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UI part of seven-site research network for Limb-Girdle Muscular Dystrophy

Oct 31, 2018 by UI Health Care

The University of Iowa is one of just seven academic medical centers in the country included in a $700,000 grant to develop a clinical research network studying a specific rare type of muscular dystrophy.

The grant, from the Muscular Dystrophy Association (MDA), will be used to establish the Limb-Girdle Muscular Dystrophy Clinical Research Network. Limb-Girdle Muscular Dystrophy describes childhood- or adult-onset muscular dystrophies that that cause weakness and wasting of the muscles in the arms and legs, specifically the shoulders, upper arms, pelvic area and thighs.

The goal of the new research network is to standardize clinical approaches and develop tools for measuring outcomes in clinical trials.

Katherine Mathews, MD, professor and vice chair for clinical investigation in the Stead Family Department of Pediatrics, UI Carver College of Medicine, and director of the Muscular Dystrophy Clinic and the Iowa Neuromuscular Program, will lead the UI involvement in the network. Her team has been studying one form of Limb-Girdle Muscular Dystrophy, measuring outcomes in anticipation of future clinical trials. The ongoing LGMD research at UI Carver College of Medicine is carried out as part of the  Iowa Wellstone Muscular Dystrophy Cooperative Research Center, funded by the National Institutes of Health  and directed by Kevin Campbell, PhD, and Steven Moore, MD, PhD. Mathews says that information gained from this ongoing study will be useful for the network.

The UI team is the only pediatric neurology team included in the network.

“No one group has taken ownership of Limb-Girdle research,” Mathews says. “This consortium will be in a place to provide the framework for clinical trials.”

Limb-Girdle Muscular Dystrophy is a group of at least 19 diseases classified by the genetic malfunctions or flaws.

The MDA grant was awarded to Virginia Commonwealth University, which will act as lead institution in the network. Other medical centers included are University of California, Irvine; University of Colorado; Brigham and Women’s Hospital; University of Kansas Medical Center; and Washington University in St. Louis, Mo.

For more information, visit https://www.mda.org/disease/limb-girdle-muscular-dystrophy.