonprofit The Speak Foundation has announced the launch of the LGMD Centers of Excellence, a novel alliance of clinics dedicated to improving care for people affected by limb-girdle muscular dystrophy (LGMD).

“LGMD has historically faced significant barriers to therapeutic development due to fragmented care infrastructure and extremely small patient populations,” Kat Bryant Knudson, founder of the Speak Foundation and an individual living with LGMD, said in a press release. “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.”

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LGMD encompasses more than 30 distinct forms of muscular dystrophy, all characterized by similar patterns of muscle weakness that primarily affect the hips and shoulders. Currently, no treatments have been approved by the U.S. Food and Drug Administration (FDA) for any form of LGMD, but that could soon change. Just a few weeks ago, Bridgebio Pharma asked the FDA to approve its experimental oral therapy, BBP-418, for the treatment of LGMD type 2i.

With the possibility of approved treatments on the horizon, the newly launched Centers of Excellence aim to create an infrastructure that enables experts in LGMD to collaborate on testing promising new therapies and determine the best way to deliver care to patients. Other advocacy groups, namely CureLGMD2i and LGMD Awareness Foundation, are also supporting the new initiative.

“Building strong clinical infrastructure is one of the most important steps in accelerating drug development for rare diseases,” Knudson said. “The LGMD Centers of Excellence represent a significant step forward in aligning patients, clinicians, and researchers as true partners as new therapies emerge.”

Founding members of the LGMD Centers of Excellence include University of Iowa Health Care, Virginia Commonwealth University and VCU Health, the University of Florida College of Medicine, the University of Minnesota, and the University of Texas at San Antonio. The network plans to expand to additional academic centers that already have expertise in LGMD care and research.

“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 Katherine Mathews, MD, of University of Iowa Health Care.

Nicholas Johnson, MD, a neuromuscular neurologist at VCU Health, added that the launch of the new network “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,” Johnson said.